Trend Watch

CAR T-cell therapy made available to all Medicare recipients

Seema Verma, MPH
Seema Verma

On Wednesday, the Trump Administration made chimeric antigen receptor T-cell therapy available to all Medicare beneficiaries via a long-awaited final coverage decision made by the CMS.

“As the first type of FDA-approved gene therapy, CAR T-cell therapies are an important scientific advancement in this promising new area of medicine and provide treatment options for some patients who had nowhere else to turn,” Seema Verma, MPH, CMS administrator, said in a press release.

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

The FDA has approved two CAR T-cell therapies that would apply to the Medicare population: axicabtagene ciloleucel (Yescarta; Kite/Gilead) and tisagenlecleucel (Kymriah; Novartis) both for adults with relapsed or refractory large B-cell lymphoma. The FDA has also approved both treatments for pediatric patients with relapsed or refractory acute lymphoblastic leukemia.

CMS said Medicare would cover CAR T-cell therapies when they are administered at facilities enrolled in its risk evaluation and mitigation strategies (REMS) program. Medicare will cover both FDA-approved indications for CAR T-cell therapies, in addition to off-label uses that are recommended by CMS-approved compendia.

Medicare coverage for off-label use of FDA-approved CAR T-cell therapies will need to be supported by recommendations from one or more CMS-approved compendia, such as ones provided by the National Comprehensive Cancer Network, policy staff at the American Society of Hematology explained to HemOnc Today.

“CMS will cover approved treatment for an off-label indication if it’s included in any of the compendia that have been approved as an authoritative source for determining medically accepted indications,” an NCCN spokesperson confirmed for HemOnc Today.

Samuel Silver
Samuel M. Silver

Off-label indications for CAR T-cell therapies that are in CMS approved compendia are now covered by Medicare, according to health policy expert Samuel M. Silver, MD, PhD, assistant dean for research and professor of internal medicine/hematology-oncology at the University of Michigan’s Rogel Cancer Center and a member of HemOnc Today’s Editorial Board.

"To my knowledge the NCCN is the only compendia where CAR T-cell therapy is discussed,” he told HemOnc Today. “This decision leaves the door open for Medicare off-label coverage without having formal FDA approval based on the results of ongoing studies.”

Silver said this could include other B-cell neoplasms not indicated on the label, such as chronic lymphocytic leukemia.

The initial FDA approvals for axicabtagene ciloleucel and tisagenlecleucel required that the manufacturers of both treatments conduct post-approval observational studies to monitor safety issues associated with the products.

Both FDA-approved CAR T-cell therapies have been associated with treatment-related toxicities that include neurotoxicity and cytokine release syndrome.

Ned Sharpless

“We know there are relatively limited data about the use of these life-saving therapies in the Medicare population,” Ned Sharpless, MD, FDA acting commissioner, said in the release.

“Our robust postmarket 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.” – by Drew Amorosi

Reference:

CMS. Trump administration makes CAR T-cell cancer therapy available to Medicare beneficiaries nationwide [press release]. August 7, 2019. Available at: www.cms.gov/newsroom/press-releases/trump-administration-makes-car-t-cell-cancer-therapy-available-medicare-beneficiaries-nationwide. Accessed August 8, 2019.

For more information:
Samuel M. Sliver, MD, can be reached at 5116 Med Sci 1, SPC 5611, 1301 Catherine St., Ann Arbor, MI 48109-5624; email: msilver@med.umich.edu.

Disclosures: Silver is on the board of directors of the NCCN and reports no relevant financial disclosures.

Seema Verma, MPH
Seema Verma

On Wednesday, the Trump Administration made chimeric antigen receptor T-cell therapy available to all Medicare beneficiaries via a long-awaited final coverage decision made by the CMS.

“As the first type of FDA-approved gene therapy, CAR T-cell therapies are an important scientific advancement in this promising new area of medicine and provide treatment options for some patients who had nowhere else to turn,” Seema Verma, MPH, CMS administrator, said in a press release.

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

The FDA has approved two CAR T-cell therapies that would apply to the Medicare population: axicabtagene ciloleucel (Yescarta; Kite/Gilead) and tisagenlecleucel (Kymriah; Novartis) both for adults with relapsed or refractory large B-cell lymphoma. The FDA has also approved both treatments for pediatric patients with relapsed or refractory acute lymphoblastic leukemia.

CMS said Medicare would cover CAR T-cell therapies when they are administered at facilities enrolled in its risk evaluation and mitigation strategies (REMS) program. Medicare will cover both FDA-approved indications for CAR T-cell therapies, in addition to off-label uses that are recommended by CMS-approved compendia.

Medicare coverage for off-label use of FDA-approved CAR T-cell therapies will need to be supported by recommendations from one or more CMS-approved compendia, such as ones provided by the National Comprehensive Cancer Network, policy staff at the American Society of Hematology explained to HemOnc Today.

“CMS will cover approved treatment for an off-label indication if it’s included in any of the compendia that have been approved as an authoritative source for determining medically accepted indications,” an NCCN spokesperson confirmed for HemOnc Today.

Samuel Silver
Samuel M. Silver

Off-label indications for CAR T-cell therapies that are in CMS approved compendia are now covered by Medicare, according to health policy expert Samuel M. Silver, MD, PhD, assistant dean for research and professor of internal medicine/hematology-oncology at the University of Michigan’s Rogel Cancer Center and a member of HemOnc Today’s Editorial Board.

"To my knowledge the NCCN is the only compendia where CAR T-cell therapy is discussed,” he told HemOnc Today. “This decision leaves the door open for Medicare off-label coverage without having formal FDA approval based on the results of ongoing studies.”

Silver said this could include other B-cell neoplasms not indicated on the label, such as chronic lymphocytic leukemia.

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The initial FDA approvals for axicabtagene ciloleucel and tisagenlecleucel required that the manufacturers of both treatments conduct post-approval observational studies to monitor safety issues associated with the products.

Both FDA-approved CAR T-cell therapies have been associated with treatment-related toxicities that include neurotoxicity and cytokine release syndrome.

Ned Sharpless

“We know there are relatively limited data about the use of these life-saving therapies in the Medicare population,” Ned Sharpless, MD, FDA acting commissioner, said in the release.

“Our robust postmarket 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.” – by Drew Amorosi

Reference:

CMS. Trump administration makes CAR T-cell cancer therapy available to Medicare beneficiaries nationwide [press release]. August 7, 2019. Available at: www.cms.gov/newsroom/press-releases/trump-administration-makes-car-t-cell-cancer-therapy-available-medicare-beneficiaries-nationwide. Accessed August 8, 2019.

For more information:
Samuel M. Sliver, MD, can be reached at 5116 Med Sci 1, SPC 5611, 1301 Catherine St., Ann Arbor, MI 48109-5624; email: msilver@med.umich.edu.

Disclosures: Silver is on the board of directors of the NCCN and reports no relevant financial disclosures.

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