VA L U E I N H E A LT H 2 1 ( 2 0 1 8 ) S 1 – S 1 1 5
S23
PCN103
A SIMULATION STUDY OF THE EFFECT OF SCREENING ON LUNG CANCER
MORTALITY IN ASIA
Chen Y1, Criss SD1, Sheehan DF1, Kong CY2
1Institute
for Technology Assessment, Massachusetts General Hospital, Boston, MA, USA,
2Harvard Medical School, Boston, MA, USA
OBJECTIVES: More than 50% of the world’s lung cancer cases occur in Asia and
more than 20% of cancer deaths in Asia are attributable to lung cancer. Lung
cancer screening has been shown to reduce lung cancer deaths. Using the Lung
Cancer Policy Model–Asia (LCPM-Asia), we estimated the potential mortality reduc-
tion achievable through implementing computed tomography-based lung cancer
screening in China, Japan, Singapore, and South Korea.  METHODS: The LCPM-Asia
was calibrated to the smoking prevalence of each of the aforementioned countries
based on published national surveys and lung cancer mortality rates from the World
Health Organization. The calibrated LCPM-Asia was then used to simulate lung
cancer deaths under screening and no-screening scenarios for the four countries.
Using screening eligibility criteria recommended by the U.S. Centers for Medicare &
Medicaid Services (CMS), which are based on participants’ ages and smoking histo-
ries, we estimated the cumulative lung cancer mortality reduction from screening
through year 2050.  RESULTS: By 2050, lung cancer screening would result in 4.82%
mortality reduction, preventing 721,589 lung cancer deaths in China; 5.75% mortality
reduction and 68,194 lung cancer deaths prevented in Japan; 3.98% mortality reduc-
tion and 2,096 lung cancer death prevented in Singapore; 5.48% mortality reduction
and 21,856 lung cancer death prevented in South Korea. As for mortality reduction by
smoker type, current smokers would have the greatest mortality reduction in each
country, from 7.04% in China to 8.02% in South Korea.  CONCLUSIONS: Among the
four countries, lung cancer screening based on CMS criteria was most effective in
Japan and least effective in Singapore. Compared to the other countries, Singapore
has the lowest smoking prevalence and Japan has the largest aging population,
partially explaining the discrepancy in effectiveness. Therefore, to perform lung
cancer screening effectively in each country, the eligibility criteria should be tailored
to the country’s demographic characteristics.
PCN104
TRANSFERRING ECONOMIC DATA FROM A DEVELOPED TO DEVELOPING
COUNTRY SETTING TO CONDUCT HTA: THE CASE OF TAMOXIFEN FOR
THE ADJUVANT TREATMENT OF ADVANCED BREAST CANCER AMONG
PREMENOPAUSAL WOMEN IN GHANA
Addo R1, Goodall S2, Hall J1, Haas M1
1Universityof Technology Sydney, Haymarket, Australia, 2University of Technology, Sydney,
Sydney, Australia
OBJECTIVES: In recent years, there has been a push for the use of health technology
assessment (HTA) in developing countries. However, the suitability of such evidence
is limited due to the lack of clinical and economic data and human capacity to con-
duct it. This study therefore sought to assess the transferability of economic data
to the Ghanaian context for an HTA, using tamoxifen for the adjuvant treatment
of advanced breast cancer among premenopausal women.  METHODS: A system-
atic review was conducted to identify economic evaluation studies transferrable
to the Ghanaian context. A cost utility analysis was conducted using a Markov
model to incorporate effectiveness, costs and utility data, with local data used to
support the model.  RESULTS: None of the economic evaluation studies identified
were transferrable to the Ghanaian context, therefore a new economic evaluation
was conducted. The best available data on clinical effectiveness on tamoxifen for
the adjuvant treatment of advanced breast cancer were not applicable to Ghana,
due to prior treatments or incorrect comparators. Indirect comparison was also not
possible. Therefore, the study used effectiveness data from two separate arms of two
studies: one from a clinical trial (Klijn et al. 2009) and the other from a cohort study
(Yang et al. 2010), to populate the model. Utility estimates were also transformed for
use in this context. Tamoxifen was cost effective at an ICER of USD 1,476 per QALY.
