ethical issues and hta

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Improving the Value of Patient Submissions on Drug Review Processes Durhane Wong-Rieger, PhD Devidas Menon, PhD Tania Stafinski, PhD Gabriel Wong-Rieger, BCom

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Improving the Value of Patient Submissions on Drug Review Processes.

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Page 1: Ethical issues and HTA

Improving the Value of Patient

Submissions on Drug Review Processes

Durhane Wong-Rieger, PhDDevidas Menon, PhD

Tania Stafinski, PhDGabriel Wong-Rieger, BCom

Page 2: Ethical issues and HTA

Purpose: How to Promote the Value of Patient Submissions

Challenge that HTA (value of therapy) did not sufficiently account for patient impact Concern: HTA (as applied to the public drug review

processes in Canada) do not adequately reflect the value of the therapy to patients

HTA theoretically includes societal values but evident

Need for direct patient input Beyond Quality of Life scales (does not sufficiently

capture all of the value to patients; quantitative summary does not convey patient perspective)

Patient is best positioned to provide experience of disease, previous therapy, new therapy

Page 3: Ethical issues and HTA

Introduction of Patient Submissions

Introduced by CADTH, Ontario, and BC (process in Quebec)

Opportunity for patients and patient representatives to provide direct input, in their own worlds

In most cases, will be presented by the patient/pubic member on the HTA committee (accurately reflect, interpret and advocate for the patient perspective)

Challenge: How does committee integrate patient input in HTA process?

Page 4: Ethical issues and HTA

Purpose of Workshop Sessions

Purpose: Provide Insight on Patient Submissions and Influence on HTA process

Questions AddressedGiven all of the data available to HTA, what

is perceived value of direct information from patients on impact of disease and therapy

What are ways in which patient data could be meaningfully considered within HTA processes?

Does the process (group deliberation) for considering patient data affect the outcome (decision on value of therapy)

Page 5: Ethical issues and HTA

Objectives of Sessions

2 workshops (Ontario and BC) with patients and other stakeholders

Objectives Learn about patient submission process: what, how. why

Learn from patient/public members of HTA committees on perceived value and process for considering patient input

Gain insights on HTA/drug review process through simulation

Compare methods for considering patient input as part of HTA/drug review process

Learn factors that potentially influence drug review

Page 6: Ethical issues and HTA

Participants

Two SessionsOntario: 90 (50% or 45 patients)BC: 75 (65% or 49 patients)

Patient representativesPatients, patient advocates, board, and staffPrevious participation in HTA training

Some experienced in making submissions to CADTH or CED

Page 7: Ethical issues and HTA

Session Agenda (Intro)

Pre-assessment: knowledge and trust of agencies

Introduction to HTA and Committees Overview of HTA Overview of CADTH (Common Drug Review) and

Patient Submission Process

Overview of provincial drug review process

Perspectives of patient/public members CADTH (CEDAC) Ontario Committee to Evaluate Drugs Patient organizations

Page 8: Ethical issues and HTA

Small Group Simulation of Drug Review Committee

Each group reviews 3 of 4 drugs for different conditions Disease (health condition) and drug information, including

safety and efficacy data, size of patient population Cost-effectiveness data, including alternative treatment

May only recommend 2 of 3 drugs Without patient input, make Initial group decision on

whether to recommend drug for funding Question: what additional patient information do you

want? Provided “patient submission” on impact of condition,

current therapy, proposed drug impact Provided with 3 of 4 formats for considering the patient

submission along with rest of data

Page 9: Ethical issues and HTA

Drugs and Condition Information

A: Progressive debilitating; affects 2 in 1000 Current: injection every 4 weeks; reduces relapses,

slows progression, mild to severe side effects New: Injection every 4 weeks, reduces relapses by

half; 1% severe disability or death; $20K/year; $68K/QALY

B: Genetic, auto-inflammatory; affects 1 in 100,000; debilitating, mental retardation, vision loss, deformities No approved therapy but RA therapy used off-label,

daily injection. New drug injected every 6 weeks; nonlife-threatening

side effects, $96K/year; $110K/QALY

Page 10: Ethical issues and HTA

Drugs and Condition Information

C: Blood cancer, affects 1 in 20,000 Current: IV chemotherapy, 2-year survival at 25% New: oral; 3 single-arm CTs = 2-year survival at 93%;

1% risk liver toxicity; life-long therapy $30K/year; $20K/QALY

D: Obesity, affects 25% Canadians, linked to diabetes, CVD, cancers New drug: oral, twice daily. RCT loss of 8kg more than

placebo; reduced metabolic CDV risks; 25% psychiatric events with 0.02% suicides; on treatment until healthy weight; $3K/year, $60K/QALY

