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Externally-Led Patient-Focused Drug Development

Our Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting was an important initiative for the ALD community. The meeting focused specifically on men with AMN, symptomatic women, and adults with cerebral ALD. It provided a unique platform for patients and caregivers to share their experiences, challenges, and hopes directly with regulators, researchers, and pharmaceutical companies.

Watch the EL-PFDD Meeting

Participants highlighted the symptoms that impact them most, the profound effects of ALD on daily life, and their experiences with existing treatments. This invaluable input is designed to inform the FDA’s decision-making both during drug development and in their review of marketing applications for new drugs. The EL-PFDD highlighted the unmet medical needs and severe disease burden of ALD. For patients, this meeting was not just about sharing their stories—it was about ensuring that their perspectives will guide future research and shape the development of more effective treatments. This meeting underscored the essential role of patient input in developing meaningful therapies for ALD, and we hope it will lead to lasting improvements in the drug development process.

Manifestaciones adultas de adrenoleucodistrofia EL-PFDD (En Español)

Voice of the Patient Report

The ALD Connect Voice of the Patient Report captures the lived experiences, challenges, and treatment priorities of individuals and families affected by adrenoleukodystrophy. It is a summary of the Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting, and it serves as a powerful testament to the realities faced by individuals and their families in the ALD community. It highlights the urgent need for more effective treatments, better management of symptoms, and greater support for patients and caregivers. By documenting these firsthand accounts, the report provides invaluable insights for regulators, researchers, and drug developers, ensuring that the patient perspective remains central in the development of new therapies and in shaping policies that impact the ALD community.

Download The Voice of the Patient Report

Thank You

Thank you to everyone who made this meeting possible. We would especially like to thank our incredible community of men with AMN, symptomatic women, and adults with cerebral ALD, as well as the caregivers and family members who bravely and candidly shared their experiences. Your participation was invaluable, making this meeting and report possible.

We are deeply thankful to the many representatives from the U.S. Food and Drug Administration who attended our meeting. Your presence provided an invaluable opportunity for us to share our experiences. Special thanks to Dr. Wilson Bryan for highlighting the importance of EL-PFDD meetings to the FDA, and to Shannon Sparklin and Karen Jackler from the FDA’s PFDD staff for expertly guiding us through this process.

Our deepest thanks go to the Dudley Digital Works media team for their outstanding support in executing this meeting, and to James Valentine and Larry Bauer from Hyman, Phelps & McNamara for their invaluable assistance throughout the entire process.

A special thank you to our generous financial sponsors, including Autobahn Therapeutics, Minoryx Therapeutics, and SwanBio. Your support made this meeting possible.

Lastly, we extend our appreciation to the many stakeholders who attended, including advocacy partners from around the world: Adrenal Insufficiency United, ALD Alliance, Alex TLC, Arrivederci ALD, ELA, Fundación Lautaro te necesita Leucodistrofias, GLIA, IndoUSrare, LRRO, Knockout ALD, Remember the Girls, ULF, and X out ALD.

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ALD Connect is here to support patients, families, and healthcare providers navigating the challenges of adrenoleukodystrophy. Whether you have questions, need guidance, or want to learn more about how we can help, we encourage you to get in touch.