PPMD is excited to share that a consensus statement providing insights from clinicians who care for patients with Duchenne into the current therapeutic landscape and access to novel therapies for Duchenne has been published in the Annals of Child Neurology Society.

The landscape of approved therapies in Duchenne is rapidly changing with eight approved therapies, including the addition of three new therapies approved in the last year. But with new approvals, comes new challenges: navigating the access landscape with multiple high-cost therapies is complex, and ensuring access and encouraging broad, flexible payer policies is critical in ensuring that patients are able to obtain the medications that their physician thinks is best for them.

Recognizing the potential barriers that lie ahead in creation of payer policies that could be restrictive, PPMD leaned on our expert community of neuromuscular physicians. Under the leadership of Dr. Aravindhan Veerapandiyan, Director of the Comprehensive Neuromuscular Program and Certified Duchenne Care Center (CDCC) at Arkansas Children’s Hospital, 43 leading neuromuscular experts from the Duchenne physician community, including many from CDCCs across the country, came together to create a consensus statement highlighting the critical importance of allowing physicians to prescribe treatments for their patients not based on payer policies, but rather based on their clinical judgment and in partnership with patients and families living with Duchenne. The statement outlines physician preferences around prescribing practices and approved therapies, empowering physician choice over payer policy limitations. 

The publication encourages payers to frequently reassess and update policies in a timely fashion to reflect advances in clinical practice and take into account the validity of the accelerated approval pathway, which is neither investigational nor experimental, but rather a full approval pathway for certain medications. Additionally, it encourages and highlights the importance of collaboration across clinical experts, policy makers, and the medical community to ensure timely access to bridge gaps in policy, enhance accessibility, and foster ongoing research and development of therapies.

Next Steps

PPMD is thrilled that leaders in our community have come together to make their stance known: all individuals with Duchenne should have access to all approved therapies for which they’re eligible, and access to one drug should not preclude access to another when safety is not a concern. We hope this publication will encourage payers to take under careful consideration the complex drug development landscape and the incredible need for access to approved therapies for all. 

Questions about your own coverage? Schedule time with the PPMD For You team to evaluate your policy and be connected with resources to help you navigate the access landscape. You can also check out our Insurance and Access Coverage Resources for sample letters of medical necessity, an overview of access pathways, and more.

Connect in Person PPMD is happy to host in-person appointments with our team to help you understand and navigate the approved product space at PPMD’s 30th Annual Conference, June 27-29 in Orlando, FL. Additionally, patient support programs from across the Duchenne space will be in attendance and happy to connect with you and answer your questions.