–Special considerations include patient age, benefit durability, and rarity of condition– 

–Report will be subject to public deliberation at January 25th Midwest CEPAC meeting–

BOSTON, January 12, 2018 – The Institute for Clinical and Economic Review (ICER) today released its revised Evidence Report assessing the comparative clinical effectiveness and value of voretigene neparvovec (LuxturnaTM, Spark Therapeutics) for treatment of vision loss associated with biallelic RPE65-mediated retinal disease.

“A scientific milestone, voretigene neparvovec is the first therapy approved in the US that inserts a new working copy of a gene into the cells of patients who have a genetic disorder,” noted David Rind, MD, MSc, Chief Medical Officer at ICER. “While the evidence is clear the therapy improves vision for patients over several years, the long-term duration of this benefit remains unknown. Assuming a 10- to 20-year period of benefit, at list price the treatment does not meet standard cost-effectiveness thresholds, even after accounting for the broader societal benefits improved vision has on productivity and education cost. The cost-effectiveness findings move into standard ranges only in analyses that consider these non-medical benefits while also assuming that only younger patients with the best baseline vision receive treatment.”

“When considering treatments intended for ultra-rare diseases such as this form of blindness, decision-makers often give special weighting to other contextual factors that may lead to coverage at prices that exceed traditional cost-effectiveness thresholds. That being said, policymakers also will need to consider how to manage pricing and payment arrangements for voretigene neparvovec to ensure long-term affordability, as well as patient access to the coming wave of genetic therapies for other conditions.”

This Evidence Report will be the subject of an upcoming public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) in Kansas City, MO on January 25, 2018. The Midwest CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report reflects changes made based on comments received from patient groups, clinicians, the manufacturers of the drugs, and other stakeholders. Detailed responses to public comments can be found here.

Key Report Findings

Evidence on voretigene neparvovec provides high certainty of at least a small net health benefit for patients with biallelic RPE65-mediated retinal disease; however, significant uncertainty remains about the long-term effects and durability of the response to treatment.

Assuming a 10- to 20-year benefit of treatment for 15-year-olds – the average age of patients in the clinical trials – economic analyses found that, at the current price of $850,000, use of the treatment would exceed common cost-effectiveness thresholds. Considering only direct health care system costs, voretigene neparvovec would require discounts between 75% and 82% to align with the benefit to patients treated at age 15, setting ICER’s value-based price benchmark between $153,000 and $217,000. When also accounting for societal benefits related to education, greater productivity, reduced caregiver burden, and other factors, voretigene neparvovec would still require a 50-57% discount to remain below these thresholds. Even when assuming an as-yet unproven lifetime benefit for the therapy, the cost remains well above commonly accepted thresholds.

Separate analyses suggest that voretigene neparvovec meets standard cost-effectiveness thresholds when treating only three-year-old patients and accounting for both direct medical costs and broader societal benefits.

When considering paying for treatments of ultra-rare conditions, decision-makers often give special weighting to other contextual factors that may lead to coverage at higher prices than would be acceptable for treatments of more common conditions. ICER’s report summarizes several additional benefits of voretigene neparvovec not captured in clinical trials. Importantly, this treatment is the only therapy available for vision loss associated with biallelic RPE65-mediated retinal disease. Further, individuals who had received the treatment cited increased mobility, self-confidence, and independence as potentially important benefits.

The Evidence Report, accompanying voting questions, all public comments received on the draft report, and ICER’s written response to comments, are available on the ICER website.During the January public meeting, pre-registered stakeholders will provide brief oral comments on the report and its findings, the Midwest CEPAC will vote on key questions raised in the report, and a roundtable of experts will discuss recommendations for applying the evidence to policy and practice.  Requests to make an oral comment were accepted during the public comment period on the Draft Evidence Report and are now closed.

Register now to attend the live meeting or to view the live webcast.

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.