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Regulating the Use of CAR-T Therapy Outside of the Drug Paradigm

Currently, chimeric antigen receptor T-cell (CAR-T) therapies are regulated as drugs—often leading to higher prices and barriers to access for patients, as well as financial strains for hospitals. However, in a recent insights article in JAMA Health Forum, researchers examine if CAR-T therapies can be regulated outside of the current drug paradigm.

According to the authors, there are two options that exist in order to shift the regulation of CAR-T products based upon the current framework. These options, which use the FDA’s rulemaking authority and discretion, include:

  • adding a specific clause to 21 CFR §1271.10 so that hospital-manufactured CAR-T therapies are regulated solely under less stringent section 361 requirements; or
  • creating a new exception under 21 CFR §1271.15 for hospital-manufactured CAR-T therapies so that they fall outside of both sections 351 and 361 and establishing new regulations addressing quality, safety, reporting, and the like.

“A more permissive regulatory landscape would enhance competition within the CAR-T therapy market, with multiple health care systems and hospitals taking therapies from bench to bedside, like other hospital services,” they explained. “This competition, coupled with elimination of the costs of premarket approval, could lower the prices of CAR-T therapies.”

According to the article, both physicians and patients believe these therapies are safe, reliable, and efficient. However, the authors explain that under different guidelines, these therapies may face similar challenges by stem cell therapies today, with stem cell clinics increasingly touting procedures with unproven benefits and unknown risks.

With this in mind, the authors question, “is there a regulatory middle ground for CAR-T therapies that promotes the benefits of a point-of-care paradigm while maintaining confidence in safety and efficacy?”

They explained that in March 2018, there was a streamlined clinical trial process proposed by the FDA. This was aimed at facilitating an accelerated development of regenerative medicine products by smaller entities, such as individual physicians and physician groups. Further, multiple sites would agree on cooperative development and receive site-specific biologic licenses on the basis of facility-specific manufacturing information and combined clinical trial efficacy data.

“Such a regulatory approach could offer a tenable middle ground for CAR-T therapies—one in which they are still regulated as drugs, but approval is offered to multiple hospitals rather than 1 commercial entity, and the costs to meet regulatory requirements are shared across parties,” the authors explained.

In conclusion, according to the authors, regulating this therapy as a drug creates a centralized, monopoly model of commercialization and manufacturing by a few pharmaceutical companies, which ultimately is driving high prices. They believe there needs to be a serious discussion as well as consideration on regulatory alternatives in order to increase access as well as decrease associated costs.

“Regulating CAR-T therapies either under the existing 361 products regime or by creating a new hospital-based exception could lower prices by enhancing competition and eliminating the costs of premarket approval,” the authors concluded. “Such change is not out of the realm of possibility, considering analogous regulation abroad and the FDA’s willingness to innovate through regulation.” 

Chalasani R, Hershey TB, Gellad WF. Cost and access implications of defining CAR-T therapy as a drug [Published online July 22, 2020]. JAMA Health Forum. doi:10.1001/jamahealthforum.2020.0868

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