Masters Degrees For Health Economists: Investigating And Evaluating Their Content

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PHP269 Dexhelpp - Aging Population: Routine Data Based Analyses Of Fractures Due To Falls Less Than Three Meters – Hospitalization, Readmission And Mortality Zauner G1, Wilbacher I2, Popper N3, Breitenecker F4 GmbH, Vienna, Austria, 2Main Association of Austrian Health Security Institutions, Vienna, Austria, 3DEXHELPP, Vienna, Austria, 4TU Wien, Vienna, Austria

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Objectives: Within DEXHELPP (Decision Support for Health Policy and Planning: Methods, Models and Technologies based on Existing Health Care Data) fractures due to falls less than three meters for the elderly are identified as one big health problem. Primary prevention strategy evaluation for new raising policies is of interest. To parameterize these models/strategies, a detailed analysis of the state of the art is essential. This part, especially readmission and mortality are the key questions.  Methods: Claims data (GAP-DRG) in the Austrian health care sector is used including many details for patient care. GAP-DRG (General Approach for Patient oriented Outpatient-based DRG) is a research data base with reimbursement data for outpatient services of sickness funds (social insurance) and Federal Ministry of Health (hospital data). Based on ICD10 diagnosis subgroups of fracture categories are analysed for the years 2006 and 2007. Diagnoses are grouped into severity classes and logistic regression as well as Cox-regressions are performed.  Results: In total 32343 patients older than 60 years with main fracture ICD10 diagnosis are identified and followed up for one year. During that time 4596 persons died. Low and moderate disease severity have a vastly reduced mortality rate. For low severity it is only about 1/3 and for moderate severity 2/3 compared to high disease severity with 21% annual mortality. In case of readmission sever fractures result in at least 19,1% cases.  Conclusions: The realized work represents the actual Austrian situation. Additional analysis including longer wash-out phase seems promising. Due to the fact, that a high number of persons is affected, the socio-economic impact is shown. The results reflect the findings from international studies and could be used for parametrization of prevention strategy evaluation models. PHP270 Attributes Defining Patient Engagement And Centeredness In Health Care Research And Practice: A Framework Developed By The Ispor Patient-Centered Special Interest Group Hanna ML1, Oehrlein EM2, Perfetto EM2, Astratinei V3, Berner T4, Burke LB5, Camp R6, Hareendran A7, Harrington R8, Houÿez F9, Patrick DL10, Scott A11, von Gizycki R12, Wheeler R13 1School of Pharmacy, University of Maryland, Baltimore, MD, USA, 2University of Maryland, School of Pharmacy, Baltimore, MD, USA, 3Melanom Romania, Bucharest, Romania, 4Baxalta, Bannockburn, IL, USA, 5Study Endpoints and Labeling Development, ONDIO, CDER, U.S. Food & Drug Administration, Silver Spring, MD, USA, 6European Patients’ Academy (EUPATI), Barcelona, Spain, 7Evidera, London, UK, 8Astellas Medical Affairs Americas, Northbrook, IL, USA, 9EURORDIS, Paris, France, 10University of Washington, Seattle, WA, USA, 11SEAS Capital Partners, Plymouth, MI, USA, 12Pro Retina Europe, Berllin, Germany, 13Leber Hereditary Optic Neuropathy, Winchester, UK

Objectives: ISPOR’s Patient Engagement in Research Working Group convened to standardize terms and definitions describing patient engagement and centeredness for use in ISPOR work and communications. To inform its work, a systematic review was conducted to identify existing definitions for “patient engagement,” “patient centeredness,” and related terms in the context of research and health care practice. Themes derived from identified terms and definitions were used to develop draft definitions for ISPOR use.  Methods: Peer-reviewed literature was searched from January 1, 2006 to March 31, 2016 using PubMed and EMBASE. The list of terms searched included “patient” with: “engagement”, “involvement”, “participation”, “centeredness”, “focused”, and “oriented”. Abstracts were screened for relevance independently by 2 researchers, followed by full text review to extract definitions, and then assembled in an Excel database. Disagreements were resolved by consensus; a third researcher resolved remaining discrepancies. A priori inclusion criteria required: a definition of a pre-specified term, association with health care research or provision, and in English language. Definitions were abstracted and classified by country, context of use, contributing stakeholder group, and whether original versus being cited. Grounded theory methods were used to identify emergent themes which were incorporated into a framework of meaningful attributes for defining “patient engagement” and “patient centeredness”.  Results: Abstracts for 1371 publications were reviewed for terms and definitions. Analyses of identified and categorized definition themes (e.g., patient-driven outcomes and assessing risk/benefit) will be discussed. Final themes will be presented (pending full-text review completion); draft definitions will be provided for discussion and debate.  Conclusions: The resulting themes lay the foundation for draft recommended ISPOR definitions for “patient engagement” and “patient centeredness” and are put forth by the Patient Engagement in Research Working Group for consideration by the ISPOR membership. PHP271 Pharmacotherapy Consultations By Clinical Pharmacists To Improve Quality And Safe Use Of Medicines: An Experience From Tertiary Care Teaching Hospital Himanshu Patel, Parthasarathi G, Madhan R JSS College of Pharmacy, Mysore, JSS University, Mysore, India

