PAYING FOR CURES- AN ANALYSIS OF INNOVATIVE ACCESS AGREEMENTS FOR LICENSED GENE THERAPIES
Author(s)
Hogg L, Richards M
Ipsos, London, LON, UK
Presentation Documents
OBJECTIVES Gene therapies are often curative one-time treatments that address the underlying cause of disease, helping patients avoid a lifetime of therapy and saving health systems the associated cost. Gene therapies typically command a significant price premium, with drug companies arguing superior effectiveness justifies the cost. As a result, health systems are increasingly balancing affordability and the needs of the general population with the ability of high-cost gene therapies to improve long-term outcomes in chronic and rare disease patient populations. This poster analyses the innovative access agreements proposed to date for licensed gene therapies in the European Union (EU) and the United States (US). METHODS We identified all gene therapies approved by the European Commission (EC) or Food & Drug Administration (FDA) and assessed those with a positive or conditional national reimbursement status in the USA, UK, Germany, France, Italy, or Spain. This was supplemented by a targeted literature review (the associated company press releases). RESULTS Eight gene therapies are currently approved: five in the US and seven in the EU. The highest list price is $US 2.1 million/patient. Four gene therapies are reimbursed in the UK, three in Germany, two in Italy, one in France, and none in Spain. Access arrangements include discounts, outcome-based rebates or refunds given if patient outcomes fail to meet pre-specified thresholds, indication-based pricing, and ‘drug leasing’. Real-world evidence collection is often required to resolve uncertainties in safety and effectiveness and to generate additional data to support reimbursement decisions. CONCLUSIONS Despite the potential of curative gene therapies, health systems cannot manage the short-term budgetary impact and risk associated with newly licensed high costs treatments and are increasingly demanding innovative pricing schemes. Therefore, gene therapy manufacturers must engage early with health providers and HTA authorities to agree innovative access schemes that balance equitable patient access with sustainable health system financing.
Conference/Value in Health Info
2019-11, ISPOR Europe 2019, Copenhagen, Denmark
Code
PBI77
Topic
Health Policy & Regulatory
Topic Subcategory
Pricing Policy & Schemes, Reimbursement & Access Policy, Risk-sharing Approaches
Disease
Genetic, Regenerative and Curative Therapies, Multiple Diseases, Oncology, Rare and Orphan Diseases