The International Society for Cellular Therapy (ISCT), the global society of clinicians, researchers, regulatory specialists, technologists and industry partners dedicated to the translation of cellular therapy into safe and effective therapies to improve patients’ lives, has announced its reasons for opposition to the current version of the REGROW Act — the US government’s legislative efforts to promote faster patient access to effective new cellular therapies.
ISCT supports, and has undertaken decades of activity with regulators in legislative efforts that seek to increase availability and financial accessibility of cell therapies whilst not compromising either safety or efficacy. In addition, ISCT has also supported progress through appropriate fast track treatments, breakthroughs and accelerated approvals as well as patient-focused tools such as compassionate use for individual patient situations. These all play a key role in addressing unmet medical needs. ISCT has additionally taken a series of initiatives to counter industry practice of unproven cell therapies (see press release here).
ISCT does support addressing the unmet need, stated by the proposed REGROW legislation, for products designated 351 in the regulatory pathways, to allow patient access to these therapies more effectively.
However, ISCT has identified a number of key features of the legislation that fail to address or provide clarity that will be needed to provide affordable and accessible treatment to patients. As a result, ISCT cannot support the current proposed legislation and strongly calls for a redraft to address the following concerns.
- All new proposed therapies must have a firm scientific basis for conditional approval relying on, for example, peer-reviewed preclinical, early clinical, or other relevant data that support a scientifically credible hypothesis of how the products might provide benefit.
- A conditional approval process predicated on preliminary clinical evidence of safety and a reasonable expectation of efficacy must provide a unique patient benefit beyond what is provided by existing regulatory processes. This must include eligibility for Medicare and Medicaid Services and insurance plans. Any conditionally approved product should be granted fast track review for CMS coverage.
- A risk/benefit algorithm must be used to assess which therapies qualify for Conditional Approval. It is critical that there is preliminary clinical evidence of safety and a reasonable expectation of efficacy. This method must consider all critical factors equally — the patients need, the ability to reasonably access alternative FDA-approved therapies and the seriousness of condition to be treated.
- Any conditionally approved product definition shall not conflict with existing guidance or interim guidance from the FDA.
- ISCT believes the rights of patients are paramount, especially in the case of individuals seeking recourse if treated with approaches for which conditional approval has been granted. Critical to this process is attention to the tenets of informed consent designed to clarify in lay terms the evidence, potential risks and potential benefits.
“ISCT has a unique role and position in the field, representing all elements of cell therapy. As a result, the Society has, and will continue to work on international consensus documents to progress approved cell therapeutics for the treatment of patients with a range of conditions. We also recognize the role for the United States Department of Health and Human Services to work with the entire industry to develop standards for manufacturing processing and controls of PHS Section 351 regenerative medicine products,” said Catherine Bollard, MBChB, MD, ISCT President.
“Last year, ISCT took the lead on tackling the problem of unproven cellular therapies with the launch of a publication and reference guide for those involved in the development of cell therapies. Therefore, in response to the proposed forthcoming REGROW Act, ISCT strongly believes it is critical that cell technologies regulated under Sections 351 as well as 361 all demonstrate a positive benefit/risk ratio through properly designed clinical trials, to provide patients the best opportunity to access demonstrably safe and effective cell therapies.”