The International Society for Cell & Gene Therapy (ISCT) has established a task force to address practical, ethical, and regulatory issues that arise from the use, and potential misuse, of expanded access within the cell and gene therapy (CGT) sector.
Expanded access is a pathway available under FDA regulations that in limited circumstances can provide patients with preapproval access to investigational products outside clinical trials, when no comparable or satisfactory alternative therapy options are available.
In the US, patient and developer interest in expanded access has increased in recent years. This trend is expected to continue or even accelerate, specifically for the CGT sector. One major reason for this is that the FDA is no longer exercising enforcement discretion for premarket approval requirements for regenerative medicine products.
ISCT has formed the ‘ISCT Expanded Access Working Group’ in response to growing concerns in the field that expanded access is being utilised in ways that fall outside its intended purpose. This Working Group will create resources with and for the CGT community on the regulatory framework for expanded access, as well as to guide ethical and appropriate uses of the pathway.
“Inappropriate use of expanded access could harm the entire CGT sector, undermine research efforts, and ultimately delay approval of products critical for many patients. As the CGT sector is continuing to grow, and the therapeutic potential of CGTs is generating increased attention from scientific and patient communities, there is also growing interest in expanded access to CGT products. Expanded access is an important mechanism for providing access to unproven, but promising, interventions for seriously and terminally ill patients who lack approved treatment options. However, at the same time, expanded access should not be understood as a mechanism for commercialising unproven, unlicensed interventions,” said Patricia Zettler, Chair of ISCT Expanded Access Working Group. “Expanded access to CGT products can pose difficult ethical and regulatory questions. This is why ISCT is launching this Working Group, which we hope will help enable expanded access to CGT products while rigorous research continues, to serve the goal of developing safe and effective therapies for patients.”