— Appraisal committee vote reveals differences in judgments on value given pricing far above traditional cost-effectiveness levels —
–Policy recommendations call attention to the growing stream of treatments for rare disorders that cannot all be priced at similar levels without threatening the financial sustainability of the health system —

BOSTON, February 14, 2018-The Institute for Clinical and Economic Review today released a Final Evidence Report and Report-at-a-Glance on voretigene neparvovec (VN; Luxturna™, Spark Therapeutics) for treatment of vision loss associated with RPE65-mediated retinal disease. Approved in 2017, VN is the first treatment for this condition.

ICER’s report was reviewed at a public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), where the Council unanimously voted that the therapy provides a net health benefit to those affected by RPE65-mediated retinal disease. Results of votes on value were mixed, with the majority of the members voting that VN provided intermediate long-term value for money. Uncertainty remains around the long-term effectiveness of the therapy, and ICER’s analyses found cost-effectiveness to far exceed commonly accepted thresholds; however, the therapy’s role as the only treatment option available for an ultra-rare condition, and as the first therapy approved in the US targeting a disease caused by mutations in a specific gene, created special considerations that weighed heavily in many Council members’ votes.

“Voretigene neparvovec has a very high cost, and the long-term durability of its effect is uncertain. However, this is an effective treatment that gives people with a serious, ultra-rare condition a treatment option where they previously had none,” noted David Rind, MD, MSc. “Payers and other policymakers must strike a difficult balance in judging the value of this therapy, recognizing the treatment’s broader benefits to affected individuals and society while simultaneously working to maintain health insurance affordability. As an increasing number of therapies for ultra-rare conditions enter the market, stakeholders must collaborate to develop new approaches to pricing and payment that can reward innovation while preserving access to high-value care for all patients.”

Policy Recommendations

Following the voting session during the Midwest CEPAC meeting, a policy roundtable of experts, including a physician, an individual who had received treatment with voretigene, a former commissioner of rehabilitative services who also has retinal disease, and pharmacy benefit manager representatives convened to discuss the implications of the evidence for policy and practice.

Key recommendations stemming from the roundtable discussion include:

  • Manufacturers should reach out to public and private payers ahead of FDA approval to negotiate innovative pricing and reimbursement strategies for high-cost therapies like VN that are delivered once but offer potentially long-term benefits. Spark Therapeutics’ development of reimbursement strategies for VN should be considered as potential best practice by other manufacturers of high cost therapies for ultra-rare conditions.
  • The Centers for Medicare and Medicaid Services (CMS) should take steps to permit private payers to use innovative payment mechanisms without triggering Medicaid Best Price constraints.
  • Clinical societies, patient groups, and the manufacturer of VN should work to educate all optometrists and ophthalmologists about RPE65-mediated retinal diseases and develop referral networks to facilitate rapid diagnosis.
  • Payers and the manufacturer should collaborate with retinal specialists to develop policies that promote appropriate access to genetic testing for individuals at high-risk of treatable genetic retinal diseases.

Additional policy recommendations are available in more detail 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.