Objectives: This study was conducted to assess the impact of pharmacotherapy consultations provided by clinical pharmacists (CPs) on improving quality and safe use of medicines.  Methods: Clinicians from various specialties referred the patients to clinical pharmacy department for consultation. All the consultation requests received by the department were categorized based on its purpose; detection & management of adverse drug reactions (ADRs); individualized drug therapy; medication counseling; assessment of drug interactions; others. CPs

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attended all the referred requests and provided their opinion(s) to respective clinicians. They were documented electronically and analyzed further to assess its impact on patient care.  Results: A total of 784 referral requests were made during 12 months period. Majority of patients were referred by dermatologist (41.5%) followed by surgeons (15%), pulmonologists (14.5%), physicians (11%) and pediatricians (7.14%). Patients were referred for assessment of suspected drug reactions (48%), individualized drug therapy and dose calculations (20%), receiving medication counseling (18%), assessment of drug-drug interactions (8%), safe use of drugs in pregnancy (4%), selection of drug therapy for high risk conditions (3%). From CPs’ consultation, 340 ADRs were detected, from which 56 patients were given alert card and 104 patients were discontinued with current medications to reduce the severity of ADRs. Ongoing therapy of the 156 patients was modified in terms of drug, dose and frequency to improvise the therapeutic benefits and patient safety. 141 patients with chronic diseases and polypharmacy were educated for appropriate use of drugs and devices. Medication orders were changed for 54 patients due to significant drug-drug interactions. Nearly for 28 patients safer drugs were suggested considering their pregnancy and lactation stage. Around 96% of the opinions given were implemented by clinicians to enhance the patient care.  Conclusions: Implementation of Pharmacotherapy referral service by CPs was beneficial in improving rational drug use and was appreciated by clinicians. PHP272 Advanced Therapies: Widening The Gap Between Payers And Regulators Hanna E1, Rémuzat C2, Auquier P1, Toumi M1 1Faculté de Médecine, Laboratoire de Santé Publique, Aix-Marseille Université, Marseille, France, 2Creativ-Ceutical, Paris, France

Objectives: Advanced Therapy Medicinal Products (ATMPs) are innovative therapies including gene therapies, cell therapies, and tissue engineered products. These therapies are expected to halt or cure many chronic, disabling diseases. While European regulators tend to speed market access of such therapies through accelerated pathways (authorisation under exceptional circumstances, conditional marketing authorisation, accelerated assessment, adaptive pathways and PRIME), health technology assessment (HTA) bodies/payers are increasingly scrutinising the incremental value of these products. The study objective was to identify potential gap in the evaluation of ATMPs between payers and regulators in Europe.  Methods: A search was conducted in European Medicines Agency (EMA) website to identify ATMPs approved in Europe; HTA assessment of these ATMPs was reviewed for France, Germany and United Kingdom through HAS, IQWiG/G-BA, NICE and SMC websites.  Results: Seven ATMPs received a marketing authorisation (MA) in Europe until June 2016: Chondrocelect® (2009), Glybera® (2012), MACI® (2013)-MA suspended in 2014, Provenge® (2013)- MA withdrawn in 2015, Holoclar® (2015), Imlygic® (December 2015), Strimvelis® (May 2016). None of these ATMPs has been recommended for reimbursement by HTA bodies in the study scope. Only Chondrocelect® is reimbursed on a case-by-case basis in Germany after negotiation between hospital and appropriate regional health insurance. HAS did not recommend Chondrocelect® and Glybera® due to insufficient actual benefit. NICE considered that Provenge® was not cost-effective and did not meet the criteria for end-of-life consideration. Glybera® and Provenge® were rated as “nonquantifiable added benefit” by IQWiG/G-BA. Holoclar®, Imlygic® and Strimvelis® have not yet been assessed.  Conclusions: EMA is accelerating the regulatory pathways for innovative products whereas HTA bodies tend not to recommend ATMPs for reimbursement mainly because of immature data. Parallel advice may help harmonizing HTA and regulators’ perspectives and provide manufacturers recommendations to achieve market access for ATMPs, while more than 900 ATMPs are in development. PHP273 Masters Degrees For Health Economists: Investigating And Evaluating Their Content Mildred M University of Sheffield, Sheffield, UK

