“Tell the FDA” is our program in conjunction with the Genetic Alliance’s Patient-Focused Drug Development (PFDD) Initiative using the Platform for Engaging Everyone Responsibly (PEER) to provide input to the Food and Drug Administration (FDA).

The FDA needs input from sickle cell patients and caregivers to help shape policies related to drug development and future therapies.

Patients and caregivers had a number of different ways to participate:

  • Online surveys developed by community-based organizations via the PFDD Initiative
  • Live or online attendance at FDA Public Meeting on February 7, 2014
  • Submitting comments online to FDA public docket

We worked with the PFDD team to create a web-based survey for people living with Sickle Cell Disease and Trait to report their perspectives in areas identified by the FDA

  • How SCD affects daily life
  • What symptoms do they experience,
    which ones impact their life the most
  • Patients’ satisfaction with current
  • Patients’ prioritization of the
    greatest needs for new treatment

Here’s WHO responded:

Here’s WHAT they said:

Top six symptoms that need better treatments:

Top six negative side effects of current treatments:

Survey Data regarding Clinical Trial Participation

The top 3 areas survey participants would like the WEPSCF to promote are:

Results presented are based on 149 total respondents to the “Tell the FDA” survey. Responses from this survey were forwarded to the FDA if permission was granted and were added to the feedback from 200 people who attended the FDA public meeting in person and approximately 150 people who participated in the FDA public meeting via webcast. After the public meeting, 41 responses were received via the FDA’s public online docket. Please refer to the FDA website for information on input received via the FDA public meeting and docket.

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