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Patient Focused Drug Development Challenge

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On Tuesday, October 8, 2013 the Genetic Alliance announced a challenge calling for Disease Advocacy Organizations to interested in collaborating with us to improve patient focused drug development.


Request for proposals released: Oct. 8th, 2013 

Question period: Oct. 8rd- 15th, 2013

Questions and answers released: Oct. 16th, 2013

Proposals due: Oct. 21st 5 pm EDT, 2013

Applicants notified of decision: Oct. 30th , 2013

Notification of participation in project: Nov. 1st, 2013

About the Proposal

The FDA periodically conducts public assessments concerning how well the Agency’s procedures and processes take into account the needs and risks of persons affected by serious or life threatening diseases or conditions. The Genetic Alliance aims to broaden the scope of these public assessments to provide well-documented and statistically representative input to focus these regulatory decisions.

Disease Areas the FDA will focus on in the future include:

  • Alpha-1 Antitrypsin Deficiency
  • Breast Cancer
  • Female sexual dysfunction
  • Hemophilia A, Hemophilia B, von Willebrand disease, and other heritable bleeding disorders
  • Idiopathic Pulmonary Fibrosis
  • Sickle cell disease
  • Irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors
  • Neurological manifestations of inborn errors of metabolism
  • Parkinson’s disease and Huntington’s disease
  • Pulmonary arterial hypertension

Genetic Alliance PFDD Challenge 

The judges will select 3 disease areas from the list above and will choose 2-3 disease advocacy groups for each disease selected. The disease advocacy groups selected to participate in this challenge will have an opportunity to contribute to the assessment of patient risks and benefits considered in drug development. Genetic Alliance will provide you with free use of our new Platform for Engaging Everybody Responsibly (PEER), through which to handle a systematic collection of patients’ perspectives based on a condition’s severity and current unmet medical need to determine benefit-risk assessments fundamental to the regulatory drug approval and development process.

Disease advocacy organizations will be evaluated and chosen based on the readiness of the disease advocacy organizations; geographic distribution of the organizations; ability to reach out to and obtain feedback from wide member base; and the diversity and attention to underserved communities.

One example of PEER can be found here: Registries for ALL ( This is the tool that will be customized for advocacy and community organizations involved in this PPRN. Please email Tetyana Murza ( for an application today!

Questions about the Application Process

arrow icon How can my organization apply?

arrow icon My organization is already planning on submitting an application for the PFDD challenge, can we submit more than one?

arrow icon My organization is small and only has volunteer staff, can we still apply?

arrow icon Can multiple organizations combine resources and submit one application?

Questions about the Application Process

arrow icon Can you provide more information about the role of the individual leading the project? Where and how often will meetings be held? Does the individual need to be somebody who can travel?

Questions about the Challenge

arrow icon What assistance will the Genetic Alliance provide the selected organizations?

Questions about PEER

arrow icon How can I get more information about the registry platform the Genetic Alliance will use for this challenge?

arrow icon To what degree will mobile technology or apps be used in collecting data or communicating with members?

arrow icon Who owns the data collected?

arrow icon Where will the data collected be stored?

arrow icon Who will have access to the data collected?


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