Objectives: To investigate the content of Masters Degrees for health economists, measuring and evaluating the perceived importance of their constituent modules.  Methods: Universities in the UK were identified using higher education funding data on all institutions which received nationally allocated funding. Potentially relevant post-graduate courses were systematically identified directly from institutions’ own websites using a pre-defined search strategy. Results were screened using inclusion/exclusion criteria, with eligibility assessed based on a detailed examination of the course curricula and syllabuses. Module level data were extracted and codified using a recursive algorithm to enable data analysis. A questionnaire was developed using the module data and an online survey was sent to 100 industry professionals via the LinkedIn professional social network.  Results: 713 potentially relevant courses from 57 institutions were identified and screened; of which 21 courses from 12 universities were deemed relevant for syllabus data extraction. 141 compulsory/core, modules were codified into 26 discrete modules. The most commonly taught modules were: (1) Economic Evaluation/Cost-effectiveness Modelling; (2) Health Economics/Pharmacoeconomics; (3) Epidemiology; (4) Health Services Research Methods; and (5) Health Policy & Regulations. Based on respondents preferences (n= 47), modules 2 and 1 were rated the most important, followed by HTA, Statistics/biostatistics, Valuing Health, and Systematic Review & Meta-analysis. In contrast, Epidemiology was rated the 10th most important module whilst Health Services Research was only ranked 13th.  Conclusions: The content of Masters Degrees for health economists varies widely; however there appears to be a set of modules fundamental to all degrees. Gathering the value-judgements

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of experts in the field of health economics has highlighted areas in which degrees could be modified to make them even more desirable or relevant, however an important matter still remains – who is best placed to determine the content of Masters degrees – academia, industry, or both in collaboration? PHP274 A Framework To Guide The Use Of Real-World Evidence To Support Evaluation Of Relative Effectiveness Of New Medicines Stegenga H1, Chambers M2, Jonsson P3, Thwaites R4, Garner S5 Institute for Health and Care Excellence, London, UK, 2MCHCE, London, UK, 3National Institute for Health and Care Excellence (NICE), Manchester, UK, 4Takeda, London, UK, 5National Institute for Health and Care Excellence (NICE), London, UK 1National

Objectives: Payers and health technology assessors (HTAs) are interested in evidence of effectiveness outside the clinical trial setting. The use of real-world data (RWD) to estimate relative effectiveness before marketing authorisation presents a variety of operational and methodological issues. An objective of the IMI GetReal project (www.imi-getreal.eu) is to develop a decision-making framework to guide the potential use and generation of RWD, and inform the design of strategies to provide better information about relative effectiveness of new medicines.  Methods: Work Package 1 of GetReal engaged multiple stakeholders to develop the decisionmaking framework, including patient organisations, clinicians, academic specialists, clinical trialists, pharmaceutical companies, European regulators, HTA bodies and payers. Case study workshops in five disease areas were used to examine different analytical methods and study designs using RWD to provide estimates of relative effectiveness, and to elicit stakeholders’ views on the usefulness and acceptability of each option.  Results: The framework consists of five key components: 1) overview of current policies related to real-world evidence (RWE) of effectiveness; 2) comprehensive review of sources of RWE; 3) catalogue of real-world study designs outlining key characteristics; 4) description of state of-the-art analytical approaches making use of RWD; and 5) stakeholder perspectives on the use of RWD for decision making. The core of the framework is a decision-making algorithm taking stakeholders through questions on the potential need for RWE and subsequently the potential RWD analyses and study designs.  Conclusions: A wide range of stakeholders provided views on the value and acceptability of different RWD study designs and analyses. The resulting framework will help pharmaceutical R&D to assess options for integrating real-world study designs in medicine development programmes, support engagements between pharmaceutical R&D and decision-makers and drive forward the scientific and policy agenda in this area. PHP275 Japan Health Care Act Reform: Toward Decentralization And Integrated Health Care System Toumi M1, Fukushima A2, Murata K2, Onishi Y2 1Faculté de Médecine, Laboratoire de Santé Publique, Aix-Marseille Université, Marseille, France, 2Creativ-Ceutical, Tokyo, Japan