It would cost USD 276,927 per year to fund tamoxifen.  CONCLUSIONS: Even though
an HTA could be done with the available data, where possible, clinical trial design
should factor in the restrictions available to low resourced settings. Further research
is needed to develop utility weights specific to these settings
PCN105
INTERNATIONAL COMPARISON OF HTA DECISIONS FOR ORPHAN AND
NON-ORPHAN ONCOLOGY DRUGS
Achour L1, Hanna E2, Borissov BN3, Ma F4, Toumi M5
1ParisDauphine University, paris, France, 2Creativ-Ceutical, Paris, France, 3Prescriptia Ltd, Sofia,
Bulgaria, 4Creativ-Ceutical, Beijing, China, 5Aix Marseille University, Marseille, France
OBJECTIVES: The aim of this study was to assess the difference in Health Technology
Assessment (HTA) requirements between Oncology Orphan Drugs (OODs) and non-
OODs by comparing HTA decisions for these products in Canada, Germany, and
France.  METHODS: Oncology products approved in Europe between 01/01/2015 and
01/02/2018 were extracted from European Medicines Agency website. HTA reports
published by HTA bodies in Canada, Germany, and France were reviewed to identify
final decisions and the main limitations criticized.  RESULTS: Forty-two oncology
products (17 OODs and 25 non-OODs) were approved in Europe. Positive recom-
mendations rates were similar for OODs and non-OODs in Germany and France
(91% and 100% respectively), whereas in Canada more non-OODs were recom-
mended than OODs (80% non-OODs versus 63% OODs). The most challenged data
by the three HTA bodies were clinical data related to efficacy, safety, and inappropri-
ate study design. In Germany, ODs evidence is, by-law, less challenged than that of
non-ODs. However, in France, all OODs dossiers were challenged especially regard-
ing trial designs and efficacy data, versus 78% of non-OODs dossiers. In addition,
France challenged significantly economic models for OODs only. Canada identified
limitations on economic modelling for OODs and non-OODs, including inappro-
priate extrapolation of immature data in the modelling of non-orphan drugs and
underestimation of utility and costs due to post-progression in the modelling of
orphan drugs. Quality of life data seems to be a more critical outcome for OOD in the
3 countries.  CONCLUSIONS: Despite less compelling evidence and substantial trials
limitations, OODs achieve similar HTA recommendation as non-OODs in France
suggesting a more flexible attitude in the deliberative process. Similar findings in
Germany are driven by mandatory additional benefit for ODs. While in Canada,
OODs achieve a lower positive recommendation than non-OOD suggesting a more
strict deliberative process.
CONCEPTUAL PAPERS - Health Care & Health Policy Conceptual Papers
PCP1
VALUE-BASED HEALTHCARE DELIVERY - A SUCCESS STORY FOR TAIWAN ?
Chen I, Hessel F
SRH University Berlin, Berlin, Germany
OBJECTIVES: Healthcare systems all over the world struggle with increasing expenses,
limitations of the financial resources of societies and organizational deficits. Although
facing these generic problems the Taiwanese healthcare system has a high reputation
amongst Asia. This research aims to analyze if and to what extent the aspects of the
value-based healthcare concept of Micheal Porter designed for the US is currently
implemented in Taiwan.  METHODS: According to the framework of Porter the six
fields of the value-based healthcare concept were identified and analyzed for the
current Taiwanese healthcare system: Integrated practice units (IPU), evaluation of
cost and outcomes on individual patient level, bundled payments, care delivery across
facilities, geographical expansion and a common IT platform. The extent of potential
gaps is described.  RESULTS: Already 20 years ago the concept of IPUs was started in
Taiwan. In the pilot project could be evaluated on a patient-level and showed lower
cost combined with outcome benefits. Despite the success demonstrated for some
pilot projects the concept was not yet rolled out. In contrary the evaluation of patient-
level data was widely implemented in the system. The main gap in the claims review
system is seen within the quality and the acceptance of the reviewers. Over the last
decades Taiwan successfully moved to a bundled payment system. A geographical
expansion and cross-sectoral care was reached mainly by telemedicine which brings
access to medical care also in rural areas. The electronic database NHIA enables com-
prehensive analyses although continuous improvement is required.  CONCLUSIONS:
Taiwan could successfully implement all aspect of a value-based healthcare system
over the last decades. First evaluations are very promising but relevant gap remain.
