systematic review of health state utility values in

30
REVIEW Open Access Systematic review of health state utility values in metastatic non-small cell lung cancer with a focus on previously treated patients Noman Paracha 1* , Ahmed Abdulla 1,3 and Katherine S. MacGilchrist 2 Abstract Background: Health state utility values (HSUVs) are an important input to economic evaluations and the choice of HSUV can affect the estimate of relative cost-effectiveness between interventions. This systematic review identified utility scores for patients with metastatic non-small cell lung cancer (mNSCLC), as well as disutilities or utility decrements relevant to the experience of patients with mNSCLC, by treatment line and health state. Methods: The MEDLINE®, Embase and Cochrane Library databases were systematically searched (September 2016) for publications describing HSUVs in mNSCLC in any treatment line. The EQ-5D website, the School of Health and Related Research Health Utilities Database (ScHARRHUD) and major pharmacoeconomic and clinical conferences in 20152016 were also queried. Studies in adults with previously treated mNSCLC were selected for further analysis. The information extracted included study design, description of treatment and health state, respondent details, instrument and tariff, HSUV or (dis) utility decrement estimates, quality of study, and appropriateness for use in economic evaluations. Results: Of 1883 references identified, 36 publications of 34 studies were included: 19 reported EQ-5D scores; eight reported HSUVs from valuations of vignettes made by members of the public using standard gamble (SG) or time trade-off (TTO); two reported SG or TTO directly elicited from patients; two reported EQ-5D visual analogue scale scores only; one reported Assessment of Quality of Life instrument scores; one reported HSUVs for caregivers to patients with mNSCLC using the 12-item Short-Form Health Survey; and one estimated HSUVs based on expert opinion. The range of HSUVs identified for comparable health states showed how differences in study type, tariff, health state and the measures used can drive variation in HSUV estimates. Conclusions: This systematic review provides a set of published HSUVs that are relevant to the experience of adult patients previously treated for mNSCLC. Our review begins to address the challenge of identifying reliable estimates of utility values in mNSCLC that are suitable for use in economic evaluations, and also highlights how varying estimates result from differences in methodology. Keywords: Health state utility values (HSUVs), Health-related quality of life (HRQoL), Metastatic non-small cell lung cancer (mNSCLC), Multi-attribute utility instruments (MAUIs), Standard gamble (SG), Time trade-off (TTO), Systematic literature review * Correspondence: [email protected] 1 F. Hoffmann-La Roche AG, Basel, Switzerland Full list of author information is available at the end of the article © The Author(s). 2018 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated. Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 https://doi.org/10.1186/s12955-018-0994-8

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Page 1: Systematic review of health state utility values in

REVIEW Open Access

Systematic review of health state utilityvalues in metastatic non-small cell lungcancer with a focus on previously treatedpatientsNoman Paracha1*, Ahmed Abdulla1,3 and Katherine S. MacGilchrist2

Abstract

Background: Health state utility values (HSUVs) are an important input to economic evaluations and the choice ofHSUV can affect the estimate of relative cost-effectiveness between interventions. This systematic review identifiedutility scores for patients with metastatic non-small cell lung cancer (mNSCLC), as well as disutilities or utilitydecrements relevant to the experience of patients with mNSCLC, by treatment line and health state.

Methods: The MEDLINE®, Embase and Cochrane Library databases were systematically searched (September 2016)for publications describing HSUVs in mNSCLC in any treatment line. The EQ-5D website, the School of Health andRelated Research Health Utilities Database (ScHARRHUD) and major pharmacoeconomic and clinical conferences in2015–2016 were also queried. Studies in adults with previously treated mNSCLC were selected for further analysis.The information extracted included study design, description of treatment and health state, respondent details,instrument and tariff, HSUV or (dis) utility decrement estimates, quality of study, and appropriateness for use ineconomic evaluations.

Results: Of 1883 references identified, 36 publications of 34 studies were included: 19 reported EQ-5D scores; eightreported HSUVs from valuations of vignettes made by members of the public using standard gamble (SG) or timetrade-off (TTO); two reported SG or TTO directly elicited from patients; two reported EQ-5D visual analogue scalescores only; one reported Assessment of Quality of Life instrument scores; one reported HSUVs for caregivers topatients with mNSCLC using the 12-item Short-Form Health Survey; and one estimated HSUVs based on expertopinion. The range of HSUVs identified for comparable health states showed how differences in study type, tariff,health state and the measures used can drive variation in HSUV estimates.

Conclusions: This systematic review provides a set of published HSUVs that are relevant to the experience of adultpatients previously treated for mNSCLC. Our review begins to address the challenge of identifying reliable estimatesof utility values in mNSCLC that are suitable for use in economic evaluations, and also highlights how varyingestimates result from differences in methodology.

Keywords: Health state utility values (HSUVs), Health-related quality of life (HRQoL), Metastatic non-small cell lungcancer (mNSCLC), Multi-attribute utility instruments (MAUIs), Standard gamble (SG), Time trade-off (TTO), Systematicliterature review

* Correspondence: [email protected]. Hoffmann-La Roche AG, Basel, SwitzerlandFull list of author information is available at the end of the article

© The Author(s). 2018 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, andreproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link tothe Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver(http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 https://doi.org/10.1186/s12955-018-0994-8

Page 2: Systematic review of health state utility values in

BackgroundNon-small cell lung cancer (NSCLC) is the most com-mon form of lung cancer, occurring in 85–90% of lungcancer cases [1], and includes adenocarcinoma (40% ofall lung cancers), squamous cell carcinoma (25–30%)and large cell carcinoma (10–15%) [2]. NSCLC is stagedaccording to the American Joint Committee on Cancer/Union for International Cancer Control system [3], andmeasurement of lesions follows the Response EvaluationCriteria in Solid Tumors (RECIST) [4]. Approximately40% of patients will have metastatic NSCLC (mNSCLC)at diagnosis [5], which includes cancers found in the lungand in the lymph nodes in the middle of the chest (definedas stage IIIA and IIIB; no distant metastasis), and cancersthat have spread to both lungs or to another part of thebody (defined as stage IV; distant metastasis) [6, 7].Treatment is recommended according to the stage of

mNSCLC, but treatment options are limited in the laterstages of disease [7, 8]. Five-year survival rates are con-siderably lower in later than in earlier stages of NSCLC(stage IA, 45%; stage IIIA, 14%; stage IIIB, 5%; stage IV,1%) [9]. Moreover, symptoms such as coughing andwheezing, chest pain, hoarseness and weight loss can se-verely reduce functional independence in patients withmNSCLC [10, 11]. Patient-reported health-related qual-ity of life (HRQoL) provides an overall evaluation ofhealth, well-being and daily functioning, and is impairedin patients with mNSCLC owing both to the disease andto treatment sequelae. Maintenance or improvement ofHRQoL is an important treatment goal [12].HRQoL can be expressed as a health state utility value

(HSUV) ranging from 0 (death) to 1 (full health). If thehealth state is considered to be worse than death, healthstates can be valued at less than 0. Utility values are keydrivers in cost-effectiveness analyses because estimatesof quality-adjusted life-years (QALYs) are obtained bymultiplying HSUVs for each health state by the timespent in that health state. Estimates of cost per QALYare highly sensitive to the choice of HSUV. It is there-fore important to identify specifically those HSUVs thathave been derived using methods acceptable to healthtechnology assessment (HTA) authorities [13].HSUVs can be derived using a range of instruments

and techniques [14, 15]. In brief, instruments include:generic preference-based measures such as the EQ-5D-3 L[16] or EQ-5D-5 L [17], Health Utilities Index (HUI) [18],6-dimension Short-Form Health Survey (SF-6D) [19], As-sessment of Quality of Life instrument (AQoL) [20], 15-di-mensional HRQoL measure [21], Quality of Well-Beingscale [22], and multi-attribute utility instrument; as well asdirectly elicited standard gamble (SG), time trade-off(TTO) and visual analogue scale (VAS, e.g. EuroQoL VAS[EQ-VAS]). Mapping algorithms may also be used to con-vert values obtained from a condition-specific questionnaire

to a generic preference-based measure; or to convert datafrom the 12- or 36-item Short-Form Health Survey (SF-12or SF-36) to SF-6D [23]. Techniques may vary in terms ofwhose health is being measured (a patient’s or a caregiver’s),who responds to the questionnaire or, if using vignettes,who considers the health-state description (the patient re-garding their own health, a patient with a different disease,the patient’s closest caregiver, another caregiver, a physicianor another healthcare provider). For preference-based mea-sures, variation can stem from who values the health state(e.g. UK general population sample) and which choice-based method is used in this valuation (SG or TTO).HTA bodies including the UK National Institute for

Health and Care Excellence (NICE) [24, 25], the ScottishMedicines Consortium (SMC) [26], the Canadian Agencyfor Drugs and Technologies in Health (CADTH) [27], theFrench Haute Autorité de Santé (HAS) [28] and the Aus-tralian Pharmaceutical Benefits Advisory Committee(PBAC) [29] have stated preferences for HSUV method-ology. Across these agencies, there is a preference forHSUVs estimated using generic preference-based mea-sures. NICE has a strong preference for EQ-5D, as thisreduces variability induced when different instruments areused between different disease areas. Agencies alsostrongly prefer patients to be the respondents, as patientscan best describe their own health state. Finally, valuationestimated using a country-specific general-population tar-iff via a choice-based elicitation technique such as SG orTTO is preferred, as this represents societal preferences.This systematic review had three main aims: first, to

identify HSUVs for adults with previously treatedmNSCLC, by treatment line and health state, and toevaluate the relevance of each health state to patients,for example, line of treatment, adverse events (AEs),response status and prognostic factors; second, to iden-tify relevant disutilities or utility decrements associatedwith adverse events (irrespective of line of treatment orhealth state). Finally, the suitability of the HSUVs ac-cording to HTA reference case was explored and thequality of the HSUVs assessed.

MethodsStudy design and search strategyA systematic review of HSUVs in mNSCLC was under-taken to identify HSUV studies in any treatment line.Studies, published either as full papers or as conferenceabstracts, in patients previously treated for mNSCLCwere selected for further analysis. The following data-bases were searched: Embase (1974 to 7 September2016); MEDLINE® (1966 to 7 September 2016); MED-LINE In-Process and e-publications ahead of print (data-base inception to 7 September 2016); and the CochraneLibrary (including the Cochrane Database of SystematicReviews, the Database of Abstracts of Reviews of Effects,

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 2 of 30

Page 3: Systematic review of health state utility values in

the Cochrane Central Register of Controlled Trials, theNational Health Service Economic Evaluation Databaseand the HTA database; 1968 to 7 September 2016).Search strings are summarized in Additional file 1:

Table S1, and were constructed not only to find utilitiesin mNSCLC (using a wide range of NSCLC and mNSCLCterms combined with the HSUV filter adapted from Arberet al. 2015 [30]) but also to identify all relevant disutilitiesor utility decrements associated with AEs/comorbidities.To ensure that estimates would be available from previ-ously treated mNSCLC populations for all AEs or comor-bidity health states relevant to the experience of suchpatients, the strings were designed to search for disutilitiesor decrements from a broader group of populations, asfollows: from lung cancer; for progressive disease disutil-ities from advanced/metastatic cancer; for disutilities asso-ciated with the most common sites of metastasis from the

lung (bone, respiratory system, nervous system, adrenalgland and liver) from advanced cancer; for disutilities as-sociated with AEs or toxicities of cancer therapy; and dis-utilities associated with specific grade 3–4 AEs known tooccur with cancer treatments from advanced cancer popu-lations (pneumonia, pneumonitis, increased aspartate ami-notransferase, febrile neutropenia, neutropenia, infection,sepsis, fatigue, lethargy, nausea, vomiting, ulcers, stoma-titis, gastrointestinal disturbance, diarrhoea, visual disturb-ance, hearing loss, hair loss, psychological/self-esteemchanges, rash, anaemia, bleeding and hypertension).From the identified disutilities/decrements for eachAE/co-morbidity health state, those from the mostrelevant population available could be selected follow-ing an order of decreasing population specificity fromfirst-line mNSCLC to NSCLC, lung cancer and ad-vanced/metastatic cancer (Fig. 1).

