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Nearly half of currently marketed ATMPs will lose their regulatory market exclusivity period by 2030. This raises the question how to ensure persistent patient access to these often life-changing therapies. In BioDrugs, reNEW’s Dr. Elena Guillen and Associate Professor Ana Hidalgo-Simon explore policy and regulatory strategies to enhance access to ATMPs. Using a EU dataset encompassing ATMP and biologic submissions to the EMA, they identified a subset of ATMPs that exhibit characteristics supportive of traditional follow-on development. Drawing lessons from biosimilars, they propose a continuum of approaches that may enable scalable manufacturing, sustainable pricing and expanded patient access to ATMP follow-on development. This publication aims to provide a starting point for further timely, in-depth discussions on the future of advanced therapies.
First author Guillen said: “We know that clinical efficacy and regulatory approval of ATMPs do not necessarily translate into market sustainability. ATMPs often target rare diseases, face standardization and logistic considerations and are challenged by high costs and pricing. A sustainable ATMP follow-on ecosystem will require flexible, fit-for-purpose pathways and solutions that leverage existing evidence while balancing scientific rigor with practical and economic considerations . to ensure continued patient access”.
The authors therefore identified several tools to increase patient access by studying the biosimilar pathway. They concluded that several principles from this approach, including tailored case-by-case approaches, totality of evidence and fit-for-purpose, hold significant promise to guide ATMP follow-on development.
“Despite this potential, we believe the biosimilar pathway may be suitable only for a subset of ATMPs. This urges the identification of alternative pathways to improve access to these clinically valuable but commercially vulnerable therapies,” said Hidalgo-Simon.
To this end, Guillen and Hidalgo-Simon discussed several alternative pathways that could potentially improve patient access for safe and efficacious ATMPs. In the manuscript they explored how approaches such as prior-knowledge applications, bio-hybrids, platform technologies or redevelopment can unlock the full potential of these life-changing therapies.
The publication aims to spark a broader discussion. “Scientific feasibility is a necessary but insufficient condition for viable follow-on development. We hope to catalyze discussions on how regulatory science, innovation and policy can work together to ensure that the promise of ATMPs translates into sustainable patient benefit,” concludes Guillen.
