A new IRDiRC feature article published in Drug Discovery Today provides a comprehensive assessment of non-oncology orphan drug development, focusing on productivity and the probability of success for therapies targeting rare diseases outside of cancer.
The article, titled “Non-oncology orphan drug development: Productivity and probability of success, highlights several important trends:
- Higher Success Rates: Orphan drug development programs have shown higher success rates than traditional drug development, particularly when considering the full regulatory and development pathway.
- Data Resource: The analysis draws on a dataset of 292 orphan drug designations made in 2017, tracked over a 7–8-year period to assess progression across development stages.
- Modality Differences: The study notes variation in success depending on therapeutic modality, with biologics (e.g., complex protein or cell therapies) generally showing higher approval rates compared to small molecules and gene therapies.
- Influence of Prioritization: Success also depends on sponsor resources, funding sources, and scientific robustness — underscoring the importance of strategic planning and collaboration in rare disease R&D.
While rare diseases collectively affect hundreds of millions of people worldwide, treatment options remain limited. These diseases often lack commercial incentives for traditional drug development. The rise of non-oncology orphan drug success signals a positive shift that could broaden therapeutic coverage for conditions such as metabolic disorders, genetic abnormalities, neuromuscular diseases, and more. Understanding the productivity and probability of success of ODDs can help policymakers, investors, and patient advocacy groups better target support mechanisms and accelerate therapeutic innovations across a wider range of rare diseases.
Read the full publication, available open access: https://www.sciencedirect.com/science/article/pii/S1359644625002818?via%3Dihub