Ways to Expedite Gene Therapy Development: Marks

CBER director Peter Marks says there are steps FDA and other stakeholders could take that would expedite the development of gene therapies. Writing in Expert Opinion on Biological Therapy, Marks says one place to start would be to consider whether the current framework for the development of small molecule drugs currently being followed for the development of gene therapies is the wholly appropriate paradigm to be following.

While there are some aspects of the paradigm that are quite applicable, he says, others may lend themselves to the application of other paradigms, such as reusing information related to the vector backbone used by gene therapies.

Marks also says that the use of novel endpoints and clinical trial designs, manufacturing process standardization, enhanced communication with regulatory authorities, and global regulatory convergence together “could go a long way toward facilitating the availability of these potentially life-saving treatments.”