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EuroScan Newsletter Web: http://www.euroscan.org.uk December 2012 Number 13
A note from the editor
Early awareness and alert activities
Welcome to issue 13 of the
EuroScan newsletter. In this
bumper issue you can read about
the work of eight EuroScan
member agencies, with articles
on emerging health technologies;
methods employed by agencies
and their networks; an evaluation
of an early awareness and alert
system; and research utilizing the
EuroScan database of emerging
technologies. There is also an
overview of the views of other
organisations on recent important
technological developments and
predictions for 2013.
This issue, we have invited
organisations who Euroscan has
a MoU with or collaborates with
to contribute to the newsletter.
The WHO has recently put out a
call to encourage the
development of innovative
technologies for use in low
resource settings, HTAi is
getting ready for its annual
meeting which is in Seoul in
2013 and you can read about
the recently funded AdHopHTA
project.
In addition to the work of
individual agencies the EuroScan
network has been busy. In
2012, the EuroScan executive
committee and members have
finalised a work programme for
the next 3 years. This will be
published on the Euroscan
website early in 2013 but work
is already ongoing to improve
the exchange of information and
share skills and experiences in
Early Awareness and Alert
activities. The year ahead
promises to be very productive.
Sue Simpson EuroScan Newsletter Editor
EuroScan’s goals include
strengthening activities for the
development of methodological
approaches to the identification,
description and assessment of
emerging technologies; and
increasing the impact of EuroScan
International Network’s output.
So it was with great enthusiasm
and pleasure that a number of
members of EuroScan accepted
the opportunity to be associate
editors for an early awareness
and alert (EAA) themed section in
the International Journal
Technology Assessment in Health
Care.
The themed section was
published in the July 2012 edition
of the journal (Volume 28 /
Issue 03) and gave a great
opportunity to disseminate the
work of EuroScan member
agencies and others working in
the field. Articles featured
covered methods employed by
EuroScan member agencies;
implementation of an EAA; an
analysis of 10 years of EAA
activities; a description of EAA
activities in Latin America,
Canada and Israel; and the use
of best-worst scaling to help
predict impact of emerging
technologies. The editorial
allowed reflection on how EAA
methods and systems had
evolved over the last decade
(and more!) and the opportunity
to present some of the
methodological challenges that
may face EAA systems with the
advent of emerging health
technologies such as
regenerative medicines,
information and communication
technologies, and personalized
health care.
EuroScan International Network
will continue to collaborate to
share and develop appropriate
methods that address the
challenges created by an ever
changing health care sector and
though our website, conference
presence and this newsletter will
share the work of the network.
In this edition
Member Profile:
Institut national
d’excellence en santé et
en services sociaux
(INESSS)
Horizon Scanning
Second Generation
Transcatheter Aortic
Valve Replacement
(TAVR) Devices
Personalised therapies
and in vitro companion
diagnostic tests for
melanoma
Methods and Evaluation
Impact evaluation of
“Horizon Scanning in
Oncology”
Providing evidence and
recommendations to
renew the benefit
package of the Basque
Health System
Evolution of the Italian
EAA system through
the RIHTA network
Horizon scanning and
burden of disease:
Does innovation in
healthcare reflect
need?
Collaboration
HTAi
WHO Call for
Innovative
Technologies
AdHopHTA
2 EUROSCAN NEWSLETTER
Member Profile: INESSS
Institut national d’excellence en santé et services sociaux Pierre Dagenais, directeur du Soutien à la qualité et à la méthode, INESSS
Since its creation, in January 2011,
the Institut national d’excellence en
santé et en services sociaux
(INESSS), a para-governmental
organization for the Province of
Québec in Canada, has been in
charge of assessing a wide range of
health technologies including drugs
and medical devices, as well as
health services and social care
delivery systems. The Institute has
also the mandate to develop
guidelines for clinical and social care
practices and to guide the optimal
use of specific technologies such as
drugs and devices.
INESSS mission is to promote
clinical excellence and efficient use
of resources in the health and social
care sectors by supporting decision
making at every level in the system.
For each technology, it may assess
various dimensions according to
decision needs including; efficacy,
effectiveness, safety, efficiency and
costs, organizational, professional,
psychosocial, ethical and legal
aspects. Based on systematic
reviews and contextual information
from stakeholders, including health
and social care professionals,
managers as well as patients and
beneficiaries, it usually issues
recommendations for adoption, use
and coverage of the technologies by
the public system. In order to
ensure that our products are up to
date, the Institute has implemented
an environmental scanning
procedure for detecting
information that could change our
recommendations and guidelines.
In regards to horizon scanning,
INESSS is a member of CNESH,
the first Canadian collaboration
dedicated to identifying new and
emerging technologies. Mainly,
our organization is involved in
horizon scanning on assistive
devices for physical disabilities.
This early awareness and alert
activity was requested by the
Ministry of Health and Social
Services for improving its
awareness of emerging
technologies in the pipeline that
may have an impact for patients
and the system in terms of
disability management, efficacy
and costs.
