In December 2018, the Institute for Clinical and Economic Review (ICER) announced that it would be conducting an assessment of three Duchenne products over the next seven months. The ICER assessment is culminating in a Roundtable tomorrow, July 25, in which a diverse panel of stakeholders will review the evidence report and vote on key issues related to each of these products (for more background about ICER, PPMD’s previous engagement with ICER, and the review process, check out our previous blog). In advance of tomorrow’s Roundtable, we wanted to provide background information about the review and make sure you knew how you could participate in the ICER meeting from home.

Thanks to decades of funding and innovation, our Duchenne healthcare landscape is rapidly changing. As new therapies emerge into the marketplace, patients, providers, and payers are faced with challenges as to how to discern which therapies are most appropriate for which patients in which combinations and at which time. In order to facilitate this decision making, an attempt to define the ‘value’ of specific interventions is made.

An entire sector exists around this definition of ‘value’ and – in the United States – value assessments for neurological products are often led by ICER.

PPMD’s Engagement with ICER

PPMD’s engagement with ICER began in November of 2015 when ICER first announced they would be reviewing three Duchenne products in 2016. While those initial reviews did not transpire, we immediately reached out to ICER around concerns that their existing assessment framework would not factor in key considerations that would need to be made for products developed for pediatric rare diseases such as Duchenne.

Following a series of discussions that took place over the next several months, ICER decided to explore this concept further by convening the ICER Orphan Drug Assessment & Pricing Summit in May of 2016 and asked us to serve on the planning committee and working group that informed the Summit agenda and the briefing paper published in advance of the summit, “Assessing the Effectiveness and Value of Drugs for Rare Conditions: A Technical Brief for the ICER Orphan Drug Assessment & Pricing Summit” (May 2017).

Several members of our Duchenne patient and clinical community provided powerful testimony throughout the Summit and helped to reinforce the fact that pediatric rare diseases should be addressed uniquely. Following the Summit, a modified framework for the assessment of pediatric rare diseases was indeed proposed by ICER. PPMD participated in the open comment period and continued to engage with ICER throughout the process as the framework was finalized. The yield was ICER’s publication of “Modifications to the ICER value assessment framework for treatments for ultra-rare diseases” (November 2017).

PPMD’s Engagement in the Review of Emflaza, Eteplirsen, and Golodirsen

Since ICER initially announced the review of the first three Duchenne-specific products in December, we have taken advantage of every opportunity to engage and provide input. In addition to submiting three formal written comments to ICER (download: February 2019, April 2019, June 2019), we have worked to connect ICER with Duchenne experts who could best inform ICER’s foundational understanding of Duchenne and the model construction. These experts included methodologists, health economists, clinicians, patient community members, patient advocacy organizations, and researchers. In addition, to inform PPMD’s comments, we sought the expertise of health economists with a deep familiarity with both ICER’s framework and Duchenne.

While our engagement with ICER over the past seven months has included extensive data points, our input on Thursday during the Roundtable will focus on key points that we feel are foundational in Duchenne and the consideration of Duchenne products — and critical for incorporation into the ICER review. They include:

• Within the Duchenne community, slowing or halting of disease progression through treatment intervention is significant.

• We have a significant concern about the ICER Assessment timing for products approved via the Accelerated Approval Pathway. From the perspective of the patient community, there is no value in conducting an assessment at a time point in which it is known in advance that ‘insufficient’ evidence will be available to conduct meta-analyses. To fundamentally damage the market in early stage is to potentially deny life-changing benefit to patients who are deserving and have no other therapeutic options.
• Among the most critical contextual considerations that must be taken into account is that the ‘yet to be fully known’ of all of these interventions must be weighted against the ‘certainty of doing nothing.’ Maintaining strength and function, slowing disease progression, reducing chance of infection, and optimizing potential outcomes for potential future interventions are all of extreme value to patients and providers within the Duchenne community.
• Therapies, which provide any incremental benefit, should be prioritized and likely will be the foundation for later combination therapies.

While we believe that — when built upon informed models and comprehensive evidence — valuations are an important resource to support stakeholder decision-making, valuations should not be utilized as a singular tool for policymakers to limit access. Such actions could have catastrophic ramifications for people with Duchenne.

On Thursday, leadership from PPMD will be present at the Roundtable and we encourage interested community members to register to attend or participate via webcast. If you do not register before the Roundtable begins, you can still watch via webcast.

ICER’s final report – integrating all public comments, stakeholder engagement, and the Roundtable proceedings – will be published on August 15.