Patient-Focused Drug Development Initiative
Check out our project partners! Read press release here.
The Patient-Focused Drug Development project provides a novel approach in gathering input from patients and caregivers regarding patient-focused questions in the hopes of creating a more complete and relevant assessment to the benefit-risk balance of new treatments undergoing regulatory review by the Food and Drug Administration.
Patient-Focused Drug Development Initiative Project
Genetic Alliance is implementing a proof of concept to generate insights into the prospective usefulness of crowd-sourcing feedback from affected persons for the Food and Drug Administration's Patient-Focused Drug Development initiative. Genetic Alliance is working with the chosen seven advocacy/support organizations to provide tools to engage the DAOs’ communities in a novel and innovative experience to elicit their thoughts and experiences regarding the benefit-risk balance of treatment options, the severity of the condition, and unmet medical needs.
For the Patient-Focused Drug Development Initiative we will be focusing on the following medical condition areas: Sickle cell disease, Idiopathic pulmonary fibrosis, and irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease
Genetic Alliance has chosen seven DAOs that provide support/advocacy 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, new tool for collecting patient input to inform the FDA’s Patient-Focused Drug Development initiative under 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) system, 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 www.reg4all.org. In this proof of concept project, the disease advocacy organizations are receiving their own customized version of the PEER system, which will reflect their brand and be seamlessly integrated into their website. 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 Food and Drug Administration (FDA) will hold at least 20 public meetings focused on different disease areas over a 5-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, in turn, may be potentially important to gathering information for the FDA. 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.
Sickle Cell Disease:
North Alabama Sickle Cell Foundation, Inc.
Sickle Cell Disease Association of America Southern CT
Sickle Cell Warriors, Inc.
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 by PhRMA. Please visit here for past RFP information.