A provincial steering committee
consisting of the Ministry of
Health and Social Services,
INESSS, a Laval University
affiliated rehabilitation institute
(IRDPQ, Institut de réadaptation
en deficiencies physiques de
Québec), the Association of
Québec physical rehabilitation
centers (AERDPQ) and the
Health Insurance Board (RAMQ)
was set up 6 months ago. It’s
remit is to establish technological
priorities for a pilot horizon
scanning project that will be
chosen from four categories of
assistive devices: mobility,
hearing, communication and
visual aids. IRDPQ will be in
charge of the horizon scanning
activities in collaboration with
INESSS, which will finance and
coordinate these activities, as
well as provide methodological
support for horizon scanning and
knowledge translation strategies.
The EuroScan International
Network welcomed INESSS to be
a member of the collaboration in
August 2012. It is anticipated
that EuroScan membership will
contribute to the general
development of Horizon
Scanning at INESSS and enable
INESSS to contribute specific
information on Horizon Scanning
for Assistive Devices to
EuroScan.
DECEMBER 2012 3
Second Generation Transcatheter Aortic Valve Replacement (TAVR) Devices Andra Morrison, CADTH
Aortic stenosis (AS) occurs when
there is the narrowing of the
aortic valve opening. Prognosis in
the absence of treatment is poor;
most patients will die within five
years. Up until recently, surgical
aortic valve replacement was the
only treatment for severe aortic
stenosis (SAS) other than medical
therapy with drugs. However,
transcatheter aortic valve
replacement (TAVR) has emerged
as a potential alternative
treatment for some patients with
SAS. Current TAVR devices are
used on SAS patients who are
inoperable or at high surgical risk,
a population that represents
approximately 10% of patients
with SAS. Second generation
TAVR devices may expand the
existing SAS population to
younger and healthier patients.
There are at least eight
transcatheter aortic valves in
clinical development that are
intended to address challenges
associated with first generation
devices:
EngagerTM(Medtronic): In
September 2011, Medtronic
announced the start of a
European non-randomized single
arm trial to pursue a CE Mark for
EngagerTM. The trial will evaluate
the safety and clinical
performance of the valve in high
risk SAS patients.
Acurate TATM(Symetis): CE Mark
approval was received in September
2011, and the transapical valve was
commercial launched in October
2011. A post market surveillance
study for its continued safety
monitoring is currently enrolling
patients. Symetis is also expected
to start a study of its transfemoral
system the Acurate TFTM by the end
of 2012.
Direct Flow Medical Aortic Valve
(Direct Flow Medical): A European
CE Mark safety and performance
trial is expected to be completed by
the end of 2012.
Sadra Medical Lotus TM (Boston
Scientific): A prospective single arm
safety and performance study,
REPRISE I, in patients with SAS was
initiated in 2012. Enrollment in
REPRISE II is anticipated next year.
Portico THV TM (St Jude Medical): A
2011 study evaluated the technical
feasibility, safety and device
deployment characteristics of this
valve in high risk SAS patients. A
multicenter observational cohort
study is currently recruiting patients
for the investigation of the safety
and effectiveness of the valve in
patients at high surgical risk.
JenaValveTM (JenaValve): CE Mark
approval was received in September
2011. The CE Mark pivotal study
was a multicentre, prospective,
single-arm study of 67 patients who
were at high risk for surgery. A new
observational cohort study is
currently recruiting patients with
SAS at increased risk of surgery
for the evaluation of the long
term performance and safety of
JenaValve.
SAPIEN 3 and CENTERA
(Edwards Lifescience): Edwards
has conducted two first-in-
human trials for these two
second generation valves. Both
are investigational devices that
are not yet commercially
available in any country.
Vitality™ and Vanguard™:
(ValvXchange): ValvXchanage
completed first-in-man clinical
studies in 2011 for its valve, and
will be pursuing clinical trials in
Europe in 2012. ValveXchange is
also developing the Vanguard™
series of valves for non-surgical
patients.
Heart Leaflet Technology valve
(Bracco Diagnostic): In 2008, a
non-randomized single arm
study was initiated to confirm
whether the dimensions of the
Heart Leaflet valve are
appropriate for patients with
aortic stenosis.
CADTH are producing an
environmental scan on TAVR.
Environmental Scans are short
reports on current or emerging
issues in health care
technology. Each report
provides information to support
health care decision-making
and policy development in
Canada.
http://www.cadth.ca/en/produc
ts/environmental-
scanning/environmental-scans
4 EUROSCAN NEWSLETTER
Impact evaluation of “Horizon Scanning in Oncology” Anna Nachtnebel, LBI-HTA, Austria
Since 2009, “Horizon Scanning
in Oncology” (HSO) is a
permanent work programme at
the Austrian Ludwig Boltzmann
Institute for Health Technology
Assessment. This programme
was set up to inform decision-
makers, foremost hospital-
based decision-makers, on
emerging and new oncologic
drugs. The underlying rationale
was to facilitate the evidence-
based use of new oncologic
drugs but also to foster the
estimation of the financial
impact on hospital budgets.
Since its implementation, 33
reports and 3 updates have
been published and were
distributed by different means.
Besides e-mail notifications via
a mailing-list for HTA-interested
persons in general, e-mail
alerts are also specifically sent
to decision-makers at Austrian
hospitals (e.g. head of
pharmacies, medical directors)
with oncologic wards as soon
as new reports are published
on the LBI’s homepage.
Furthermore, all reports are
entered to the EuroScan
database, they are freely
available on our institute’s
homepage, and short
summaries of the reports are
irregularly published in our
newsletter.
Despite these various
dissemination methods, it
remained unclear whether
reports had been used at all
and if, for what purposes.
