Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. The community has anxiously awaited an update on both access and pricing ever since the deal between PTC and Marathon was announced.
On today’s call, we learned:
We know that, like PPMD, many of you are very concerned about the new price that has been announced. While the price is lower than the proposed Marathon price point there remains confusion about the weight based dosing and how that is tied to pricing. PTC also indicated that it anticipated that the process from point of start form initiation to payer access could take many weeks and likely require prior authorization.
This makes navigating access issues more important than ever. And as we are already doing with families who are navigating the complicated landscape of EXONDYS 51 access, PPMD will do everything we can to help assist families who want access to EMFLAZA. We will also work closely with PTC to better understand the progress made with payers to ensure a favorable access environment.
PPMD will update our online Access Resource to reflect EMFLAZA access, as information becomes available. For now, check out some of the useful general information we have pulled together.
Many Questions Remain
PTC has a long history with our community. It is this long history and deep understanding of Duchenne that continues to give us hope that PTC will truly expand access of deflazacort in the U.S. For decades, PPMD has wanted options for families, including deflazacort as an approved steroid option.
We hope that EMFLAZA will be that option, but we still have a number of outstanding concerns that we will address with PTC, including:
As PTC mentioned on their public calls today, the company met with various stakeholders between the time the deal with Marathon was announced and today’s update. PPMD was one of the stakeholder groups that PTC met with. We shared with the company the importance of landing on a fair price for this therapy that was not only sustainable for the Duchenne community, but that protected future pricing in the rare disease space as a whole.
We reminded the company that pricing concerns may force families to make impossible decisions, choosing one therapy over another in a disease that we know will take a combination of therapies to eventually slow disease progression.
And we emphasized that once this drug goes to market, issues of access – in a payer landscape that has perhaps never been more uncertain – is a crucial concern. Families are changing insurance, losing benefits, and/or navigating new premiums multiple times a year in our community. We need to ensure that the people who need deflazacort the most are able to get it in a timely and affordable manner.
The price announced today does not reflect our conversations with PTC, nor the concerns we expressed. We hope that going forward, programs like EMFLAZACares will help families affordably access EMFLAZA without limiting coverage of other critical therapies currently approved as well as those in the pipeline.
PPMD will continue to monitor and remain engaged in the access environment in order to understand the impact of every new approval, providing materials and resources to help guide families.
Accessing Deflazacort from Other Sources
There a lot of questions about what this means for those who receive deflazacort from sources outside of the U.S. PPMD will be exploring available options and will share with the community our findings in the coming weeks.
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