(Nafees [69])

(Tabberer [52])

(Doyle [65])

(Nafees [68])

(Handorf [70])

Grade III/IV neutropenia, febrile neutropenia, fatigue, nausea and vomiting, diarrhoea, hair loss, rash

Neutropenia, febrile neutropenia, nausea, diarrhoea, rash, stomatitis and neuropathy

Severe symptoms for cough, dyspnoea, pain

Grade III/IV neutropenia, febrile neutropenia, fatigue, nausea and vomiting, diarrhoea, hair loss, rash, bleeding, hypertension

Neutropenia, pneumothorax, haemorrhage, thrombocytopenia, thrombosis

• Stage IV 2L: UK

• Stage IV LNS: UK

• Stage IV LNS: UK

• Stage IV 1L: UK + multinational

• Stage IV 1L: USA

Advanced/mNSCLC

Early stage NSCLC (Grutters [44])• LNS/early stage

Advanced/mLC (NSCLC + SCLC)

(Yokoyama [55])• Stage IIIB/IV LC with BM: Japan

Not reported(Westwood [71])• Lilly and Roche NSCLC

NICE submissions

Cancer(Lloyd [59])• Cancer unclear stage:

UK

LC + BC(Grunberg [58])• LC + BC: USA

Advanced cancer

(Matza [67])

Grade III+ dyspnoea

Average disutility for SREs (pathologic fracture, radiation or surgery to bone lesion, spinal cordcompression or hypercalcaemia)

Anaemia (single disutility), i.v. treatmentmode, oral treatment mode

Anaemia (by Hb level)

Chemotherapy-related limited nausea, limited vomiting, limited nausea and vomiting, continuous nausea and vomiting

Specific SRE disutilities for spinal cord compression with/without paralysis, fracture of leg, fracture of rib, fracture of arm, radiation treatments, surgery to stabilize bone

• Stage IV cancer with BM: UK, Canada

Fig. 1 Studies reporting adverse event health state (dis) utilities by patient population and country. Abbreviations: 1L first line, 2L second line, BCbreast cancer, BM bone metastasis, Hb haemoglobin, i.v. intravenous, LC lung cancer, LNS line of treatment not specified, mLC metastatic lungcancer, mNSCLC metastatic non-small cell lung cancer, NICE National Institute for Health and Care Excellence, NSCLC non-small cell lung cancer,SCLC small cell lung cancer, SRE skeletal-related event

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 3 of 30

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Using the term “NSCLC” or “non-small cell lung can-cer”, manual searching of the EQ-5D website, of theSchool of Health and Related Research Health UtilitiesDatabase (ScHARRHUD) and of major pharmacoeco-nomic and clinical conferences in 2015–2016 was con-ducted on 3 and 5 December 2016. Conferences included:the International Society for Pharmacoeconomics andOutcomes Research (ISPOR) International Meetings andEuropean Congresses; the HTA International AnnualMeetings (HTAi); the Society for Medical Decision Mak-ing (SMDM) North American Meetings and EuropeanConferences; the American Society of Clinical Oncology(ASCO) Meetings; and the European Society for MedicalOncology (ESMO) Congresses. Bibliographic referencelists of relevant systematic reviews from 2010 onwardswere searched and of relevant cost-utility analyses, andHTA reports from various bodies identified in a paralleleconomic systematic review, including: NICE; SMC; AllWales Medicines Strategy Group (AWMSG); PBAC;CADTH; Institut National d’Excellence en Santé et enServices Sociaux; pan-Canadian Oncology Drug Review(pCODR); and HAS.The PICOS (patient, intervention, comparator, outcome,

study) statements for study inclusion and exclusioncriteria are summarized in Table 1. Although, second- andlater-line data were of primary interest, studies thatreported utilities for patients with mNSCLC who wereeither treatment-naïve or in receipt of maintenancefirst-line treatment were included for reference at the firstscreening but data were not extracted. These studies arelisted in Additional file 2: Table S2.Mapping from condition-specific to preference-based

studies was not sought because it was anticipated thatsufficient published utility and EQ-5D data would beavailable to populate the health states of an economicmodel, and because results based on mapping algo-rithms sit lower in the acceptance hierarchy used bysome HTA authorities (Additional file 3: Figure S1). Wehave acknowledged NICE’s stated preference for EQ-5D-3 L data over EQ-5D-5 L (Additional file 3: FigureS1) and provide detailed information of the instru-ment used for generating data for each identifiedstudy in Table 2 [31].

Study selectionThe screening process complied with the 2009 PreferredReporting Items for Systematic Reviews and Meta-Ana-lyses (PRISMA) guidelines [32]. Publications werede-duplicated using EndNote (Clarivate Analytics, Phila-delphia, PA, USA) and using Rayyan (Qatar ComputingResearch Institute, Doha, Qatar) [33], an internet-basedreference management system endorsed as suitable forsystematic review screening by the European Network forHTA [34]. Abstracts and titles of papers were screened by

one reviewer, and a 50% sample check conducted by asecond reviewer; exclusion criteria are summarized inTable 1. The full texts of papers potentially meeting theselection criteria were screened by one reviewer, and a50% sample check was conducted by a second reviewer.Discrepancies were discussed between reviewers, and anyunresolved disputes were referred to a third reviewer.

Data extractionData were collected using a piloted data-extractionsheet. Extraction was conducted by one reviewer, andpriority data elements were quality checked by a secondreviewer. The information extracted included studydesign, whether the selection criteria yielded a popula-tion that matched the target population (i.e. previouslytreated adult patients with mNSCLC), health state de-scription, instrument type, instrument scale, HSUV or(dis) utility or decrement estimates and measure of vari-ability (median with interquartile range or mean withstandard error, standard deviation or 95% confidenceinterval), derivation methods and if the data presentedwere appropriate for use in HTA submissions to NICE,SMC, CADTH, HAS and PBAC.

Quality and relevance assessmentThe appropriateness of utilities reported for use in eco-nomic evaluations was determined by whether data metthe requirements of the HTA body reference case; andthe quality of utility estimates (based on sample size, re-sponse to the questionnaire, loss to follow-up, handlingof missing data, and reporting of point and variance esti-mates, as discussed in NICE Decision Support UnitTechnical Support Document 11 and its related publica-tion [25, 35]; Additional file 4: Table S3). Any recom-mendation for, or rationale against, the use of specificutilities in a cost–utility analysis model in previouslytreated patients with mNSCLC was also taken into consid-eration in line with preliminary guidance from the ISPORHealth State Utility Good Practices Task Force [36].

ResultsSearch yieldsElectronic database searches identified 1883 citations(1521 from MEDLINE/Embase, 144 from MEDLINE In-Process/e-publications and 218 from the Cochrane Li-brary databases). After de-duplication (51 citations: 30via Endnote and 21 via Rayyan) and title/abstract screen-ing (1557 exclusions), 275 full-text papers werereviewed. Of these, 250 were excluded (21 of which weretagged as reporting first-line treatment; Additional file 2:Table S2), yielding 25 citations that were included fromelectronic sources. Manual searching identified 11 cita-tions. In total, 36 articles were included, reporting 34studies (Table 2). Two articles [37, 38] were linked to

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 4 of 30

Page 5: Systematic review of health state utility values in

other publications [39, 40], and were retained becausethey provided additional information. The study selec-tion is summarized in a PRISMA flow chart in Fig. 2.

Description of studies identifiedAmong the 36 articles (34 studies) identified, 19 reportedEQ-5D scores [37–55] (three studies further specified theinstrument as EQ-5D-3 L [39, 41, 56] and two asEQ-5D-5 L [44, 57]; Table 2), two reported SG or TTOdirectly elicited from patients [58, 59], two reportedEQ-VAS scores only [57, 60], and one reported AQoL

scores [61] (Table 2). Moreover, one study reported SF-12scores for caregivers to patients with mNSCLC [62], eightreported HSUVs from valuations of vignettes made bymembers of the public using SG or TTO [59, 63–69] (oneof which reported both general public-elicited SG andpatient-elicited TTO) [59], and one reported disutility esti-mates based on expert opinion for pneumothorax,thrombocytopenia and thrombosis, adverse event healthstates for which disutilities were not available from otherHSUV derivation methods [70]. A further two articles re-ported HSUVs but were unclear about how these were

Table 1 Inclusion criteria

Characteristic Inclusion criteria Exclusion criteria

Population Adult patients (aged ≥16 years)Locally advanced NSCLC or mNSCLC, second/subsequent lineLocally advanced or metastatic NSCLC, line unspecified

Population not of interest, e.g.• in vitro data• animal data• mixed adult/child population or child population• mixed disease populations without mNSCLC datareported separately

• not disease of interest• 1 L mNSCLC data (treatment-naïve or maintenancefirst-line treatment) were excluded but taggeda

Interventions/comparators

Not relevant for QoL SR selection. Intervention-specificutility data were noted as such during data extraction

N/A

Outcomes For mNSCLC patients:• individual (patient or caregiver) derived mean or medianhealth state utilities from indirect generic HRQoL measure(EQ-5D (-3 L and -5 L), SF-6D, HUI2, HUI3, AQoL, QWB, 15D,MAUI) or direct valuation by TTO, SG or EQ-VAS

• SF-36 or SF-12• general public valuations of vignettes using TTO or SGFor NSCLC or wider population:• disutilities or decrements for AEsb or progressive disease

No outcome of interest:• expert or healthcare provider (doctor, nurse) valuationsof utilities

• utilities not relating to a specific health state

Study design RCTs, non-RCTs, observational data Study design not of interest:• case reports, n = 1 before-and-after studies• PK/PD study only• (Non-systematic) reviews• SRs/NMAsc

Date limits Unlimited –

Child citation Citation linked to another paper but with unique data Child citation or sub-study with no unique data, determinedat first or second pass

Duplicate citation Duplicate/copy

Publication type Publication type not of interest e.g. editorials, commentaries,letters, notes, press articles, unless relevant data has beenpublished in a letter, for example, that does not appearelsewhere in the literatureConfidential reports where unable to use report, or HayesInc. reports requiring purchase

Language English or FrenchAny foreign language paper with an English abstractwere included if sufficient information is present in theEnglish abstract to ensure the eligibility criteria are met

Full text in language other than English or French with noEnglish abstract or no abstract; or insufficient information inEnglish language abstract of foreign language full paper toassess eligibility

aTo enable listing in the reportbDisutilities may be included for AEs, inconvenience of treatment or progressive health states from diseases outside NSCLC (preferably from lung cancer or fromadvanced/metastatic cancer) where no such data are available from patients with NSCLCcSRs were kept in until the second pass, where the full paper’s included studies were examined, after which the SR itself was excludedAbbreviations: 1 L first line, 3 L 3-level, 5 L 5-level, 15D 15-dimensional health-related quality of life measure, AE adverse event, AQoL Assessment of Quality of Lifeinstrument, EQ-VAS EuroQoL visual analogue scale, HRQoL health-related quality of life, HUI2/3 Health Utilities Index Mark 2/3, MAUI multi-attribute utilityinstrument, mNSCLC metastatic non-small cell lung cancer, N/A not available, NMA network meta-analysis, NSCLC non-small cell lung cancer, PD pharmacodynamic,PK pharmacokinetic, QoL quality of life, QWB Quality of Well-Being scale, RCT randomized controlled trial, SF-6D 6-dimension Short-Form Health Survey, SF-12/3612/36-item Short-Form Health Survey, SG standard gamble, SR systematic review, TTO time trade-off, VAS visual analogue scale

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 5 of 30

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Table

2Iden

tifiedutility

stud

iesby

lineof

treatm

ent

Autho

r,year,cou

ntry

Line

oftreatm

ent

Health

state

Instrumen

tTreatm

ent

Firstlinea

Hando

rf2012

USA

[70]

1L

StageIV

aden

ocarcino

maSD

is,PD,SDis+AEs

(neutrop

enia,

pneumotho

rax,haem

orrhage,thrombo

cytope

nia,thrombo

sis)

Expe

rtop

inionestim

ates,bpu

blishe

dsources

NTS

Nafees2016

Multin

ational

andUK[68]

1L

Metastatic

NSC

LCcommon

gradeIII/IV

toxicities(neutrop

enia,

febrile

neutrope

nia,fatig

ue,nauseaandvomiting

,diarrhe

a,hair

loss,rash),b

leed

ing,

hype

rten

sion

TTO(gen

eralpu

blic)

NTS

≥Firstlinec

Che

valier2013

France

and

nine

othe

rcoun

tries[38]

1L,2L,3/4LandBSC

Advanced/metastatic

NSC

LC1L,2L,3/4LPF

andPD

EQ-5D

NTS

Cho

uaid

2013

Multin

ational[39]

1L55.1%

2L24.7%

3/4L17.9%

BSC2.3%

Advanced/metastatic

NSC

LC1L,2L,3/4L,BSCandmixed

linePF

andPD

EQ-5D,EQ-VAS

NTS

Iyer

2013

France,G

ermany[46]

1L52%

2LL

48%

Advanced/metastatic

NSC

LCEQ

-5D

NTS

Second

line

Blackhall2014Multin

ational[41]