Given that considerable
resources, mostly in terms of
staff, are dedicated to the HSO
programme, the question arose
whether pursuing this
programme line proofed
worthwhile. Thus, an impact
analysis consisting of a
download analysis and an
online-questionnaire was
conducted.
The download analysis yielded
that HSO reports had been
viewed about 14,000 times
(from October 2009 – February
2012) and had been
downloaded about 7,000 times.
Huge differences exist between
individual reports as, for
example, the most popular
report (bendamustine for non-
Hodgkin’s lymphoma , chronic
lymphocytic leukaemia and
multiple myeloma) had been
downloaded approximately 900
times, whereas axitinib for
renal cell carcinoma ranged
with only 37 downloads at the
other side of the spectrum.
However, these numbers have
to be interpreted against the
background that the temporal
availability of reports differed
substantially; bendamustine
had been online for about two
years and axitinib had been
published only one month
prior to the download
analysis. Besides overall
downloads, the average
monthly download rates per
year were calculated. Starting
with an average of 11
downloads per month in 2009,
this number increased to 40 in
2012. Despite this trend, one
has to bear in mind that also
the number of published
reports rose from 7 in 2009 to
24 in 2012. Thus, the sheer
availability of an ever
increasing number of reports
has definitely contributed to
this gain.
“According to the download
rates and the satisfaction of
the primary target audience,
continuation of our HSO
programme is justified”
Table 1: Satisfaction with HSO reports
completely satisfied
satisfied fairly satisfied
somewhat dissatisfied
very dissatisfied
not able to judge
structure of the
reports44% 50% - 6% - -
breadth of the reports
59% 35% 6% - - -
quality of the reports
50% 50% - - - -
timing of the publication
47% 35% 18% - - -
type of notification
56% 39% - - - 6%
DECEMBER 2012 5
To investigate whether the
actual target audience of the
HSO programme used the
reports and how quality and
content was perceived, an
online questionnaire was sent to
those Austrian decision-makers
who also receive the e-mail
notifications on new reports -
approximately 120 individuals.
Despite repeated reminders, the
response rate did not exceed
30% with the majority of
respondents being pharmacists.
Interestingly, only a little over
50% knew the programme.
Nonetheless, respondents were
overall satisfied with the quality,
structure and depth of
information provided within the
reports (see table 1). Only with
the timing of the reports, about
20% were “fairly satisfied” but
it is unclear whether earlier or
later reports, for example closer
to actual reimbursement
decisions, would have been
appreciated. When questioned
about the usage of the reports,
most respondents valued them
as an information source rather
than as decision documents (see
figure 1). However, this may be
a result of the rather low
response rate as well as of the
fact that survey participants
came mainly from the same
professional background.
According to the download rates
and the satisfaction of the
primary target audience,
continuation of our HSO
programme is justified.
Nonetheless, several issues
remain unresolved. For one, it is
unclear who the actual users of
our reports are, because the
download analysis did not allow
identification of the users’
professional backgrounds. Also
the international impact, for
example usage within the
EuroScan database, is unknown.
Lastly but foremost, whether
HSO reports are used for those
purposes set out in the
programme’s objectives is,
due to the low survey
response rates, uncertain.
However, based on this
impact analysis a couple of
actions arose. Besides
improved methods for a more
detailed analysis of the usage,
efforts for further increasing
the awareness of the HSO
programme are clearly
needed. In addition, adaption
of the underlying processes as
well as of the outputs of our
horizon scanning project
might be in order to better
meet our target audience’s
needs. Out of a broader
scientific perspective and in
absence of commonly
accepted frameworks more
refined methods for fully
capturing the impact of policy-
oriented research activities
need to be developed.
Figure 1: Reasons for using HSO reports
6 EUROSCAN NEWSLETTER
Personalised therapies and in vitro companion diagnostic tests for melanoma C. Poggiani, D. Pase, R. Joppi – Italian Horizon Scanning Project
Background
In the oncology setting, the
need of drugs associated with a
lower incidence of side effects
together with a well-
demonstrated efficacy is
leading to an increased move
to develop personalised
therapies. These treatments
are directed to a specific
molecular target or mutation of
tumour cells that become a
predictor of therapy response.
In this context, molecular
diagnosis is fundamental to
select patients who are most
likely to respond to the
therapy, thus rendering it
necessary to co-develop with
each new personalised therapy,
for which a specific diagnostic
test does not already exist, the
matching companion in vitro
diagnostic test1.
Molecularly targeted assays are
usually specific for DNA
mutations, for expression of
transcripts or protein or to
determine functional properties
of molecular complexes. Two
early examples of companion
diagnostic tests are the HER2-
directed therapy in breast
cancer with trastuzumab and
EGFR-targeted therapy in
colorectal cancer with
cetuximab or panitumumab1.
Aim
This article will describe new
oncology personalised therapies
co-developed with related in
vitro companion diagnostic
tests for melanoma and will
evaluate regulatory aspects
concerning in vitro companion
diagnostic tests.
Method
Medical literature, websites of
early awareness and alert
systems, company websites
and regulatory agencies were
searched.
Results
Recently, in the EU2 and US3,
vemurafenib was approved with
its companion diagnostic test
for the treatment of BRAFV600-
positive metastatic melanoma.
