Early Payer Scientific Advice Procedures In The Eu: Which One Is Most Suited For Pharmaceutical Compaines?

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PHP212 Early Payer Scientific Advice Procedures In The Eu: Which One Is Most Suited For Pharmaceutical Compaines? Wolfram V1, Bailey S1, Foxon G1, Craddy P2 Consulting, Cheshire, UK, 2Remap Consulting, Zug, Switzerland

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Objectives: To understand the procedures and outputs from different European payer scientific advice procedures (PSAPs) to determine the most suitable approach for pharmaceutical companies.  Methods: The procedures for four major European PSAPs (NICE, G-BA, EMA joint scientific advice, and SEED’s multi payer pilot program) were analyzed. Data relating to: evidence submission requirements; processes; timelines; fees; submissions conducted; outputs were extracted and compared between the four PSAPs.  Results: There are multiple ways to seek payer guidance, each with distinct procedures for securing advice. Across Europe, the number of PSAPs procedures increased > 3 fold between 2011 and 2015. All PSAPs are resource and time intensive, requiring input from clinical development, regulatory, health economic, and market access teams. PSAPs take between 8 weeks in Germany to ~20 weeks for the SEED program to complete. PSAP fees vary significantly (between € 0 to ~€ 40,000) depending on the number of questions sought and payers involved. All PSAPs provide insights into clinical endpoints, level of clinical benefit required, and clinical trial design. NICE, either individually or within a multi-payer approach, also provides health economic guidance. All PSAPs have a face-to-face meeting with payers. The G-BA, NICE and SEED provide written meeting minutes, whereas in the EMA PSAP, the company submits the meeting minutes to payers for comments.  Conclusions: PSAPs can provide pharmaceutical companies with valuable insights into phase III trial design, health economics, and payer expectations at time of launch. The level of product and therapy area uncertainty can drive PSAP selection. If there is significant uncertainty, national advice procedures (e.g. NICE, G-BA) should be sought to gain detailed country specific insights, but this must be balanced against the additional cost and resource implications. EMA PSAP is most suited for products with limited uncertainty where companies are looking for confirmation that the clinical program is acceptable to payers across multiple markets. PHP213 Comparing Eq-5d-3l And Eq-5d-5l In A General Population Ferreira LN1, Ferreira PL2, Pereira LN3, Antunes P2 1Universidade do Algarve, Faro, Portugal, 2CEISUC, Coimbra, Portugal, 3Research Centre for Spatial and Organizational Dynamics (CIEO), Faro, Portugal

Objectives: The EQ-5D is a generic preference-based measure of health for use in economic evaluation. To improve this instrument’s sensitivity and discriminatory power and to reduce the ceiling effect reported for some respondents, the EuroQol Group has recently expanded this instrument by adding two intermediate response levels (EQ-5D-5L) defining a total of 3,125 health states. This study aimed to compare both EQ-5D versions using data from two representative samples of the Portuguese general population.  Methods: EQ-5D-3L data was available from a sample of 1,500 respondents and EQ-5D-5L data was available from 1,000 participants. Both samples were representative of the general Portuguese population according to age, gender and regions. Data was compared in terms of feasibility (missing values), inconsistency, ceiling effect, level of agreement, discriminative properties, knowngroups validity and health status reported in the descriptive system.  Results: Though both instruments showed a good feasibility, the EQ-5D-5L had a higher completion rate, which is consistent with other studies. Our findings show that participants used all new levels within each of the dimensions. Quantitative results showed a ceiling effect reduction and a high level agreement between both indices. Known-groups validity was confirmed for both indices. Regarding the discriminative properties, the indices did not present statistically significant differences in their discriminatory capability. A wider spread of health states was reported in the EQ-5D-5L.  Conclusions: The EQ-5D-5L version contributed to a significant reduction in the ceiling effect. The EQ-5D-5L performed better than the EQ-5D-3L and appears a valid and reliable extension of the three-level system.

PHP214 Validation Of Comorbidity Indices (Charlson And Elixhauser) Computed In Cross Sectional Hospital Billing Data Wei D1, Narain A1, Lerner J2, Yoo A1, Kalsekar I3 1Epidemiology & Health Informatics, Medical Devices, Johnson and Johnson Co., New Brunswick, NJ, USA, 2DePuy Synthes, Inc., Raynham, MA, USA, 3Johnson & Johnson Co., New Brunswick, NJ, USA

Background: Charlson and Elixhauser comorbidity indices are frequently used in observational studies for patient comorbidity risk adjustment. Cross sectional hospital billing databases may underestimate patient comorbidities when compared to longitudinal databases such as administrative claims.  Objectives: To compare Charlson and Elixhauser comorbidity indices and the individual comorbid categories calculated from a cross sectional hospital billing data with longitudinal claims data.  Methods: Data source included two data files with linked patients: IMS Health Real-World Data Hospital (billing data) and IMS Health Real-World Data Adjudicated Claims (claims data). Patients who had a total hip/knee replacement surgery from 07/2011 to 06/2015 were identified using billing data. The hospital admission date was termed as the index date and administrative claims data for a one year pre-index period were obtained. Charlson and Elixhauser comorbidity indices and their disease component categories were calculated from claims across all care settings (gold standard) and compared to the scores calculated with the cross-sectional hospital billing data. Charlson and Elixhauser indices from the two databases were compared using generalized estimating equations (GEE) model. Sensitivity and specificity of assessing individual comorbid conditions was also examined.  Results: A total of 11,708 patients (age: 59.1 ± 5.8 years) were identified. Their average Charlson comorbidity index was 0.47 (95% CI: 0.45-0.48) from hospital bill data, and 1.09 (1.06-1.12) from claims data. Average Elixhauser comorbidity