2Lafterprog

ressionon

platinum

-based

1L

therapy

Locally

advanced

/metastatic

ALK+NSC

LCBL

andtreatm

ent-

specificutilities(not

PSS)

EQ-5DEQ

-VAS

CRZ

PEM

DOC

Huang

2016

Worldwide[45]

2Lafterplatinum

-based

therapy

AdvancedPD

-L1+

NSC

LCNTS

PF,PD

NTS

timeto

death

EQ-5D

PEMB

DOC

Lang

ley2013

UK,Australia[48]

2Ld

Treatm

ent-specificstageIV

NSC

LCwith

BMat

BLandafter

certaintim

epo

intson

treatm

ent

EQ-5D

OSC

WBRT+OSC

Nafees2008

UK[69]

2L

Metastatic

NSC

LCPD

,RES,SDis,com

mon

gradeIII/IV

toxicities

(neutrop

enia,feb

rilene

utrope

nia,fatig

ue,nauseaandvomiting

,diarrhea,hairloss,rash)

SG(gen

eralpu

blic)

NTS

Novello

2015

Multin

ational[49]

2L

StageIII/IV

recurren

tNSC

LC(SQandNSQ

)treatm

ent-specificat

BLandcertaintim

epo

intson

treatm

ent(≤

30weeks)

EQ-5D,EQ-VAS

NIN

+DOC

PLA+DOC

Reck

2015

Multin

ational[50]

2L

AdvancedSQ

NSC

LCtreatm

ent-specificat

BLrepo

rted

.Collected

also

forup

to1year

butvalues

NRin

abstract

EQ-5D,EQ-VAS

NIVO

DOC

Rude

ll2016

USA

,Canada,Hon

gKo

ng,Italy,Japan,Rep

ublic

ofKo

rea,Spain,Taiwan

[57]

2L

AdvancedEG

FR+NSC

LC,treatmen

t-specificat

BLand36

weeks

onOSI

EQ-VAS

OSI

Schu

ette

2012

Germany,

Austria[51]

2L

StageIII/IV

NSC

LCtreatm

ent-specificat

BL,6

weeks

(secon

dcycle)

andsixthcycle

EQ-5D

EQ-VAS

PEM

Vargas

2009

Mexico[72]

2Lafterprevious

CHEM

ONSC

LC,stage

NR(assum

edadvanced

),treatm

ent-specificno

tPSS

GlobalQ

oLinde

xERL

Taxane

s

≥Second

line

Che

n2010

UK/multin

ational[64]

2L,3LandBSC

StageIIIb/IV

EGFR+NSC

LCtreatm

ent-specific

(not

PSS)

SG(gen

eralpu

blic)

DOC

PEM

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 6 of 30

Page 7: Systematic review of health state utility values in

Table

2Iden

tifiedutility

stud

iesby

lineof

treatm

ent(Con

tinued)

Autho

r,year,cou

ntry

Line

oftreatm

ent

Health

state

Instrumen

tTreatm

ent

On/afterDOC2L

On/afterPEM

2L

OnERL3LBSC

ERL

BSC

Griebsch

2014

Multin

ational[37]

2LLe

andtreatm

ent-

naïve

StageIIIbwith

pleuraleffu

sion

orstageIV

NSC

LCaden

ocarcino

matreatm

ent-specificandNTS

effect

ofprog

ression

EQ-5D,EQ-VAS

AFA

BSC

CIS/PEM

Hirsh2013

Multin

ational[40]

2LLf

StageIIIb/IV

NSC

LCBL

andtreatm

ent-specificon

oralAFA

50mgq.d.

+BSCor

PLA+BSC

EQ-5D

AFA

+BSC

PLA+BSC

Stew

art2015

Canada[56]

Targeted

therapy84%

3LL25%

RCT22%

Metastatic

EGFR+NSC

LC,allpatientsno

tPSS

PR/SDisEG

FRTKI

RESCHEM

ORESGEF

RESERL

RESOSI

PDEG

FRTKI

EQ-5D-3

LGEF

ERL

OSI

Schw

artzbe

rg2015

USA

,Canada[60]

2LL

Squamou

sandno

n-squamou

sstageIIIb/IV

NSC

LCtreatm

ent-specificweeks

6–30

Treatm

ent-specificPR,SDisandPD

weeks

6–30

EQ-VAS

NIVO

Treatm

entlineno

tspecified

Bradbu

ry2008

Canada[42]

Unclear

AdvancedNSC

LCTreatm

ent-specific(not

PSS)

EQ-5D

ERL

BSC

Chang

2016

SouthKo

rea[63]

NR

AdvancedNSC

LCfro

m>360days

before

deathto

<30

days

before

death(not

PSS)

TTO(gen

eralpu

blic)

NTS

Dansk

2016

UK[43]

NR

Synthe

sizedadvanced

NSC

LCPF,PDused

inNICEHTA

sTrial-b

ased

PF,PD

Non

-trialb

ased

PF,PD

EQ-5D

NTS

Doyle2008

UK[65]

NR

Metastatic

NSC

LCSD

is,RES,severesymptom

s(cou

gh,d

yspn

oea,pain)

SG(gen

eralpu

blic)

NTS

Grunb

erg2009

USA

[58]

NR

Mixed

cancer

popu

latio

nchem

othe

rapy-related

nausea,

vomiting

,and

nausea

andvomiting

,ofd

ifferen

tseverities

SG(patient)

CHEM

O

Grutters2010

Nethe

rland

s[44]

NR

NSC

LCwith

grade3+

dyspno

eaEQ

-5D

NTS

Jang

2010

Canada[47]

NR

StageIV

NSC

LCandlocally

advanced

NSC

LCEQ

-5D

NTS

Linn

et2015

Den

mark[62]

Unclear

Metastatic

NSC

LCsecond

andthird

CHEM

Ocycles

onoralVINO

Patient

andcaregiverutilitiesrepo

rted

SF-12

VINO

Lloyd2005

UK[66]

NR

StageIV

NSC

LCRES,SD

isi.v.treatmen

t,SD

isoraltreatm

ent,

PD,end

oflife

SG(gen

eralpu

blic)

NTS

Lloyd2008

[59]

Previous

CHEM

OAnaem

iaby

haem

oglobinlevel

Gen

eralpu

blicSG

,patient

TTO

NTS

Manser2006

Australia[61]

NR

StageIV

NSC

LCAQoL

NTS

Matza

2014

UKandCanada[67]

NR

StageIV

cancer

with

BMsanddifferent

type

sof

SRE

(spinalcordcompression

with

/with

outparalysis,fracture

TTO(gen

eralpu

blic)

NTS

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 7 of 30

Page 8: Systematic review of health state utility values in

Table

2Iden

tifiedutility

stud

iesby

lineof

treatm

ent(Con

tinued)

Autho

r,year,cou

ntry

Line

oftreatm

ent

Health

state

Instrumen

tTreatm

ent

ofleg,

fractureof

rib,fractureof

arm),radiationtreatm

ent

(2weeks,5

appo

intm

ents/w

eek),radiatio

ntreatm

ent

(2appo

intm

ents),surgeryto

stabilize

bone

Tabb

erer

2006

UK[52]

NR

AdvancedNSC

LCRES,SD

is,SDisoraltreatm

ent,SD

isi.v.

treatm

ent,PD

,nearde

ath,AEs

(neutrop

enia,feb

rile

neutrope

nia,nausea,d

iarrho

ea,rash,stom

atitis,ne

urop

athy)

EQ-5D(gen

eralpu

blic)

NTS

Trippo

li2001

Italy[53]

NR

Metastatic

NSC

LCEQ

-5D,EQ-VAS

NTS

Westw

ood2014

[71]

NRforothe

rdisutilities

AdvancedNSC

LCDisutility

foranaemiaandfori.v./o

raltreatmen

tmod

eSG

NRforothe

rdisutilities

NTS

ERL

i.v.tx

Yang

2014

Taiwan

[54]

NR

NSC

LCop

erable(I–IIIA)andNSC

LCinop

erable(IIIB/IV

)EQ

-5D

NTS

Yokoyama2013

Japan[55]

NR

StageIIIB/IV

mixed

NSC

LC/SCLC

with

bone

metastasis

andSRE(patho

logicfracture,radiationor

surgeryto

bone

lesion

,spinalcordcompression

orhype

rcalcaem

ia)

EQ-5D

NTS

a Studies

wereretained

,despite

repo

rtingfirst-line

treatm

enton

ly,b

ecau

sethey

repo

rted

prog

ressivediseaseutility

estim

ates

similarto

thoseseen

inasecond

-line

popu

latio

n,or

repo

rted

AEdisutility

estim

ates

from

popu

latio

nsbroa

derthan

mNSC

LCbAlth

ough

theutilitie

swereba

sedon

expe

rtop

inion,

thesewereretained

,asthey

prov

idedisutility

estim

ates

forthead

verseeven

tspn

eumotho

rax,thrombo

cytope

niaan

dthrombo

sis,no

tavailableelsewhe

rec Studies

repo

rted

data

onfirst-line

treatm

entan

dsubseq

uent

treatm

entlin

esdPrevious

treatm

entwith

system

icCHEM

Oor

EGFR

inhibitors

allowed

e Lux-Lun

g1triald

atawerein

patie

ntsprog

ressed

on1–

2lin

esof

treatm

ent,on

eof

which

was

platinum

based(cou

ldinclud

ead

juvant

settingtreatm

entlin

e),and

hadPD

afterat

least12

wks

ofER

Lor

GEF.Lux-Lun

g3triald

atawerein

treatm

ent-na

ïvepa

tients,so

not2L.

f Progressedon

1–2lin

esof

treatm

ent,on

eof

which

was

platinum

based,

andha

dPD

afterat

least12

wks

ofER

Lor

GEF

Abb

reviations:1

Lfirst

line,

2Lsecond

line,

2LL

second

andsubseq

uent

line,3LLthird

andsubseq

uent

line,

3/4Lthird

andfourth

line,

AEad

verseeven

t,AFA

afatinib,A

QoL

Assessm

entof

Qua

lityof

Life

instrumen

t,BL

baselin

e,BM

bone

metastasis,BSCbe

stsupp

ortiv

ecare,C

HEM

Ochem

othe

rapy

,CIScisplatin

,CRZ

crizotinib,D

OCdo

cetaxel,EG

FRep

idermal

grow

thfactor

receptor,EQ-VASEu

roQol

visual

analog

uescale,

ERLerlotin

ib,

GEF

gefitinib,G

EMge

mcitabine

,i.v.intraveno

us,N

INninted

anib,N

IVOnivo

lumab

,NRno

trepo

rted

,NSC

LCno

n-sm

allcelllun

gcancer,N

SQno

n-squa

mou

s,NTS

nottreatm

ent-specific,OSC

optim

alstan

dard

care,O

SIosim

ertin

ib,P

Dprog

ressivedisease,

PEM

pemetrexed,

PEMBpe

mbrolizum

ab,P

Fprog

ression-free,P

LAplaceb

o,PR

partialrespo

nse,

PSSprog

ression-status-spe

cific,q

.d.o

nceda

ily,Q

oLqu

ality

oflife,

RCTrand

omized

controlledtrial,RESrespon

se,SCLCsm

allcelllun

gcancer,SDisstab

ledisease,

SF-1212

-item

Short–Fo

rmHealth

Survey,SGstan

dard

gamble,

SREskeletal-related

even

t,TKItyrosinekina

seinhibitor,TTOtim

etrad

e-off,

VASvisual

analog

uescale,

VINOvino

relbine,

WBR

Twho

le-brain

radiothe

rapy

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 8 of 30

Page 9: Systematic review of health state utility values in

derived; one reported disutilities used in previous NICEsubmissions, for anaemia and for oral and intravenoustreatment modes [71], and one reported a “global qualityof life index” for second-line NSCLC [72].Among the dataset, two studies were retained despite

reporting first-line treatment only, because they reportedAE disutility estimates from populations broaderthan mNSCLC [68, 70]; three further studies that re-ported first-line data also reported on subsequenttreatment lines [38, 39, 46]. Eleven studies focusedexclusively on HSUVs associated with second-linetreatment [41, 45, 48–51, 57, 59, 69, 71, 72], and fivereported HSUVs in second-line and subsequent treatment[37, 40, 56, 60, 73]. Line of treatment was unspecified in15 studies [42–44, 47, 52–55, 58, 61–63, 65–67].

