Your Voice. Their Blueprint.

Patient-Focused Drug Development(PFDD) means a powerful way for families, caregivers, and people living with a disease to directly tell regulatory decision-makers and treatment developers what living with that condition is really like.

PFDD is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation.

This is your chance to shape future treatment options with your voice.

Read on below or download our info sheet here.

  • The Goal

    To host an externally-led PFDD (El-PFDD) meeting for Alexander Disease, with regulatory decision-makers attending in a listening role.

    Uncover the real-life impact, unmet needs, and hopes for the future.

    Result in a formal “Voice of the Patient” report that becomes part of the public record.

  • Who should participate?

    Patients living with AxD - newly diagnosed, early onset, adolescent, young adult, adult

    Caregivers and family members of all ages

    Siblings, loved ones, and advocates

    Bereaved families whose voices still deeply matter

    Anyone with lived experience, regardless of diagnosis timeline or disease stage

  • What happens next?

    Immediately

    Planning kicks off

    Identification of participants who express interest in sharing their voice to support the EL-PFDD process

    July through December

    Monthly check-ins with the End AxD board.

    Organize panels, survey the community, and prep materials.

    Align with the End AxD board to plan a virtual Zoom session with regulatory participants based on the provided timeframe

  • How you can help?

    Share your story

    Join our contact registry

    Add your name to the Patient Advocacy support list by emailing: info@endaxd.org

    Help spread the word about the EL-PFDD to fellow AxD families

    Reach out

  • Key takeaways

    This meeting does not Involve giving approval or clinical direction - it’s for sharing and listening; for looking into the lives of those affected by AxD

    It’s hosted by the AxD community, not regulators

    Sharing your story increases our chances for the Alexander Disease Community

    to be heard and to be prioritizedfor consideration of medical treatments and breakthroughs

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