Objectives: Japanese health care system experienced important changes to cope with population ageing. The government passed a new Health bill in 2015 to complement the 2012 one. The objective of this research is to describe how those two reforms will impact the health care landscape in Japan.  Methods: We did a literature review and analysed health care reform acts of 2012 and 2015  Results: Main directions are: Enhanced decentralisation with transfer of power to Prefecture (regional government). Regional governance will be established to accountably manage health plan. Prefectures are responsible for health care plan for cancer, stroke, acute myocardial infarction, diabetes mellitus, psychiatric disease, pediatric care. But also to develop home care delivery. Integrated health care are under Prefecture responsibility with case managers who coordinate health care for chronic and multimorbid patients. More than 300 diseases are eligible to case management. Equity in health care access is high on the agenda to ensure equitable accessibility to health services. the two markers will be: mortality and use of expensive therapies (with special focus on oncology). New incentives to encourage patients to use GP for hospital referral and discourage patients self-decision. At the same time large hospitals will be requested to ensure health care coordination with all community based providers to prevent admission and re-admision. Hospital ensuring postdischarge follow-up in coordination with ambulatory care providers will receive subsidies The unique social security and tax number will be established in 2018 to support the extension of E-health record networks currently piloted in targeted catchment area.  Conclusions: The Japanese Health care system is operating a reform toward prefecture accountability, and health care integration supported by development of unique E-Health record, incentive for information sharing, case managers, and strengthening GP’s role. This process is established smoothly but should improve health care quality and cost.

HEALTH CARE USE & POLICY STUDIES – Health Technology Assessment Programs PHP276 Understanding Health Technology Assessment (HTA) Bodies in Major European Markets: Systematic Evaluation in 10 EU Countries Nanavaty M, Gala S, Nyandege A, Ramesh V, Mwamburi M Market Access Solutions LLC, Raritan, NJ, USA

Objectives: Value of new drugs are evaluated through HTAs in at least 48 countries. Their influence on evidence-based healthcare decision-making includes determining level of coverage in Europe. The characteristics, attributes and methodologies employed by HTA bodies vary significantly. Navigation through the HTA application process in multiple countries is a challenging endeavor. The objective was to characterize the different HTA methodologies used in major European markets.  Methods: We conducted a systematic review of HTA bodies’ documentation of 10 markets in Europe (England & Wales, France, Germany, Italy, Netherlands,

Poland, Scotland, Spain, Sweden and Switzerland) and on peer-reviewed publications through PubMed. For Italy and Spain, a prominent regional HTA was evaluated, Lombardy and Catalonia respectively. Over 350 HTA characteristics, attributes assessed and methodologies used were assessed.  Results: Among 10 HTA systems evaluated, England & Wales (NICE), Netherlands (CVZ) and Poland (AOTM) use explicitly stated incremental cost-effectiveness ratio (ICER) thresholds of £20,000-£30,000/QALY, € 40,000/QALY and € 26,500/QALY respectively. Three markets use implicit ICER thresholds based on the cost-effectiveness studies of previously approved drugs/technologies. Seven HTA bodies involve the general public, 6 publish reports online, 5 review within 2-6 months, and 3 allow for some form of appeal. We describe the various characteristics, attributes, and methodologies by payer architypes across Europe and discuss the associated pros and cons. Numerous HTA bodies provide support during submission process but others lack important information in the public domain.  Conclusions: HTA bodies vary significantly and can be categorized as ones using explicit ICER thresholds and ones using implicit ICER thresholds in addition to other factors. Understanding the nuances related to HTA evaluation processes in multiple countries regarding involvement the general public, duration of review process, and ability to appeal, methods of technology assessment, preference on active controls, or availability of submission support will help manufacturers plan accordingly. PHP277 Concordance of Health Technology Assessment Recommendations of Diabetes, Hepatitis, Oncology and Epilepsy Pharmaceutical Products by Five Agencies within Europe: An Interrater Reliability and Association Analysis Abugri I1, Braun D1, Manfred E1, Gerbsch N2 1Cardiff University in collaboration with Hochschule Fresenius, Idstein, Germany, 2Bundesverband der Pharmazeutischen Industrie e.V, Berlin, Germany

