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Patient-Focused Drug Development Initiative

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Check out our project partners! Read the press release.

Mission Statement

The Patient-Focused Drug Development (PFDD) initiative provides a novel approach to gathering input from patients and caregivers. PFDD asks these individuals patient-focused questions to create a more complete and relevant assessment of the benefit-risk balance of new treatments undergoing regulatory review by the Food and Drug Administration.

Patient-Focused Drug Development Initiative


Genetic Alliance is implementing a proof of concept on the usefulness of crowd-sourcing feedback from affected persons for the Food and Drug Administration's Patient-Focused Drug Development initiative. Genetic Alliance is providing tools to seven support groups and disease advocacy organizations (DAOs) to help elicit the DAOs' community members' thoughts and experiences regarding the benefit-risk balance of treatment options, the severity of the condition, and unmet medical needs.

Disease Areas

For the Patient-Focused Drug Development initiative we will focus on the following medical condition areas: Sickle cell disease, Idiopathic pulmonary fibrosis, and irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease.

Project Process

Using a Request for Proposals process, Genetic Alliance chose seven DAOs that provide support, advocacy, or both to the medical condition areas of sickle cell disease, idiopathic pulmonary fibrosis, and irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease. Each organization and its members have the opportunity to contribute to the customization of an innovative tool for collecting patient input to inform the FDA’s Patient-Focused Drug Development initiative under the Prescription Drug User Fee Act V (PDUFA V). Genetic Alliance offers a new, technology-enabled approach to ascertain the views of patients and caregivers. Participating organizations will receive a customized version of this Platform for Engaging Everybody Responsibly (PEER), through which they will engage in a systematic collection of affected individuals’ perspectives. Each DAO will participate in determining the specific questions, beyond those required by the PFDD process, to be administered to the members of their community. Questions will address a condition’s severity and current unmet medical needs to determine benefit-risk considerations fundamental to a patient-focused drug development and regulatory review process. The results of the study will be published in a peer-reviewed journal.


Genetic Alliance is excited to offer the Platform for Engaging Everyone Responsibly (PEER) to capture the perspectives of DAOs’ members about the benefit and risks of medicines that are most relevant and important to them. To learn more about the platform and see a version of the technology, please visit In this proof of concept project, the disease advocacy organizations receive their own customized versions of the PEER system, which will reflect their brands and be seamlessly integrated into their websites. With the help of Genetic Alliance and our technology partner, Private Access, the disease advocacy organizations will be able to customize the sharing options (levels of privacy their members may wish to consider when deciding preferences about what information to share and with whom) and three guide videos to help educate their members (Genetic Alliance will provide scripts and editing).

FDA Meetings Background

As required by the Prescription Drug User Fee Act V (PDUFA V) commitments, the FDA will hold at least 20 public meetings focused on different disease areas over a five-year span to prospectively inform future benefit-risk assessments that are at the core of regulatory decisions about new medicines. Genetic Alliance seeks to ascertain if its new crowd-sourced, cross-disease, privacy-assured technology platform is useful in gathering patient perspectives and if it therefore may be important to help the FDA gather information. If this proof of concept is successful, a deeper understanding of participant perspectives will be achieved. Our goal is to collect a robust dataset of information and to offer a diverse range of perspectives to the FDA and to the participating DAOs.

Project Partners

Genetic Alliance
Private Access

Sickle Cell Disease
North Alabama Sickle Cell Foundation, Inc.
Sickle Cell Disease Association of America Southern CT
The William E. Proudford Sickle Cell Fund, Inc.
Citizens for Quality Sickle Cell Care, Inc.

Idiopathic Pulmonary Fibrosis
Coalition for Pulmonary Fibrosis

Irritable Bowel Syndrome, Gastroparesis, and Gastroesophageal Reflux Disease
Gastroparesis Patient Association for Cures and Treatments, Inc.
Share and Care Cockayne Syndrome Network


Funding for the Patient-Focused Drug Development initiative is from PhRMA.

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