Finalized CMS Rule Supports Medicare Coverage for Gene Therapy

CMS finalized a decision to provide Medicare coverage for chimeric antigen receptor (CAR) T-cell therapy, which is an FDA-approved gene therapy used to treat non-Hodgkin lymphoma and B-cell precursor acute lymphoblastic leukemia.

“Today’s coverage decision provides consistent and predictable patient access nationwide,” said CMS Administrator Seema Verma. “CMS will work closely with our sister agencies to monitor outcomes for Medicare patients receiving this innovative therapy going forward.”

Four percent of all cancer patients in the US are diagnosed with non-Hodgkin lymphoma, making it one of the most prevalent cancers nationwide. While the disease largely affects older populations, with over fifty percent of those diagnosed being 65 years old or above, it is also one of the most common cancers among minors and young adults.

For payers, cancer remains one of the costliest diseases for chronic disease management in the US.

CAR T-cell therapy is the first type of FDA-approved gene therapy. The treatment genetically alters the patient’s T-cells to enable them to fight cancer cells. CAR T-cell therapies are classified as biologics and have been covered under Medicare’s policies that apply to biologicals. There are two FDA-approved CAR T-cell therapies on the market, tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta).

CMS will cover CAR T-cell therapies when they are provided in a healthcare facility that complies with FDA’s risk evaluation and mitigation strategies (REMS). The treatment must be used for an FDA-approved indication as directed by the FDA’s label.

CMS will also cover off-label uses if the therapy is used in accordance with CMS-approved compendia.

Lastly, the rule extended its coverage of routine costs in NCD 310.1-compliant CAR T-cell clinical trials.

Use of CAR T-cell therapies that have not been FDA-approved will not be covered under the finalized rule, CMS made clear. In cases when an autologous treatment is used, such as chemotherapy, with T-cells that express one or more CARs but the Section A requirements are not satisfied, the treatment will not be covered.

Because these treatments are still new and have been associated with serious toxicities, the agency recognized the need for continued evaluation of the Medicare population undergoing CAR T-cell therapy. It therefore will use FDA post-approval safety studies and mandated that the drug manufacturers track patients post-marketing.

“We remain committed to supporting the efficient development of safe and effective CAR T-cell therapies,” said acting FDA Commissioner Ned Sharpless, MD. “We know there are relatively limited data about the use of these life-saving therapies in the Medicare population. Our robust post-market surveillance programs will continue to monitor for potential risks, as we do for all licensed and approved medical products. We will also continue to carefully assess the benefits and risks when considering whether to approve new CAR T-cell products.”

Earlier versions of the rule were met with controversy from different quarters of the industry. The rule’s critics expressed the fear that there would not be enough flexibility for reimbursement beyond Kymriah and Yescarta.

“We are concerned that the agency’s draft coverage determination is not flexible enough to accommodate advances in this rapidly developing field, recognize the progress made towards decreasing toxicity, account for ongoing research on new indications and earlier lines, and adapt existing frameworks to deliver the promise of innovation to patients in the most robust way,” the Community Oncology Alliance (COA) commented.

Community oncologists pushed back significantly against the Coverage With Evidence With Development (CED) portion of the original model, a data collection method. The oncologists believed it would place too much administrative burden on physicians and disincentivize them from using the treatments, particularly among community practices.

“The CED requirements would add additional administrative processes and patient consents on top of those that are required by other entities,” the COA continued. “We are concerned that the fact that the studies outlined in the NCD would not be funded by CMS, resulting in providers assuming additional costs to administer CAR-T treatments, which may further adversely restrict access.  The strict and complex registry approval requirements that are outlined in the NCD would require additional infrastructure investments from providers.”

As a result, CMS removed the CED from the finalized rule entirely. In the agency’s response to public comments, it underscored that data collection will continue to occur through drug manufacturers’ mandatory post-marketing and federally-supported NCD 310.1 clinical trials.

As part of its policymaking process, CMS conducted a study on CAR T-cell therapy which produced uncertain results, due to the small sample-size and short-term nature of the study. However, further research provided by other sources reported high and durable remission rates.

Private payers have also been making strides in providing improved care, primarily by expanding their coordinated care programs.