Input has been drawn from various stakeholders, including consultations with product innovators, sponsors, researchers and patients, to help make the development and review of these products more efficient, while putting in place regulatory controls.
There are currently 900 investigational new drug applications in the pipeline for ongoing clinical studies, with more anticipated, the FDA has developed these policies to offer regulatory clarity for drug makers developing and manufacturing gene therapy products.
'As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs,' said Dr Peter Marks, director of the FDA's Centre for Biologics Evaluation and Research.
Gene therapy products aim to treat illness by inserting new genetic material into a patient's cells to replace or supplement genes that are not functioning adequately. Some deliver the genetic material into cells using viruses as vectors. They can be used to treat rare conditions such as muscular atrophy. Only four gene therapy products have been approved by the FDA to date, but the administration is keen to see an increase in treatment choices for patients.
'We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases,' said Dr Stephen Hahn, FDA Commissioner.
The documents include recommendations for safe product manufacturing and quality, testing, and long-term follow-up such as post-market clinical trials.
'Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time', said Dr Marks.
Their draft guidance document, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations outlines how they will determine whether products qualify as orphan drugs, which treat rare diseases that would not be profitable to produce without state funding. It also includes whether products under review should receive seven-year market exclusivity.
The FDA stresses that it does not want to discourage the development of multiple gene therapy products to treat the same disease or condition. Rather, their policy should lead to a competitive marketplace with more choices for patients.
Stakeholders who participated in the development of these guidelines are being encouraged to make further comment on this issue.