Accelerating Access to Gene Therapies for Rare Disease Patients: A Normative Approach

There is an increasing demand to ease regulatory barriers concerning gene therapies, aiming to expedite their development, accessibility, and approval. This urgency is particularly critical for individuals who suffer from rare diseases and often endure significant suffering or premature death, and who could substantially benefit from gene therapies in certain cases. In this article, we explore the normative criteria that should guide the discussion on whether current regulatory processes should persist or undergo streamlining to facilitate quicker drug access and approval for investigational gene therapies. Our proposed framework outlines five specific criteria, namely, 'intention', 'urgency', 'evidence', 'risks', and 'standardization'.