Having lost two sons to Duchenne muscular dystrophy (DMD), Pat Furlong, President, and CEO of Parent Project Muscular Dystrophy (PPMD), has experienced the damage a lethal rare disease can incur firsthand.

Furlong described her journey as a caregiver for her two sons both diagnosed with DMD at an early age. Her experience led to the creation of the PPMD in 1994 – a not-for-profit organization dedicated to advocating for those affected by DMD.

The core message of Furlong’s talk was the need for a greater role for patient advocacy in drug development, describing the process as an ecosystem consisting of various stakeholders all with a shared interest: “Like every other ecosystem, we all belong together. Patients, drug companies, payers, government – all of us together – and the value is that we want to improve the quality and lives of all patients with disease.”

Everyone involved wants better access to good care, therapies that make a difference and for those therapies to be reimbursed, said Furlong, “but to do so, we need to be there from the beginning and through the whole drug development process.”

When Furlong founded the PPMD, there was no standard of care for DMD, there was very little investment in the space and, as a result, very few companies were interested in developing treatments for the condition.

To make meaningful change, PPMD began engaging with the U.S. Food and Drug Administration (FDA) at every stage of the drug development process, from discovery to market, to help drive more effective therapies for DMD patients.

In 2013, the PPMD hosted a Duchenne Policy Forum to help gather DMD patient insights and, within six months of engaging with the patient community, presented the FDA with a 46-page draft guidance document. The literature was then used by the FDA as a basis for guidance later returned to the community.

Ultimately, Furlong’s journey is one that continues to be a positive message for others to learn from in the patient advocacy community and one that emphasizes the importance and necessity of the patient voice in instigating change.

“Improving quality of life, working across the drug development paradigm, understanding your patient population, working with them to get their opinion and presenting it is a top priority,” said Furlong. “It’s about all of us and the value proposition of having drugs that increase the quality and length of our lives and the need to really drive access for all the patients that need them.”

Engaging regulators to instigate change in muscular dystrophy treatment access

Astellas Patient Advocacy is a function within Corporate Affairs at Astellas that focuses on creating, building and maintaining third-party relationships. We serve as a conduit between Astellas and external stakeholders to help improve patient outcomes, improve access issues and address patients’ unmet needs head on.