— Independent appraisal committee highlights importance of non-clinical benefits and contextual considerations when evaluating AR101 and Viaskin Peanut —

— Potential short-term budget impact leads ICER to issue an Access and Affordability Alert —

BOSTON, July 10, 2019 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance assessing the comparative clinical effectiveness and value of two investigational technologies that treat children with peanut allergy by building their immune tolerance to peanuts. The report evaluates Viaskin® Peanut (DBV Technologies) and AR101 (Aimmune Therapeutics), as well as non-commercialized oral immunotherapy (OIT). An FDA decision on AR101 is expected by January 2020. A biologics license application for Viaskin Peanut was withdrawn in December 2018, but another is expected to be resubmitted later this year.

ICER’s report was reviewed at the June 2019 public meeting of the California Technology Assessment Forum (CTAF), one of ICER’s three independent evidence appraisal committees. During the meeting, CTAF found that the evidence was inadequate to demonstrate a superior net health benefit of either AR101 or Viaskin Peanut compared to strict peanut avoidance. While the panel recognized that desensitization as measured by an oral food challenge may be promising, they noted several countervailing concerns: 1) requirement to continue treatment indefinitely without longer-term evidence of safety or effectiveness; 2) desensitization does not eliminate the need for caution to avoid inadvertent peanut exposure; and 3) the expected yet burdensome increase in the risk of allergic reactions and epinephrine use in the one-year clinical trials for AR101 and Viaskin Peanut. The CTAF panel emphasized the need for better evidence to demonstrate that desensitization is linked to fewer reactions related to accidental peanut exposure after the trial duration, as well as improvement in quality of life for patients and their caregivers. The CTAF panel also voted unanimously that the evidence was insufficient to demonstrate a net health benefit of AR101 over non-commercialized OIT.

During their deliberation, CTAF panel members weighed the therapies’ other benefits and contextual considerations. They noted that these interventions target an area of large unmet need, may impact health disparities, and may significantly reduce anxiety among patients and caregivers. However, they also highlighted the significant uncertainty related to the therapies’ long-term benefits and risks.

ICER’s evaluation of long-term cost-effectiveness found that the fair value-based price benchmark range would be $4,800-7,200 per year for AR101 and $3,000-$4,500 per year for Viaskin Peanut. CTAF did not vote on either treatment’s long-term value for money because the actual launch prices remain unknown at this time.

“Currently, there are no FDA-approved treatments for patients with peanut allergy, and the potential for accidental exposure to peanuts can create tremendous anxiety for patients and their caregivers,” said David Rind, MD, ICER’s Chief Medical Officer. “However, while Viaskin Peanut, AR101, and OIT have all been shown to desensitize patients to the allergen, each one of these approaches is also associated with increased allergic reactions and use of epinephrine. Caregivers and patients must remain vigilant about avoiding peanuts even while on desensitization therapy, and future clinical trials will need to demonstrate whether long-term benefits of these approaches outweigh their short-term risks.”

Affordability & Access Alert

Given that the clinical goal for uptake might exceed the potential budget impact threshold at the national level, ICER is issuing an Affordability and Access Alert for both AR101 and Viaskin Peanut. While the actual prices of both therapies are unknown, industry analysts have estimated that AR101 may receive an annual price of $4,200, and Viaskin Peanut may receive an annual price of $6,500. Using these estimates, ICER’s potential budget impact threshold of $819 million per year would be crossed after treating only 41% of eligible patients with AR101, or after treating only 71% of eligible patients with Viaskin Peanut.

The purpose of an ICER Affordability and Access Alert is to signal to stakeholders and policy makers that the amount of added health care costs associated with a new service may be difficult for the health system to absorb over the short term without displacing other needed services or contributing to rapid growth in health care insurance costs that threaten sustainable access to high-value care for all patients.

Key Policy Recommendations

Following the voting session, a policy roundtable of experts – including clinicians and patient advocates, as well as representatives from DBV Technologies, Aimmune Therapeutics, and payer organizations – convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • Manufacturers should pursue further evidence development in order to provide better certainty of the long-term safety and effectiveness of desensitization therapies. Evidence should not rely on short-term surrogate outcome measures like desensitization, but focus instead on outcomes that matter most to patients.
  • Specialty societies should collaborate with patient organizations to develop unbiased materials for both patients and clinicians to support appropriate shared decision making that does not create stigma for patients who decide for or against treatment with the current generation of desensitization therapies.
  • Prescribing peanut desensitization therapies should be restricted to specialists (i.e., Allergy and Immunology Specialists); or for those patients with inadequate access to allergists, by primary care physicians only in consultation with a specialist.
  • The FDA should update its guidance for the assessment of outcomes in food allergy therapy to require a common definition of desensitization.

ICER’s detailed set of policy recommendations, including considerations for establishing prior authorization criteria, is available in the Final Evidence 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.