Relevant HSUVs by line of treatmentUtilities were reported for a range of health state types:treatment-specific or not, RECIST response-based ornot, time-on-treatment, time-till-death, or a combin-ation of these. Details of HSUV estimates by treatmentline are given in Table 3. Among patients receivingsecond-line or subsequent treatment for advanced

NSCLC or mNSCLC, mean HSUV estimates based onEQ-5D for stable/progression-free disease and for pa-tients at baseline or pre-treatment were in the range0.66–0.76 [38, 39, 41, 45, 49, 50]; in the same group,mean values for patients with progressive disease weregenerally lower (0.55–0.69) [38, 39, 45]. Among patientson treatment at this stage of disease and treatment line,the range of mean HSUVs based on EQ-5D was broad(0.53–0.82) [40, 41, 46, 51, 56], the highest value beingassociated with treatment with tyrosine kinase inhibitors[41, 56]. A similar range of HSUV values was seenamong patients being treated for advanced NSCLC ormNSCLC when the treatment line was unspecified (0.53–0.77) [42, 47, 52, 53]. Only three papers specified usingEQ-5D-3 L [39, 41, 56] and only two EQ-5D-5 L [44, 57].Disutilities for progression from a stable state were

− 0.056 or − 0.065 by EQ-5D, both from Griebsch et al.[37], or − 0.1798 by general population-derived SG [69].Overall, HSUVs varied not only by treatment line and dis-ease state, but also by the treatment received under thesame health state (potentially reflecting differences insafety profiles) and by the instrument/tariff used to derivethe HSUV.

Duplicate papers removed: 51Via Endnote: 30Via Rayyan: 21

Citations excluded: 1557Excluded design: 657

Excluded population: 414No outcome of interest: 255No relevant camparator: 129

Secondary source: 51Duplicates: 51

Child: 0

Citations excluded: 250Duplicate: 21

Sub-study/child citation: 12No relevant outcome: 93

Population: 40Design: 50

Language: 4Publication type: 2

First-line: 21Mapping algorithm or validation: 5

Full paper unavailable: 1Mapped utility: 1

Total number of papers identified: 1883Embase and MEDLINE: 1521

MEDLINE In-Process and e-publications: 144Cochrane: 218

Title/abstract screening: 1832

Included from manual search: 11

Full-text articles assessed for eligibility: 275

Included in systematic review: 36(34 unique studies)

Fig. 2 PRISMA flow chart for study selection. Abbreviation: PRISMA Preferred Reporting Items for Systematic Reviews and Meta-Analyses

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 9 of 30

Page 10: Systematic review of health state utility values in

Relevant disutilities and decrementsEleven studies identified in this systematic review re-ported disutilities or decrements for AE health states[44, 52, 55, 58, 59, 65, 67–71]. Only two studies reporteddisutilities specifically associated with second-line treat-ment [69, 71], and another two studies did not specifythe treatment line [44, 65]; disutility and decrement dataare summarized in Table 4. Utility-incorporating decre-ments were identified for the following AEs in thecontext of second-line “stable disease” or second-line“responding”: diarrhoea, fatigue, febrile neutropenia, hairloss and nausea/vomiting. Disutilities associated withsecond-line treatment were reported for the followingevents [69]: “moving from stable to progressive state”(− 0.18), neutropenia (− 0.09), febrile neutropenia (− 0.09),fatigue (− 0.07), nausea/vomiting (− 0.05), diarrhoea(− 0.05), hair loss (− 0.04) and rash (− 0.03).Further recommended sources of AE health state (dis)

utilities were as follows (Fig. 1): in 2 L from generalpopulation SG in Nafees et al. 2008 [69]; in metastaticNSCLC (line unspecified) from general population SG inDoyle et al. 2008 [65]; in 1 L from patients withoutNSCLC using directly elicited TTO in Nafees et al. 2016[68]; in 2 L in NSCLC as reported in Westwood et al.2014 [71]; in cancer with bone metastases forskeletal-related events from general population TTO inMatza et al. 2014 [67]; stage IV NSCLC in 1 L fromexpert opinion estimates in Handorf et al. 2012(expert-opinion-derived utilities from this study wereincluded, as they are the only source of estimates forpneumothorax, thrombocytopenia and thrombosis dis-utilities) [70]; and anaemia from general population SGor from patient-derived TTO in Lloyd et al. 2008 [59].

Description of HTA-relevant HSUVs and disutilitiesOf the 36 publications, 13 provided HSUVs that meetthe NICE reference case or are considered acceptable tothe HTA agencies of interest [37–40, 42, 45, 46, 49, 53,56, 58, 64, 69], based on the measurement technique forgeneration of HSUVs, as outlined in Additional file 3:Figure S1. The main characteristics of these studies arepresented in Table 3. These 13 publications reporteddata from multinational studies [37–40, 45, 49, 64], andfrom Canada [42, 56], France/Germany [46], USA [58],Italy [53] and the UK [69]. In these studies, HRQoL wasmeasured using EQ-5D [37–40, 42, 45, 46, 49, 53, 56],EQ-VAS [37, 39, 40, 49] and SG [58, 64, 69]. The HTAsuitability of disutilities and decrements for AE healthstates in previously treated patients are reported in Table 4.

DiscussionEconomic evaluation, particularly cost–utility analysis,provides important information for guiding decision-making in health care, and its use in HTA is increasing

globally. Such evaluation includes examination of thetime spent in different disease states and uses an HSUVfor each disease state to calculate QALYs; HSUVs there-fore play a key role in economic evaluation. As summa-rized in Additional file 3: Figure S1, NICE, SMC,CADTH, HAS and PBAC prefer utilities to be estimatedusing a generic preference-based instrument, with healthstates described by patients through use of a question-naire, and with the health state valued using acountry-specific tariff that reflects societal preferences.As the aim of this systematic review was to evaluate theexperience of adults with previously treated mNSCLC,the synthesis of health state utility estimates was outsideits scope. However, the findings presented here may pro-vide a basis for generation of an accurate estimate of themean HSUV for use in economic evaluations [74, 75].This systematic review identified HSUVs relevant to

the experience of previously treated adult patients withmNSCLC. Search strings were designed to allow (dis)utilities from a broader population (including lung can-cer, advanced/metastatic cancer and specific metastasescommon in patients with lung cancer). In the absence ofsecond-line mNSCLC (dis) utilities, alternatives were se-lected with decreasing population specificity and rele-vance from first-line mNSCLC, NSCLC, lung cancer oradvanced/metastatic cancer, as outlined in Fig. 1. Order-ing the HSUVs by line of treatment reflects the practiceof switching treatment at progression. However, for thenewer immunotherapies, patients may remain on treat-ment post-progression, and their HRQoL may remain atpre-progression levels. Thus, HSUVs estimated for progres-sion status-specific health states from patients receivingchemotherapy may not be suitable to apply to the equiva-lent health states when patients receive immunotherapy.In total, the 36 identified articles reported 591 HSUVs

relevant to the experience of previously treated adult pa-tients with mNSCLC, and 11 of these studies reported atotal of 195 disutilities or decrements for AE healthstates that are relevant to the experience of patients withmNSCLC. The range of HSUVs identified for compar-able health states, such as progression-free/stable diseaseamong patients treated second-line for advanced/meta-static NSCLC [39, 45], highlights how differences instudy type, tariff, health state and the measures used candrive variation in HSUV estimates. For instance, disutil-ities for progression from a stable state were − 0.056 or− 0.065 using EQ-5D, [37] or − 0.1798 by general-popu-lation-derived SG. [69] To overcome such variations,where possible, HSUV studies should seek to use instru-ments, respondents and valuation populations that aremost acceptable to HTA bodies. However, there are in-stances where variation in methods can be justified. Forexample, disutility values derived from vignettes and ageneral public sample were used by Nafees et al. [69],

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 10 of 30

Page 11: Systematic review of health state utility values in

Table

3Health

stateutility

values

bytreatm

entline,he

alth

stateandinstrumen

t

Stud

yHealth

state

Utility

valuea

Instrumen

tTariff

Respon

dent

details

HTA

suitability

1stline

Nafees2016

[68]

Metastatic

NSC

LCb

PDvs

BLstate

0.095

TTO

N/A

Patients(but

notNSC

LCpatients)fro

mthege

neral

publicin

UK,Australia,France,

China,S.Korea,Taiwan

No

RESno

side

effectsvs

BL0.773

SDisno

side

effectsvs

BL0.460

Che

valier2013

c[38]

Advanced/metastatic

NSC

LC1LPF

0.69

(0.26)

EQ-5D

Fren

ch(TTO

)StageI–StageIII/IV

PFandPD

MeetsHASrequ

iremen

ts

1LPD

0.61

(0.24)

Cho

uaid

2013

[39]

Advanced/metastatic

NSC

LC1LPF

0.71

(0.24)

(95%

CI,

0.67–0.76)

EQ-5D-3

LUK

Attim

eof

advanced

diagno

sis,meanage

64.8years

Ade

nocarcinom

a:65.2%

Large-cellcarcinom

a:6.8%

SQcellcarcinom

a:17.1%

Other:9.9%

Clinicalstageat

timeof

survey:

IIIb:

17.9%

IV:82.1%

MeetsNICErequ

iremen

ts

69.31(18.33)(95%

CI,

65.9–72.8)

EQ-5DVA

SN/A

No

1LPD

0.67

(0.2)(95%

CI

0.59–0.75)

EQ-5D-3

LUK

MeetsNICErequ

iremen

ts

58.67(17.4)

(95%

CI

51.3–66.0)

EQ-5DVA

SN/A

No

Iyer

2013

[46]

Advanced/metastatic

NSC

LCFrance,G

ermany1L

0.63

(0.31)

EQ-5D

UK1

Patientswith

:Ade

nocarcinom

a:56.3%

Large-cellcarcinom

a:11.8%

SQcellcarcinom

a:29.3%

Other:2.5%

StageIIIb:

15.4%

Stage:IV

84.6%

MeetsNICErequ

iremen

t

60.8(19.9)

EQ-5DVA

SN/A

No

≥1stline

Iyer

2013

[46]

Advanced/metastatic

NSC

LCFrance,G

ermany1L/2L

0.58

(0.35)

EQ-5D

UK

Patientswith

:Ade

nocarcinom

a:56.3%

Large-cellcarcinom

a:11.8%

SQcellcarcinom

a:29.3%

Other:2.5%

StageIIIb:

15.4%

Stage:IV

84.6%

MeetsNICEandSM

Crequ

iremen

tsFrance,1

L/2L

0.57

(0.41)

Germany,1L/2L

0.59

(0.31)

France,G

ermany1L/2L

58.0(19.9)

EQ-5DVA

SNo

France,1

L/2L

57.1(21.1)

Germany,1L/2L

58.6(19.1)

2ndline

Blackhall2014[41]

Advanced/metastatic

ALK+NSC

LC

2LBL

CRZ

0.73

(0.24)

EQ-5D-3

LNR

Multin

ationalp

atients,locally

advanced

/metastatic

ALK+

NSC

LC,2

L

Unclear

astariffNR

2LBL

chem

othe

rapy

(PEM

orDOC)

0.70

(0.26)

2LBL

PEM

0.73

(0.24)

2LBL

DOC

0.67

(0.29)

2Lon

CRZ

0.82

(SE,0.01)

(95%

CI,0.79–0.85)

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 11 of 30

Page 12: Systematic review of health state utility values in

Table

3Health

stateutility

values

bytreatm

entline,he

alth

stateandinstrumen

t(Con

tinued)

Stud

yHealth

state

Utility

valuea

Instrumen

tTariff

Respon

dent

details

HTA

suitability

2Lon

Che

mothe

rapy

0.73

(SE,0.02)

(95%

CI,0.70–0.77)

2Lon

PEM

0.74

(SE,0.02)

(95%

CI,0.70–0.79)

2Lon

DOC

0.66

(SE,0.04)

(95%

CI,0.58–0.74)

Che

valier2013

c[38]

Advanced/metastatic

NSC

LC2LPF

0.70

(0.22)

EQ-5D

Fren

ch(TTO

)StageI–StageIII/IV

PFandPD

MeetsHASrequ

iremen

ts

2LPD

0.55

(0.35)

Cho

uaid

2013

[39]

Advanced/metastatic

NSC

LC2LPF

0.74

(0.18)

(95%

CI,0.68–0.80)

EQ-5D-3

LUK

Attim

eof

advanced

diagno

sis,meanage

64.8years

Ade

nocarcinom

a:65.2%

Large-cellcarcinom

a:6.8%

SQcellcarcinom

a:17.1%

Other:9.9%

Clinicalstageat

timeof

survey:

IIIb:

17.9%

IV:82.1%

MeetsNICErequ

iremen

ts

65.0(19.6)

(95%

CI,59.2–70.8)

EQ-5DVA

SN/A

No

2LPD

0.59

(0.34)

(95%

CI,0.42–0.77)

EQ-5D-3

LUK

MeetsNICErequ

iremen

ts

53.5(23.3)

(95%

CI,41.5–65.4)

