New Resource to Help Navigate EXONDYS 51 Access

Prior to the approval of EXONDYS 51™, we knew that access to emerging therapies would be a process – and an often challenging process at that.


Based on the previous experiences of other rare disease communities such as Pompe Disease and Cystic Fibrosis, we were applying best practices and preparing to engage with stakeholders in the access environment post-approval. What we didn’t expect was the way in which the circumstances around the approval of EXONDYS 51, specifically, might impact the access environment that would follow.

Accelerated Approval is Approval

Once again, in Duchenne, we find ourselves charting new territory. The accelerated approval of this therapy has some insurance companies interpreting this still being an experimental therapy – it’s not.

The fact that so much detail was released in the label and background dossiers from the FDA has provided insurance companies with unprecedented insight into the academic discussions of select data. And we are in an environment where these insurance companies are trending towards the application of outcomes-based contracts in the absence of established outcomes.


New Data May Prove Our Case

For this reason, PPMD and clinical experts in our community have been working to pro-actively engage insurance companies.

One such example was the engagement between by California MediCal (California’s Medicaid program) and three of the world’s top Duchenne experts based out of California, led by Dr. Craig McDonald of UC Davis with support from Dr. Perry Shieh of UCLA and Dr. John Day of Stanford.


Dr. McDonald has graciously shared the data and presentation that was compiled for their successful engagement with MediCal and recorded an explanation of the data and the context of the engagement, as well as details as to how he and his team have been engaging with private insurance companies and empowering families to work with insurers.


Watch Dr. McDonald's California MediCal (Medicaid) Presentation: 


How to Use this Information

We believe that this data and accompanying presentation will serve as a resource for clinicians working with public and private insurers on your behalf. If you have received a denial and you are working with your clinician to appeal the decision, please make sure he or she has the opportunity to review this data. If you are appealing a decision from your insurance company on your own, insist that they review this data and presentation as it will confirm that EXONDYS 51 is not an experimental therapy but an approved drug.


We hope that you will regard this as a powerful tool as you navigate the complicated and often frustrating health insurance landscape.


PPMD will continue to share resources and tools to empower our community through the PPMD Access Resource Center.

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