Speed Versus Safety for Cell Therapy

Faster translation into the clinic is a worthy goal, but not at the cost of patient safety and public trust.

By Karen Nichols, Chief Regulatory Officer, ISCT, and Jacques Galipeau, Chair, Mesenchymal Stem Cell Committee, ISCT.

November 2016

The Reliable and Effective Growth for Regenerative Health Options that Improve Wellness (REGROW) Act, proposed by US Senators Mark Kirk, Joe Manchin, and Susan Collins, seeks to speed up access to experimental cell therapies. The International Society for Cell Therapy (ISCT) welcomes efforts to accelerate clinical translation of promising cell therapies, but has grave concerns over the REGROW Act in its current form.

Cell therapies in the US fall under either section 351 or section 361 of the Public Health Service Act. Section 361 applies to cellular and tissue products that are minimally manipulated and often autologous – typically well-established procedures such as fat grafts. These products have less intensive regulatory requirements and would be unaffected by the proposed legislation. Section 351 covers cell products that are meaningfully manipulated; in other words, cells grown in culture, cells that have undergone genetic or physiological modification, or any cell product that involves a manufacturing step. Section 351 products are more highly regulated by the FDA – you need to get an Investigational New Drug license (IND), present rigorous evidence of safety and efficacy, and receive market authorization. The REGROW Act seeks to introduce a conditional approval pathway for these 351-designated products that would allow them to be administered to patients based on preliminary safety data and a “reasonable expectation of effectiveness” – in practice, meaning that critical Phase III clinical trials could be bypassed. Unlike other accelerated approval pathways, REGROW would not require the product to address an unmet medical need or life-threatening illness.

ISCT supports conditional approval, but only if it remains under FDA oversight and there is a clear path towards final approval, predicated on the unambiguous demonstration that the cell therapy is effective. Our concern is that REGROW would allow manufacture and marketing of very expensive therapies, for which there is no demonstrable proof of efficacy.

If this legislation is enacted, patients could legally pay for conditionally approved therapies that have no clear efficacy data. Insurers may refuse copayment on the grounds that the therapies have no proven efficacy, so the therapies are likely to be limited to the one percent of the population who can afford to pay privately for them.

Unfortunately, there is a perception that cell therapies are a magic bullet for a wide array of ailments, from baldness to autism, which obviously isn’t the case. In the Internet era, desperate patients are already just a few clicks away from clinics offering unproven therapies at astronomical costs – the REGROW Act could be seen as endorsement for these therapies from the FDA.

Despite excoriating the agency publicly, there is an implicit trust amongst the public and scientists alike that the FDA’s mandate is to protect the public. When talking to people around the world about a new product, one of the first things they ask is: do you have FDA approval or an IND? An unintended side effect of the legislation would be decreasing the moral and intellectual authority of the FDA, which not only affects the public’s trust in existing therapies and efficacious cell therapies going forward, but also in other products that the FDA approves, such as food and drinks.

For an emerging field like cell therapy, public trust is critical. Without trust, there is no funding, and without funding there is no research. We believe that REGROW could damage that trust and delay legitimate medical research in the field.