Advanced Therapies: Widening The Gap Between Payers And Regulators

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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