Vemurafenib is a first-in-class
inhibitor of oncogenic BRAF
kinase and, according to
European guidelines, it is
considered an optimal first-line
choice for metastatic melanoma
in a subset of patients with
mutated BRAF4. In the pivotal
phase III trial, 675 patients
with untreated stage IIIc-IV
metastatic melanoma
presenting the BRAF V600E
mutation, as detected by the
companion cobas 4800 BRAF
V600 Mutation Test, were
treated with vemurafenib or
dacarbazine. Vemurafenib
significantly prolonged both
overall survival (value not reached
after a median follow-up of 6.21
months for vemurafenib vs. 7.89
months with dacarbazine,
p<0.0001) and progression-free
survival (5.3 vs. 1.6 months,
p<0.001)5.
Both US and EMA labels indicate
that before taking vemurafenib,
patients must have undergone a
molecular diagnosis with the
companion cobas 4800 BRAF
V600 Mutation Test. This test,
which is CE marked, was
designed to detect the
predominant BRAF V600E
mutation with high sensitivity
(down to 5% V600E sequence in
a background of wild-type
sequence from FFPE-derived
DNA) and it is used to assess the
BRAF mutation status of DNA
isolated from formalin-fixed,
paraffin-embedded (FFPE) tumour
tissue. The test was co-developed
with vemurafenib and it was used
to select patients eligible for
phase II and III trials at
designated laboratories in the US,
Europe and Australia2,3,6.
During vemurafenib’s
development program, the cobas
test was validated through
retrospective sequencing analyses
with the Sanger method. Four
hundred and sixty seven
specimens were positive with the
DECEMBER 2012 7
cobas test in non-clinical and
clinical studies. The BRAF V600-
mutation positive status was
further confirmed with “deep”
sequencing of 454 out of all
those selected specimens2,3.
Recently, two other BRAF-
targeted therapies are being
developed with companion tests,
the BRAF inhibitor dabrafenib
and the MEK kinase inhibitor
trametinib. In phase III studies,
both trametinib and dabrafenib
significantly prolonged
progression-free survival (PFS)
vs control (trametinib vs
paclitaxel or dacarbazine: 4.8 vs
1.5 months, p<0.0017;
dabrafenib vs dacarbazine: 5.1
vs 2.7 months, p<0.00018). In
parallel, in a phase I/II trial in
247 patients with metastatic
melanoma and BRAF V600
mutations, the combination
dabrafenib+trametinib
prolonged PFS vs dabrafenib
monotherapy (9.4 vs 5.8
months, p<0.001). For both
drugs a companion test for BRAF
mutation detection is being co-
developed and was used to
select patients in the phase III
trials. One of the secondary
objectives of both trials was the
validation of the tests9,10.
Clinical validation of companion
tests co-developed with
personalised therapies is
required for regulatory approval
by the FDA11. However, it is not
mandatory for CE marking12. On
the other hand, the validation
process in clinical setting is
recommended by the EMA. This
is in order to collect all the
information necessary to
describe the companion test in
both the assessment report
and in the summary of product
characteristics of the related
matching drug. This helps to
prepare for and facilitate the
transition of testing
methodology in post-approval
clinical use13.
Conclusions
BRAF-directed therapies are
new treatment options for a
subset of patients with
metastatic melanoma, for
which other specific treatments
do not exist4. The use and the
possible efficacy of these
therapies is strictly related to
the selection of patients with
mutated BRAF, as detected by
companion diagnostic tests.
Global definition and
harmonisation of regulatory
requirements for the co-
development of companion
tests with matching therapies
is necessary to introduce in
clinical practice rigorously
validated companion tests, that
are able to support the
appropriate utilisation of the
related personalised therapies.
References 1Zieba A et al. Molecular tools for companion diagnostics. N Biotechnol 29(6):634‐640 2http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002409/human_med_001544.jsp&mid=WC0b01ac058001d124. Accessed on 04/12/2012 3http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.DrugDetails. Accessed on 04/12/2012 4Dummer R et al. Cutaneous
melanoma: ESMO clinical practice guidelines for diagnosis, treatment and follow‐up. Annals of Oncology 23 (Supplement 7): vii86–vii91, 2012 5Chapman PB et al. Improved survival with vemurafenib in melanoma with BRAF V600E mutation. N Engl J Med 2011, 364:2507‐2516 6Cheng S et al. Co‐development of a companion diagnostic for targeted cancer therapy. N Biotechnol 2012, 29(6):682‐688 7Flaherty KT et al. Improved survival with MEK inhibition in BRAF‐mutated melanoma. N Engl J Med 2012, 67:107‐114 8Hauschild A et al. Dabrafenib in BRAF‐mutated metastatic melanoma: a multicenter, open‐label, phase 3 randomised controlled trial. Lancet 2012, 380:358‐365 9Protocol for: Flaherty KT, Robert C, Hersey P, et al. Improved survival with MEK inhibition in BRAF‐mutated melanoma. N Engl J Med 2012;367:107‐14. DOI: 10.1056/NEJMoa1203421. 10Supplement to: Hauschild A, Grob
J‐J, Demidov LV, et al. Dabrafenib in BRAF‐mutated metastatic melanoma: a multicentre, open‐label, phase 3 randomised controlled trial. Lancet 2012; published online June 25. http://dx.doi.org/10.1016/S0140‐6736(12)60868‐X. 11http://www.fda.gov/medicaldevic
es/deviceregulationandguidance/guidancedocuments/ucm262292.htm. Accessed on 04/12/2012 12http://ec.europa.eu/enterprise/p
olicies/european‐standards/harmonised‐standards/iv‐diagnostic‐medical‐devices/index_en.htm. Accessed on 04/12/2012 13Refelection paper on co‐
development of pharmacogenomic biomarkers and assays in the context of drug development. EMA/CHMP/641298/2008, 24/06/2010
The Italian Horizon Scanning
Centre (IHSP) is an Early
Warning System for
identification and assessment of
emerging medicines and medical
devices with medicated coating.
