Wednesday, May 16, 2018

Brief Blog; Medicare Opens NCD Process for CAR-T Therapies

On May 16, 2018, CMS opened a National Coverage Determination process for CAR-T therapies, based on a request from United Healthcare dated February 22, 2018.
  • The NCD tracking sheet is here.
  • The UHC letter is here.
    • The letter specifically refers to creating a "level playing field" for Medicare Advantage plans in regard to these costly drugs.
    • The letter proposes on-label coverage with a flexibility for important valid uses that may not be on the FDA label.   
  • Coverage at BioPharmaDive, here.
The timetable includes an initial open comment period (on the topic; there is no draft NCD yet) from May 16-June 15, 2018.   CMS will hold a "MedCAC" public meeting on August 22, 2018.

The draft NCD is expected February 26, 2019, and the final NCD (after a comment and review period) is expected May 17, 2019.

Of note, CMS had a relatively extended and skeptical range of criticism in April 2018 inpatient rulemaking, in response to a request for new technology add-on payments (NTAPs) for Kymriah and Yescarta.  See the relevant pages in the cloud, here.  (Somewhat scarily, on page 20189, CMS referred to the diseases under treatment as "hematopoietic carcinomas."  Ouch.  Carcinomas are normally other classes of cancers, excluding leukemia and lymphoma.)

The last NCD and MedCAC on a cancer drug covered Provenge for on-label uses in 2011, here, here.

I've clipped the CMS announcement of the NCD process below.  The lead analyst is Katherine Szarama, PhD; the lead medical officer is Lori Paserchia MD.

National Coverage Analysis (NCA) Tracking Sheet for CHIMERIC ANTIGEN RECEPTOR (CAR) T-cell Therapy for Cancers (CAG-00451N)

CAR T-cell therapy is a precision cancer treatment wherein each treatment dose is individually manufactured for the patient using their own T-cells, a type of white blood cell known as a lymphocyte. CAR T-cell therapy is a rapidly emerging adoptive cell transfer immunotherapy for select patients with relapsed or refractory cancers. Treatment protocols vary, but may be summarized in five steps:

  • lymphocyte harvesting from the patient with cancer;
  • creation of cancer-targeting lymphocytes in vitro using various immune modulators;
  • selection of lymphocytes with reactivity to cancer antigens using enzyme-linked immuno-assay;
  • depletion of the patient's remaining lymphocytes using immunosuppressive agents;
  • transfusion of the cancer-targeting lymphocytes back into the patient with cancer-this transfusion represents one treatment.

In 2017, the Food and Drug Administration (FDA) approved tisagenlecleucel (Kymriah®) and axicabtagene ciloleucel (Yescarta®) CAR T-cell therapies for the treatment of certain relapsed or refractory leukemias and lymphomas. The two FDA-approved therapies include boxed warnings (calling attention to serious and life-threatening risks) and safety monitoring through an FDA Risk Evaluation and Mitigation Strategy (REMS).

To date, few Medicare patients have been studied and follow up has been limited. The majority of patients who received CAR T-cell therapy also experienced adverse events including cytokine release syndrome and neurologic effects. Initial studies were also confined to the inpatient hospital setting.

On August 22, 2018, we are convening a Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) meeting to review the available evidence on this topic.