CMS To Make Hospitals Study Patients Who Get CAR-T
CMS will require hospitals that administer the expensive new CAR-T gene therapy to participate in a clinical registry or study aimed at determining whether real-world results mirror those from clinical trials used to get the treatments approved, according to a national coverage determination published Friday (Feb. 15) for chimeric antigen receptor (CAR) T-cells therapy. Drug makers were hoping to avoid such requirements, but consultants said the so-called coverage with evidence development plan was expected. CMS hasn’t released the study design, which will play a big role in determining whether hospitals participate, and one consultant said hospital participation also will depend on whether the agency increases pay for the procedure.
FDA approved two CAR-T treatments: Yescarta for the treatment of diffuse large B-cell lymphoma and Kymriah for adults with certain types of non-Hodgkin lymphoma. The technology is expensive and is expected to be applied to many other types of cancer.
Given the newness of the treatment and its potential cost, UnitedHealthcare, which has a major presence in Medicare Advantage, requested the national coverage decision. CMS has issued seven such national coverage policies in as many years. Typically, local Medicare Administrative Contractors determine coverage policies.
UnitedHealthcare did not immediately respond on whether it is happy with the coverage determination.
Hospitals that provide CAR-T must be part of a registry or clinical study that monitors patients for two years following treatments. The agency says evidence gathered in this way will help CMS identify the types of patients that benefit from the therapy, which will inform the likely future policy of covering certain types of patients without registry or trial requirements. However, coverage with evidence development programs tend to be open-ended; CMS started a evidence development-development program for transcatheter aortic valve replacement in 2012 that is still running.
CMS is only covering the treatment for FDA-approved indications and has yet to say how many patients the study will accept. If the study limits enrollment, as some past coverage with evidence development programs have done, it may be difficult for patients to get the treatment, a consultant said. It also isn’t clear whether patients will be able to get covered for the treatment during the time that results from the study are being analyzed, which can take a year or more, the consultant said, even though FDA approved the drug.
There is a 30-day comment period for the proposed coverage decision, and the agency will receive advice on the study design during that time.
However, the coverage decision outlines how the study will work. Hospitals will have to monitor how patient outcomes compare to either the clinical trials that the companies ran for FDA approval or to a cohort of controls receiving standard of care treatment. They’ll compare clinical characteristics of registry patients to those in the FDA trials and determine how those characteristics affect how well the treatment works.
The study also will include patient-reported outcomes on quality-of-life questions. -- John Wilkerson ([email protected])