- Medicare and Medicaid will cover CAR-T cell therapies nationally, a decision that helps address, but doesn't fully solve, the reimbursement challenges that have hampered uptake of the pricey cancer drugs.
- The change, finalized Wednesday by CMS, was a long time in the making, arriving nearly two years after the Food and Drug Administration approved the first CAR-T therapy, Novartis' Kymriah.
- Hospitals still won't be fully reimbursed for the cost of administering CAR-T therapies, which carry price tags in the hundreds of thousands of dollars and typically involve overnight stays in a hospital. But the finalized rules replace with a nationwide policy a patchwork of local determinations on whether to pay for the drugs.
CMS' decision gives CAR-T cell therapy a firmer footing and could help drive use of the cutting-edge treatments, which consist of patient immune cells genetically engineered to target tumors.
CMS also broadened the settings in which CAR-T would be covered, applying its final rule to use in "healthcare facilities" rather than explicitly in hospitals.
For all of the technology's clinical promise — CAR-T can spur lasting remissions in some lymphoma and leukemia patients — its commercial impact has been modest.
Sales of the two approved products, Kymriah (tisagenlecleucel) and Gilead's Yescarta (axicabtagene ciloleucel), have grown slowly, and some eligible patients reportedly are seeking out CAR-T clinical trials instead.
One major hang-up is reimbursement. Kymriah costs $475,000 for pediatric and young adult patients with leukemia and both therapies are priced at $373,000 to treat lymphoma in adults. Patients given CAR-T therapy are at risk of serious side effects, requiring intensive monitoring and potentially other drugs to control.
All of that makes CAR-T a challenging financial proposition for hospitals, which have been losing as much as $200,000 per patient treated.
Under regulations published this month, Medicare will reimburse at least 65% of the treatment's cost, or about $242,000, through Part B. The coverage determination issued Wednesday applies that nationwide, instead of leaving the decision on whether to pay up to local administrative contractors, as it was previously.
That will give hospitals surety they will receive payment for the drug, yet doesn't necessarily make whole the full cost of treating a patient with CAR-T. The cell therapies are also still reimbursed via billing codes for bone marrow and stem cell transplants, rather than using a separate identifier.
CMS administrator Seema Verma has acknowledged the difficulties the agency has in solving reimbursement questions around CAR-T.
"Technology is moving faster than government policies," she said in April, indicating development of a unique billing code for CAR-T could take as long as three years.
Wednesday's announcement is more than two months after CMS was expected to release its determination, a delay reportedly due to clashes between politically appointed agency staff and career employees.
The finalized rules also differ from a proposed determination unveiled in February. CMS dropped plans to cover CAR-T through a process that required hospitals to collect information on real-world CAR-T use, instead opting to rely on the FDA's risk plans for the therapies.
The determination applies only to autologous, or derived from the same patient being treated, therapies that rely on T cells engineered to express a chimeric antigen receptor. Any allogeneic, or "off-the-shelf," therapies later approved by the FDA would not be covered.
Finalization of the national determination could help accelerate Gilead's efforts to expand Yescarta's use in lymphoma patients over 65, a group CEO Daniel O'Day recently described as a particular focus.