Taiwan is seen as a flagship example for many other countries.
PCP3
NAVIGATING THE USE OF REAL-WORLD EVIDENCE IN EFFECTIVENESS
RESEARCH AND PATIENT ACCESS DECISIONS
Thwaites R1, Stegenga H2, Chambers M3, Garner S4, Jonsson P5
1Takeda, London, UK, 2National Institute for Health and Care Excellence, London, UK, 3MCHCE,
London, UK, 4World Health Organisation, Geneva, Switzerland, 5National Institute for Health and
Care Excellence (NICE), Manchester, UK
OBJECTIVES: Reimbursement and patient access decisions for new medicines
should be guided by relevant and good-quality evidence. Randomised controlled
trial (RCT) data are used to support these decisions, however real-world evidence
(RWE) could be considered too, especially in cases of accelerated regulatory access
where RCT data is less mature, in orphan disease areas where randomised trials
are difficult to conduct, or where RCTs do not reflect the patients or clinical prac-
tice outside of the trial setting. Stakeholders have different views of the accept-
ability of using RWE, therefore the GetReal Initiative’s (www.imi-getreal.eu) key
aims are to educate and stimulate discussion on the appropriate use of RWE in
medicines decision making. RESULTS: The Initiative has developed the “RWE
Navigator” (rwe-navigator.eu) which explains key evidence challenges in relative
effectiveness research and indicates potential uses for non-randomised evidence,
accompanied by overviews of data sources, study designs, quality assessment and
bias adjustment of RWE. The navigator also provides links to case studies that
demonstrate the potential application of RWE to support regulatory and patient
access decisions. It will continue to serve as a gateway to the ongoing work of the
GetReal Initiative.  CONCLUSIONS: The work of the GetReal Initiative indicates that
further dialogue is needed to develop policies and perspectives on RWE. The RWE
Navigator has been developed to stimulate and inform the debate on the use of
RWE in effectiveness research, and it has created a platform for a dialogue between
industry and decision-makers. It is a unique source of information on alternative
study designs and analytical methods, informed by key decision makers’ views on
the acceptability and usefulness of RWE, to inform health technology assessments
andpatient access decisions in Asia and elsewhere.
PCP4
A QUALITATIVE INVESTIGATION OF INFORMATION ASYMMETRY FOR OBESITY
SURGERY: DIVERSITY OF PATIENT EXPERIENCES IN THE INFORMATION AGE
AND DEMAND-INDUCED SUPPLY
Campbell JA1, Ezzy D2, Neil A1, Hensher M3, Venn A1, Sharman M1, Wilkinson S4,
Palmer AJ1
1University of Tasmania, Hobart, Australia, 2University of Tasmania, Sandy Bay,
Australia, 3Tasmanian State Government, Hobart, Australia, 4Royal Hobart Hospital, Hobart,
Australia
OBJECTIVES: A key market failure in healthcare is information asymmetry between
the consumer and supplier where the level of knowledge and expertise is weighted to
the supply-side (physician/surgeon). However, in the information-age, bariatric surgery
patients may be more empowered in their negotiated relationship with healthcare
providers. Importantly, information that empowers the consumer potentially enables
‘demand-induced-supply’ (patients demanding and receiving care that their clinician
would not otherwise have offered) and has implications for healthcare resource allo-
cation to bariatric surgery. The main objective of our study was to employ qualitative
research methods to investigate the concept of information asymmetry for bariatric
surgery patients in the information-age.  METHODS: Our study used the pre-existing
S24 VA L U E I N H E A LT H 2 1 ( 2 0 1 8 ) S 1 – S 1 1 5
health economics theory of information asymmetry to inform qualitative inductive
and deductive theory building about information drivers, sources and needs for bari-
atric surgery patients pre- and postoperatively. Ten semi-structured focus groups of
people who were waitlisted or had undergone bariatric surgery were conducted (n= 49).