EQ-5DVA

SN/A

No

Huang

2016

c[45]

AdvancedPD

-L1+

NSC

LC2LPF

0.76

(95%

CI,0.75–

0.77)

EQ-5D

NR

Multin

ationalp

atientswith

advanced

NSC

LCandPD

-L1+

tumou

rsin

2Lon

PEMBor

DOC,after

platinum

-based

chem

othe

rapy

Unclear

astariffNR

2LPD

0.69

(95%

CI,0.66–

0.71)

AdvancedPD

-L1+

NSC

LC,

2L,>

360days

from

death

0.81

(0.79,0.83)

Patientswith

advanced

NSC

LCandPD

-L1+

tumou

rsin

2Lon

PEMBor

DOC,after

platinum

-based

chem

othe

rapy

AdvancedPD

-L1+

NSC

LC,

2L,180–360days

from

death

0.73

(0.71,0.75)

AdvancedPD

-L1+

NSC

LC,

2L,90–180

days

from

death

0.69

(0.66,0.72)

AdvancedPD

-L1+

NSC

LC,

2L,30–90days

from

death

0.60

(0.56,0.64)

AdvancedPD

-L1+

NSC

LC,

2L,<

30days

from

death

0.40

(0.31,0.48)

Iyer

2013

[46]

Advanced/metastatic

NSC

LCOntreatm

ent:2Lon

ly0.53

(0.38)

EQ-5D

UK

Fren

chandGerman

patients

MeetsNICEandSM

Crequ

iremen

ts

54.9(19.3)

EQ-5DVA

SN/A

No

Lang

ley2013

[48]

StageIV

NSC

LCwith

brain

metastases

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

+WBRT0days

0.63

EQ-5D

NRd

UKandAustralianNSC

LCpatientswith

brain

metastases

No,as

VAStariffused

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

+WBRT28

days

0.49

NSC

LCwith

BM,p

reviou

stx

0.39

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 12 of 30

Page 13: Systematic review of health state utility values in

Table

3Health

stateutility

values

bytreatm

entline,he

alth

stateandinstrumen

t(Con

tinued)

Stud

yHealth

state

Utility

valuea

Instrumen

tTariff

Respon

dent

details

HTA

suitability

allowed

,OSC

+WBRT56

days

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

+WBRT

112days

0.36

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

+WBRT

168days

0.16

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

alon

e0days

0.60

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

alon

e28

days

0.49

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

alon

e56

days

0.44

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

alon

e112days

0.38

NSC

LCwith

BM,p

reviou

stx

allowed

,OSC

alon

e168days

0.36

Lloyd2008

[59]

Cancerwith

chem

othe

rapy-

relatedanaemiaor

fatig

ue

Anaem

ia,H

blevel,≥12.0

g/dL

0.708(95%

CI,0.057)

SGN/A

Gen

eralpu

blicsample

from

UK

No

0.611(95%

CI,0.112)

TTO

UKcancer

patientswho

have

recentlyexpe

rienced

chem

othe

rapy-related

fatig

ueandanaemiacompleting

vign

ette-based

TTO

MeetsNICE/SM

Crequ

iremen

tsbu

tstill

vign

ette-based

health

state

rather

than

patient

ratin

gow

nhe

alth

Nafees2008

[59]

mNSC

LC2LStablediseasee

0.65

(SE,0.02)

SGN/A

100mem

bersof

gene

ral

publicin

UK

No,bu

tused

inmultip

leHTA

subm

ission

s2LRespon

ding

diseasef

0.67

2LRespon

segain

0.02

(SE,0.01)

2LProg

ressivediseaseg

0.47

Novello

2015

[49]

StageIII/IV

recurren

tNSC

LC(SQandNSQ

)h

2LNIN

+DOC,b

efore

treatm

ent(week0)

0.72

EQ-5D

UK

Multin

ationalp

atientswith

stageIII/IV

recurren

tNSC

LC(SQandNSQ

)in2Lafter

chem

othe

rapy

Ade

nocarcinom

a:50.1%

MeetsNICE/SM

Crequ

iremen

ts

2LNIN

+DOC,after

treatm

ent(week30)

0.61

2LPLA+DOC,b

efore

treatm

ent(week0)

0.72

2LPLA+DOC,after

treatm

ent(week30)

0.62

2LNIN

+DOC,b

efore

treatm

ent(week0)

69.0

EQ-5DVA

SN/A

No

2LNIN

+DOC,after

treatm

ent(week30)

63.2

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 13 of 30

Page 14: Systematic review of health state utility values in

Table

3Health

stateutility

values

bytreatm

entline,he

alth

stateandinstrumen

t(Con

tinued)

Stud

yHealth

state

Utility

valuea

Instrumen

tTariff

Respon

dent

details

HTA

suitability

2LPLA+DOC,b

efore

treatm

ent(week0)

69.0

2LPLA+DOC,after

treatm

ent(week30)

63.1

Reck

2015

[50]

AdvancedSQ

NSC

LC2LNIVOat

BL0.68

(0.208)

EQ-5D

NR

Multin

ationalp

atientswith

advanced

SQNSC

LCUnclear

astariffNR

2LDOCat

BL0.66

(0.284)

2LNIVOat

BL63.7(18.2)

EQ-5DVA

SN/A

No

2LDOCat

BL66.3(20.5)

Rude

ll2016

c[57]

AdvancedNSC

LC,EGFR+

2LOSIat

BL65.2(20.33)

EQ-5D-5

LVA

SN/A

Multin

ationalp

atientswith

EGFR+advanced

NSC

LC,

2Lafterprevious

TKI

No

2LOSIat

36weeks

73.7(17.33)

Schu

ette

2012

[51]

NSC

LCStageIIIB–IV

2L,PEM

atBL

0.66

(0.256)

EQ-5D

UKTTO

AustrianandGerman

advanced

/mNSC

LC2L

patientsmainlyafterprior

platinum

treatm

ent

(IIIa,6.7%;IIIb,19.8%

;IV,73.5%

)

MeetsNICE/SM

Crequ

iremen

ts2L,PEM

at6weeks

(2nd

cycle)

0.02

(0.214)

EQ-5Dgain

2L,PEM

at6thcycle

0.11

(0.228)

2L,PEM

atBL

59.3(17.8)

EQ-5DVA

SN/A

No

2L,PEM

at6weeks

(2nd

cycle)

3.3(12.58)

EQ-5DVA

Sgain

N/A

2L,PEM

at6thcycle

12.8(17.62)

Vargas

2009

c[72]

AdvancedNSC

LC2L,on

ERL

0.81

GlobalQ

oLinde

xNR

Patientswith

advanced

NSC

LC,2

Lafterprevious

chem

othe

rapy

No

2L,on

taxane

s0.62

≥2n

dline

Che

n2010

c[73]

AdvancedNSC

LCd

2L,DOC,d

uringtreatm

ent

0.45

iSG

N/A

UKge

neralp

ublic

(as

algo

rithm

basedon

Nafees

2008

data

used

tocalculate

utilities)

Accep

tabledata

forSM

C

2L,DOC,after

treatm

ent

0.57

2L,PEM,d

uringtreatm

ent

0.54

2L,PEM,after

treatm

ent

0.59

3L,ERL,du

ringtreatm

ent

0.48

BSC,d

uringtreatm

ent

0.47

Che

valier2013

[38]

Advanced/metastatic

NSC

LC3/4LPF

0.61

(0.3)

EQ-5D

Fren

ch(TTO

)StageI–StageIII/IV

PFandPD

MeetsHASrequ

iremen

ts

3/4LPD

0.42

(0.40)

Griebsch

2014

[37]

StageIIIb(with

pleural

effusion

)/IV

NSC

LCaden

ocarcino

ma

(LUX-LU

NG1)j

Week4,prog

ressioneffect

long

itudinalm

odel

−0.1

EQ-5D

UK

Multin

ationaladvanced/

metastatic

NSC

LC,2

LLMeetsNICErequ

iremen

ts

Mixed

effect

long

itudinal

mod

elIRC

−0.056(95%

CI,

−0.083to

−0.028)

Mixed

effect

long

itudinal

mod

elIN

−0.065(95%

CI,

−0.092to

−0.039)

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 14 of 30

Page 15: Systematic review of health state utility values in

Table

3Health

stateutility

values

bytreatm

entline,he

alth

stateandinstrumen

t(Con

tinued)

Stud

yHealth

state

Utility

valuea

Instrumen

tTariff

Respon

dent

details

HTA

suitability

Mixed

effect

long

itudinal

mod

elIRC,A

FA−0.06

Mixed

effect

long

itudinal

mod

elIRC,BSC

−0.046

Mixed

effect

long

itudinal

mod

elIIN

V,AFA

−0.081

Mixed

effect

long

itudinal

mod

elIIN

V,BSC

−0.033

Week4,prog

ressioneffect

long

itudinalm

odel

−7.3

EQ-5DVA

SN/A

No

Mixed

effect

long

itudinal

mod

elIRC

−3.76

(95%

CI,−5.19

to−2.32)

Mixed

effect

long

itudinal

mod

elINV

−3.83

(95%

CI,−5.21

to−2.44)

Mixed

effect

long

itudinal

mod

elIRC,A

FA3.63

Mixed

effect

long

itudinal

mod

elIRC,BSC

−4.11

Mixed

effect

long

itudinal

mod

elINV,AFA

−4.42

Mixed

effect

long

itudinal

mod

elINV,BSC

−2.55

Hirsh2013

[40]

StageIIIB/IV

NSC

LC3LL

onAFA

+BSC

0.71

EQ-5D

UK

98%

aden

ocarcino

ma

PDfollowingtreatm

entlines

1–2,on

eof

which

was

platinum

based,

plus

PDafter

atleast12

weeks

ofERLor

GEF

MeetsNICErequ

iremen

ts

3LL

onPLA+BSC

0.67

3LL

onAFA

+BSC

67.4

EQ-5DVA

SN/A

No

3LL

onPLA+BSC

65.2

Schw

artzbe

rgc2015

[60]

StageIIIb/IV

NSC

LC(SQ

&NSQ

)

Allpatientswk6

1.0(21.7)

EQ-5DVA

SN/A

Patients,2LL,N

IVO3mg/kg

i.v.q

2wNo

wk12

5.8(21.3)

wk18

8.2(22.3)

wk24

8.2(23.9)

wk30

8.4(29.2)

SDiswk6

3.8(19.8)

wk12

6.4(21.9)

wk18

8.2(20.9)

wk24

5.2(21.9)

wk30

7.2(28.5)

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 15 of 30

Page 16: Systematic review of health state utility values in

Table

3Health

stateutility

values

bytreatm

entline,he

alth

stateandinstrumen

t(Con

tinued)

Stud

yHealth

state

Utility

valuea

Instrumen

tTariff

Respon

dent

details

HTA

suitability

PRwk6

7.3(22.4)

wk12

6.6(24.7)

wk18

8.1(27.6)

wk24

18.1(31.0)

wk30

13.7(38.2)

PDwk6

−5.8(21.1)

wk12

−3.0(19.8)

wk18

3.9(24.3)

wk24

6.8(12.2)

wk30

5.5(15.7)

Treatm

entlineno

tspecified

Bradbu

ry2008

c[42]

AdvancedNSC

LCOnERL

0.772

EQ-5D

NR(possibly

Canadian)

Canadianpatients

Potentially

relevant

toCADTH

OnBSC

0.754

Chang

2016

c[63]

AdvancedNSC

LC>360days

from

death

0.904

(95%

CI,0.892–0.917)

TTO

NR

Gen

eralpu

blic,Sou

thKo

rea

No

180–360days

from

death

0.720

(95%

CI,0.692–0.748)

90–180

days

from

death

0.627

(95%

CI,0.598–0.655)

30–90days

from

death

0.379

(95%

CI,0.349–0.409)

<30

days

from

death

0.195

(95%

CI,0.172–0.218)

Dansk

2016

c[43]

AdvancedNSC

LCSynthe

sizedPF

Med

ian,0.706

Rang

e,0.620–0.815

Synthe

sizedutility

across

>1

instrumen

ttype

NR

Utilities

synthe

sizedinclud

edthosewhe

rerespon

dents

werepatientsandthose

whe

rethey

werethege

neral

publicconsideringa

hypo

theticalhe

alth

state

No

Synthe

sizedPF

trial-b

ased

Med

ian,0.750

Rang

e,0.627–0.815

Synthe

sizedPF

non-trial-

based

Med

ian,0.653

Rang

e,0.620–0.653

Synthe

sizedPD

Med

ian,0.565

Rang

e,0.470–0.688

Synthe

sizedPD

trial-b

ased

Med

ian,0.599

Rang

e,0.550–0.688

Synthe

sizedPD

non-trial-

based

Med

ian,0.473

Rang

e,0.470–0.530

Doyle2008

[65]

