P335: Learning and Education to ADvance and Empower Rare Disease Drug Developers (LEADER 3D)
Poster Presenter
Andrea Bell-Vlasov
Science Policy Analyst
U.S. Food & Drug Administration United States
Objectives
LEADER 3D is a regulatory initiative with the goal to develop educational content that addresses complexities in bringing rare disease (RD) products regulated by the Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) to the market.
Method
CDER conducted a landscape assessment via interviews and a public docket to obtain feedback from RD drug developers on complexities faced in bringing RD products to market. Feedback was analyzed to identify topics of interest for which educational content could be developed.
Results
As part of the Accelerating Rare disease Cures (ARC) Program, CDER’s Rare Diseases Team (RDT) inaugurated the LEADER 3D initiative to better understand and address the complexities in bringing RD products regulated by CDER to market. RD drug development can be complex for many reasons, such as small patient populations, genotypic/phenotypic heterogeneity within a disease, and novel endpoint development and selection, all in the context of often serious and life-threatening diseases without approved therapies. These complexities can make study design, conduct, and interpretation of data challenging when developing a RD drug program for future regulatory assessment.
To achieve the goal of LEADER 3D, CDER obtained feedback from stakeholders involved in RD drug development to better understand their needs, and the unique complexities in bringing RD products regulated by CDER to market. Feedback was obtained through interviews and a public docket for which an analysis was completed. The analysis identified several topics of interest; specifically, they included non-clinical, dose-finding, natural history studies/registries, novel endpoints and biomarker development, clinical trial design and analysis, and RD drug development regulatory considerations. The analysis also noted that stakeholders were unaware of and/or had difficulties in finding currently available resources (e.g., FDA guidance, CDER Biomarker Qualification Program, etc.). Stakeholders also noted their interest in educational materials, such as case studies that provide examples of RD drug approvals.
Based on this feedback, currently available resources relevant to RD drug development were organized and made accessible on the LEADER 3D website. Additionally, several new relevant educational materials, including case studies and videos, were developed.
Conclusion
The stakeholder feedback was key in identifying topics of interest for which drug developers desired regulatory information. Such information could ultimately improve the regulatory fitness of RD drug programs and submissions. To provide this information, RDT and CDER subject matter experts across clinical, pharmacology/toxicology, statistics, and clinical pharmacology developed educational materials, including several case studies and videos regarding RD drug development. In total, three videos, two case studies, and a case study user guide were published on the LEADER 3D website. In addition, a public report of external stakeholder analysis, links to the FDA guidance tab that organizes guidances relevant to RD drug development by topic, and links to funding opportunities for RD drug developers are made available on the website.
Currently available videos and case studies are:
Video: Challenges, Strategies, and Regulatory Considerations for the Design of Rare Disease Clinical Trials
Video: Understanding the Importance of Endpoints in Rare Disease Drug Development
Video: Considerations for Collecting and Using Natural History Study Data that are Fit For Use in the Regulatory Setting
Case Study: Fosdenopterin: Use of a Single Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence to Demonstrate Substantial Evidence of Effectiveness for a Rare Disease
Case Study: Olipudase alfa-rpcp: A clinical Dose Escalation Strategy for a Rare Disease Drug Program.
Educational content can be found on the LEADER 3D website. The webpage will be updated with new materials as they become available. The RDT intends to obtain feedback on the new educational materials and continue to improve and develop content that meets the needs of the RD drug developer community.
