Orphan drugs are therapeutic agents developed to treat rare diseases. In the US, the Food and Drug Administration (FDA) determines rare medical conditions are those with a prevalence below 200,000 people for a potential drug to be eligible for designation. In the EU, the European Medicines Agency (EMA) designated rare medical conditions as those with a prevalence below 5 in 10,000 people within the EU. Orphan designation programs at the FDA and the EMA offer incentives for sponsors to stimulate drug development for rare diseases, which otherwise would not be profitable due to small patient populations. Additionally, the FDA offers rare pediatric disease (RPD) designation and priority review voucher programs to further incentivize drug development for rare diseases that primarily affect the pediatric population. This presentation will focus on what the orphan designation and RPD programs are, the incentives for obtaining such designations, and the required data needed to position your drug for regulatory success.
Key Learning Objectives:
Understand the criteria for orphan drug designation in the US and EU, including prevalence thresholds set by the FDA and EMA.
Explore the incentives provided by orphan designation programs to sponsors, such as financial incentives and market exclusivity, to encourage drug development for rare diseases.
Learn about additional incentives like the rare pediatric disease (RPD) designation and priority review voucher programs offered by the FDA to further stimulate drug development for pediatric rare diseases.
Gain insights into the necessary data and strategies required to position a drug for regulatory success under orphan designation and RPD programs.
Who Should Watch:
Regulatory Affairs Professionals,
Quality Assurance (QA) Professionals,
Compliance Officers,
Clinical Research and Development Teams,
Pharmaceutical Manufacturers,
Quality Control (QC) Professionals,
Clinical Operations Professionals,
Legal and Compliance Professionals
Darius J. Devlin, Ph.D., Senior Consultant, Regulatory Affairs
Dr. Darius J. Devlin is a Senior Consultant of Regulatory Affairs at BioPharma Global, a division of Pace® Life Sciences. Darius has experience in drug discovery, pharmaceutical quality, vaccine development, assay development, and molecular biology. He works to provide high-quality regulatory deliverables and advice to clients, including writing, drafting, editing, conducting literature research, and submitting orphan drug designation (ODD) applications, annual reports for granted ODDs, investigational new drug (IND) submissions and amendments, development safety update reports (DSUR), indication prevalence assessments, client data reviews, and other specialized reports for regulatory advice. He also performs quality control reviews of various regulatory applications. Darius is particularly interested in drug development and regulations for diseases with high unmet medical needs.
Prior to joining Pace®, Darius worked as an ORISE Postdoctoral Fellow at FDA CDER studying the impact of excipients on monoclonal antibody drug stability, quality, and immunogenicity. Darius received his Ph.D. in Translational Biology & Molecular Medicine from Baylor College of Medicine in 2020, studying the genetics of male infertility and targeting male-reproductive-tract-specific proteins for non-hormonal contraceptive development. He received his B.S. in Biological Sciences from Georgia State University in 2014, where he worked in the development of novel anti-microbial agents and vaccine development for group A streptococcus.