Metastatic

NSC

LCSD

is,noadditio

nalsym

ptom

s0.626

SGN/A

Gen

eralpu

blic

No

Treatm

entrespon

se,no

0.712

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 16 of 30

Page 17: Systematic review of health state utility values in

Table

3Health

stateutility

values

bytreatm

entline,he

alth

stateandinstrumen

t(Con

tinued)

Stud

yHealth

state

Utility

valuea

Instrumen

tTariff

Respon

dent

details

HTA

suitability

additio

nalsym

ptom

s

Grunb

erg2009

c[58]

BC/LC

Che

mothe

rapy-in

duced

nausea

andvomiting

ofdifferin

gseverity

Repo

rted

graphically

SGN/A

PatientsBC

/LC

MeetsNICErequ

iremen

ts

Grutters2010

c[44]

NSC

LC(stage

unspecified

)NSC

LCwith

grade3+

dyspno

ea,stage

unspecified

Med

ian,0.52

EQ-5D-5

LNR

Patientsat

anearly

treatm

ent

stage

No

Jang

2010

[47]

StageIV

NSC

LCStageIV

NSC

LC0.75

(0.15)

EQ-5D

US

Patientswith

NSC

LCattend

ingamajor

Canadian

cancer

center

outpatient

clinic

No

Linn

et2015

c[62]

StageIV

NSC

LCon

oralVINO

PCS,cycle2

37.0

SF-12

N/A

Patients

No

PCS,cycle3

38.6

MCS

,cycle2

47.7

MCS

,cycle3

44.2

PCS,cycle2

52.9

Careg

ivers

Potentialtoestim

ateSF-6D

forcaregiversto

mNSC

LCpatients,forSM

Cor

CADTH

PCS,cycle3

53.4

MCS

,cycle2

46.2

MCS

,cycle3

44.6

Lloyd2005

c[66]

StageIV

NSC

LCRES

0.70

SGk

N/A

Gen

eralpu

blic

No

SDis,oraltreatmen

t0.63

SDis,i.v.treatmen

t0.58

PD0.42

Endof

life

0.33

Manser2006

[61]

StageIV

NSC

LCStageIV

Med

ian,0.68

(IQR,0.54–0.82)

AQoL

Australia

Mixed

stageen

rolled:

I,31.5%;II,17.4%;IIIa,16.3%

;IIIb,

7.6%

;IV,25.0%

No

Matza

2014

[67]

StageIV

cancer

with

bone

metastases

Cancerwith

bone

metastases

andno

SRE

0.47

(0.41)

TTO

N/A

Gen

eralpu

blic,U

K(Edinb

urgh

andLond

on)

No

0.47

(0.45)

Gen

eralpu

blic,C

anada

(Mon

trealand

Toronto)

0.47

(0.42)

Gen

eralpu

blic,U

Kand

Canada

Stew

art2015

[56]

EGFR+StageIV

NSC

LCPR/SDison

EGFR

TKIs

(GEF,ERL,A

ZD9291)

0.82

(SE,0.16)

EQ-5D-3

LNR

Patients,eligibleforor

onTKI

tx,55%

Asian,45%

male,

med

ianage60,66%

never

smokers.StageIV:

atdiagno

sis,80%

whe

nsurveyed

,100%

Unclear

Respon

dedto

standard

chem

othe

rapy

0.80

(SE,0.12)

EGFR+,respo

nded

toGEF

0.84

(SE,0.14)

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 17 of 30

Page 18: Systematic review of health state utility values in

Table

3Health

stateutility

values

bytreatm

entline,he

alth

stateandinstrumen

t(Con

tinued)

Stud

yHealth

state

Utility

valuea

Instrumen

tTariff

Respon

dent

details

HTA

suitability

EGFR+,respo

nded

toERL

0.82

(SE,0.17)

EGFR+,respo

nded

toAZD

9291

0.83

(SE,0.16)

EGFR+,PDdu

ringTKI

treatm

ent(GEF,ERL,

AZD

9291)

0.74

(SE,0.08)

EGFR+,allpatients(PR/SD

is/

PD),25%

3LL

0.802

Tabb

erer

2006

[52]

AdvancedNSC

LCRES

0.49

EQ-5D

NR

Gen

eralpu

blic,U

K(Cardiff,Glasgow

,Lon

don

andOxford)

No

SDis

0.46

SDis+oraltreatm

ent

0.45

SDis+i.v.treatmen

t0.43

PD0.22

Nearde

ath

0.15

Trippo

li2001

[53]

Metastatic

NSC

LCMetastatic

NSC

LC0.53

(0.36)

EQ-5D

UK(TTO

)Italianpatients

MeetsNICEandSM

Creferencecases

0.55

(0.22)l

EQ-5DVA

SN/A

No

Yang

2014

[54]

StageIIIB/IV

NSC

LCStageIV

inop

erable,

perfo

rmance

status

0–1

0.75

(0.22)

EQ-5D

Taiwan

Patients,mixed

NSC

LCstages:

I,0.8%

;II,0%

;IIIA,4.5%;

IIIB,16.9%;IV,77.8%

No

StageIV

inop

erable,

perfo

rmance

status

0–4

0.75

(0.22)

Yokoyama2013

c[55]

AdvancedNSC

LC/SCLC

StageIIIB/IV

NSC

LC/SCLC

with

bone

metastasisand

SRE

NR

EQ-5D

NR

Patients,advanced

NSC

LC,

72%,SCL

C,28%

NSC

LCandSC

LC:IIIB,37%

;IV,63%

No

a Mean,

ormean(SD)un

less

stated

othe

rwise

bVA

Sscores

werealso

repo

rted

inthisstud

ybu

tun

clearwhe

ther

thiswas

EQ-VAS

c The

sestud

ieswerepu

blishe

das

abstractsor

posters

dTh

isreferenced

article

(https://www.ncb

i.nlm

.nih.gov

/pub

med

/101

0980

1)isforaVA

Svaluation

e SDisvign

ette:

•Yo

uha

vealife-threaten

ingillne

ssthat

isstab

leon

treatm

ent.Yo

uarereceivingcycles

oftreatm

entthat

requ

ireyo

uto

goto

theou

tpatient

clinic

•Yo

uha

velost

weigh

t,an

dyo

urap

petiteisredu

ced.

Yousometim

esexpe

riencepa

inor

discom

fortin

your

chestor

unde

ryo

urrib

s,which

canbe

treatedwith

painkillers.Y

ouha

veshortnessof

breath,and

breathing

canbe

painful.Yo

uha

veape

rsistent

nagg

ingcoug

h•Yo

uareab

leto

washan

ddressyo

urselfan

ddo

jobs

arou

ndtheho

me.

Shop

ping

andda

ilyactiv

ities

take

moreeffortthan

usua

l•Yo

uareab

leto

visitfamily

andfriend

sbu

toftenha

veto

cutitshortbe

causeyo

uge

ttired

•Yo

usometim

esfeel

less

physically

attractiv

ethan

youused

to.Y

ourillne

ssha

saffected

your

sexdrive

•Yo

uworry

abou

tdy

ingan

dho

wyo

urlovedon

eswillcope

f Secon

d-linerespon

ding

vign

ette:

•Yo

uha

vealife-threaten

ingillne

ssthat

isrespon

ding

totreatm

ent.Yo

uarereceivingcycles

oftreatm

entwhich

requ

ireyo

uto

goto

theou

tpatient

clinic

•Yo

uarega

iningba

ckyo

urweigh

tan

dyo

urap

petiteisreturning.

Youoccasion

ally

expe

riencepa

inor

discom

fortin

your

chestor

unde

ryo

urrib

swhich

canbe

treatedwith

painkillers.Y

ousometim

esha

veshortness

ofbreath.Y

ouoccasion

ally

have

ana

ggingcoug

h•Yo

uareab

leto

washan

ddressyo

urselfan

ddo

jobs

arou

ndtheho

me.

Shop

ping

andda

ilyactiv

ities

cansometim

esbe

tiring

•Yo

uareab

leto

visitfamily

andfriend

sbu

tsometim

esha

veto

cutitshortbe

causeyo

uge

ttired

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 18 of 30

Page 19: Systematic review of health state utility values in

•Yo

uoccasion

ally

feel

less

physically

attractiv

ethan

youused

to.Y

ourillne

ssha

ssomew

hataffected

your

sexdrive

•Yo

usometim

esworry

abou

tdy

ingan

dho

wyo

urlovedon

eswillcope

gSecond

-line

PDvign

ette:

•Yo

uha

vealife-threaten

ingillne

ss,and

your

cond

ition

isge

ttingworse

•Yo

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hTh

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hasutilitie

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foralltreatmen

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althou

ghitisno

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inthepa

per

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taalso

repo

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from

LUX-LU

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lcou

ntry

values

arealso

availablein

thispu

blication

l Thisvalueisrepo

rted

asin

theoriginal

publication

Abb

reviations:1

Lfirst

line,

2Lsecond

line,

2LL

second

andsubseq

uent

line,3LL

third

andsubseq

uent

line,

AFA

afatinib,A

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aplasticlymph

omakina

semutationpo

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Assessm

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instrumen

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breast

cancer,B

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Can

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andTechno

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inHealth

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Europe

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visual

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HTA

health

techno

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assessmen

t,IQRinterqua

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cancer,M

CSmen

talcom

pone

ntsummary,mNSC

LCmetastatic

non-sm

all

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cancer,N

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ent-

specific,OSC

optim

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care,O

SIosim

ertin

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CSph

ysical

compo

nent

summary,PD

prog

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PEM

pemetrexed,

PFprog

ression-free,P

LAplaceb

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partialrespo

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PSSprog

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specific,

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ceevery2weeks,Q

oLqu

ality

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RESrespon

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all-celllun

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deviation,

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dard

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Short-Fo

rmHealth

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icines

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sortium,SQsqua

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Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 19 of 30

Page 20: Systematic review of health state utility values in

Table

4Disutilitiesandde

crem

entsforadverseeven

the

alth

states

inpatientswith

previouslytreatedmNSC

LC

Relevance

Autho

r,year,cou

ntry,

referencea

Instrumen

tand

respon

dent

Utility

type

Health

state/disutility

MeanHSU

V(SD)[SE]

{95%

CI}

HTA

suitability

Advanced/mNSC

LCNafees2008,U

K[69]

SG Gen

eralpu

blic

UID

StageIV

NSC

LC,2

L,stable

disease

Doe

sno

tmeetHTA

body

referencecase

asvign

ette-

basedutility

completed

byge

neralp

ublic

respon

dents.

Has

been

used

inmultip

leHTA

subm

ission

s,ho

wever.

Specifically

2LandUK,go

odsamplesize

(n=100)

and

measure

ofdispersion

available.

+Diarrho

ea0.61

+Fatig

ue0.58

+Febrile

neutrope

nia

0.56

+Hairloss

0.61

+Nausea/vomiting

0.61

+Neutrop

enia

0.56

+Rash

0.62

StageIV

NSC

LC,2

L,respon

ding

disease

+Diarrho

ea0.63

+Fatig

ue0.60

+Febrile

neutrope

nia

0.58

+Hairloss

0.63

+Nausea/vomiting

0.62

+Neutrop

enia

0.58

+Rash

0.64

DStageIV

NSC

LC,2

L,movingfro

mstableto

prog

ressive

−0.18

[0.022]

Neutrop

enia

−0.09

[0.015]

Febrile

neutrope

nia

−0.09

[0.016]

Fatig

ue−0.07

[0.018]

Nauseaandvomiting

−0.05

[0.016]

Diarrho

ea−0.05

[0.016]

Hairloss

−0.04

[0.015]

Rash

−0.03

[0.012]

Respon

segain

0.02

[0.007]

Tabb

erer

2006,U

K[52]

EQ-5D(tariff

NRbu

tlikelyUKTTOtariffas

UKsample)

Gen

eralpu

blic

DCom

paredwith

stable

disease(advancedNSC

LC,

lineno

tspecified

)

Not

suitableas

gene

ralp

ublic

respon

dents,lineof

treatm

entno

tspecified

,and

nomeasure

ofdispersion

repo

rted

.Goo

dsamplesize,

however

(n=154)

andNafees

etal.2008in

2Ldo

esno

t

Febrile

neutrope

nia

−0.27

Rash

−0.06

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 20 of 30

Page 21: Systematic review of health state utility values in

Table

4Disutilitiesandde

crem

entsforadverseeven

the

alth

states

inpatientswith

previouslytreatedmNSC

LC(Con

tinued)

Relevance

Autho

r,year,cou

ntry,

referencea

Instrumen

tand

respon

dent

Utility

type

Health

state/disutility

MeanHSU

V(SD)[SE]

{95%

CI}

HTA

suitability

providedisutilities

for

neurop

athy

orstom

atitisso

thesevalues

from

Tabb

erer

etal.m

aybe

thebe

stavailable.

