Legislative Framework for Reimbursement Conditions for Gene Therapies

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Introduction: In Central-Eastern Europe (CEE) access to high priced biologic therapies is associated with barriers. Biosimilars offer therapeutic alternatives to original products at reasonable price. Therefore, to fulfill positive effect of authorised biosimilars on healthcare budgets and optimise patient access to modern biological treatment in CEE their usage would be obvious solution. Objectives of this policy research were to: 1) Map barriers of patient access to modern biologic treatments in CEE, 2) Explore how biosimilars may reduce these hurdles and 3) Identify factors limiting the market penetration of biosimilars.  Methods: A survey was developed and filled in by local experts in 10 CEE countries on topics of registration, pricing, purchasing, reimbursement, patient access, clinical decision making and collection of real world evidence of biological medicines, with special focus on biosimilars.  Results: Limited number of treated patients, waiting lists and limits for prescribers were reported as key barriers for using biologics. Clinicians especially in the field of oncology are expected to raise more concerns about biosimilar indication extrapolation than policy makers. According to respondents, both clinicians and payers consider that treating more patients is the primary benefit of switching patients to biosimilars. However, concerns with therapeutic equivalence and fear of immunogenicity related to potential interchange from original biologicals to biosimilars were reported. Therefore, patients treated with originator products are not expected to switch/interchange, unless the physician decides otherwise for medical reasons. Nevertheless, in countries with central purchasing, tender winning products with the lowest price are likely to be used even for patients on maintenance therapy with a patented product.  Conclusions: As access limits for patients were similar for original products and biosimilars, the appropriateness and success of current biosimilar policies are questionable in CEE countries. Uncertainties related to increased uptake of biosimilars should be reduced by more appropriate biosimilar drug policies. PHP368 Beyond Risk-Based Vaccination Policies - A Life-Course Approach to Vaccination Lach K1, Jaroslawski S2, Pisarczyk K1, Toumi M2 University, Marseille, France

1Creativ-Ceutical, Krakow, Poland, 2Aix-Marseille

Background: Vaccination policies in EU Member States (MS) are mainly focused on at-risk groups i.e. children and elderly above 65 years, especially susceptible to vaccine-preventable diseases (VPDs) such as influenza, while adult vaccination remains underused; age of ≥ 65 is the predominant target group for recommended influenza vaccination, being adopted in 22 MS while the age of 50+ is set in Austria and Ireland only. A life-course approach to immunisation is a proposed means for the improved epidemiological control of VPDs.  Discussion: A wide variability in the recommended target groups for VPDs is seen in MS which could exacerbate inequalities at international level. Vaccination schedules targeted at adults aged ≥ 50 in merely a few MS pose a missed opportunity to promote healthy ageing given that this group is characterized by a sizable prevalence of risk factors. In the light of recognized benefits of vaccination beyond solely at-risk groups, life-course approach to immunization has been partly put in place in some MS. However, vaccination coverage rate (VCR) for influenza did not reach expected threshold of 75% by the 2014-2015 winter season in most of the MS from a number of reasons (e.g. lack of awareness among health-care workers). Sub-optimal uptake of routinely recommended adult immunisations is reported which entails great burden associated with VPDs. An approach targeted at healthy adults and promoted throughout the life-course is expected to reduce risk of VPDs and contain health-care costs while increasing the economic productivity.  Conclusions: Life-course approach to vaccination could become the future EU vaccination policy upon an important prerequisite being the systematic evaluation of its benefits and (cost-)effectiveness in different VPDs to determine its added-value over the current approach. This could mean a switch in the understanding and the implementation of vaccination policies from “risk-based” to “age-based” and improve VCRs.

PHP370 Balancing Commercial Confidentiality with Accountability: A Proposal for Enhanced Disclosure of Cost-Effectiveness Decisions O’Mahony JF Trinity College Dublin, Dublin, Ireland

Purpose: To propose two new reporting conventions regarding cost-effectiveness evidence that would enhance accountability in the allocation of scarce healthcare resources, while preserving commercial confidentiality regarding the price of specific products.  Background: The negotiated prices for new drugs are often not disclosed in the interest of commercial confidentiality. Keeping prices confidential helps manufacturers pursue separate pricing strategies in different markets. An unfortunate consequence of price confidentiality is that it hampers transparency in the allocation of healthcare resources. The absence of published prices makes it difficult to assess how closely decision makers adhere to efficient spending policies. This compromises the accountability of decision makers’ funding choices, which may result in more spending on cost-ineffective treatments than would otherwise be the case.  Proposals: The first proposed convention is that decision makers publish an annual review of funding approvals made over the previous year that reports a histogram showing the distribution of incremental cost-effectiveness ratios (ICERs) of the funded interventions. The histogram bins could be at various fractions of the cost-effectiveness threshold such as 0.5, 1, 1.5, 2 and so on. This would allow analysts understand how many interventions are being approved above and below the threshold without disclosing the price or ICER of any individual product. The second proposal is that an aggregated cost-effectiveness ratio is published annually, which aggregates the incremental cost and effects of all products approved over the previous year. Again, this would provide some quantification of the efficiency of funding approvals without disclosing sensitive information on any specific product.  Conclusions: Our proposals provide a simple way to enhance