http://horizon.cineca.it/
8 EUROSCAN NEWSLETTER
Providing evidence and recommendations to renew the benefit package of the Basque Health System Iñaki Gutiérrez-Ibarluzea, Gaizka Benguria-Arrate, Rosa Rico-Iturrioz and Marta López de Argumedo Osteba, Basque Office for HTA. Department of Health and Consumer Affairs. Basque Government.
One of the main goals of Early
Awareness and Alert Systems is to
improve the implementation /
diffusion processes of new and
emerging technologies by providing
evidence and recommendations to
decision-makers at the different
levels of decision. Osteba started its
early awareness and alert system
(EAAS) 14 years ago when it was
invited to a meeting where
EuroScan was founded. Since then,
Osteba has updated its methods
including the concept of Life cycle of
Health Technologies that led to the
analysis of not only new but low-
added value technologies.
Osteba has recently started a new
initiative to improve its impact in the
benefit basket or package of the
Basque Health System. To this end,
a new unit has been created called
the Comparative Effectiveness Unit,
Osteba-EKU. EKU is an advice or
consultation body that aims to
provide information on both
emerging and low added-value
health technologies. The aim is to
include in the benefit package of
health services, those technologies
with maximum benefit for health to
make better use of available
resources.
EKU has started a process this year
to identify technologies that could
be included or delisted in the benefit
package. The project started with a
transparent identification process
followed by a prioritisation
scheme that resulted in the final
list. The following sources were
used to identify technologies:
GenTecS, EuroScan, Cochrane,
INAHTA, NICE, ECRI and
Cleveland Clinic Databases for
new and emerging technologies;
the Spanish National Guidelines
Clearing House and NICE
databases for low-added value
technologies. In terms of the
prioritisation process, we used
PriTec software coordinated by
the Galician HTA Agency Avalia-T
which is available at:
http://pritectools.es/index.php?idi
oma=en.
We identified 910 new and
emerging health technologies
initially that were reduced after
different cycles of prioritisation to
a total of 11. In the case of low
added value technologies, 375
technologies were identified and
finally 9 were included for the
assessment. This prioritisation
process was conducted by the
advisory board of EKU involving
clinicians, medical directors,
hospital managers, decision-
makers and health care service
managers.
Osteba-EKU is currently
conducting a process of
assessment that will provide
evidence, including an analysis of
variability of health care services
in the case of low-added value
technologies, and
recommendations and a plan for
the implementation of the
promising technologies in clinical
practice. Some preliminary
results are already available at
the Osteba webpage
http://www.osakidetza.euskadi.n
et/osteba .
Osteba has designed different
type of documents and diffusion
processes, tailoring them to the
needs of various stakeholders.
http://www.osakidetza.euskadi.n
et/r85-
pkoste13/en/contenidos/informa
cion/osteba_eku/en_osteba/oste
ba_eku.html
Further studies will be required
to analyse the impact of this
initiative in health services
provision and resources
allocation.
DECEMBER 2012 9
Evolution of the Italian EAA system through the RIHTA network
Antonio Migliore and Marina Cerbo, , Agenas, Italy
According to its core mission,
supporting Regional
Governments to establish,
improve and maintain HTA
activities, in 2009 Agenas
created RIHTA: the Italian
Network for HTA. RIHTA is a
collaborative network among
Regions and Regional
Healthcare Agencies aimed at
spreading the HTA/EAA culture
across Italy, promoting the
launch of new HTA/EAA
programmes and improving the
ones already running, and
facilitating collaboration among
Regions on common issues.
In 2012, Agenas focused on the
evolution of the EAA system
created within the research
project named COTE (concluded
in 2011) [1]. The new
framework, proposed by Agenas
and discussed with RIHTA
members, was aimed at
promoting a more active
participation of members in EAA
activities. The agreed workflow
was then tested in real-life.
The new features are related to
the prioritisation and
assessment phases. As the EAA
became a regular Agenas
activity (reports are not
committed anymore by the
Ministry of Health but produced
with Agenas’s own budget) the
prioritisation is carried out by
RIHTA members. Every 2
months all the members receive
a list of the new and emerging
technologies that have been
identified and are asked to
prioritise 3 technologies of
interest. The criteria used for the
choice are internal to each Region
(a range of different methods,
qualitative/quantitative, has been
observed; see EuroScan
Newsletter n.12 [2]).
On the basis of the number of
preferences obtained by the single
technology, working groups are
defined to perform the
assessment. The multi-regional
teams are then coordinated by
Agenas.
The first assessment experience
with this new model was the
Agenas HS report n.10 [3]. Two
Regions were asked to collaborate:
Liguria (with one epidemiologist)
and Piemonte (one clinical
engineer and one librarian).