Page 11: Ethical issues and HTA

Group Decision-Making Formats

Each group assigned Open Discussion + 2 other formats

Open Discussion: Individuals freely share opinions, beliefs, and preferences to arrive at a group decision. Pro’s: Equal voice, relevant attributes specific to current case,

unrestricted conversation

Con’s: Leader/champion influence, lack of prioritization or systematic consideration of all attributes

Nominal Group Technique: Individuals formulate and express own opinions prior to influence from others Pro’s: Initial personal opinion shared; al options considered; identify

and discuss personal biases

Con’s: Support own “uninformed” opinion; polarization of views

Page 12: Ethical issues and HTA

Group Decision-Making Formats

Each group assigned Open Discussion + 2 other formats

Dialectical Inquiry/Deliberative Dialogue: All aspects of “funding or not funding” explored prior to making decision. Pro’s: Attributes relevant to key stakeholders strongly positioned

Con’s: Arriving at consensus; need for representative views

Simple Multi-attribute Rating Technique: Systematically analyze the attributes of the drug and arrive at a decision based on a “weighted” assessment of the criteria Pro’s: Consistent and systematic approach across drugs

Con’s: Restricted discussion of perspectives; too reductionist

Page 13: Ethical issues and HTA

Patient Submission Summaries

Drug A for Progressive neuromuscular Impact: Devastating, severe attacks, relapses Treatment outcomes desired: reduce severity,

frequency of attacks; less severe side effects Drug characteristics: small fatal risk acceptable

Drug B for Rare Genetic Disorder Impact: Fever, pain, joint and tissue deterioration Treatment outcomes desired: short-term reduction of

flare-ups, fever, pain; long-term damage reduction; Drug characteristics: fewer injections

Page 14: Ethical issues and HTA

Patient Submission Summaries

Drug C for Blood Cancer Impact: devastating, low survival, helpless, hopeless Treatment outcomes desired: survival, reduced blood

counts Drug characteristics: oral, nausea manageable

Drug D for Obesity Impact: Physical and psychological, low self-esteem Treatment outcomes desired: sustainable weight loss

which is very difficult to attain Drug characteristics: most effective, least risky

options; immediate and steady weight loss

Page 15: Ethical issues and HTA

Results: Group Decisions

Total 19 groups and 57 decisions Each group: may approve maximum 2 of 3 drugs Groups segregated as patients and non-patients

Small difference in recommendations from patients and non-patients Patients: approved 67% drugs (2 out of 3) Non-patients approved 59% (fewer than allowed)

Small difference between Ontario and BC groups BC: 71% approval (1 group approved all 3 drugs) Ontario: 58% (2 groups approved only 1 drug)

Page 16: Ethical issues and HTA

Results by Condition and Drug

Approvals (all groups combined)

Drug C (Blood Cancer) approved by 100% of groups

Drug B (Rare Genetic) approved by 86% of groups

Drug A (Progressive) approved by 67% of groups

Drug D (Obesity) approved by 0% of groups

Page 17: Ethical issues and HTA

Rationale for Drug Decisions

Important Rationale

Drug C: Increased chances for survival; everyone relates to cancer; acceptable $/QALY

Drug B: No other treatments; no proof of long-term benefit; small numbers; approve despite very high $/QALY

Drug A: Reduced relapses but no impact on progression; severe disease; high $/QALY

Drug D: Not significant benefit; no direct disease impact; should not be public funding

Page 18: Ethical issues and HTA

Approvals by Discussion Format

No difference in Approvals by Discussion Format

Multi-Attribute Rating: 67% Approvals

Open Discussion: 63% Approvals

Nominal Group: 64% Approvals

Dialectic Dialogue: 58% Approvals

Page 19: Ethical issues and HTA

Approvals by Drug and Discussion Format

Progressive

Debilitating

Rare Geneti

c

Blood Cancer

Obesity Overall

Open Discussion

60% 80% 100% 0% 63%

Nominal Group 50% 100% 100% 0% 64%

Mutli-Attribute Rating

75% 75% 100% 0% 67%

Dialectic Dialogue 75% 100% 100% 0% 58%

OVERALL 67% 86% 100% 0% 63%

Page 20: Ethical issues and HTA

Frequency of Decision Reasons

For positive decisions, frequently cited: Safety (lack of or manageable side effects) Cost, cost-effectiveness, or cost mitigated by other

benefits Meeting patient needs and survival benefits Quality of life; lack of alternatives