Neuropathy

−0.15

Neutrop

enia

−0.14

Nausea

−0.14

Stom

atitis

−0.14

Diarrho

ea−0.13

Doyle2008,U

K[65]

SG Gen

eralpu

blic

UID

Metastatic

NSC

LC,lineno

tspecified

,SDisno

additio

nal

symptom

s

Doe

sno

tmeetreference

case

asge

neralp

ublic

respon

dents.How

ever,the

searetheon

lydisutilities

for

severe

symptom

sforcoug

h,dyspno

eaandpain

inmNSC

LC,soarebe

stop

tion

inspite

ofno

tmeetin

gHTA

derivationmetho

dpreferen

ces(n=101)

+Cou

gh0.58

+Dyspn

oea

0.58

+Pain

0.56

+Cou

gh,d

yspn

oeaandpain

0.46

DCou

gh−0.05

[0.011]

Dyspn

oea

−0.05

[0.012]

Pain

−0.07

[0.012]

Cou

gh,d

yspn

oeaandpainc

−0.17

b

Respon

ding

diseasegain

vsSD

is0.09

[0.015]

Aslineno

tspecified

,data

from

Nafeeset

al.2008

shou

ldbe

used

inpreferen

ce.

Hando

rf2012,U

SA[70]

Expe

rtop

inion

UID

StageIV

NSC

LCaden

ocarcino

ma(1

LSD

is)

+ne

utrope

nia

0.67

Doe

sno

tmeetreference

case

asexpe

rtop

inion-

derived

.ThisAEiscoveredby

Nafeeset

al.2008,which

uses

abe

tter

derivationmetho

dthan

expe

rtop

inion.

+pn

eumotho

rax

0.63

Doe

sno

tmeetreference

case

asexpe

rtop

inion-

derived

,but

thesearethe

onlyestim

ates

fortheseAE

health

states.SDisestim

ate

was

0.670(oralthe

rapy)and

0.653(i.v.chem

othe

rapy)for

disutility

calculation.

+haem

orrhage

0.63

+thrombo

cytope

nia

0.65

+thrombo

sis

0.56

Earlier

stageNSC

LC(curativeintent)

Grutters2010,cou

ntry

NR[44]

EQ-5D-5

L(tariff

NR)

Patients

UID

NSC

LC,curativeintent

stage,

lineno

tspecified

,grade

III+

dyspno

ea

0.52

(med

ian)

Patient-derived

EQ-5Dbu

ttariffandmeasure

ofdisper

sion

NR.Onlysource

ofgrade

III+dyspne

a.Utility

forNSC

LC

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 21 of 30

Page 22: Systematic review of health state utility values in

Table

4Disutilitiesandde

crem

entsforadverseeven

the

alth

states

inpatientswith

previouslytreatedmNSC

LC(Con

tinued)

Relevance

Autho

r,year,cou

ntry,

referencea

Instrumen

tand

respon

dent

Utility

type

Health

state/disutility

MeanHSU

V(SD)[SE]

{95%

CI}

HTA

suitability

patientswith

outdyspno

eain

thissamplewas

0.81,i.e.

disutility

−0.29

Advanced/mLC

(NSC

LC+SC

LC)

Yokoyama2013,Japan

[55]

EQ-5D(tariff

NR)

Patients

DStageIIIB/IV

NSC

LC/SCLC

with

bone

metastases+

skeletalrelatedeven

t(patho

logicfracture,radiation

orsurgeryto

bone

lesion

,spinalcord

compression

orhype

rcalcaem

ia)

−0.05

dProvides

NSC

LC/SCLC

(mixed

)patient-derived

EQ-5Dde

cre

men

tfor(m

ixed

)SREs.D

ata

fortheseAEs

arelim

ited,

soalthou

ghthisestim

ateisno

trobu

st(n=9andrespon

se%

low

at32%)itdo

esprovide

anindicatio

n.Novariability

measure

repo

rted

Breastcancer

and

lung

cancer

Grunb

erg2009,U

SA[58]

SG Patients

UID

Base

state:continuo

usnausea

andvomiting

0.53

fNafeeset

al.2008provide

data

fornausea

andvomiting

butifdifferent

levelsof

nausea

andvomiting

need

tobe

discerne

dthen

these

utilitiescanbe

considered

.Patient-derived

SGbu

tmixed

lung

/breastcancers.Goo

dsamplesize

(n=96)bu

tno

measure

ofdispersion

.Asthe

Cop

yright

feeforGrunb

erg

2009

was

high

,the

sedata

arerepo

rted

from

Shabarud

din2013

[79]

(aprevious

SRthat

extractedthegraphical

data

from

Grunb

erg2009)

Increm

ent

Limitednausea

andlim

itvomiting

vscontinuo

usnausea

andvomiting

+0.53

f

Increm

ent

Limitednausea

vscontinuo

usnausea

andvomiting

+0.55

f

Increm

ent

Limitedvomiting

vscontinuo

usnausea

and

vomiting

+0.50

f

AdvancedCancer

Matza

2014,U

K[67]

TTO

Gen

eralpu

blic

UStageIV

cancer

with

bone

metastases(noskeletal-

relatedeven

ts)

0.47

(0.41)

Doe

sno

tmeetreference

case

asge

neralp

opulation

respon

dents.How

ever,as

thereareno

alternative

utilitiesforbo

nemetastases

theseUKutilitiescouldbe

considered

forNICEor

SMC.

Goo

dsamplesize

(n=126),

SDavailable.

UID

+spinalcord

compression

with

outparalysis

0.25

(0.50)

+spinalcord

compression

with

paralysis

0.13

(0.49)

+fractureof

theleg

0.42

(0.41)

+fractureof

therib

0.44

(0.42)

+fractureof

thearm

0.44

(0.41)

+radiationtreatm

ent

(2weeks,5

appo

intm

ents/

week)

0.42

(0.42)

+radiationtreatm

ent

(2appo

intm

ents)

0.45

(0.41)

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 22 of 30

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Table

4Disutilitiesandde

crem

entsforadverseeven

the

alth

states

inpatientswith

previouslytreatedmNSC

LC(Con

tinued)

Relevance

Autho

r,year,cou

ntry,

referencea

Instrumen

tand

respon

dent

Utility

type

Health

state/disutility

MeanHSU

V(SD)[SE]

{95%

CI}

HTA

suitability

+surgeryto

stabilize

bone

0.40

(0.44)

Matza

2014,C

anada[67]

TTO

Gen

eralpu

blic

UStageIV

cancer

with

bone

metastases(noskeletal-

relatedeven

ts)

0.47

(0.45)

Doe

sno

tmeetreference

case

asge

neralp

opulation

respon

dents.How

ever,as

thereareno

alternative

utilitiesforbo

nemetastases

theseCanadianutilitiescould

beconsidered

forCADTH

.Reason

ablesamplesize

(n=61),SD

available.

UID

+spinalcord

compression

with

outparalysis

0.25

(0.54)

+spinalcord

compression

with

paralysis

0.19

(0.53)

+fractureof

theleg

0.40

(0.48)

+fractureof

therib

0.43

(0.47)

+fractureof

thearm

0.43

(0.48)

+radiationtreatm

ent

(2weeks,5

appo

intm

ents/

week)

0.41

(0.50)

+radiationtreatm

ent

(2appo

intm

ents)

0.45

(0.45)

+surgeryto

stabilize

bone

0.39

(0.50)

Matza

2014,U

Kand

Canada[67]

TTO

Gen

eralpu

blic

UStageIV

cancer

with

bone

metastases(noskeletal-

relatedeven

ts)

0.47

(0.42)

Doe

sno

tmeetreference

case

asge

neralp

opulation

respon

dents.How

ever,as

thereareno

alternative

utilitiesforbo

nemetastases

theseUK+

Canadianutilities

couldbe

considered

forNICE,

SMCor

CADTH

.Goo

dsample

size

(n=187),SDavailable.

UID

+spinalcord

compression

with

outparalysis

0.25

(0.21)

+spinalcord

compression

with

paralysis

0.15

(0.50)

+fractureof

theleg

0.41

(0.43)

+fractureof

therib

0.44

(0.43)

+fractureof

thearm

0.43

(0.43)

+radiationtreatm

ent

(2weeks,5

appo

intm

ents/

week)

0.41

(0.45)

+radiationtreatm

ent

(2appo

intm

ents)

0.45

(0.42)

+surgeryto

stabilize

bone

0.40

(0.46)

Matza

2014,U

K[67]

TTO

Gen

eralpu

blic

DStageIV

cancer

with

bone

metastases

Asabove

+spinalcord

compression

with

outparalysis

−0.22

(0.31)

+spinalcord

compression

−0.34

(0.36)

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 23 of 30

Page 24: Systematic review of health state utility values in

Table

4Disutilitiesandde

crem

entsforadverseeven

the

alth

states

inpatientswith

previouslytreatedmNSC

LC(Con

tinued)

Relevance

Autho

r,year,cou

ntry,

referencea

Instrumen

tand

respon

dent

Utility

type

Health

state/disutility

MeanHSU

V(SD)[SE]

{95%

CI}

HTA

suitability

with

paralysis

+fractureof

theleg

−0.05

(0.09)

+fractureof

therib

−0.03

(0.08)

+fractureof

thearm

−0.03

(0.07)

+radiationtreatm

ent

(2weeks,5

appo

intm

ents/

week)

−0.05

(0.12)

+radiationtreatm

ent

(2appo

intm

ents)

−0.02

(0.07)

+surgeryto

stabilize

bone

−0.07

(0.17)

Matza

2014,C

anada[67]

TTO

Gen

eralpu

blic

DStageIV

cancer

with

bone

metastases

Asabove

+spinalcord

compression

with

outparalysis

−0.22

(0.32)

+spinalcord

compression

with

paralysis

−0.28

(0.30)

+fractureof

theleg

−0.07

(0.19)

+fractureof

therib

−0.04

(0.17)

+fractureof

thearm

−0.04

(0.07)

+radiationtreatm

ent

(2weeks,5

appo

intm

ents/

week)

−0.06

(0.21)

+radiationtreatm

ent

(2appo

intm

ents)

−0.02

(0.11)

+surgeryto

stabilize

bone

−0.08

(0.21)

Matza

2014,U

Kand

Canada[67]

TTO

Gen

eralpu

blic

DStageIV

cancer

with

bone

metastases

Asabove

+spinalcord

compression

with

outparalysis

−0.22

(0.31)

+spinalcord

compression

with

paralysis

−0.32

(0.34)

+fractureof

theleg

−0.06

(0.13)

+fractureof

therib

−0.03

(0.12)

+fractureof

thearm

−0.04

(0.11)

+radiationtreatm

ent

(2weeks,5

appo

intm

ents/

week)

−0.06

(0.15)

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 24 of 30

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Table

4Disutilitiesandde

crem

entsforadverseeven

the

alth

states

inpatientswith

previouslytreatedmNSC

LC(Con

tinued)

Relevance

Autho

r,year,cou

ntry,

referencea

Instrumen

tand

respon

dent

Utility

type

Health

state/disutility

MeanHSU

V(SD)[SE]

{95%

CI}

HTA

suitability

+radiationtreatm

ent

(2appo

intm

ents)

−0.02

(0.08)

+surgeryto

stabilize

bone

−0.07

(0.18)

Cancer,un

clearstage

Lloyd2008,U

K[59]

SG Gen

eralpu

blic

UID

Anaem

iaassociated

with

cancer

treatm

ent

Doe

sno

tmeetreference

case

asge

neralp

opulation

samplerespon

dent

forSG

exercise.

