Institute for Clinical and Economic Review Final Report on CAR-T Therapies Highlights Need for Systemwide Changes to Ensure Affordability and Access to Innovative, One-Time Therapies

-Policy recommendations highlight that even though the pricing of current CAR-T therapies aligns with patient benefit there will be changes needed in future pricing, payment, and delivery mechanisms to ensure patient access without threatening health system affordability-

BOSTON, March 23, 2018 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance on tisagenlecleucel (KymriahTM, Novartis) and axicabtagene ciloleucel (YescartaTM, Kite Pharma/Gilead), two CAR-T therapies for treatment of B-cell cancers.

ICER’s report was reviewed at a public meeting of the California Technology Assessment Forum (CTAF), where a majority of the panel voted that evidence is sufficient to show that tisagenlecleucel provides a net health benefit for children with B-cell acute lymphoblastic leukemia (B-ALL) and that both tisagenlecleucel and axicabtagene ciloleucel provide a net health benefit for adults with certain B-cell subtypes of non-Hodgkin’s lymphoma (NHL). The two therapies were compared to commonly used chemotherapy regimens for each indication. Evidence was not sufficient to distinguish between the two CAR-T therapies for treatment of NHL.

A majority of the panel further voted that tisagenlecleucel provides intermediate long-term value for money when treating B-ALL. While ICER’s assessment determined that the price of the therapy aligns with its clinical value, the panel noted that the significant uncertainty surrounding the long-term risks and benefits of the therapy precluded a high-value vote. After deliberating on the value of axicabtagene ciloleucel to treat NHL, the panel’s votes were split between low value and intermediate value, driven by similar concerns about long-term uncertainty.

“Given the currently available evidence, these therapies appear to be effective options for those with B-ALL or NHL, though uncertainty in the evidence raised questions around the long-term value for money,” noted Dan Ollendorf, PhD, ICER’s Chief Scientific Officer. “Based on current evidence, both therapies appear to be priced in alignment with their clinical value, but there are potential short-term affordability concerns – for axicabtagene ciloleucel under its current indication, and for both treatments should they receive future approvals for broader patient populations. With many other potentially transformative therapies in the pipeline, stakeholders must collaborate now to develop payment and delivery systems that can ensure timely patient access, manage short-term affordability for expensive one-time treatments, and continue to reward the innovation that brings these new treatments to market.”

Affordability & Access Alert

As part of its final report, ICER is issuing an Affordability and Access Alert for axicabtagene ciloleucel used to treat adults with NHL. This alert is intended to signal when the added costs associated with a new treatment may be difficult for the health care system to absorb over the short term without displacing other needed services or contributing to unsustainable growth in health care insurance costs. At current prices, only 38% of eligible patients could be treated before the potential budget impact would cross ICER’s threshold of $915 million per year.

Policy Recommendations

Following the voting session, ICER convened a policy roundtable of experts comprising physicians, patient advocates, manufacturer representatives, and payer representatives. Based on this discussion, ICER developed key recommendations for enhanced stakeholder communication, innovative payment models, generation of additional evidence, settings of care, and patient education, including:

  • For treatments with the potential to have a major impact on patterns of care and costs, manufacturers, insurers, and providers should meet prior to FDA approval to discuss the drug’s potential role in therapy, key patient subpopulations, pricing parameters, and payment arrangements, a step that will reduce unnecessary delays in delivering care to patients by addressing financial uncertainties for insurers and providers.
  • When launching novel therapeutics that are approved with limited clinical evidence, manufacturers and payers should consider one of two options: a lower launch price with the potential for increase should substantial clinical benefits be confirmed, or a higher initial price tied to requirement for refunds or rebates if real-world evidence fails to confirm high expectations.
  • Value-based pricing should be viewed in context with the affordability of a new treatment for different health insurers and payers based on the size of the population eligible to receive the therapy.
  • All patients treated with CAR-T therapy should enter into a registry with planned long-term follow-up.

Additional policy recommendations and more details are available in ICER’s Final Report.

About ICER
The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.