FDA Issues Set of Gene Therapy Draft Guidances

Rare Daily Staff

The U.S. Food and Drug Administration today issued six draft guidance documents, part of an ongoing effort to provide a regulatory framework for the emerging area of gene therapy.

The new documents follow the issuance last year of the FDA’s comprehensive policy framework for regenerative medicine, which included a draft guidance that describes the expedited programs that may be available to sponsors of regenerative therapies. It also comes as the agency has approved three gene therapy products during the past 12 months and a growing number of experimental gene therapies advance through development.

The new documents are expected to serve as a framework for how the FDA will review gene therapies and provide the agency’s current thinking for how these products should be developed, reviewed, and reimbursed. The FDA is soliciting public input on these policies and will take that feedback into account prior to finalizing the documents.

“Today, we’re taking a step toward shaping this modern structure for the regulation of gene therapy,” said FDA Commissioner Scott Gottlieb in a statement issued in conjunction with the release of the documents. “The agency is issuing a suite of six scientific guidance documents intended to serve as the building blocks of a modern, comprehensive framework for how we’ll help advance the field of gene therapy while making sure new products meet the FDA’s gold standard for safety and effectiveness.”

The guidances include three documents that address the development of gene therapy products for specific disease categories including hemophilia, retinal disorders, and rare diseases. The other three guidances address broader issues surrounding gene therapy. The guidances are linked below.

• Human Gene Therapy for Hemophilia
• Human Gene Therapy for Retinal Disorders
• Human Gene Therapy for Rare Diseases
• Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)
• Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up
• Long Term Follow-Up After Administration of Human Gene Therapy Products

“In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can’t be fully answered in any reasonably sized pre-market trial,” said Gottlieb. “For some of these products, we may need to accept some level of uncertainty around these questions at the time of approval.”

Because of that, he said that effective tools for reliable post-market follow-up, such as required post-market clinical trials, are going to be one key to advancing the gene therapy field.

Once finalized, the draft guidances will replace previous guidances issued by the FDA in April 2008.

July 11, 2018
Photo: FDA Commissioner Scott Gottlieb