Gene Therapy: BIO Comments on FDA Draft Guidance, Human Gene Therapy for Rare Diseases

November 14, 2018

Re: Docket No. FDA–2018-D-2258: FDA Draft Guidance, Human Gene Therapy for Rare Diseases

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding Human Gene Therapy for Rare Diseases.

BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial, and environmental biotechnology products.

We applaud the FDA for putting forth this guidance to assist stakeholders developing human gene therapy (GT) products for the treatment of rare diseases. The Draft Guidance is reflective of the Agency’s experience gathered over the past decade on the evaluation of safety of gene therapy products.

In line with the ongoing efforts by the FDA to further modernize the regulatory review process, FDA should keep in mind that complex innovative designs might be fit-for-purpose to execute clinical trials of GT products for rare diseases. Consequently, BIO suggest the Agency provide regulatory flexibility in its guidances to allow for innovative clinical trial designs, and to not use otherwise limiting language such as those recommending specific number of treatment arms, utilizing different doses but the same product administration procedures, or the inclusion of a sham control group. Use of innovative clinical trial design can decrease unnecessary and unethical patient exposure to clinical procedures and experimental products while still advancing clinical development. Along these lines the Agency should consider inclusion of GT Programs into the recently launched Complex Innovative Designs Pilot Meeting Program.[1]

Regarding inclusion of patient experience, BIO believes additional guidance from the Agency would be helpful on what types of patient experience data may be the most useful to inform regulatory decision-making. We recognize the Agency is working on developing separate guidance on patient experience and would welcome the opportunity to work with the Agency on incorporating the patient voice and provide additional thinking on this topic. 

BIO appreciates this opportunity to submit comments regarding FDA’s Draft Guidance, Human Gene Therapy for Rare Diseases. Below we have provided additional detailed comments. We would be pleased to provide further input or clarification of our comments, as needed.