Since the first chimeric antigen receptor T-cell (CAR-T) launched in 2017, the Food and Drug Administration (FDA) has mandated Risk Evaluation and Mitigation Strategy (REMS) requirements to ensure safe treatment delivery and that providers are educated on how to recognize and manage adverse events. REMS requirements have played a pivotal role in shaping CAR-T delivery by requiring treatment centers to complete product-specific training and maintain supply of supporting medications. Additionally, the FDA mandated that patients remain within a 30-minute drive of the site of care for four weeks after infusion. While these requirements introduced important safety monitoring features, they have also presented an additional layer of care coordination complexity and barriers to scale CAR-T therapy.

In a significant regulatory shift, the FDA announced the elimination of REMS requirements for CAR-T products on June 27, signaling a new era for cell and gene therapies and increased confidence in real-world clinical management of CAR-T toxicities and broader integration into standard oncology practices. The announcement means that there will be:

• No more FDA-mandated requirements for hospital certification tied to REMS, including on-site immediate access to tocilizumab
• Updated product labeling without a separate REMS program
• Streamlined post-treatment monitoring guidelines:
  • Daily monitoring for approximately one week, then continued monitoring through two weeks post-infusion (down from four)
  • Patients will be required to stay near the treatment center for two weeks (down from four)
  • Patients are to avoid driving for two weeks (down from eight)

These updates have far-reaching implications, from the patient journey (including access), coverage and Center of Excellence (COE) networks, and outpatient/community expansion efforts.

Implications for the Patient Journey and Access

While financial assistance for ancillary costs associated with the monitoring period post-infusion are available to a subset of patients, resources are often fragmented across multiple vendors and may not cover all expenses. Shortening the required follow-up monitoring period from 30 days to two weeks may help alleviate some of the financial and logistical challenges faced by patients and their caregivers, particularly for patients living in suburban or rural areas who must travel long distances or secure and pay for extended lodging. A prior Avalere Health analysis found that more than 70% of potential CAR-T patients across different indications are located >30 minutes from a treatment site.

The recent FDA changes not only have the potential to reduce the financial and logistical burden associated with CAR-T treatments such as lodging, transportation, and lost wages, but also support a faster return home for the patient and clearance to drive. In combination, these changes could improve treatment accessibility, especially for patients in underserved or remote communities where travel and proximity to COEs may have historically been a barrier to-date.

Considerations for Coverage and COE Networks

While the removal of REMS requirements for B-cell maturation antigen- and CD19-directed CAR-T therapies is a positive driver toward enabling outpatient expansion, the access impact depends in part on how payers adjust their network requirements and whether they expand current networks to include CAR-T providers in the outpatient setting.

Although outpatient sites will need to ensure that appropriate infrastructure and clinical training are implemented for CAR-T, many potential outpatient sites may not meet Foundation for the Accreditation of Cellular Therapy (FACT) standards. FACT accreditation is a voluntary accreditation process denoting that a site adheres to comprehensive guidelines related to the collection and processing of cells and CAR-T infusion. Most national commercial payers that maintain COE networks for CGTs require FACT accreditation as a condition for participation, leaving questions around what role these standards will continue to play in the establishment of treatment networks for CGTs.

Additionally, broader adoption of CAR-T in outpatient settings may shift referral patterns as providers increasingly consider administering CAR-T within their scope of care. These changes could alter how patients access care and which sites become key access points in the evolving CAR-T landscape.

Next Steps

Avalere Health’s subject matter experts in policy, market access, and evidence assessment can help manufacturers and sites of care understand what each of these updates mean for your organization and how to respond accordingly to increase access to CAR-T therapies and enhance the landscape of patient care.

To better prepare for and shape the evolving CGT ecosystem in 2025 and beyond, connect with us.