Sickle Cell Community Encouraged to Share Perspective with FDA

Genetic Alliance Launches First Pilot Program to Gather Perspectives on Benefit-Risk Assessment in Development of New Treatment

02.07.14 | By Kristin Van Goor

As highlighted in an earlier Catalyst post, PhRMA is partnering with Genetic Alliance on an exciting project to enhance patient engagement in the Food and Drug Administration’s (FDA) Patient-Focused Drug Development initiative.  Genetic Alliance recently launched the first pilot program, partnering with four trusted organizations serving the sickle cell community.  With the consent of survey respondents, the findings will be submitted to the docket as a follow-up to the public meeting being held by FDA today on Patient-Focused Drug Development for Sickle Cell Disease

In the guest blog below, Sharon Terry, President and CEO of Genetic Alliance, explains the goal of the pilot program and how each participant can choose how their data are shared.


The voices of those affected by diseases are critical to accelerating the quest to alleviate suffering from those diseases.  The FDA’s Patient Focused Drug Development (PFDD) initiative seeks the perspectives of patients and caregivers about benefits and risks in the development of new treatments.

PFDD is a terrific opportunity for the individuals and families affected by the 20 or so conditions chosen by the FDA. We know that for many people it is hard to attend an actual hearing at FDA, or to write one’s thoughts for submission to the docket.  

Platform for Engaging Everyone Responsibly (PEER)

With funding from an unrestricted grant from PhRMA, Genetic Alliance created a technology tool to make this opportunity more accessible to more people and to more diverse communities based on the Platform for Engaging Everyone Responsibly (PEER).  PEER is unique in allowing individual respondents to set their own sharing, privacy and data access preferences.  They indicate ‘Allow’, ‘Deny’ or ‘Ask Me’ for each of three kinds of access:  “Discover Me’, ‘Contact Me’, and “Use my Data’.  The participant makes these decisions relative to several categories: ‘Advocacy Organizations’, ‘Researchers’, and ‘Data Analysis Platforms’ (like ClinVar, or Oracle Health Sciences Network).  There are several choices within each of these categories, for example in the ‘Researcher’ category: “FDA’, ‘Researchers who Study My Condition’, or ‘All Researchers’.

Undoubtedly research is accelerated when health data are available, and especially when it is collected longitudinally.  As various studies have shown, real people have diverse views about sharing and privacy; there is no one size fits all.  These views must and can be respected.  PEER enables individuals to choose the level of sharing with which they are comfortable, and to change those preferences over the course of their life.  The participant defines all of that – they are in control. 

Sickle Cell Chosen for First Pilot

For this first PFDD pilot, Genetic Alliance partnered with organizations in the sickle cell community to give individuals affected by sickle cell disease and their caregivers a portal to share their perspectives: Citizens for Quality Sickle Cell Care; North Alabama Sickle Cell Foundation; Sickle Cell Disease Association of America, Southern Connecticut; and William E. Proudford Sickle Cell Fund.  The organizations helped craft the questions to ask of participants to make the results meaningful, and are hosting the portal on their websites.  

Each organization chose three representatives to tape a short video to put the request to share feedback with the FDA in context.  In the videos, each guide shares personal experience, describes the goal of the PFDD initiative and offers recommendations about sharing, privacy and access.  Survey participants can accept the guide’s recommendations for various categories or modify them.   The platform is dynamic: participants can change their settings over time, as their life situations changes.  

The questions in the survey are designed to elucidate what it is like to live with sickle cell disease, what type of treatments are helping individuals with sickle cell now and what type of treatments individuals with sickle cell think are most important to be developed.  

We are very excited to give people all over the country, urban and rural, in community clinics, and community based organizations, the opportunity to share their perspective with the FDA.  Individuals will tell the FDA what it is to live with sickle cell, whether treatments are effective, if side effects are intolerable, and how much risk they are willing to accept in drug development for sickle cell disease.

We are looking forward to rolling this out for more diseases, perhaps even beyond the ones FDA has named.  It is time to let the data speak, and point the way to better drug development.  Each of us is an important piece of the puzzle, let’s join together to make a difference: http://www.diseaseinfosearch.org/peer/sicklecell.

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