SMA Patient-Focused Drug Development Meeting with the FDA Set for Next Week

By Cure SMA | Published On April 12, 2017

SMA Patient-Focused Drug Development Meeting with the FDA Set for Next Week

The SMA community is eagerly looking forward to next week’s Patient-Focused Drug Development (PFDD) Meeting with the FDA.

Over 400 members of the community are registered to participate in this event. This includes individuals and families affected by SMA, representatives from patient advocacy groups and industry, clinicians, and more than a dozen leaders from the FDA.

Though in-person registration has reached capacity, registration for the livestreaming webcast will remain open through Friday, April 14.

Register for the webcast▶

Resources for Participants

This is just the second externally led PFDD meeting to be granted by the FDA. To make the most of this unique opportunity, we’ve created the following resources to help our community effectively make our voices heard.

Webinar on FDA Interactions

On March 8, we hosted a webinar on interactions between the FDA and the SMA community. We were joined by two panelists from the FDA, and one from the patient advocacy sphere with experience in hosting a successful PFDD meeting. The webinar included specific information and details on the PFDD meeting. If you were not able to attend the live webinar session, you can view a recording on our YouTube channel:

Discussion Guide

We have created a guide for participants that covers many of the topics that will be discussed at this meeting. The guide includes questions for panelists and the audience. We're providing this guide in advance so that both in-person and webcast attendees have an opportunity to reflect on these questions prior to the PFDD meeting. We hope that this further lays the groundwork for thoughtful, open dialogue.

Download the guide▶

Download polling questions▶

More About the PFDD Meeting

The Patient-Focused Drug Development Initiative is part of FDA’s commitments under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), which aims to more systematically obtain the patient’s perspective on the burden of specific diseases and current treatments available.

At the meeting, 20 panelists, representing all types, ages and stages of SMA, will bring their voices and stories to depict the real and specific ways in which their lives are impacted by SMA. Each round of panelists will be followed by polling questions and a period of facilitated discussion with participants here in Washington DC, and from across the US via our livestreaming webcast.

The goal of this meeting is to increase the FDA’s understanding of how patients, families and caregivers manage SMA, and the factors that are taken into account when a treatment is chosen. This in turn, will inform the FDA regarding the benefit-risk balance of treatment options, the severity of the condition, and the urgency of unmet medical needs. Ultimately, your voice and feedback will directly inform decisions made about the drug development process, and the overall assessment of current and future therapies for SMA.

Thank You

We would like to thank and acknowledge the members of the SMA Industry Collaboration for their hard work and support of this meeting. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory topics that are critical for the broader SMA community.

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