Gene Therapy Approval Standards: Available Treatments Influence US FDA’s View On Durability
CBER’s Peter Marks says agency assesses the importance of durability of effect differently for a gene therapy that treats a disease that has no other available therapies versus a condition for which there are multiple approved treatments. Marks’ comments shed more light on the complete response letter for BioMarin’s hemophilia gene therapy Roctavian.
You may also be interested in...
COVID-19 Vaccines: ACIP’s Allocation Proposal Prioritizes Long-Term Care Residents, Essential Workers
First group to receive vaccine would comprise health care personnel and residents of long-term care facilities; next in line are non-health care essential workers, who would take priority over adults with high-risk medical conditions and those ages 65 years and older, according to the CDC advisory committee’s proposed allocation framework.
Although US FDA wants sponsors to maintain blinded Phase III follow-up as long as possible after emergency use authorization or licensure, vaccine researcher Larry Corey says it will become ‘untenable’ to keep individuals in placebo arm once they qualify for vaccine access under the allocation framework.
Industry principles for enhancing diversity in clinical trials call for use of digital data collection tools and flexible scheduling of study site visits to reduce barriers to enrollment, as well as greater involvement of under-represented communities in the trial design planning process and recruitment of investigators with diverse ethnic and racial backrounds.