Institute for Clinical and Economic Review Posts Draft Scoping Document on Voretigene Neparvovec, First Gene Therapy Submitted for FDA Approval

-Report expected to review clinical effectiveness, value of the therapy; Scoping Document will be open to public comment until July 31, 2017-

Boston, Mass., July 10, 2017 – The Institute for Clinical and Economic Review (ICER) has posted a Draft Scoping Document that will shape an upcoming report on voretigene neparvovec (Spark Therapeutics), a gene therapy currently under FDA review for people with vision loss associated with biallelic RPE65-mediated retinal disease. The Draft Scoping Document will be open to public comment for three weeks until July 31, 2017.

Consideration of gene therapy brings with it a number of important questions, including the durability of the clinical benefits provided, as well as long-term safety. In addition, the possibility of one-time treatment with interventions such as voretigene poses unique challenges related to value assessment, pricing, and payment options. ICER’s report will assess the clinical effectiveness of the therapy and will include analyses of cost-effectiveness and potential budgetary impact.

The Draft Scoping Document was developed with input from key stakeholders, including groups representing the visually-impaired community and clinical experts, who provided important insights into the context of treatment decisions. For example, conversations with representatives of the visually-impaired community highlighted the importance of outcomes beyond improvements in vision, including how available therapies and services can contribute to increased independence and productivity. Costs were raised as a concern, particularly as they affect a person’s access to therapies and services.

ICER will continue to engage with groups working in the visually-impaired community, clinicians, drug manufacturers, and other stakeholders to assess appropriate outcomes and comparators and to enrich the evidence available from clinical trials.

All interested stakeholders, including people living with visual impairment, providers, payers, and manufacturers, are encouraged to submit comments and suggested refinements to the scope to ensure all perspectives are adequately considered. All comments must be received by July 31, 2017 at 5pm ET.Comments can be submitted by email to publiccomments@icer-review.org.

More information about submitting a public comment, including formatting specifications, is available on ICER’s website. ICER’s Patient Participation Guideand Manufacturer Engagement Guide provide additional guidance for submitting public comments. If formatting requirements pose a burden to individuals submitting comments, ICER is happy to make accommodations.

Following the public comment period, a revised scoping document will be posted on or about August 7, 2017.

The Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) will evaluate the report assessing voretigene neparvovec at a public meeting on January 25, 2018. The Midwest CEPAC is an independent, regional body of practicing physicians, methodological experts, and leaders in patient advocacy and engagement that provides objective, independent guidance on the application of medical evidence to clinical practice and payer policy decisions.

During the January 2018 public meeting, the Council will vote on key questions raised in the report, and a policy roundtable of individuals from the visually impaired community, clinical, payer, and policy experts will discuss recommendations for how best to apply the evidence to policy and practice.

Additional information regarding the report and meeting are available on ICER’s website.

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.