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indices were 1.72 (1.69-1.74), and 2.95 (2.92-2.99) from two databases respectively. Average sensitivity for individual Charlson categories was 0.321 (range: 0.100–0.710), and 0.374 (0.070-0.802) for Elixhauser categories. Conditions with low sensitivity were cerebrovascular, liver disease, AIDS/HIV, cancer, and psychosis. Average specificity was 0.999 for Charlson, and 0.997 for Elixhauser categories.  Conclusions: Comorbidity indices were underestimated by 57% for Charlson and 42% for Elixhauser when calculated from hospital billing data. Individual comorbidity categories had high specificity, but low sensitivity. PHP215 Estimation Of Value Of Life With A Model Approach Depending On Net Present Value For Turkey Kockaya G1, Yenilmez FB2, Tuna E3 Economics and Policy Association, Ankara, Turkey, 2Hacettepe University, Ankara, Turkey, 3Polar Health Economics and Policy Consultancy, Ankara, Turkey

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Objectives: Value of life is a marginal benefit of preventing death in a given lifetime. Human life is priceless. However, under limited supply of resources or infrastructure, it is important to estimate value of life. The Human Capital Theory(HCT) based on investments to human`s health which can reduce morbidity and mortality, but also improve macro and micro economic outcomes of societies. The aim of this analysis is to conduct a model for estimating value of life of Turkey depending on HCT.  Methods: Net present value(NPV) of the taxes and spending of governments were calculated depending on population demographics and economic data as population distribution by age, employment compliance by age, age of school enrollment, schooling years, year of retirement, unemployment rate, entry annual wage, healthcare spending, education spending, etc. The estimated produced economic value for a life time term for Turkey was assumed as value of life. In additional, each decades NPV were calculated. The economic data for the model was taken from World Bank, UNESCO, OECD and WHO.  Results: Estimated produced economic value for a life time for Turkey was found to be US$ 59.159 which was 5.39 times of GDP per capita. Estimated produced economic value for Turkey were calculated for each decades as US$ -1,069.91, US$ -2,658.13, US$ 2,217.27, US$ 16418.36, US$ 36,081.26, US$ 56,250.61, US$ 68,034.85, US$ 64,900.00, US$ 59,246.64, US$ 59,158.74 for 1., 2., 3., 4., 5., 6., 7., 8., 9 and 10. decades, respectively.  Conclusions: Value of life is estimated by a hypothetical model based on NPV with taxes and spending of government for each individual for a life-term. Model approaches included value of life for each decades of age, which can guide for marginal benefit of preventing pre-mature deaths. This may be a guide for health policy authorities to make prioritization for treatment options for different decades. PHP216 Participation Of External Medical Societies In The Benefit Assessment Of Pharmaceuticals In Germany Bleß H, de Millas C, Kern B IGES Institut GmbH, Berlin, Germany

Objectives: Every new molecule entity undergoes a benefit assessment by the Federal Joint Committee (G-BA) in Germany. After an assessment of the dossier by the Institute for Quality and Efficiency in Health Care (IQWiG) or the G-BA, external experts can comment during an appraisal process. This research aims to explore how specific external experts (medical societies (MS) and the Drug Commission of the German Medical Association (AkdÄ)) differ from the opinion of the assessment and whether the G-BA agrees or disagrees with them regarding additional benefit, appropriate comparator (AC) and endpoints.  Methods: All 136 benefit assessments regularly completed between January 2011 and August 2015 were systematically reviewed. It was determined if MS and AkdÄ recommended a higher, lower or equal additional benefit compared to the assessment. Regarding the AC, experts’ agreement or disagreement with the choice in the assessment was determined. Endpoints were evaluated (agree/ disagree) when at least one expert disagreed with the assessment.  Results: AkdÄ or MS disagreed with 74/136 of all assessments concerning the additional benefit. MS recommended a higher additional benefit more often (118/140) than the AkdÄ (8/45). A lower additional benefit was recommended in 2/140 (MS) respectively 9/45 (AkdÄ) of all statements. When MS/ AkdÄ recommended a higher benefit, the G-BA accorded with 54/118 respectively 6/8 of all statements. Regarding the other subjects, the concurrence rate was 14/79 respectively 4/21 in case of AC and 33/135 respectively 16/35 in case of controversial endpoints.  Conclusions: The high number of divergent opinions between assessment, medical experts and final appraisal demonstrate the need for discourse. The authors recommend to improve the process: involvement of medical experts in the choice of the AC; earlier access for medical experts to dossiers; greater transparency towards considered external opinions by the G-BA; including medical experts as voting members of the appraisal board.

PHP217 Patients’ Acceptance And Adherence Of Their Medication: Results From A European Multi-Disease Study With Online Patient Community de Bock E1, Chekroun M2, Morisky DE3, Arnould B4 1Mapi Group, Patient-Centered Outcomes, Lyon, France, 2carenity.com, Paris, France, 3UCLA Fielding School of Public Health, Los Angeles, CA, USA, 4Mapi Group, Lyon, France

Objectives: Lack of adherence and persistence are major barriers to treatment efficiency. Measuring patients’ acceptance of their medication can help better understand and predict patients’ behavior towards treatment. The objective of this study was to evaluate the level of acceptance and adherence for various chronic diseases in real life using a patient online community in 5 languages.  Methods: An observational, cross-sectional study was conducted through the French, English, German, Spanish and Italian Carenity platforms between October 2015 and February 2016.