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Non-oncology orphan drug development: Productivity and probability of success
Samantha Parker1, Jida El Hajjar2, Anneliene H Jonker3
1International Rare Diseases Research Consortium, Paris, France; Italfarmaco SpA, Milan, Italy.
Abstract:
There are >6000 rare diseases (RDs), affecting >300 million people worldwide. Despite increases in orphan drug designations (ODDs), approved treatments remain scarce. This study analyzes the outcomes of non-oncology ODs designated by the FDA and European Medicines Agency (EMA) in 2017, providing a 7-8-year window to evaluate progression. This analysis of 292 ODDs examined therapeutic modalities, sponsor size, and funding factors influencing success, as defined by development phase transitions. Biologics showed higher approval rates whereas gene therapies displayed lower phase transition success. Smaller companies and academic groups demonstrated lower success rates. This research identifies drivers and gaps in OD development, emphasizing the need for sustained multistakeholder investment.
Insights
Developing treatments for rare diseases (RDs) remains challenging. This study found biologics have higher approval rates, while gene therapies and smaller sponsors face hurdles in orphan drug development.
Area of Science:
- Pharmacology
- Biotechnology
- Drug Development
Background:
- Over 6000 rare diseases (RDs) affect 300 million globally, with limited approved treatments.
- Orphan drug designations (ODDs) have increased, yet treatment scarcity persists.
- Non-oncology orphan drugs face significant development and approval challenges.
Purpose of the Study:
- To analyze the 7-8 year outcomes of non-oncology orphan drugs (ODs) designated by the FDA and EMA in 2017.
- To identify factors influencing the success of orphan drug development.
- To pinpoint drivers and gaps in the orphan drug pipeline.
Main Methods:
- Analysis of 292 non-oncology orphan drug designations from 2017.
- Evaluation of therapeutic modalities, sponsor size, and funding.
- Assessment of development phase transition success rates.
Main Results:
- Biologics demonstrated higher approval rates compared to other modalities.
- Gene therapies exhibited lower phase transition success rates.
- Smaller companies and academic institutions showed reduced success rates in development.
Conclusions:
- Therapeutic modality, sponsor size, and funding significantly impact orphan drug development success.
- Gaps exist in translating designations into approved treatments, particularly for gene therapies and smaller entities.
- Sustained, multi-stakeholder investment is crucial for advancing rare disease therapies.
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