PPM7 Unlocking EARLY Access for One-Time Treatments

Abstract

The rise of one-time therapies introduces new challenges for regulatory and payer bodies, including the often-overlooked considerations for early access (defined as access prior to Health Technology Assessment (HTA) or national coverage/reimbursement). This study aims to understand the current early access landscape in Europe and assess the adequateness/relevance of existing frameworks for innovative one-time therapies. A systematic review of local/regional early access frameworks across Europe was conducted by reviewing laws, guidelines and publications relating to early access at both the centralized and regional levels. Recent case studies of one-time therapies were then used to evaluate key challenges and the relative adequateness of existing frameworks for innovative one-time therapies. While a number of early access mechanisms exist in Europe to enable early access, the vast majority are designed to provide treatment free-of-charge: except for France and Italy, where the ATU and ‘326/’648 programs enable financial remuneration for early access, countries in Europe are broadly unaccustomed to paid early access. In fact, local regulations frameworks often mandate a free-of-charge period or make it challenging for financial remuneration prior to HTA outcomes as is exemplified by the EAMS compassionate use program in England or the 210-day mandatory free of charge period for PAP in Portugal. For one-time therapies, providing such products free-of-charge directly removes patients from the future patient pool and thus potentially deters cell and gene therapy (C&G) manufacturers from pursuing such programs (unlike the case for chronic therapies, where early access patients continue treatment post-early access). There is a clear need for payer/HTA bodies to evolve in order to accommodate new early access mechanisms for curative, one-time therapies. As demonstrated through recent successes with early access for C&G therapies in Europe, payers have demonstrated openness to adapting the system to accommodate the new treatment paradigm.