For negative decisions, frequently cited: Lack of comparator (placebo only) Lack of adequate information Risk of side effects Lack of sustainability and long-term outcomes Lack of cost-effectives (cited by only 1/3)

Page 21: Ethical issues and HTA

Average Number Reasons Cited by Variable

Variable Avg. # Reasons

Discussion Format Open 2.2

Nominal Group 2.5

Multi-Attribute Rating 1.6

Deliberate Dialogue 1.4

Drug Progressive Disease 2.1

Rare Genetic 1.4

Cancer 2.1

Obesity 2.3

Decision Yes 1.9

No 2.1

All 2.0

Page 22: Ethical issues and HTA

Summary—Number of Reasons

By decision, more reasons cited for “no” (2.1) than for “yes” (1.9)

By deliberation format, fewer reasons with more structured group format (multi-attribute (1.6) and deliberate dialogue (1.4)) than with more open discussions (open format (2.3) and nominal group technique 2.5))

By type of drug, fewest reasons given for rare genetic drug (1.4) than for all others (2.1 – 2.3)

Page 23: Ethical issues and HTA

Preferences for Discussion Format

Preferences for Discussion Formats

MA Rating: best liked; easy to do; sorted options for easy decision making

Open: Usual process; best when high consensus; leader may set direction

Nominal: Assured all opinions heard; allowed differences to surface

Dialectic: Difficult to “role play”; helpful in hearing patient perspective

Page 24: Ethical issues and HTA

Advocare Simulation: Patient Input Shifts Discussion

Groups felt patient information was very important for conditions that were unfamiliar to them, specifically the progressive debilitating disease and the rare genetic condition.

Information about the patient experience of the challenges of the conditions were cited as important, as was information about the current therapies, or lack thereof.

Prior to receiving patient information, groups said they were more focused on cost, cost per QALY, and affordability.

Introduction of patient submission focused discussion, regardless of the format, on the patient’s perception of the burden of the condition, the challenges of current therapy, and the potential benefit on hope, manageability, and quality of life.

Page 25: Ethical issues and HTA

Advocare Simulation: How Does Patient Input Impact Discussion?

Groups reported that the patient submission helped them to visualize the patient, see him/her as a person rather than statistic

The “lack of concern” about potentially fatal (but very rare) side effect was important

Risk of imminent death (blood cancer) was a significant factor and had greater weight than debilitating condition. Drug C for blood cancer always chosen regardless of alternatives.

Slowing progression was not as important as reducing risk of developing organ, joint, tissue damage. Drug A for progressive condition NOT chosen if alternatives were B (genetic disorder) AND cancer.

Page 26: Ethical issues and HTA

Advocare Simulation: Findings of Patient Submission Workshops

Direct patient input (submissions) enhance discussion of patient impact and consideration of patient values and preferences

Cost factors (individual cost/patient and $/QALY) are important factors but can be superseded by patient impact (especially if rare disease or not other treatments)

Survival (even for limited time) given more weight than reducing risk of future serious organ/joint damage and reducing progression of debilitating disease

Groups liked rating tool; easy way to integrate variety of factors and rank options.

Mutli-attribute Rating technique also resulted in the highest percentage of approvals overall

Drug fo common condition (obesity) not given priority over serious diseases.

Page 27: Ethical issues and HTA

Patient Trust in Drug Review Agencies Pre-and Post-Workshop

Trust knowledgeable and fair process by… (agree somewhat and stronglY

Ontario Pre-

OntarioPost

BCPre

BCPost

Health Canada approving drug 90% 100% 95% 92%

CADTH (Common Drug Review) recommending 65% 84% 43% 65%

Provincial Drug Program funding drug 55% 68% 45% 425

Physician recommending drug 90% 74% 95% 92%

Patient Group informing re: drug and options 90% 100% 100% 92%

Page 28: Ethical issues and HTA

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Recommendations for Patient Engagement with HTA

Dialogue with patient groups to define submission: what information, how to collect, how to present

Engage patients in CT design to ensure patient values included in measures

Train potential HTA patient-public members on technical processes of HTA and decision making

Provide means for patient-public members of HTA committees to dialogue with patient representatives

Train all HTA committee members on methods for integrating qualitative information

Promote transparent decision-making (records of deliberation as well as outcomes); open meetings.

COMING: Patient Experts in Health Technology!

Page 29: Ethical issues and HTA

Contact:

Durhane Wong-Rieger

Consumer Advocare Network

www.consumeradvocare.org

416-969-7435

[email protected]