Thematic analyses were employed to analyse verbatim transcripts.  RESULTS: The core
finding of our interpretive analysis was that irrespective of the sources and types of
information that the participants used in their decision-making, most participants’
decisions to undergo bariatric surgery would be unlikely to change . We also found
that psychosocial ‘drivers’ for information gathering and interpretation were funda-
mentally important before surgery and that the quality and consistency of validated
objective healthcare information became more important to participants after their
bariatric surgery. We also found that information sources relevant to participants pre-
operatively (e.g. family and friends, and the Internet) were different postoperatively
(surgeon, allied-health professionals e.g. psychologist).  CONCLUSIONS: It was only
after bariatric surgery that many participants fully acknowledged the complexity of
their psychosocial needs. We recommend that high-quality and consistent patient
information be more targeted towards the psychosocial domains of health preopera-
tively and ongoing postoperatively.
PCP6
ORPHAN DRUGS: HEALTH TECHNOLOGY ASSESSMENT AND PRICING IN EUROPE
Jarosławski S1, Toumi M2, Korchagina D3, Jadot G4
1Aix-MarseilleUniversity, Marseille, France, 2Creativ-Ceutical, Paris, France, 3Cr, Paris, France,
4Lyon 1 University, Villeurbanne, France
OBJECTIVES: We sought to review the approaches to health technology assess-
ment (HTA) and pricing of orphan drugs in Europe. METHODS: Literature
review.  RESULTS: There is no universal HTA decision framework for orphan drugs
and different jurisdictions focus on various HTA criteria, such as cost-effectiveness,
budgetary impact, disease severity, therapeutic need, social benefits etc. However,
standard HTA methods, such as cost-effectiveness analysis are not compatible
with orphan drugs due to: 1) very limited clinical data and the use of surrogate
endpoints, rather than patient-relevant endpoints; 2) a comparative assessment
may be complicated by the lack of data on the natural history of the disease and the
absence of an established standard-of-care treatment with known efficacy; 3) cost-
effectiveness ratios are above acceptable thresholds due to a very high per patient
cost. Consequently, lower significance levels for p-values (e.g. 10% significance lev-
els) for small sample sizes, as well as the evidence from surrogate endpoints rather
than only patient-relevant endpoints can be accepted and special legal frameworks
for orphan drugs have been developed in some countries. These may consider
the profits made by the company, the nature of the condition, the impact of the
new technology, the cost for the national healthcare system, value for money, and
the impact of the technology beyond direct health benefits.  CONCLUSIONS: HTA
and pricing of orphan drugs cannot follow the standard pathways and individual
countries apply various ad hoc frameworks to enable the reimbursement of these
drugs in their national health systems. This has led to inequality in access to these
drugs among jurisdictions while the prices of the drugs remain very high. This calls
for the development of universal guidelines on HTA methods for orphan drugs and
for policy changes in order to ensure the sustainability of health care systems.
