To expand the field of gene therapy, Peter Marks, director of the FDA’s Center for Biologics Evaluation (CBER), believes that the current clinical development framework is in need of rethinking in terms of trial design, endpoints, communication and other areas.

As it stands now, gene therapy developers commonly refer to the clinical development framework for small-molecule drugs, Marks wrote in an editorial published in Expert Opinion on Biological Therapy. While many components of this framework apply to gene therapies — good manufacturing practices (GMP), understanding nonclinical aspects of the investigational drug and demonstrating safety and effectiveness, for instance — other aspects of gene therapies might be better served by other models, the CBER chief believes.

Marks posed that greater use of novel trial designs and endpoints, global regulatory harmonization, standardized manufacturing processes and improved communication with regulators would have a big impact on getting more gene therapies to market.

Marks believes that sponsors should consider the use of Bayesian clinical trial designs that include reassessments of therapeutic benefit probability as each patient goes through the trial process, as these measures can help to lower the number of participants needed in gene therapy trials and the small pools of rare disease patients that can make enrollment a struggle.

Additionally, because current gene therapies use a device-like “vector backbone,” most often an adeno-associated virus or lentivirus, to deliver an artificial gene, Marks thinks that the reuse of backbone-related information should be considered, as appropriate, for other gene therapies in development. Successfully doing this could accelerate development for multiple products.

Access Marks’ article here: https://bit.ly/3NPD1Vv.