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accountability in resource allocation without compromising commercial confidentiality. In principle, the proposed measures have only modest data requirements and there can be little reason to oppose their introduction. PHP371 Legislative Framework for Reimbursement Conditions for Gene Therapies Ligri D1, Kani C2, Souliotis K2 1Aristotle University of Thessaloniki, Thessaloniki, Greece, 2University of Peloponnese, Corinth, Greece

Objectives: In April 2016 European Medicines Agency (EMA) approved an autologous CD34+ ex vivo gene therapy product for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). As this product would be administered in some specialist accredited transplant centers through Europe, classic reimbursement criteria should be revised.  Methods: Available EU and national legislation has been searched in order to identify available negotiation tools for the following two cases: a)integration of gene therapies under the general framework of a national policy (leading to national pricing and reimbursement) b) adaptation of the benefit package provided by reimbursement funds to gene therapies and c) study of the existing administrative procedure of approval and reimbursement in the field of transplantations of human tissues, which presents important similarities to the ex vivo gene therapy studied.  Results: According to Regulation (EC) no 883/2004 a prior authorization is needed in all cases of programmed treatment in another EU Member State (MS). The authorization shall be accorded where the treatment in question is among the benefits provided for by the legislation in the MS where the person concerned resides and where he cannot be given such treatment within a time-limit which is medically justifiable, taking into account his current state of health and the probable course of his illness. Another approach is as all candidates for the gene treatment will be treated in an accredited center in other MS discounts and price-volume agreements could be applied according to national legislation.  Conclusions: The lack of a common framework for the reimbursement of gene therapies could lead to the non-reimbursement of new therapies. Furthermore, the development of specialized centers throughout MS could be more efficient than the development of a network in each MS. That perspective leads national healthcare systems in joint negotiation procedures. PHP372 Managed Access Agreements: A New Model Pathway for The Reimbursement of Non-Oncology Drugs in England Approved Under European Adaptive Pathways? Macaulay R PAREXEL Access Consulting, London, UK

Managed Access Agreements (MAAs) are agreements between NHS England and manufacturers to enable a drug to become available for a limited time period at a discounted price. This allows patients to access the drug whilst further evidence is gathered on its real-world effectiveness. The National Institute of Health and Care Excellence (NICE) subsequently re-issues guidance on whether this drug should be funded. In December 2015, elosulfase alfa (Vimizim), which treats Morquio A syndrome became the first therapy to be recommended by NICE with an associated MAA. In April 2016, NICE issued draft guidance recommending ataluren (Translarna) in Duchenne muscular dystrophy, conditional on an MAA. MAAs can provide an access route for drugs for very rare diseases, which typically have a limited supporting clinical evidence base and significant associated uncertainties alongside high treatment costs. Indeed, elosulfase alfa (costing £395,000 per patient annually 3 people are born with this condition every year in the UK) and ataluren (costing £222,000 per patient annually; 50 patients are estimated to receive ataluren under the 5-year MAA) had previously been rejected by the Scottish Medicines Consortium (SMC). The number of therapies approved in Europe supported by a clinical data package lacking large multicentre, randomized, comparative Phase 3 trials will likely increase, driven by the EMA Adaptive Pathways pilot alongside very promising new pipeline therapy classes (e.g. CAR-T and gene therapies). For cancer therapies lacking supportive Phase 3 data, under new arrangements for the Cancer Drugs Fund (CDF), promising drugs for which the evidence is not strong enough can temporarily enter the CDF to collect real-world evidence to help inform subsequent NICE appraisals. MAAs therefore can similarly provide a model that allows temporary patient access whilst additional evidence is generated for non-oncology drugs that treat severe, rare diseases.

PHP373 Using Social Finance to Fund Generic Drug Repurposing for Rare Diseases: A Social Impact Bond Proof of Concept Thompson RS1, Potter J2, Griffiths A3, Eljamel S3, Raffai F1, Sireau NT1 for Good, London, UK, 3Costello Medical Consulting Ltd, Cambridge, UK

1Findacure, Cambridge, UK, 2Numbers

Repurposing existing generic pharmaceuticals to treat rare diseases with an unmet medical need has a number of clear benefits; reducing the investment required in drug discovery, and leveraging known information on drug behaviour and safety often in multiple patient populations. This makes repurposing a relatively quick and inexpensive route to deliver new treatments to those patient populations who need them most. Sadly, such projects have little appeal to industry, in part due to the small patient populations and the challenge of securing intellectual property in generic medicines, which limits potential financial returns. Findacure, a charitable organisation, have completed a proof of concept study into the use of social finance to fund generic drug repurposing research. Our proposed social impact bond aims to repay private investment into clinical trials on repurposed drugs, using savings