Pink Sheet: global policy and regulatory coverage
By John Davis 31 Mar 2020
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the...
The US Centers for Medicare and Medicaid Services has initiated a national Medicare coverage analysis to evaluate the clinical data on the chimeric antigen receptor T-cell (CAR-T) therapies for cancer, which may be followed by development of a national Medicare coverage policy for such treatments.
A national policy could ensure consistent coverage for the therapies across the US because it would supersede potentially incongruent decisions by regional Medicare contractors.
CMS is scheduled to release a draft decision memo on the results of the CAR-T analysis Feb. 16, 2019, according to an agency “tracking sheet” on the initiative posted May 16. The decision may take the form of a national coverage determination (NCD), which will set out the conditions of coverage, such as whether Medicare will pay for off-label uses.
The process involves a public comment period, ending June 15, and CMS will convene a Medicare Evidence Development & Coverage Advisory Committee (MedCAC) meeting Aug. 22 to consider the available data and coverage possibilities.
Because the process will take several months, stakeholders are expected to urge CMS to ensure that patient access doesn’t suffer while a final decision is being deliberated.
Novartis AG, marketer of the CAR-T drug Kymriah, told the Pink Sheet it plans to provide comments to the agency as part of the coverage analysis process and “we support the open dialogue around coverage of CAR-T cell therapies to ensure patient access to this innovative treatment.” But “in the meantime, Novartis also recommends that CMS implement measures to ensure appropriate patient access to these innovative products while the broader NCD process continues.”
Similarly, American Society for Blood and Marrow Transplantation Health Policy Director Stephanie Farnia told the Pink Sheet that “while an NCD provides uniform national standards for coverage, the current process for the initial decision and for subsequent changes is lengthy and cumbersome. We are concerned that access may be limited during the NCA process and hope that CMS issues guidance that beneficiaries should be able to continue to receive the therapy during the NCA process.”
UnitedHealthCare requested that CMS issue an NCD for CAR-T therapiesin a Feb 22 letter, arguing that the treatments present unique issues that could complicate coverage policy.Kymriah (tisagencleucel) andGilead Sciences Inc./Kite Pharma Inc.’sYescarta(axicabtagene ciloleucel) are the only CAR-T therapies on the US market as yet, both approved for B-cell lymphoma.
The insurer also pointed to the high cost of the treatments as an issue. List prices for Kymriah and Yescarta are $475,000 and $373,000, respectively.
A national coverage policy could “clarify the circumstances under which the therapies will be covered and to create consistent patient access to the therapies across the country and financial sustainability in the Medicare Advantage program with regard to these therapies,” according to the insurer.
“Given the complexity of the therapy, treating patients with acute life-threatening disease requiring the manufacture of an individualized product, the potential for severe and also life-threatening side-effects necessitating specialized expertise to manage, and the high cost of the products and associated care required, a [NCD] is essential to ensure that coverage is available to the Medicare population and that the criteria used to determine eligibility for coverage are evidence-based and are consistent regardless of the state of residence of the beneficiary,” it added.
UnitedHealthcare proposed that coverage for CAR-T therapy be limited to the FDA-approved indications.However, the firm also said “given that CAR-T cell therapy is an innovative therapy, and ongoing clinical trials are likely to identify new patient populations who may benefit, which may not all be reviewed by the FDA, we urge CMS to develop a process to update the NCD as new evidence emerges.”
NCDs set a national coverage policy for products or services provided under the Medicare Part A hospital inpatient benefit or the Part B physician outpatient benefit which includes physician-administered drugs; they do not apply to the Part D drug benefit.
NCDs are rarely issued for therapeutic drugs and biologics. Typically CMS relies on its local Medicare contractors to make coverage decisions for such products.
The last NCD for a therapeutic drug or biologic was issued in 2011 for Dendreon Corp.’s prostate cancer vaccine,Provenge(sipuleucel-T). In that decision, CMS said it would cover Provenge for its labeled uses and would allow its local contractors to decide whether to cover off-label uses. However, off-label coverage could only be allowed in the context of a clinical trial, the decision said. (Also see "Medicare Will Cover Provenge Labeled Uses, CMS Proposes; Off-Label Is Up To Contractors" - Pink Sheet, 30 Mar, 2011.)
Provenge and the CAR-T therapies are cutting edge and highly expensive treatments (Provenge launched at a list price of $93,000). But CMS is not authorized to base coverage decisions on cost. Instead the agency indicates it will focus on the safety issues surrounding Kymriah and Yescarta and limitations in the data.
“The two FDA-approved therapies include boxed warnings … 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,” CMS said in the tracking sheet. “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.”
The coverage parameters that may be addressed by an NCD are separate from the actual payment mechanics of covering CAR-T drugs, like reimbursement coding and assignment of the treatment to a diagnosis-related group (DRG) for claims. CMS addressed those issues in its recent hospital inpatient prospective payment system draft rule for 2019. (Also see "CAR-T Reimbursement: Medicare Weighing Bundle For Kymriah, Yescarta In 2019" - Pink Sheet, 25 Apr, 2018.)
Pink Sheet: global policy and regulatory coverage
By Ian Schofield 31 Mar 2020
As part of its efforts to preserve patient access to medicines amid the COVID-19 pandemic, Russia is planning to cap...
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