Relaxing Rules For Pharmaceuticals And Benefit Coverage Expansion In Korea: The Impact On The Pharmaceutical Expenditure

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potential to close the gap between internal validity of explanatory trials and real-life practice. The objective of this study was to assess the current use of PCTs to support drug launch.  Methods: Peer-reviewed articles on PCTs were searched in Medline/ Embase through Ovid. In addition, search of the EUnetHTA and EU Commission websites was performed, supplemented by Google search of grey literature.  Results: PCTs are currently on the rise; however, their use to support drug launch is uncommon. PCTs have several limitations, i.e., 1) methodological/analytical issues inherent to the potential risk of bias of more flexible designs, 2) operational issues with coordinating the recruitment of large patient populations from different settings, 3) concerns of regulatory and ethical nature, e.g. securing rights and interests of participants while remaining pragmatically flexible, 4) substantial costs incurred to conduct large PCTs. In the United States, PCTs are currently mainly promoted through public sector funding, while their potential use by regulators and payers is still uncertain. Before PCTs can be used for decision-making, academic experience (e.g. from the National Institutes of Health Health Care Systems Research Collaboratory, the National Patient-Centered Clinical Research Network, the Clinical Trials Transformation Initiative) with these trials should broaden. In Europe, PCTs are usually considered by decision-makers as complementing (but not replacing) standard randomised clinical trials. Evidence from PCTs seems more acceptable for drugs with a known benefit/risk profiles, while less acceptable for drugs with novel mechanisms of action.  Conclusions: PCTs are currently underused for initial drug regulatory/reimbursement dossier filing. Currently, multi-stakeholder initiatives are undertaken to enhance pragmatism in clinical trials and reduce decisionmaking uncertainty through developing tools, guidelines, and research on new trial designs and statistical methodologies. PHP53 A Typology-Based Decisional Framework To Support Market Access And Reimbursement Decisions For Personalised Medicines Govaerts L1, Geldof T1, Simoens S1, Huys I1, Van Dyck W2 Leuven, Leuven, Belgium, 2Vlerick Business School, Brussels, Belgium

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Objectives: New co-development approaches in personalised medicine challenge current decisional frameworks of health-technology access and reimbursement procedures. We aim to conceptualize an efficient typology-based decisional framework which takes into account the development and market access synchronism between therapeutic (Tx) and diagnostic (Dx) components of personalized medicines.  Methods: Following systematic literature review, a focus group discussion study was conducted with Belgian personalised medicine industry stakeholders; BeMedTech (Dx-component), Pharma.be (Tx-component), and the Belgian health payer (INAMI). The discussions resulted in Tx-Dx cases to be used to support the personalized medicine access and reimbursement decision-making.  Results: A Tx-Dx typology-based decision support framework was derived and agreed upon by the stakeholders for implementation in the Belgian healthcare system. Within the personalised medicine industry, different strategic development approaches were unfolded for either in vitro diagnostics and therapeutics. The proposed Tx-Dx co-development and market access typology takes into account the synchronism of the strategic development approaches and translates them into access and reimbursement pathways to be used by assessment committees. In the framework, we distinguish different access and reimbursement pathways based on personalized medicine development strategies; (1) The co-development of an innovative therapeutic and companion diagnostic combination (e.g. Vemurafenib and BRAF-case), (2) the novel therapeutic development on a targeted patient population characterized by an already marketed diagnostic (e.g. Olaparib and BRCA-case). (3) The novel diagnostic development on a patient subpopulation within an already marketed therapeutic treatment (e.g. Cetuximab and KRAS/NRAS-case) and (4) the development of an innovative improved diagnostic on an already marketed diagnostic stratification technology [e.g. Immunohistochemistry products and Fluorescence in situ hybridization products-case].  Conclusions: The proposed typology-based decisional framework might allow for a more efficient and effective assessment and budget impact analysis of personalised medicine products. Accepted for guiding decisions in the Belgian healthcare system, the framework can function as a conceptual basis for other agencies outside Belgium. PHP54 Relaxing Rules For Pharmaceuticals And Benefit Coverage Expansion In Korea: The Impact On The Pharmaceutical Expenditure Oh R, Na Y HIRA (Health Insurance Review & Assessment Service), Wonju-si, Gangwon-do, Korea, Republic of (South)

Objectives: Korea has gradually extended the benefit coverage of National Health Insurance. For pharmaceuticals, reimbursement restriction that limits use on disease or patient for approved indication is to manage new medicines or oncology treatments. Strengthening benefit for serious diseases has been forwarded by reimbursing new drugs and relieving the restrictions. This study aims to investigate the effects of the coverage expansion on the pharmaceutical expenditure trends.  Methods: The National Health Insurance Claims data for year 2015 and 2016 was used. We analyzed the increase of total pharmaceutical expenditure by dividing it into 1) newly reimbursed drugs in the year 2015~2016, 2) drugs which have alleviated the reimbursement restrictions during 2015~2016, and 3) the residual which has not changed their reimbursement area.  Results: Korea’s national drug expenditure has been increased by 118 million dollars (9.8%) during 2015 and 2016. The expenditure of the newly reimbursed drugs accounted for 20.7% of the total pharmaceutical increase. The drugs reimbursed in the year 2015 and 2016 have contributed 12.0% and 8.7% respectively. The drugs which have lifted the reimbursement restriction between 2015 and 2016 have contributed to the 23.1% of the total increase of expenditure. Anti-tumor drugs accounted for 30% among them expanded on use. The increase of the expenditure due to the residuals which have remained the reimbursement conditions accounted for 56.2% of the total increase. As an individual product, Herceptin(trastzumab) treating breast cancer showed the

