ALPE participates in FDA Advisory Committees meeting on achondroplasia

Two of FDA Advisory Committees: the Pediatric Advisory Committee and the Endocrinologic

and Metabolic Drugs Advisory Committee met on the 11th May 2018, in Maryland, USA, to discuss drug development for the treatment of children with achondroplasia. 

FDA is the Food and Drug Administration in the USA and is an agency within the Department of Health and Human Services. Among various topics, for advancing the public health by helping to speed innovations that make medical products more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medical products and foods to maintain and improve their health.1

Image 1. FDA Advisory Committees meetings

meetings The advisory committees provide FDA with independent advice from outside experts on issues related to human and veterinary drugs, biological products, medical devices, and food. And although the committees provide advice to the Agency, final decisions are made by FDA.1

This FDA meeting was oriented to discuss the value and the design of clinical trials for achondroplasia,  the topics considered for discussion were the following:

  • Evidence required to establish dose-response,
  • Study design,
  • Study duration,
  • Intended population
  • Endpoints

There were two working sessions for this meeting: one was closed for experts and one was open, for public participation and presentation. ALPE Foundation requested to do an oral presentation, with a 3 minutes speech slot. There were a total of 33 individuals and patient organizations representatives doing oral presentations besides the letters FDA received for this working sessions.

ALPE participation and presence was supported by Global Genes, one of the leading rare disease patient advocacy organizations in the world, that covered traveling and accommodation costs. ALPE was represented by Estefánia González, our secretary, and translator, who presented ALPE statement and was accompanied by Susana Noval, the team coordinator. Before this open working session, ALPE sent an informative letter to FDA prior the meeting with data, information, and perspectives on issues related to achondroplasia which you can read in full here.


Image 2: Estefánia González presenting ALPE statement. Screenshot of the FDA webcast on the meeting for achondroplasia.

ALPE oral statement can be watched in this FDA webcast link between 2:16:30 and 2:19:30. Estefánia ended by saying "We are in your hands".

Image 3: Seth Fritts, from Global Genes, Estefánia González, Amy Grover, from Global Genes and Susana Noval, ALPE team coordinator.

ALPE attendance at this meeting enabled us to offer our vision as a patient organization as acknowledge our key position as patients representative and intermediaries between clinical and scientific agents and international regulatory institutions. It was also a great privilege for us to be able to participate in a decision-making process, which can have a fundamental value in the lives of people with achondroplasia around the world, now and in the future.

This event has been an example of transparency and participation and we trust that the decisions these committees will be reflected in future clinical trials for achondroplasia and that drugs that will be approved will have an increased efficacy to improve patients lives. With this, we believe that history can change: our children and grandchildren with achondroplasia will probably face lives fewer efforts and less suffering.

We thank Global Genes for this opportunity to put our grain of sand in this process and we are looking forward to reading the conclusions od the Advisory committees Meeting on achondroplasia clinical trials design.