REMS Removal Likely to Spur CAR-T Growth – and 340B Financial Risk
In a previous post, BluePath reported on the FDA elimination of REMS programs for all currently approved BCMA- and CD19-directed autologous CAR-T cell therapies. The removal of the REMS programs implies that future CAR-T utilization may be subject to a change in site of care, as CAR-Ts are increasingly administered in the outpatient setting of care. While increased access to additional sites of care is beneficial, it does expose manufacturers to a potential increase in 340B discount liability for outpatient use.
To better understand the financial risk, BluePath evaluated 2023 and 2024 CAR-T outpatient utilization in the Medicare Fee-for-Service population at the facility level. CAR-T claims include all brands commercially available at the time and 340B pricing was identified through analysis of 340B modifier codes (JG or TB) associated with each claim. Facility types may include, but not be limited to, children’s hospitals, critical access hospitals, and both long and short-term acute care hospitals among other types.
To provide a benchmark, BluePath also evaluated utilization of two market-leading PD-1s. The PD-1s were selected as a proxy for a mature market which has existing broad exposure to 340B discounts. Results of the CAR-T and PD-1 analyses are seen below:
In 2024, 45% of facility outpatient CAR-T claims were associated with 340B discount pricing. The average of PD-1 claims associated with 340B discounts was 68%, suggesting that increased outpatient use of CAR-Ts may lead to higher 340B discount exposure over time if a mature market such as the PD-1s represents a potential ceiling on 340B discount liability. Both CAR-T and PD-1 claims associated with 340B discounts grew 2% on an absolute basis year-over-year. Notably, CAR-T growth in outpatient use grew to 68 facilities in 2024, an increase of 55% over the 2023 facility count of 44 as outpatient utilization increased prior to the change in REMS.
Implications
Similar to the gradual transition of chemotherapy from inpatient to outpatient care, it is expected that CAR-T administration will also experience a shift in site of care. Trends already show an increase in outpatient utilization despite the previous presence of REMS programs and limitations on site of care from some commercial payer or institutional protocols which require FACT site accreditation. As confidence in CAR-T safety grows, administration may eventually shift from outpatient facilities to outpatient clinic settings. Patients should be a primary beneficiary of these changes as the increase in CAR-T outpatient administration of CAR-Ts increases convenience and access to care.
For CAR -T manufacturers, the expected increase in outpatient utilization should boost overall net revenue growth. However, manufacturers should be mindful that the brand gross-to-net forecast will be impacted as 340B discounts reduce net revenue in outpatient use – it will be important to assess the impact of 340B discount mix by brand in order to accurately capture the impact of this emerging outpatient utilization trend on the forecast.
Additional Information:
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