Since the Orphan Drug Act was passed 40 years ago, there have been significant change in the size and composition of the rare disease drug development pipeline. Between 1983-2019, the Office of Orphan Products Development (OOPD) within the US Food and Drug Administration (FDA) granted 5,099 orphan drug designations. Of this total, 58% of designations (2,979) were granted between 2010-2019—a  three-fold increase in total designations from the prior decade. Of these 5,099 designations, 59% (3,010) were small molecules and 41% (2,089) were biologics. These designations represent 3,269 unique drug or biologic products. Within these products, we see multiple instances of repurposing and repositioning: 26 unique products had ten or more associated designations, representing 379 designations. Approximately one-third of these most designated products were antineoplastics, and an additional third were immune system products (including immunostimulants and immunosuppressants). The top five most designated products were: interferon alfa (30 designations), alpha-1 proteinase inhibitor (21), immune globulin (21), somatropin (21), cannabidiol (19), and paclitaxel (19). In recent years, we have seen the emergence of novel new drug types being designated, such as gene, cell, and tissues therapies, as well as antisense oligonucleotides. Preliminary analyses indicates that between 2010-2019, 660 unique diseases were designated. The most frequently designated diseases in this period were pancreatic cancer, acute myeloid leukemia, and multiple myeloma. The non-oncologic disease with the most designations between 2010-2019 was amyotrophic lateral sclerosis (ALS) with 57 designations. These analyses show the changing landscape of rare disease drug development: a substantial increase in the number of products being developed; novel product platforms being investigated for rare diseases; and sustained investment in oncology products.

Trends in rare disease drug development

Where thousands of chemistry professionals meet to share ideas and advance scientific and technical knowledge. Spring 2023 abstracts, posters, and presentations will launch at the end of March.

ACS Spring 2023

The official archive and repository for all ACS Meetings & Expositions abstracts and select posters or presentations.