Last night, the Institute for Clinical and Economic Review (ICER) released their final report assessing three Duchenne products: Emflaza, EXONDYS 51, and golodirsen. This report integrates all public comments, stakeholder engagement, and the Roundtable proceedings that took place late last month.

On behalf of Parent Project Muscular Dystrophy (PPMD) and the Duchenne community, we have taken advantage of every opportunity to engage, inform, and provide input in the current review of Duchenne products, including submission of a summary of our oral remarks from the July 25 Roundtable. And while we do see some of our inputs throughout the assessment impacted the underlying modeling and some of the recommendations we advocated for included within the final ICER report, we remain concerned that the report fails to reflect many of the key items that we believe are foundational in Duchenne.

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. We are committed to collaborating on the development of evidence and resources to facilitate this decision-making – and recognize that ICER’s construct is an attempt to define the ‘value’ of specific interventions.

That said, we are concerned that ICER’s current framework and construct does not yet consider patient experience and input meaningfully into the underlying equation. Patient experience and innovative qualitative and quantitative patient experience data is valid – and is more than a contextual consideration or controversy. Indeed, it is the lens through which value-based decisions are made and it is our belief that these inputs must be a more-central element of ICER’s Value Assessment Framework for Ultra-Rare Diseases.

We feel strongly that patients and providers must continue to have access to all therapeutic options approved for the treatment of Duchenne and that valuations, like that provided by ICER, should be seen as an additional resource to empower individual patient/provider-shared decision-making. Valuations should not be utilized as a singular tool for policy-makers to limit access; such actions could have catastrophic ramifications for people with Duchenne.

Therefore, PPMD has drafted and shared an open letter to payers that we hope will help insurance companies better understand the experiences and priorities of our patient community, as well as the limitations of this recent valuation.

PPMD’s Open Letter To Payers

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. We are committed to collaborating on the development of evidence and resources to facilitate this decision-making – and recognize that valuations are simply an additional construct within the access ecosystem that attempt to define the ‘value’ of specific interventions with the ultimate goal of informing such decision-making. However, valuations should not be utilized as a singular tool for policy-makers to limit access; such actions could have catastrophic ramifications for people with Duchenne.

PPMD would like to underscore key foundational concepts we urge payers to consider when approaching decision-making around Duchenne products:

• Slowing or halting of disease progression through treatment intervention is significant and the impact of such – on the lives of both patients with Duchenne and their caregivers – should be reflected.
• When only one approved on-label product exists within a therapeutic class, individuals with Duchenne should not have to go through the process of prior authorization or N-of-1 trials to compare toxicities of approved products with off-label therapies. Care decisions should be made by patients and providers. Gaps or delays in care undermine the effectiveness, and therefore the value, of treatments.
• Step Therapy policies are unethical in conditions such as Duchenne where step therapy requirements will cause irreversible loss of function.
• Therapies – which provide even incremental benefit – should be prioritized and considered foundational.

Patients and providers must continue to have access to all therapies approved for the treatment of Duchenne and valuations should be seen as an additional resource to empower individual patient/provider shared decision-making. Among the most critical considerations that must be taken into account is that the ‘yet to be fully known’ characteristics of these interventions which must be weighed against the ‘certainty of doing nothing.’ Even incremental advances are the foundation for combination therapy and long- term progress in Duchenne. Maintaining strength and function, slowing disease progression, reducing hospitalizations and chance of infection, and optimizing outcomes for potential future interventions are all of extreme value to patients and providers within the Duchenne community.

Learn more

• Click here to read the full Final Evidence Report.
• Click here to read the Report at a Glance.
• To better understand ICER and the process that culminated in this report, click here.
• If you missed last month’s ICER Roundtable, click here.