Gene Therapy, Digital Health Boost in PDUFA Talks
FDA is seeking to advance efforts associated with its Cell and Gene Therapy Program (CGTP) and Digital Health Program, according to FDA summaries of meetings with industry on negotiating the reauthorization of the Prescription Drug User Fee Act. The agency has proposed to “enhance resources for the existing CGTP in CBER due to the rapid growth in cell and gene therapies development, which has resulted in a continuous and sustained increase in the number of applications and meeting requests for a variety of diverse and complex products,” a CBER meeting summary says. “This increased workload has forced CBER to limit sponsor interactions, convert many meetings to ‘written responses only,’ and limit external interactions, among other impacts.”
At the meeting, industry asked FDA to hold a public meeting and develop guidance on how sponsor-specific prior knowledge could be leveraged to streamline submissions, manufacturing, and potentially pre-clinical and clinical data for gene therapy products. “FDA and Industry agreed that guidance development on specific areas to address frequently asked questions heard from multiple sponsors would be beneficial,” the summary says. Industry representatives also stated that workshops would be helpful to discuss manufacturing issues specifically and how to advance the development of gene therapies.
Additionally, industry requested that FDA hold a public workshop to focus on the regenerative medicine advanced therapy (RMAT) program to update current FDA guidance, including adding potential uses of real world evidence (RWE) for regulatory decision making. The summary notes that FDA said there challenges with developing meaningful guidance on the use of RWE since there have been limited cell or gene therapy products approved under accelerated approval and none with a RMAT designation. “Industry stated interest in understanding the reasons for denials of RMAT designation requests and that a guidance could be useful in explaining expectations and common reasons for denials,” the summary says, adding that such discussions will continue.
At the digital health and informatics meeting, FDA proposed an “integrated cloud-based technology environment for collaboration and as a framework for regulatory submission innovation, using pilot projects to test feasibility and to collaboratively design and implement cloud-based capabilities that could be utilized in the regulatory process,” a meeting summary says. In discussing potential pilot programs, FDA identified information requests as a “common pain point.”
The agency also proposed a Digital Health Technologies (DHT) framework for “strengthening its capability to leverage DHT-generated data in submissions, including identification and development of needed guidance, building staff capacity to enhance review consistency and efficiency, and ensuring IT support for identifying, storing and utilizing DHT data,” the summary says. FDA clarified that efforts would align with the recently announced Digital Health Center of Excellence being established in CDRH to harmonize on policy and practices where applicable.