In either case, the Alliance for Regenerative Medicine’s Research Foundation (ARM Foundation) has issued a “landscape analysis” of current evaluation and reimbursement structures for cellular and genetic therapies just getting onto the market now. This landscape analysis will be followed by a stakeholder panel, possibly meeting in the fall, and a white paper or other position statement after that, according to Morrie Ruffin, executive director of the ARM Foundation. The project was announced earlier this year, and IQVIA is onboard as a contractor.
A standards coordination group has identified and is working to fill gaps in standards for cell and gene therapies in a congressionally mandated effort to strengthen regulatory predictability for manufacturers working to develop and launch commercial cell and gene therapies.
The agency’s move reinforces its sustained support for the technology, a type of regenerative medicine treatment that involves modifying a person’s genes or introducing new ones to treat, prevent, or possibly cure disease, Alliance for Regenerative Medicine and co-founder and senior policy counsel Michael Werner told Bloomberg Law in an interview.
The article outlines some of issues and recommendations to improve the regulatory landscape for ATMPs in Europe, with a focus on hospital exemption, GMO requirements for clinical trials with gene therapies and other potential improvements in the regulatory framework, including a convergence of requirements for cells and tissues, new initiatives to simplify or facilitate regulatory processes, and to address the lack of standards in the field.
The RMAT (regenerative medicine advanced therapy) status has been given to NSR-REP1, the London-based biotech’s therapeutic for choroideremia, a rare, degenerative retinal disorder that mainly affects males and causes blindness. Last month, FDA Commissioner Scott Gottlieb told the Alliance for Regenerative Medicine in the US that the RMAT designation is a key part of its plans to help bring forward cell and gene therapies.
The Alliance for Regenerative Medicine (ARM) website has a new look that is worth checking out. ARM is pushing on PR too with a new foundation to “Promote Understanding and Acceptance of Potentially Curative Cell and Gene Therapies.”
The Alliance for Regenerative Medicine (ARM) announced the launch of the ARM Foundation for Cell and Gene Medicine, which will be dedicated to increasing public awareness and understanding of the clinical and societal benefits of cell and gene medicine — including gene therapy, gene editing, cell therapy, tissue-engineering, and organ regeneration — and supporting its development through education and research projects.