PCP7
THE EUROPEAN ADPKD FORUM (EAF): TRANSLATING SCIENCE INTO POLICY TO
IMPROVE ADPKD PATIENT CARE IN EUROPE
Roebuck D
Otsuka Pharmaceutical Europe Ltd, Wexham, UK
To demonstrate the long-term impact of implementing an international, multi-
disciplinary, expert working-group; dedicated to translating scientific and clinical
advances into policy, to improve health and quality of life of people living with
autosomal dominant polycystic kidney disease (ADPKD). Between 2013-2018 leading
patient advocates, healthcare professionals and academics, representing nephrol-
ogy, hepatology and genetics, have come together as the EAF, to develop policy-
focused approaches to address unmet needs in ADPKD care in Europe. Focusing on
low-level disease awareness amongst policymakers, practitioners and payers; varia-
tions in care; and absence of approved treatment options; the EAF has engaged with
patient advocacy organisations and professional society representatives through
publications, multi-stakeholder policy-events, conference activities, media releases
(press, social, video), targeted communications and advocacy toolkits. The EAF
Report and Brussels Declaration on ADPKD (co-published with PKD International
and EGAN. 2015) provides strategic, policy-focused recommendations empowering
stakeholders to become active partners in healthcare, policymaking, service design
and research. Endorsed by 32 national and international societies they informed
multiple national ADPKD collaborations. The Multidisciplinary Position Statement
on ADPKD Care (Nephrol Dial Transplant. 2017) advocates for holistic patient-cen-
tered care services, referencing EU policy initiatives and achieving strong impact
metrics. The ADPKD Patient Route Map (2018) enables patients to navigate their
ADPKD services and provides checklists for self-management enhancing shared
and informed decision-making. The EAF has provided a collaborative platform
for clinical experts and patient advocates to raise awareness and develop impact-
ful policy-focused recommendations, together with communications and tools
designed to improve real-world patient care and empower patients. Multi-sector
and multi-channel outreach has aided impact across clinical, academic, patient
and policy-making communities. Advances in ADPKD services have included estab-
lishment of additional specialist centers and the European Reference Network for
Rare Kidney Diseases, both advocated for by the EAF Report. However, variations in
ADPKD services remain across Europe and warrant further work.
PCP8
EXTERNAL REFERENCE PRICING IN UKRAINE: IMPLEMENTATION AND IMPACT
Piniazhko O1, Zaliska O1, Irynchyna I2, Irynchyn H3, Brezden O4
1Danylo Halytsky Lviv National Medical University, Lviv, Ukraine, 2Kyiv National Economic
University named after Vadym Hetman, Kyiv, Ukraine, 3Ukrainian Center for Scientific Medical
Information and Patent License of Ministry of Health of Ukraine, Kyiv, Ukraine, 4Jagellonian
University, Krakow, Poland
OBJECTIVE: In the international practice external reference pricing (ERP) is a power-
ful tool implemented by governments to inform or set pharmaceutical prices in a
given country. The study aimed to analyze reimbursement programs and quality
of ERP system implemented by Ministry of Health of Ukraine in 2017.  METHODS:
We benchmarked findings from the literature review against 14 basic principles
that an optimal ERP system should follow (Kanavos et al. 2017). Literature review
was conducted examining a number of defined endpoints relating to the structural
elements of ERP.  RESULTS: EPR and reimbursement program “Affordable Medicines”
launched in April 2017 for cardiovascular diseases, type 2 diabetes and asthma with
the objective to lower pharmaceutical prices and increase affordability of medicines
in this program (according to Resolutions of the Cabinet of Ministers of Ukraine
No.862 “On state regulation of prices on medicines” dated 09.11.2016 and No.863 “On
implementation of reimbursement” dated 09.11.2016). Ministry of Health is compe-
tent authority in ERP implementation. There is presence of an appeals process for
stakeholders. Reference prices are calculating based on the prices in 5 neighboring
countries: Poland, Latvia, Slovakia, Hungary and Czech Republic. Marginal wholesale
prices are calculating as a median of reference prices for such medicines in the
reference countries with terms DDD as recommended by WHO. Price list obtained
from official sources of authorized state bodies of reference countries: official prices,
ex-factory prices, reference prices. There is fixed exchange rate and no inclusion of
wealth adjustments.  CONCLUSIONS: Implementation of EPR and reimbursement
program has already demonstrated its first positive results according to interna-
tional good practice: impact on cost-containment of medicines, decrease by 10.5%
the average weighted value of 1 DDD of medicines in 2017, comparing to the same
period of previous year and intention to expand the reimbursement list by govern-
ment in Ukraine.