Logistically, e-mails and e-
meetings have been utilised
instead of face-to-face meetings to
avoid travel costs. Agenas
coordination work consisted of:
general project management,
interaction with external clinical
experts, interaction with
manufacturers, internal review,
translation of the final document
(from English to Italian), upload of
the document on the EuroScan
database and Agenas website.
The report was produced within
the expected deadline and
without relevant organisational
problems. Thanks to the internal
review by Agenas experts and
extra support by the Agenas
coordinator, the final document
was produced according to the
previous quality standard. A new
assessment is ongoing.
SUGGESTED READING
[1] Migliore A, Perrini MR,
Jefferson T, Cerbo M.
Implementing a national early
awareness and alert system for
new and emerging health
technologies in Italy: the COTE
Project. Int J of Technol Assess
Health Care. 2012 Jul, 28(3):
321-326.
[2] Migliore A. “Prioritisation in
the Italian Regions”. EuroScan
Newsletter. June 2012 Issue 12.
http://euroscan.org.uk/mmlib/inc
ludes/sendfile.php?id=130
[3] Migliore A, Bonelli L, Giani E,
Romano V. Raman spectroscopy
for early detection of skin cancer.
Agenas, Agenzia nazionale per i
servizi sanitari regionali. Rome,
July 2012.
http://www.agenas.it/agenas_pdf/spectroscopy_english.pdf
10 EUROSCAN NEWSLETTER
Health Technology Assessment International (HTAi) Chris Sargent, Managing Director, HTAi
HTAi is a global society focused on
supporting those involved with and
practicing health technology
assessment in every part of the
world. HTAi Annual Meetings, held
in May-June each year, attract
thousands of researchers from
around the globe, with the most
recent meeting in Bilbao, Spain
providing an opportunity for
members to share the latest
thinking and cutting-edge
techniques in HTA.
HTAi’s up-coming Annual Meeting in
Seoul, in June 2013 offers a
thought-provoking agenda that
examines the balance between art
and science in the practice of HTA
and will bring together the brightest
minds in HTA from Asia and around
the world. The call for abstracts is
now out; more information can be
found on the HTAi website at
www.htai.org.
HTAi also continues to serve the
needs of its growing membership in
emerging markets in Asia and Latin
America and convenes the HTAi
Policy Forum. A clear example of
HTAi supporting its members is
HTAi’s second Latin America
regional meeting that took place in
early December in Bogota,
Colombia, which brought together
over 200 HTA experts from across
Latin America. The meeting focused
on topics ranging from good
practice principles for HTA agencies
to methodological challenges when
conducting HTA to looking at
managed entry schemes and
disinvestment. It was also an
exciting moment for HTA as this
meeting coincided with the
establishment of Colombia’s HTA
agency, IETS.
The continued growth and
success of HTAi in these regions
is one of Dr. Clifford Goodman’s
main area of focus on during his
tenure as President of the HTAi
Board. Dr. Goodman says, “as in
other parts of the world with a
range of rapidly evolving
economies, the need for HTA in
Latin America is driven by greater
health care demand of an
expanding middle class, rapidly
rising prevalence of chronic
disease, and ongoing
technological innovation and
marketing. Many decision makers,
including those at ministries of
health, hospitals, and private
insurers increasingly see the need
for making transparent, evidence-
based, and accountable policies
and guidelines. Although there is
great diversity in the roles,
resources, and maturity of HTA
programs in Latin America, they
are very involved in regional and
global HTA networks, and they
are making strong contributions
to advancing the field.”
HTAi will soon return to Spain for
its annual Policy Forum meeting
in Barcelona in February 2013.
The HTAi Policy Forum provides a
venue for senior leaders from the
not-for-profit and for-profit
sectors to discuss issues of
emerging global importance and
to share the findings of those
discussions with the wider HTA
community. This year’s topic is
HTA and Value, and discussions
will examine the various
dimensions of value, including
innovation, how these are
viewed and prioritised by
different stakeholders, and how
this information can best be
factored into decision making in
health care systems. A paper will
be published on the main
themes discussed at the meeting
in IJTAHC, HTAi’s peer-reviewed
journal. All papers of the HTAi
Policy Forum are available on
the HTAi website.
As a global society, HTAi
continues to ensure it stays the
course as a neutral forum for
diverse stakeholders engaging in
multidisciplinary, multi-sector
collaboration. This requires
remaining responsive to the
needs and priorities of our
individual and organizational
members across the globe. As
always, Dr. Clifford Goodman
and his colleagues on the HTAi
Board, look forward to continued
collaboration with EuroScan and
its members to support and
promote health technology
assessment.
EuroScan signed a Memorandum
of Understanding with HTAi in
June 2011.
DECEMBER 2012 11
Horizon scanning and burden of disease: Does innovation in healthcare reflect need? Orsolina Martino, NIHR-HSC
Studies that have used research
funding to estimate the amount of
innovation in healthcare have
raised concerns that some disease
areas are neglected despite
having a high burden on society.
A study carried out within the
NIHR Horizon Scanning Centre in
England used data from the
EuroScan network as a novel way
to measure innovation.1
Technologies reported on the
EuroScan database between 2000
and 2009 by 21 agencies were
compared with the burden of
related diseases among their
respective countries. 17 countries
were represented in all, but the
data were combined to give
figures for EuroScan as a whole.
