Relationship Between The Price Differences In Orphan Drugs With More Than One Benefit Assessment In Amnog And The Potential Influencing Factors

VA L U E I N H E A LT H

PSY138 Review Of Reimbursement Decision Drivers For Rare Cancer Therapies Across EU5 Markets Gupta J1, Wahal VP2 1DRG Abacus, Gurgaon, India, 2DRG Market Access Insights, Gurgaon, India

Objectives: To review health technology assessment (HTA) decisions for rare cancer therapies and rationale for these decisions across EU5 countries.  Methods: We used the Global Market Access Solutions (GMAS) database to assess HTA decisions for seven rare cancers across EU5 countries. Additional information was extracted from individual HTA reports. Decisions were categorised as recommended, restricted, or rejected. Clinical and economic factors influencing decisions were analysed.  Results: In total, 24 HTA reports were identified, which assessed 12 interventions across seven rare cancers. Across HTA agencies, 50% of submissions resulted in an intervention being recommended. No relevant reports were published by the Spanish HTA agencies. The French HTA agency (Haute Autorité de Santé [HAS]) was most likely to recommend an intervention (100% recommended; 7 submissions) based on demonstration of clinical effectiveness (overall survival and response rates) and generic substitution of branded drugs. Demonstration of clinical effectiveness was the key criterion for a ‘recommended’ status (in 66% submissions). All interventions for hairy cell leukaemia and malignant pleural mesothelioma were recommended. The Scottish Medicines Consortium (SMC) was most likely to reject an intervention (88% rejected; 8 submissions) due to lack of robust economic analysis data resulting in high incremental cost-effectiveness ratios (ICERs) versus comparator therapies. In other countries, interventions were rejected mainly due to no additional benefit being demonstrated, absence of submission, and inappropriate choice of comparator/patient population.  Conclusions: Manufacturers developing new therapies in rare cancers should consider providing robust clinical data in HTA submissions. Provisions are made by HTA agencies for early access and managed entry programmes, which allow for some uncertainty when unmet need is high and few or no alternative therapies are available. However, robust economic analysis that adjusts for uncertainty and incorporates appropriate cost and utility values could facilitate reimbursement, particularly in markets driven by cost-effectiveness. PSY139 Determinants Of The Optimal Route To Reimbursement For Orphan Medicinal Products (OMPS) In England Sattar S, Marshall JD, Hill CA MAP BioPharma Limited, Cambridge, UK

Objectives: NICE appraise approximately half of new medicines and there is uncertainty regarding the alternative options for obtaining reimbursement in England. We identify possible routes to market for OMPs in England and conduct a critical appraisal of which route is most suited to appropriately evaluate OMPs.  Methods: The route to reimbursement for OMPs launched between January 2012 and June 2017 was evaluated via online searches of NICE and NHS England (NHSE) websites. The assessments were reviewed to ascertain key elements of the analysis and their outcomes to help identify successful strategies.  Results: OMPs may undergo a NICE Highly Specialised Technology (HST) appraisal, however these are specifically reserved for ultra-orphan (not “just” orphan) treatments and NICE only have scope to evaluate 3 HSTs per year. When a product does not meet HST criteria, it can only undergo a standard NICE appraisal that limits cost-effectiveness to an ICER of £30,000. If a product is not appraised by NICE, OMPs may be reimbursed through an NHSE Commissioning Policy but the process for determining commissioning policies relies on published evidence and prioritises treatments by net cost per patient per year, thereby penalising orphan treatments which tend to be costlier due to limited population size. Alternatively, reimbursement may be gained via individual funding requests made by clinicians on a per-patient basis for 5 patients per region, up to a maximum of 20 patients across England.  Conclusions: Despite increasing research and development of orphan treatments, current reimbursement assessment processes are not well suited to OMPs as they are typically assessed against the same criteria as non-orphan products. The gap between non-orphan and HSTs is commonly referred to as the “doughnut hole”. Non-HST products with an ICER above £30,000 currently have no chance of success in a NICE appraisal and the only chance of success is via a NHSE Commissioning Policy. PSY140 Statistical Trend Analysis of German Benefit Ratings And Their Correlation With Negotiated Rebates Focusing on Orphan Oncologics Dehnen J, Jonas F, Wenzel R Charles River Associates, München, Germany

Objectives: We evaluated the development of the Early Benefit Assessments (benefit rating) of the German Federal Joint Committee (G-BA) and their influence on the outcome of the later net price negotiations across all therapeutic areas and for orphan and oncology drugs in particular.  Methods: We collected and analyzed the outcome of all 230+ G-BA resolutions until June 2017. We evaluated them by therapeutic area and by orphan status over 2 year time bins and tested their relation with linked rebate levels of 100+ marketed pharmaceuticals (pPU, Lauer-Taxe) with Pearson correlation coefficient.  Results: The share of quantifiable positive benefit ratings, e.g. minor, considerable or mayor benefit, for orphan drugs (42%) is similar to that of non-orphan drugs (44%) across all therapeutic areas. Over time, the distribution of benefit ratings of orphan drugs shows a trend towards non-quantifiable benefit. Oncology therapies represent the major share of submissions to the G-BA (~34%, non-orphan and orphan). Comparing different therapeutic areas, we find that therapies in oncology achieve more positive ratings (59%) than therapies in any other therapeutic areas (13%-42%), but the share of positive benefit ratings in oncology decreased from ~65% to ~50% in the past 2 years. The number of orphan oncology assessments increased from 4 in the first two years to more than 10 in the past two years with the share of nonquantifiable.  Conclusions: German benefit ratings of orphan oncology therapies worsened over time but are not correlated to the negotiated rebate. This trend may have contributed to the worsened perception of orphan drugs.

