First gene therapy to be submitted for approval to FDA; potential for life-time benefits with single treatment raises questions for value assessment, pricing, and payment options –

Boston, Mass., June 15, 2017 – The Institute for Clinical and Economic Review (ICER) will develop a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (Spark Therapeutics), a potentially one-time gene therapy for vision loss associated with biallelic RPE65-mediated retinal disease. Voretigene neparvovec is the first gene therapy to be submitted for approval by the US Food and Drug Administration (FDA).

An “Open Input” period begins today, and comments on the topic will be accepted from all interested stakeholders until July 6, 2017 at 5 PM ET. The Open Input period is intended to allow stakeholders to share key information relevant to the development of the evidence report. During this time, ICER will also contact key patient groups and clinical experts to gain further insights on the patient perspective and clinical context of this new treatment option.

For more information about the Open Input period, including further explanation of the types of information that may be most helpful to development of the report, visit ICER’s website. ICER’s Manufacturer Engagement Guide and ICER’s Patient Participation Guide and Patient Guide to Open Input provide additional information for manufacturers and patient groups.

There are no page limits to Open Input submissions, and input received will be incorporated throughout report development. All input can be emailed to and must be received by 5 PM ET on July 6, 2017 in order to be considered.

A Draft Scoping Document, which will provide more detail on ICER’s proposed scope for the review, will be available on or about July 10, 2017 and will be open to public comment for three weeks.

The report will be the subject of a January 2018 meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC). During the meeting, the independent panel will deliberate and vote on the comparative clinical effectiveness, benefits or disadvantages, and contextual considerations of the therapy. The panel will discuss the implications of the votes for policy and practice with a roundtable including clinical experts and patients to provide guidance to patients, clinicians, insurers, and policymakers in making informed, evidence-based decisions related to use of the therapy.

The key dates for ICER’s review of voretigene neparvovec are included below:

  • Open Input Period: June 16, 2017-July 6, 2017
  • Draft Scoping Document Posted: July 10, 2017
    • Public Comment: July 10-July 31, 2017
  • Revised Scoping Document Posted: August 7, 2017
  • Draft Evidence Report Posted: November 14, 2017
    • Public Comment: November 14, 2017-December 13, 2017
  • Evidence Report Posted: January 11, 2018
  • Public Meeting: January 25, 2018
  • Final Report Posted: February 7, 2018