Drug Development

From WikiAdvocacy

Externally Led Patient Focused Drug Development Meeting - Experiences from Groups

  • Friedreich’s Ataxia Research Alliance

FARA has hosted an ELPFDD as a stand alone meeting, held a reception the night prior and a board meeting following the ELPFDD. FARA leadership felt the merit of the ELPFDD was important and sufficient to stand on its own. A great deal of time and attention was given to identify appropriate panelists and in preparing panelists for their role in this meeting. Preparation was also provided to members of the audience who wished to make statements. The meeting was described by Ron Bartek as monumental in building relationships with the FDA and in providing insights about Friedreich's ataxia from the patient's perspective. The group hired a consultant. Participation was not restricted to US residents. Anyone from anywhere was included. Many members of the community joined remotely. All responses provided via cell phone were tallied together and responses feedback to all regardless of whether the participant was attending the face-to-face meeting or joining online.

  • Genetic Alliance

Genetic Alliance led ELPFDD for 3 diseases using the Platform for Engaging Everyone Responsibly (PEER) system. Some communities use PEER for registries, some for campaigns and some for trials. The six disease groups (including three orgs representing sickle cell disease) developed videos and copy placed on GA portal. Tablets were used by volunteers in infusion clinics to get people’s preferences. These three groups were selected to be included in the first phase of the FDA Patient Focused Drug Development.

  • Tuberous Sclerosis Alliance

Hosted a ELPFDD, see resources provided below.

  • Organic Acidemia Association and XLH Network are currently planning a ELPFDD meeting.

Externally Led Patient Focused Drug Development Resource Links

Acute Porphyrias



Friedreich's Ataxia (FA)

Friedreich's Ataxia (FA)#2

Global Genes



Myotonic Dystrophy

CG3, National Kidney Foundation


Spinal Muscular Atrophy

Tuberous Sclerosis