Burden of disease was measured
as deaths and disability-adjusted
life years (DALYs), taken from the
WHO’s 2004 Global Burden of
Disease estimates.2 These were
grouped by 21 main disease
areas, which were split further
into 102 specific causes. Each
technology entered onto the
EuroScan database was given a
code so that it could be matched
to the relevant disease.
The results showed only a weak
relationship between technologies
reported by EuroScan members
and the burden of related
diseases. Most notably, there
was more innovation than
expected for cancer and less for
neuropsychiatric conditions.
Dividing the main groups into
specific diseases weakened the
association further, as illustrated
in Figure 1.
It was concluded that outputs
from horizon scanning activity do
not always reflect areas of the
greatest need, as factors other
than disease burden drive
innovation.
References: 1. Martino OI, Ward DJ, Packer C, Simpson S, Stevens AJ. Innovation and the burden of disease: Retrospective observational study of new and emerging health technologies reported by the EuroScan network from 2000 to 2009. Value in Health 2012;15:376-80. 2. World Health Organization. The Global Burden of Disease: 2004 Update. Geneva: WHO, 2008.
Figure 1- Relationship between DALYs and technologies for 102 specific causes.
12 EUROSCAN NEWSLETTER
Contact person:
Laura Sampietro-Colom
Hospital Clinic Barcelona
(Coordinator)
AdHopHTA: a new European Project on Hospital Based Health Technology Assessment
The EU has provided a grant for the
AdHopHTA (Adopting Hospital
Based Health Technology
Assessment in EU) project under the
7th Framework Research
Programme. AdHopHTA aims to
strengthen the use and impact of
excellent quality HTA-results in
hospital settings, making available
pragmatic knowledge and tools to
facilitate adoption of hospital based
HTA-initiatives. As a secondary aim,
the project will build an adequate
ecosystem where formal
coordination amongst existing
hospital based HTA-initiatives and
liaisons with national and regional
agencies will flourish.
AdHopHTA has 10 partners with
different, but complementary,
backgrounds: five hospitals with
HTA-programs (Hospital Clinic
Barcelona/FCRB-Spain
[Coordinator]; Odense University
Hospital-Denmark; Hospital District
of Helsinki and Uusimaa-Finland;
Hospices Cantonaux CHUV-
Switzerland; Università Catolica
del Sacro Cuore-Italy), two
hospitals without a HTA program
(Ankara Numune Training and
Research Hospital-Turkey; Tartu
Univeristy Hospital-Estonia), two
national HTA agencies/institutes
(The National Health Center for
Health Services-Norway; Ludwig
Boltzmann Institute for HTA-
Austria) and one business school
(IESE-Spain). It also has an
advisory board where
international societies and key
institutions are represented
(HTAi, INAHTA, EuroScan,
EUnetHTA, ISQua, CEDIT-
Assistance Publique Hôpitaux
Paris, HTA Centrum at
Sahlgenska University Hospital).
The final output of the project will
be a handbook on best practices
for hospital based HTA as well as
a hospital based HTA deployment
toolkit. Another relevant output will
be the design and creation of a
pilot repository on hospital based
HTA-products which aims to be
aligned as much as possible with
the EUnetHTA-POP Database, in
order to ensure that the
AdHopHTA-repository
complements it. These outputs will
be built through five main technical
work packages supported by
dissemination and management
work packages. Figure 1 shows the
PERT diagram of the project.
It is intended that the results of
AdHopHTA will be applied across
the EU and worldwide, and will
facilitate the start of hospital based
HTA-programmes, make improved
tools available for hospital based
HTA (e.g. mini-HTA 2.0), improve
the quality and efficiency of current
hospital based HTA-programmes,
improve the liaison with
national/regional HTA-programmes
and set up the basis for a
European network of hospital
based HTA. AdHopHTA is a
complementary initiative for
EUnetHTA Joint Action, bringing
forward a more comprehensive
HTA-strategy across health system
levels in Europe.
EuroScan looks forward to
supporting the AdHopHTA Project
as a member of the advisory
board.
Fig. 1. Graphical presentation of the interdependencies of work packages and project components (Pert diagram).
DECEMBER 2012 13
The Israeli Center for Emerging Technologies Orna Tal and Nina Hakak, ICET
Technology advances in 2012 & predictions for 2013
The Israeli Center for Emerging
Technologies (ICET), formerly
known as DMTP, is currently
concentrating on 2 major trends:
A) Hospital-based HTA
1. An in-depth assessment of
hospital technologies in Israel is
required since the annual
updating process of the National
List of Health Services focuses
mainly on technologies provided
within the framework of the
community.
2. Hospitals are considered to be
technology promoters due to
several factors:
a) Hospitals have additional
sources of funding for research
and invest in developing
specialists who concentrate on
very specific issues.
b) The existence of competition
between hospitals is a powerful
tool to encourage innovation. This
entails the capacity of each
hospital to develop services
according to staff members
especially specialists in fields such
as genetics, imaging and
nanotechnology procedures.
c) Hospitals encourage specialists
to focus on rare diseases such as
genetic disorders e.g. Fabry
disease.
3. Hospitals are challenged by
unique populations such as ethnic
subgroups e.g. specific groups of
immigrants from Africa or highly
orthodox populations requiring
sensitive ethical approaches.