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largest increase.  Conclusions: Korea’s new government is likely to drive force to expand the national health insurance coverage. It is not inconclusive whether relaxing pharmaceutical restriction contributes to the goal of the benefit extension plan policy yet. We examined only short term effect and presented the estimates maximizing the effect on expenditure rather than optimizing. PHP55 Early Acess But Then What? The Uk Early Access To Medicines Scheme Three Year Report Card Macaulay R PAREXEL International, London, UK

Objectives: In April 2014 the UK Medicines and Healthcare products Regulatory Agency (MHRA) launched the Early Access to Medicines Scheme (EAMS) to enable patients with severe, life-threatening diseases without adequate treatment options to access medicines prior to their marketing authorization. EAMS is a voluntary scheme whereby the medicine is provided free of charge by the manufacturer. EAMS comprises two key steps: (i) Promising Innovation Medicine (PIM) designation and (ii) EAMS scientific opinion. We aimed to systematically evaluate all EAMS appraisals to date.  Methods: Publically-available EAMS documentation was screened from the MHRA website (to 08/06/2017).  Results: Of 50 PIM designation applications, 68% were granted; 16% refused; 4% withdrawn, with 12% pending. Only 40% (20/50) of medicines granted PIM status had applied for an EAMS scientific opinion, 75% (15/20) of which were awarded. 73% (11/15) of awarded opinions were in oncology. 82% (9/11) oncology agents were for anti-PD-1/L1 therapies: nivolumab, pembrolizumab and atezolizumab. Only 1/15 was from a small-/medium-size enterprise (SME). 73% (11/15) awarded opinions had expired when EU marketing authorization was granted. Medicines were available under EAMS for an average of 93 days (range: 18–327). In the mean of 441 days (range: 128–720) since these EAMS opinions had expired, only 6/11 had been NICE-appraised (all recommended) and 8/11 SMC-appraised (75% accepted/restricted) at an average delay of 202 and 270 days post-EAMS expiry, respectively.  Conclusions: Over the past three years, EAMS has enabled medicines for nine drugs across fifteen indications (mostly within oncology) to be made available to patients with severe unmet prior to marketing authorization. However, this was for an average time period of only three months and was followed by an average delay of over six months until NICE/SMC appraisal completion. Reforming EAMS to ensure patients can continue to access therapies post-marketing authorisation until a NICE/SMC appraisal should be considered. PHP56 Identification Of Important Criteria For Drug Reimbursement Decision-Making And Their Relative Importance Funagoshi M, Murasawa H, Shimozuma K Ritsumeikan University, Kusatsu, Japan

Objectives: In healthcare technology assessment for reimbursement decisions, it is important to consider multiple aspects and public preferences. We aimed to identify important decision-making criteria for reimbursement of drugs to treat life-threatening diseases and to reveal their relative importance for the general public (GP) and healthcare professionals (HCPs) comprising physicians and pharmacists.  Methods: We selected some decision-making criteria for drug reimbursement from a literature review and conducted a two-step online cross-sectional survey. In the first step, participants were asked to rate each criterion on a 7-point scale. The criteria with high ratings were selected based on means for each group. In the second step, the same participants were asked to allocate 100 points among these criteria to measure their relative importance.  Results: The first survey was completed by 719 participants (GP: 499, HCPs: 220) and 8 criteria were selected: out-of-pocket cost (Mean= 5.73 [GP], 5.47 [HCPs]), adverse event (5.66, 5.74), symptom relief (5.61, 5.79), cost effectiveness (5.57, 5.73), productivity loss (5.54, 5.39), life expectancy (5.41, 5.55), significant innovation (5.30, 5.49), and budget impact (5.29, 5.36). There were 613 respondents to the second survey (GP: 421, HCPs: 192). The most important criterion among the GP was symptom relief (relative importance= 16.2 vs. 15.6), but the difference with HCPs was not significant. Similarly, no significant differences were found in budget impact (9.7, 8.7) and life expectancy (8.8, 10.5). The criteria in which there were significant differences were out-of-pocket cost (15.2, 9.7), productivity loss (12.7, 10.0), adverse event (11.8, 9.3), significant innovation (11.3, 16.2), and cost effectiveness (14.2, 20.1).  Conclusions: This study shows the relative importance of reimbursement decision-making criteria both for the GP and HCPs. These findings will provide valuable insight for an appropriate method for drug reimbursement decisions. PHP57 Review Of The Reimbursement Environment For Advanced Therapeutic Medicinal Products (AtmpS) In The Uk Hughes T, Harries M, Sattar S MAP BioPharma Limited, Cambridge, UK

Objectives: To review current processes for reimbursement of ATMPs in the UK and consider mechanisms to improve patient access to these therapies.  Methods: Structured desk research was conducted to assess the health technology assessment (HTA) status and reimbursement of products classified by the European Medicines Agency (EMA) as ATMPs in the UK. A review of decision drivers and consideration of potential policy solutions to support wider access was undertaken.  Results: Appraisal processes in the UK are no different for ATMPs than non-ATMPs. The only determinant of which reimbursement process applies to an ATMP is the size of the eligible patient population. Eight ATMPs have received marketing authorisation in Europe, seven launched commercially, but four were subsequently withdrawn from the market due to low uptake. Imlygic was recommended for restricted use by NICE with a patient access scheme, ChrondoCelect was not recommended by NHS England and Strimvelis is scheduled for a NICE Highly Specialised Technology appraisal. Provenge was not recommended by NICE and the company has subsequently withdrawn marketing authorisation. Scottish Medicines Consortium has