Objectives: In recent years, a “fourth hurdle” is increasingly adopted by health care systems within Europe that requires product sponsors to show value for their products, usually at national levels. Thus, the same product is assessed by different national agencies. Therefore, this study seeks to assess the level of agreement HTA recommendations of similar product-indication pairs across five countries, test the association between HTA agencies and HTA recommendations. Methods: HTA recommendations on Diabetes, Hepatitis, Oncology and Epilepsy pharmaceutical products appraised by IQWiG, HAS, NICE, SMC and ZIN between January 2011 and 15th March 2016 were comparatively analyzed. Products were included if at least two HTA agency appraises it within the study timeline. Absolute percent agreement and Kappa statistic were used to measure the level agreements. The null hypothesis of independence between HTA agencies and recommendations they issue were explored using correspondence analysis and the strength of association evaluated.  Results: There was a significant discordance in HTA recommendations across study agencies with an average percentage of divergence of 69.2%. The level of agreement varied between agencies and across therapeutic areas. Agreement between IQWiG and other agencies was poor to slight in all therapeutic areas except with HAS in the appraisal of Diabetic products. Between NICE and SMC was moderate to strong agreements. The strength of association between agencies and their HTA decisions, measured in Cramer` V, were moderate for diabetes and hepatitis products (0.639 p< 0.000 and 0.535 p< 0.000) and low for oncology products (0.327 p< 0.000).  Conclusions: It was concluded that disparity in HTA recommendations among agencies exists leading to a possible non-uniform market access of pharmaceutical products across study countries. Differences in methodology, value judgment, and preferences were the major drivers of divergence. Thus, appreciation of agency-specific preference and value judgment are important to the MAH in obtaining positive HTA outcomes. PHP278 Cadth’s $50,000 Cost-Effectiveness Threshold: Fact or Fiction? Griffiths EA1, Vadlamudi NK2 1PAREXEL, London, UK, 2PAREXEL, Hyderabad, India

Objectives: The Canadian Agency for Drugs and Technologies in Health (CADTH) does not formally state a cost-effectiveness threshold for new medicines. A threshold of CAD$50,000 per quality-adjusted life-year (QALY) is often cited as a benchmark, but there is limited evidence to confirm the accuracy of this figure. To inform future submissions, CADTH appraisals were reviewed to determine whether a cost-effectiveness threshold can be inferred.  Methods: All publicly-available CADTH appraisals (assessed under the common drug review [CDR] or pan-Canadian Oncology Drug Review [pCODR]) from January 2010 to May 2016 were reviewed. The decision, rationale, and economic analysis presented were extracted.  Results: Between January 2010 and May 2016, CADTH published 279 drug appraisals: 215 under CDR and 64 under pCODR. 29 submissions (10%) were recommended, 163 (58%) were recommended with restrictions, and 87 (31%) were not recommended. Cost-utility analyses were presented in 63/64 pCODR submissions and 83/215 CDR submissions, with other forms of economic analysis (e.g. cost-minimization, cost comparison) in the remaining submissions. Incremental cost-effectiveness ratios (ICER, expressed in cost per-QALY) were often revised upwards by the review committee, and submissions were commonly recommended with restrictions conditional on improved cost-effectiveness. In several appraisals, CADTH included wording such as “a [X%] price reduction would be required for the ICER to fall to [~$50,000]” or “the ICER was < $50,000 per-QALY in most scenarios”, suggesting that a $50,000 threshold may have been considered. However, the range of ICERs for recommended submissions was dominant to $131,100, with several being accepted with ICERs > $50,000.  Conclusions: There is some evidence to suggest that CADTH’s cost-effectiveness threshold is ~$50,000 per-QALY, but this is neither consistently applied nor explicitly stated. Further, CADTH will often make decisions based on alternative forms of economic analysis. Although cost-effectiveness analysis has limitations, formally stating a cost-effectiveness threshold would improve transparency and may improve equitable access to new medicines.