Researchers Highlight Restrictive Coverage of Gene, Cell Therapies

As one-time gene and cell therapies continue to enter the market with larger upfront costs and potentially larger patient populations, the health care system, particularly Medicaid, has had to adjust to these new types of treatments. Among the challenges created by these advanced therapies, write the researchers of a new paper in Molecular Therapy: Methods & Clinical Development, are inconsistencies between gene and cell therapy indications and payor coverage.

The researchers focused on coverage trends across an array of disease states, including lymphoma, vision loss, and spinal muscular atrophy, finding discrepancies in coverage across 16 states and Medicaid Care Organizations (MCOs).

“Medicaid coverage for some of the first gene and cell therapies on the market has sometimes been inconsistent with federal legal requirements, with delayed and narrowly defined coverage,” detailed the researchers in their paper. “Even when covered by a Medicaid program, various factors in the appeals and administration process cause delays in time to treatment. For gene and cell therapies specifically, this delay can have a negative impact on patient health outcomes and often leads to disqualification from treatment, and it could deny patients the only or last option for treating their disease.”

With over 3600 cell and gene therapies in the pipeline, the findings have important implications for payers, explained the group, noting that Medicaid often covers large portions of patients on these treatments.

The researchers focused on coverage trends for Kymriah (tisangenlecleucel), Luxturna 9voretigene neparvovec), and Zolgensma (onasemnogene abeparvovec).Across the 3 treatments, coverage was often more restrictive than the FDA labels of the treatment. For example, 14 states/MCOs included more restrictive coverage for Zolgensma and 9 had more restrictive coverage for Luxturna. No payors included in the analysis covered Zolgensma according to its approved indication.

Restricted coverage was less common for Kymriah, which was implemented in 4 states/MCOs; 9 payors covered the treatment according to its labeled indication. Greater restrictions were implemented for pregnancy, and one was for required documentation of lymphocyte counts.

“Unfortunately, our study suggests that state coverage policies, including those of MCOs, are not always aligned with the spirit of the law,” wrote the researchers. “Despite Medicaid patients meeting the ‘medically accepted indication’ standard, authors have anecdotal experience with many of these patients receiving a denial of insurance approval. This denial can be accompanied by requests for additional information, likened to ‘eligibility criteria,’ that is above and beyond simply requesting confirmation that a patient’s condition corresponds to the labeled indication of the commercial product.”

Outside of prior authorizations, coverage disputes and delays can affect a patient’s timely access to gene therapies. Due to the rare nature of certain diseases treated by gene or cell therapies, such treatment is often administered in centers of excellence, which Medicaid and Medicaid MCOs cover costs for even if out of state. However, negotiating these payments with the centers of excellence may require many resources and can be time consuming, creating treatment delays particularly for patients receiving treatment out of state.

Reflecting on the patterns identified in their paper, the researchers outlined several opportunities for the federal government to play an active role in ensuring appropriate access to gene and cell therapies:

• Provide additional guidance to states that outline federal requirements for providing coverage according to a label
• Reinforce timely coverage when prescribed according to label and limit requirements of certain additional criteria to be met outside of the label
• Create clear avenues for stakeholders to report noncompliance

Reference

Allen J, Berry D, Cook F, et al. Medicaid coverage practices for approved gene and cell therapies: existing barriers and proposed policy solutions. Mol Ther Methods Clin Dev. 2023;29:513-521. doi:10.1016/j.omtm.2023.05.015