Thus, specific elements of HTA
are used to identify emerging
technologies.
B) Personalized Medicine
The current trend throughout
the world and in Israel involves
focusing on the identification and
adoption of genetic markers to
diagnose and treat different
diseases. This can be
demonstrated in:
• BRAF & CTLA4 for identifying
a treatment response for
malignant melanoma
• MLH1, PMS2, MSH2 and MSH6
for Lynch Syndrome.
Nature Medicine recently
summarized notable advances in
biomedicine in 2012 (Vol 18, No.
12 p 1732-3). These included the
discovery of new candidate genes
for autism spectrum disorders
offering new directions for autism
research; research showing how
the plant–derived compound
resveratrol might imitate the
benefits of caloric restriction; the
identification of the novel
hormone irisin which is thought to
turn white fat ‘beige’ , thus
burning energy rather than
storing it; and the findings of 2
studies of the inhibitory co-
receptor PD-1 (protein cell death
protein 1) that further validated
immune modulation of T cell
activity as a therapeutic approach
to cancer treatment.
The Lancet reviewed 2012 (Vol
380, Dec 8 p1979-80) and
highlighted the Encyclopedia Of
DNE Elements (ENCODE) project,
which has assigned biochemical
functions to 80% of our genome
and will transform the future of
medical research.
At the end of October 2012
Cleveland Clinic announced its list
of Top 10 Medical Innovations
that will have a major impact on
improving patient care in 2013.
The Top 10 Medical Innovations
for 2013 selected by a panel of
Cleveland Clinic physicians and
scientists are:
1. Bariatric Surgery for Control of Diabetes
2. Neuromodulation Device for Cluster and Migraine Headaches
3. Mass Spectrometry for Bacterial Identification
4. Drugs for Advanced Prostate Cancer
5. Hand-held Optical Scan for Melanoma
6. Femtosecond Laser Cataract Surgery
7. Ex Vivo Lung Perfusion
8. Modular Devices for Treating Complex Aneurysms
9. Digital Breast Tomosynthesis (DBT)
10. Health Insurance / Medicare Program / Rewards for Better Health
http://www.clevelandclinic.org/INN
OVATIONS/SUMMIT/topten.html
14 EUROSCAN NEWSLETTER
WHO Call for Innovative Health Technologies
EuroScan Executive Committee
Do you have a new technology
solution to a well-known health
problem?
Do you have a technology
solution to a health problem not
yet addressed?
Medical devices and eHealth
solutions have the potential to
improve lives. However, too
many people worldwide suffer
because they don’t have access
to the appropriate health
technologies. The Medical
Devices and eHealth units of the
World Health Organization invite
you to submit your solution to
the Call for innovative health
technologies for low-
resource settings.
Deadline for submission is
March 15th, 2013. Selected
submissions will be published in
the WHO Compendium of
innovative health technologies
2013.
WHO aims to raise awareness of
the pressing need for appropriate
design solutions. The Compendium
series specifically focuses on
showcasing innovative medical
devices and eHealth solutions that
are not yet widely available in
under-resourced regions. The
annual publication serves as a
neutral platform to introduce
health technologies that have the
potential to improve health
outcomes or to offer a solution to
an unmet medical need. It is
designed to help developing
countries to become aware of
appropriate health technologies in
support of their environment. The
Compendium 2012 can be viewed
at:
www.who.int/medical_devices/inn
ovation/compendium202
About EuroScan
EuroScan - the International Information Network on New and Emerging Health Technologies - is a collaborative network of member agencies for the exchange of information on important new and emerging health technologies.
Mission
EuroScan International Network is the leading global collaborative network that collects and shares information on innovative technologies in healthcare in order to support decision-making and the adoption and use of effective, useful and safe health-related technologies. We are also the principal global forum for the sharing and development of methods for the early identification and early assessment of new and emerging health-related technologies and their potential impact on health services and existing technologies.
EuroScan International Network is committed to work with a high level of transparency and professionalism, and in partnership with researchers, research centres, governments and international organisations to produce high quality information and effective early awareness and alert systems for our respective constituencies. We are also committed to support the development of existing and new not-for-profit public agencies working in the early awareness and alert field.
Position Current Post Holder End of termChair Professor Brendon Kearney, HPACT 31/12/13 Vice Chair Dr Marianne Klemp, NOKC 31/12/13 Registrar Dr Kees Groeneveld, GR 31/12/12 Dr Roberta Joppi, IHSP From 1/1/13-31/12/14Treasurer Christoph Künzli, SFOPH 31/12/12 Dr Anna Nachtnebel, LBI-HTA From 1/1/13-31/12/14Head of Secretariat Dr Claire Packer, NIHR HSC n/a
Visit http://www.euroscan.org.uk to find out more about EuroScan, its work and how to become a
member.
If you have any feedback, questions, would like to know more or have any articles that would be of interest for the next edition of the newsletter please contact Dr Sue Simpson, EuroScan Network Co-ordinator, NIHR HSC, Department of Public Health, Epidemiology and Biostatistics, University of Birmingham, B15 2SP [email protected] +44 121 414 7496
Views of individual authors are not necessarily the views of the editor or EuroScan members
EuroScan has a Memorandum of
Understanding with the World
Health Organisation and has an
agreed collaboration plan. As such
EuroScan members have
contributed to the previous
Compendiums by evaluating
submissions using agreed criteria.