Haemog

lobinlevel(g/dL)

7.0–8.0

0.58

{0.067}

8.0–9.0

0.61

{0.064}

9.0–10.0

0.64

{0.060}

10.0–10.5

0.64

{0.062}

10.5–11.0

0.66

{0.061}

11.0–12.0

0.70

{0.056}

>12.0

0.71

{0.057}

VAS

Gen

eralpu

blic

UID

Haemog

lobinlevel(g/dL)

7.0–8.0

16.9{2.6}

8.0–9.0

22.3{3.0}

9.0–10.0

27.6{2.9}

10.0–10.5

32.9{3.4}

10.5–11.0

38.8{3.6}

11.0–12.0

45.9{4.2}

>12.0

51.2{4.3}

TTO

Cancerpatientswith

recent

expe

rienceof

chem

othe

rapy-related

anaemiaor

fatig

ue

UID

Haemog

lobinlevel(g/dL)

7.0–8.0

0.30

{0.127}

8.0–9.0

0.36

{0.126}

9.0–10.0

0.41

{0.125}

10.0–10.5

0.45

{0.122}

10.5–11.0

0.45

{0.111}

11.0–12.0

0.55

{0.105}

>12.0

0.61

{0.112}

UOwncurren

the

alth

0.85

{0.034}

EQ-5Dcurren

the

alth

0.87

{0.076}

VAS

Cancerpatientswith

recent

expe

rienceof

chem

othe

rapy-related

anaemiaor

fatig

ue

UID

Haemog

lobinlevel(g/dL)

7.0–8.0

21.7{5.7}

8.0–9.0

32.4{6.6}

9.0–10.0

34.2{6.7}

10.0–10.5

41.9{6.6}

10.5–11.0

44.7{6.6}

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 25 of 30

Page 26: Systematic review of health state utility values in

Table

4Disutilitiesandde

crem

entsforadverseeven

the

alth

states

inpatientswith

previouslytreatedmNSC

LC(Con

tinued)

Relevance

Autho

r,year,cou

ntry,

referencea

Instrumen

tand

respon

dent

Utility

type

Health

state/disutility

MeanHSU

V(SD)[SE]

{95%

CI}

HTA

suitability

11.0–12.0

52.2{6.8}

>12.0

62.4{7.9}

UOwncurren

the

alth

87.6{4.9}

EQ-5Dcurren

the

alth

84.2{4.57}

NR

Westw

ood2014,N

R[71]

NR

DAnaem

ia0.073[0.018]

Disutilitiesforanaemiaand

treatm

entmod

ehave

been

used

inprevious

NICE

subm

ission

s.How

ever,the

reisno

inform

ationconcerning

theirde

rivation.

2LNSC

LCOralthe

rapy

(ERL)

0.014[0.012]

2LNSC

LCi.v.the

rapy

0.043[0.020]

Patientswith

out

NSC

LC1Lsetting

Nafees2016,

Multin

ationaland

UK

[68]g

TTO

Patients(but

not

NSC

LCpatients)fro

mthege

neralp

ublic

inUK,Australia,France,

China,S.Korea,

Taiwan

DBleeding

vsBL

(stableno

side

effects)

−0.25

No

Hypertensionvs

BL(stableno

side

effects)

−0.03

UID

Respon

ding

+bleeding

vsBL

0.534

Respon

ding

+hype

rten

sion

vsBL

0.749

Stable+bleeding

vsBL

0.508

Stable+hype

rten

sion

vsBL

0.729

a Som

estud

iesiden

tifiedin

thissystem

aticreview

wereno

tinclud

edin

thistableforthefollowingreason

s:Grunb

erg20

09[58]

andGrutters20

10[44]

didno

trepo

rtvalues

bTh

eita

licsindicate

acalculated

utility.Thisiscalculated

from

thevalues

repo

rted

inDoy

le20

08[65]

Table3(differen

cebe

tweenstab

lediseaseno

othe

rsymptom

san

dstab

lediseasewith

coug

h,dy

spno

eaan

dpa

in,

toob

tain

thedisutility

).Itisno

tcalculated

byad

ding

thedisutilities,asthiswou

ldno

tbe

valid

c Value

swerecalculated

from

‘SDis+coug

h,dy

spne

aan

dpa

in’u

tility

minus

‘SDisno

additio

nalsym

ptom

s’utility

dTh

estud

ydidno

tindicate

ifmeanor

med

ian

e Som

estud

iesiden

tifiedin

thissystem

aticreview

wereno

tinclud

edin

thistableforthefollowingreason

s:Grunb

erg20

09[58]

andGrutters20

10[44]

didno

trepo

rtvalues,and

Nafees20

16[68]

repo

rted

varia

tionin

twode

crem

ents

(bleed

ing,

hype

rten

sion

)ba

sedon

thedifferen

tpo

pulatio

nsin

which

valuationwas

unde

rtaken

f Asrepo

rted

inSh

abarrudd

in20

13[79],b

asestatean

dutility

increm

ents

werepresen

tedon

differen

tscales:b

asestatewas

basedon

stan

dard

gamblescalebe

tweenpe

rfecthe

alth

(arbitraryscoreof

100)

orim

med

iate

death(arbitraryscoreof

0)while

theutility

increm

ents

wereba

sedon

ascalebe

tweenpe

rfecthe

alth

(arbitraryscoreof

100)

andthesurrog

atene

gativ

ean

chor

ofcontinuo

usna

usea/vom

iting

(re-setto

anarbitraryscoreof

0)gVa

lues

arepresen

tedforglob

alpo

pulatio

n(UnitedKing

dom,A

ustralia,France,

China

,Taiwan

,Korea).Notethat

coun

try-specificda

taarealso

available

Abb

reviations:1

Lfirst

line,

BLba

selin

e,CI

confiden

ceinterval,D

decrem

ent,ERLerlotin

ib,H

SUVhe

alth

stateutility

value,

HTA

health

techno

logy

assessmen

t,i.v.intraveno

us,LClung

cancer,m

LCmetastatic

lung

cancer,

mNSC

LCmetastatic

non-sm

allcelllun

gcancer,N

ICENationa

lInstituteforHealth

andCareExcellence,

NRno

trepo

rted

,NSC

LCno

n-sm

allcelllun

gcancer,SCLCsm

allcelllun

gcancer,SDstan

dard

deviation,

SEstan

dard

error,SG

stan

dard

gamble,

TTOtim

etrad

e-off,Uutility,U

IDutility

incorporatingde

crem

entforad

verseeven

ts,V

ASvisual

analog

uescale

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 26 of 30

Page 27: Systematic review of health state utility values in

because asking patients suffering such toxicities tocomplete HRQoL questionnaires was considered to betoo burdensome for patients and potentially unethical.Moreover, although the variation may be large, it helpsdecision makers to identify where variability exists andinforms the design of sensitivity analyses.In the 36 publications identified, 13 provided

HSUVs that meet the NICE reference case or areconsidered acceptable to the HTA agencies of interest[37–40, 42, 45, 46, 49, 53, 56, 58, 64, 69]. These weredeemed suitable because HRQoL was measured using theEQ-5D [37–40, 42, 45, 46, 49, 53, 56] or SG [58, 64, 69],both measures preferred or accepted by several HTA au-thorities. This endeavour fills an important gap in the fieldbecause hitherto, only two reports had described HSUVsin mNSCLC [68, 69]; neither was a systematic review ofthe literature, nor did they assess their appropriateness foruse in economic evaluations.This systematic review did not identify an HSUV

report based on data from the OAK trial(NCT02008227), because it was published as a congressabstract after the cut-off date for literature searching[76]. However, the HSUVs are relevant to the aims ofthis systematic review, and a brief description isprovided below for completeness. Patients with locallyadvanced NSCLC or mNSCLC after failure ofplatinum-containing chemotherapy were randomized ina phase 3 trial to receive atezolizumab or docetaxel [76,77]. As part of the trial, patients completed the EQ-5D,and the resultant HSUVs were presented by time pointbefore death. This study is similar to Huang et al. 2016,which also presented time-to-death EQ-5D utilities fora similar patient group receiving immunotherapy, ex-cept comparing pembrolizumab and docetaxel [45].Overall, HSUVs were very similar between studies atapproximately equivalent time points. In the OAKstudy, the following HSUVs were reported by timepoint before death: 0.77 (> 210 days), 0.71 (105–210 days), 0.61 (35–105 days) and 0.39 (< 35 days). Forcomparison, HSUVs published by Huang et al. 2016were 0.73 (180–360 days), 0.69 (90–180 days), 0.60(30–90 days) and 0.40 (< 30 days). A further studyevaluating the efficacy of immunotherapy in patientswith NSCLC showed that baseline mean EQ-VAS andEQ-5D index scores were similar for nivolumab (63.7and 0.68, respectively) and docetaxel (66.3 and 0.66,respectively) [50].Strengths of this systematic review include the wide

range of data sources searched and the search stringdesign, which enabled identification of disutilities andutility decrements for a wide range of AEs and progres-sive disease states (e.g. common sites of metastasis fromlung cancer) of relevance to the experience of patientspreviously treated for mNSCLC. We have presented

HSUVs by line of treatment, allowing use in economicmodelling, and have discussed HSUVs likely to be ac-cepted by the HTA bodies of interest. Inadequate or in-consistent reporting is common, and low sample sizesand response rates considerably impact on the reportedconfidence intervals of the reported results. However,among the studies identified here, most reported samplesize (over 100 respondents in most cases), many pro-vided a measure of variability for the values reported,and several were based on response rates greater than80% (although response rates were unreported in morethan half of the studies). Moreover, the use of only pub-lished HSUVs can be a limitation, as HTA submissionsmay use HSUVs that have not been previously published.As part of this systematic review, we have thereforesearched HTA submissions for any relevant utilities;most HTAs use data reported by Nafees et al. [69]Limitations of this review include that the label for the

upper bound of the utility scale (e.g. “full health” or “per-fect health”) was not recorded. This has been shown tobe a significant predictor of utility in lung cancer [78],so variation in utilities due to a different upper boundlabel cannot be explored. A further limitation concernsdata extraction from some studies presented as congressabstracts or posters. Owing to the word restrictionsplaced on conference proceedings they may not be con-sidered a robust data source in comparison with fullpublications. Furthermore, both screening and dataextraction were conducted primarily by a single re-viewer, and only 50% of studies were checked by asecond reviewer. The exclusion of studies that usedmapping to derive EQ-5D and utility values is a fur-ther limitation of this study; however, sufficient dataobtained through direct measurement were identifiedto be informative.

ConclusionsThis systematic review begins to address the challengeof identifying reliable estimates of utility values inmNSCLC that are suitable for use in economic evalua-tions. Our work has also highlighted that these estimatesare vulnerable to variations in study type, tariff, healthstate and the measures used, and that shortcomings inreporting are common.

Additional files

Additional file 1: Table S1. Search strings. (DOCX 90 kb)

Additional file 2: Table S2. Listing of first-line mNSCLC studies withutility data excluded at second pass [80–100]. (DOCX 84 kb)

Additional file 3: Figure S1. Hierarchy of preferred methodology forgeneration of HSUVs for different HTA agencies. (PDF 1172 kb)

Additional file 4: Table S3. Quality assessment of identified studies.(DOCX 111 kb)

Paracha et al. Health and Quality of Life Outcomes (2018) 16:179 Page 27 of 30

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AbbreviationsAE: Adverse event; AQoL: Assessment of Quality of Life instrument;CADTH: Canadian Agency for Drugs and Technologies in Health; HAS: HauteAutorité de Santé; HRQoL: Health-related quality of life; HSUV: Health stateutility value; HTA: Health technology assessment; mNSCLC: Metastatic non-small cell lung cancer; NICE: National Institute for Health and Care Excellence;NSCLC: Non-small cell lung cancer; PBAC: Australian Pharmaceutical BenefitsAdvisory Committee; PICOS: Patient, intervention, comparator, outcome,study; PRISMA: Preferred Reporting Items for Systematic Reviews and Meta-Analyses; QALY: Quality-adjusted life-year; RECIST: Response EvaluationCriteria in Solid Tumors; SF-12/SF-36: 12-/36-item Short-Form Health Survey;SF-6D: 6-dimension Short-Form Health Survey; SG: Standard gamble;SMC: Scottish Medicines Consortium; TTO: Time trade-off; VAS: Visualanalogue scale

AcknowledgementsMedical writing support was provided by Christian Eichinger ofPharmaGenesis London, London, UK and was funded by Roche.

Availability of data and materialsNot applicable. The data presented in this systematic review are publishedand can be found in the cited original references.

DisclosuresNP is an employee of F. Hoffmann-La Roche. AA was an employee of F.Hoffman-La Roche at the time the study was conducted. KSM is anemployee of Epidemica Ltd.

Authors’ contributionsNP, AA and KSM carried out the conception and design, participated in thereview and critique process, drafted the manuscript, and revised it criticallyfor intellectual content. The systematic review was carried out by KSM fromEpidemica Ltd., UK and was funded by Roche. All authors read and approvedthe final manuscript.

Ethics approval and consent to participateNot applicable.

Consent for publicationNot applicable.

Competing interestsThe authors declare that they have no competing interests.

Publisher’s NoteSpringer Nature remains neutral with regard to jurisdictional claims inpublished maps and institutional affiliations.

Author details1F. Hoffmann-La Roche AG, Basel, Switzerland. 2Epidemica Ltd, Bicester,Oxfordshire, UK. 3Present address: Digipharm, Zug, Switzerland.

Received: 8 February 2018 Accepted: 8 August 2018

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