PCP9
NON-PROFIT DRUG RESEARCH AND DEVELOPMENT AT A CROSSROADS: THE
CASE STUDY OF GENETHON
Jarosławski S1, Toumi M2, Auquier P1, Dussart C3
1Aix-Marseille University, Marseille, France, 2Creativ-Ceutical, Paris, France, 3Lyon 1 University,
Lyon, France
OBJECTIVES: In wealthy nations, non-profit drug R&D has been proposed to reduce
the prices of innovative medicines. We sought to analyse Genethon, a French non-
profit biotherapy R&D organisation focused on rare diseases. We discuss the ethical
and economic issues concerning non-profit drug R&D companies, and the possible
impact that their pricing strategy may have on the innovation efforts from for-profit
companies targeting the same segment of the pharmaceutical market.  METHODS:
Published and grey literature review.  RESULTS: There are two possible approaches
to pricing drugs developed by non-profit R&D programs: pricing that maximises
profits and “affordable” pricing that reflects the cost of manufacturing and distri-
bution, plus a margin that ensures sustainability. Genethon has opted for in-house
production and distribution in order to maintain control over the supply of its prod-
ucts and sell them at a ‘fair and controlled price’. The firm has decided to occupy
the whole chain of value creation, from discovery to large-scale manufacturing
and sales. However, it lacks the experience in launching, selling, and distributing
medicines, which may delay or jeopardize patient access to Genethon’s innovative
therapies. Ethical issues include spending public funds on commercial sales activity
and investing them into a sales venture that will bring a return on investment much
below the potential return in the for-profit sector. Further, lower pricing constitutes a
disincentive for the for-profit sector to develop further products in the same disease
area and the risk of drying out the for-profit sector R&D is high.  CONCLUSIONS:
Since the antitrust law does not permit non-profits to impose prices on poten-
tial licensees, these companies face ethical challenges when making decisions on
licensing and pricing their inventions. Licensing their products to for-profit com-
panies will bring high royalties that could be reinvested into further R&D, but will
also result in high drug prices.
PCP10
EVALUATION OF INCREASING TREND OF PHARMACOECONOMICS AND
OUTCOMES RESEARCH IN LEADING INSTITUTES OF INDIA
Ligade VS, Udupa N
Manipal College of Pharmaceutical Sciences, Manipal Academy of Higher Education, Manipal,
India
Pharmacoeconomics, health outcomes research and patient-reported outcomes
in particular focuses on understanding patient value in terms of impact of dis-
ease and its treatment on physical functioning and psychosocial wellbeing.
Pharmacoeconomics and health outcomes research is developing field in India. To
study and find out the trend of pharmaceconomics and outcome research contrib-
uted by Indian institutions. In order to meet the objective, required information
was collected through scientific presentations database of International Society
for pharmacoeconomics and outcomes research (ISPOR). The scientific presenta-
tions database was searched with key word “India”. The website includes presen-
tations from all ISPOR international meetings since 1998. The abstracts published
till 2017 were included in the study. From the ISPOR scientific database search
it was found that research abstract paper in ISPOR meetings have been steadily
increasing with around 573 papers being published by Indian authors. 198 research
abstract papers were published with foreign authors, which is most encouraging
trend. In India, some of the leading group of institutions that had published research
abstract papers were - Manipal Academy of Higher Education (142), NIPER (47),
Novartis Healthcare Pvt. Ltd. Hyderabad (28), JSS College of Pharmacy (31), PAREXEL
International, Chandigarh (18), Tata Consultancy Services, (12), and Chalapathi
Institute of Pharmaceutical Sciences, Guntur (10). Other leading institutes are also
discussed in the study. Indian researchers at some leading institutes are carrying
out research along with foreign researchers, which is encouraging trend. At present