A569

20 (2017) A399–A811

PSY141 Relationship Between The Price Differences In Orphan Drugs With More Than One Benefit Assessment In Amnog And The Potential Influencing Factors Hinnenthal M1, Hofmann-Xu L2, Bonduelle D3 1QuintilesIMS, München, Germany, 2QuintilesIMS

Health, Munich, Germany, 3QuintilesIMS,

Munich, Germany

Objectives: Since the Act on the Reform of the Market for Medicinal Products (German: Arzneimittelmarktneuordnungsgesetz, AMNOG) has been implemented in 2011, medicinal products including orphan drugs with active ingredients or indication extension after 2011, have been undergone a benefit assessment (Rules of procedure (VerfO) of the Federal Joint Committee (FJC), chapter 5, §8). Whilst for non-orphan drugs the price is based on the comparator, the pricing of orphan drugs is unclear, since no comparative assessment is required (VerfO, chapter 5, §12). The objective of this analysis is to identify any pattern in the pricing of orphan drugs by analyzing the relationship between the price differences and potentially influencing factors from two benefit assessments of the same active ingredient.  Methods: Therefore, all orphan drugs with at least two benefit assessments have been identified by the IMS HTA Datenbank. For orphan drugs with re-submission, a descriptive analysis regarding the change in the extent of the additional benefit has been conducted. For orphan drugs with indication extension, the relationship between the price differences in several benefit assessments and the change in possible influencing parameters has been analyzed.  Results: Using a correlation method, the weighted European costs of an orphan drug was identified as the factor with the biggest influence on the percentage price change, followed by the percentage change in the size of the target population and in the annual therapy costs. The change in the extent of the additional benefit seems to have no verifiable effect.  Conclusions: For orphan drugs the relationship between the price differences from two benefit assessments and the potential influencing factors, except the weighted European costs, is difficult to identify. The pricing mechanism for orphan drugs remains non-transparent but seems to be dependent on a variety of factors and is not based on the additional benefit alone. PSY142 Adaptation of International Cost-Effectiveness Analyses To A Single Country - The Case of Bariatric Surgery Versus Conventional Treatment For Obesity And Overweight Ademi Z1, Tomonaga Y2, van Stiphout J1, Glinz D3, Gloy VL3, Raatz H2, Schwenkglenks M1 1European Center of Pharmaceutical Medicine - University of Basel, Basel, Switzerland, 2University of Zurich, Zurich, Switzerland, 3Universitätsspital Basel, Basel, Switzerland

Objectives: The aims of this study were to a) identify published cost-effectiveness studies regarding bariatric surgery versus conventional treatment for obesity and overweight, b) assess their reporting quality, c) assess their transferability to Switzerland, and d) to adapt transferable cost-effectiveness results to Switzerland.  Methods: A systematic literature search was performed in MEDLINE, Embase and other databases. Two reviewers independently undertook the screening, extraction of studies, assessment of reporting quality utilising the Consolidated Health Economic Evaluation Reporting Standards, assessment of transferability, and adaptation of cost-effectiveness results. Adaptation of cost data was performed in three steps: correction for different levels of resource utilisation, for different prices of healthcare services, and for changes in level of resource utilisation and prices over time.  Results: Fifteen studies (Europe, the United States and Australia) fulfilled criteria for numerical adaptation of incremental cost effectiveness ratio (ICER) results to Switzerland. Adapted ICERs for patients with body mass index (BMI) > 35 kg/m2 indicated a cost-saving (dominant) situation or showed ICERs of CHF 8,000-44,000 per quality adjusted life years (QALYs) gained for bariatric surgery versus conventional treatment. Adapted results for patients with BMI < 35 kg/m2 showed ICERs of CHF 3,000-50,000 per QALY gained for bariatric surgery versus conventional treatment. Procedure-specific differences showed that gastric bypass appeared better than gastric banding in terms of benefits, but was more expensive.  Conclusions: Nearly all studies found bariatric surgery to be cost saving or cost-effective, compared to conventional treatment. Differences were due to approaches to the modelling of effectiveness (in terms of duration of BMI changes and dependent effects on morbidity, mortality) costs, time horizon, population studied, exact type of intervention studied, and possibly other unidentified reasons. The adaptation strategy described may be useful for individual countries in which financing or capacity for economic analyses is scarce. PSY143 A Comparison of The Nice Highly Specialised Technology (HST) Programme with Assessment By The National Authority For Health (HAS; France), And The Federal Joint Committee (G-BA; Germany) Hann G1, Wordsworth J1, Ralston S2, Cork D1 1SIRIUS Market Access, Newcastle upon Tyne, UK, 2SIRIUS Market Access, London, UK

Objectives: NICE defines ultra-orphan drugs as those treating life-threatening or seriously debilitating conditions affecting ≤ 1:50,000 people and developed the HST programme to assess these drugs, which are unlikely to meet standard HTA criteria. We compare outcomes of HST evaluations with assessment by HAS and the G-BA.  Methods: HST final evaluations published by June 2017 were identified (n= 7). HAS and G-BA evaluations of the same drugs were then identified.  Results: The HST programme assessed unmet need, cost to NHS/personal social services, and value for money. NICE recommended six of the seven drugs, three outright, two with managed access agreements (MAAs) requiring additional data collection to account for uncertainty, and one for a subpopulation with an MAA. Despite acknowledgement of clinical need and likely benefit, sebelipase-α  was not recommended because of high costs. HAS consider additional benefit (ASMR) of orphan drugs with expected budget impact (BI) < € 30 million/year to be proven at marketing authorisation. However, the drugs reviewed here were assessed through normal processes. HAS considered six drugs to provide additional benefit (two ASMR II,