Perspectives on the FDA platform technology designation program for the approval of gene therapies: a Swiss multi-stakeholder exploratory interview study

TL;DR

This study explores Swiss stakeholders' views on the FDA's platform technology designation program for gene therapies and considers its potential adoption in Europe.

Contribution

The paper provides new insights into the feasibility and acceptability of the FDA's platform designation program from a European stakeholder perspective.

Findings

Participants identified benefits like reduced redundancy in testing and standardization of manufacturing.

Concerns were raised about clinical assessment and global regulatory alignment.

There is potential for innovation in rare diseases, but challenges like technological obsolescence remain.

Abstract

The U.S. Food and Drug Administration (FDA)’s platform technology designation program aims to streamline the development and approval process for advanced therapy medicinal products and is anticipated to be particularly beneficial for the development of gene therapies for rare and ultra-rare diseases. This is an exploratory interview study of Swiss-based perspectives, including insights from stakeholders in industry, academia, regulation, and reimbursement. The objective of our study is to document Swiss professionals’ views on the US platform designation program and to assess the acceptability and feasibility of a similar program in Europe.

Participants identified benefits such as reduced redundancy in pre-clinical testing, standardization of manufacturing, and increased predictability of regulatory requirements. Concerns were raised about clinical assessment, commercialization strategies, and global regulatory alignment. Though participants anticipate it to increase innovation in the rare disease area, some raised the possibility that technologies may stagnate around a platform, or that designations may quickly become obsolete due to the speed of technological development.

The introduction of the platform technology designation program in the US is a step towards increasing treatment options for ultra-rare diseases. While there is potential for platform designation to enable development in this area, its success will depend on addressing the outlined challenges.

The online version contains supplementary material available at 10.1186/s13023-025-04038-y.