FDA Lifts Key Barriers to Immunotherapies Access, Signaling Regulatory Shift

The FDA has eliminated special safety restrictions for a class of immunotherapies widely used to treat patients with certain blood cancers, due to growing evidence that concerns about serious side effects occurring weeks after administration weren’t seen in the real world.

The agency announced on June 27 that its Risk Evaluation and Mitigation Strategies, or REMS, requirements for CAR T cell therapies, used to treat patients with multiple myeloma or certain types of leukemia or lymphoma, would no longer be required. These rules had mandated that patients remain near treatment centers with a caregiver for a month after administration, and restrict their driving for up to eight weeks.

The change received wide praise across social media by industry organizations, physicians and patient advocates. The Alliance for Regenerative Medicine (ARM) celebrated the move in a LinkedIn post, saying the change would help more patients receive cell and gene therapies.

“ARM applauds the FDA’s leadership in making a flexible, evidence-based decision that will help more patients access these life-changing therapies,” the organization said. “We believe this patient-centric change will dramatically lower the cost and inconvenience of receiving potentially lifesaving CAR-T care, helping patients and their caregivers overcome geographic and logistical barriers.”

Surbhi Sidana, MD, a hematologist and an Associate Professor at Stanford Medicine posted that the policy change “is huge in terms of easing patient burden and CART access,” by decreasing the time patients have to stay near the treatment center.

The American Society for Transplantation and Cellular Therapy (ASTCT) took credit for helping bring about the decision, citing their advocacy efforts and a white paper the organization produced arguing for the removal of the REMS restrictions.

“These are wonderful advances,” said Sarah Nikiforow, MD, PhD, cochair of the ASTCT subcommittee that worked on the white paper, in a YouTube video the organization posted. “It really reflects the collaboration between FDA accrediting bodies, clinical sites and our commercial partners, and we expect to keep that collaboration going.”

“Patients will be able to get access to CAR T cell therapy more broadly now that we don't have the administrative burdens of the REMS programs to contend with,” said Frederick L. Locke, MD, Chair of the Department of Blood and Marrow Transplant and Cellular Immunotherapy in the video.

Anti-Cancer Therapies Manufactured with the Patient’s Own T Cells

CAR Ts are therapies typically manufactured with a patient’s own T cells, with genetic modifications to help them to better recognize and attack cancer cells. These medicines have shown great promise in helping patients achieve long-term remission, however, they can cause serious and even potentially deadly side effects called cytokine release syndrome (CRS) and neurotoxicity syndromes.

These side effects can be managed with the right treatment plan and medication, which is why the FDA has been requiring patients to remain near their treatment center for a month following administration. However, because of the complex nature of the drugs, they are mainly available at large academic medical centers, meaning patients would need to live away from home with a caregiver. This restriction, as well as the high cost of these medications, has caused many patients to forgo this form of treatment.

A study published last year showed that around 60 percent of patients experienced CRS, the symptoms typically occurred in the first week after treatment, with few, if any serious side effects popping up after the second week. The study concluded that the four-week monitoring period was adding unnecessary physical and financial restraints on patients.

In its announcement, the FDA justified the change citing growing evidence that the anti-cancer treatments can be safely managed without additional regulatory oversight. Since the first CAR T was approved in 2017, physicians have grown more accustomed to treating patients and managing the side effects. This decision could also make these drugs more widely available by lowering the regulatory requirements for hospitals and providers, the FDA said.

“Eliminating the REMS that is no longer needed also expedites the delivery of potentially curative treatments to patients and reduces burden on providers,” said Vinay Prasad, MD, MPH, chief medical and scientific officer at the FDA’s Center for Biologics Evaluation and Research, in the news release.

The policy change impacts all currently approved CAR T therapies, including Abecma, Breyanzi, Carvykti, Kymriah, Tecartus, and Yescarta.