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Medicine Development and Access for Rare Diseases: Can We Do Better?
Carla E M Hollak1,2,3, Noa Rosenberg2, Colinda Post1,2,4
1Department of Endocrinology and Metabolism, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Expertise Center for Inborn Errors of Metabolism, University of Amsterdam, Amsterdam, the Netherlands.
Journal of Inherited Metabolic Disease
|February 17, 2026
Summary
Academic researchers must strengthen their role in orphan medicine development to improve access and affordability. Greater engagement across all stages, regulatory training, and sustainable pricing frameworks are crucial for rare disease therapies.
Area of Science:
- Molecular biology and genomics
- Rare disease research
- Orphan medicine development
Background:
- Advances in genomics enhance rare disease understanding and targeted therapies.
- Orphan medicine development faces complexities in regulation and accessibility.
- High prices and uncertain effectiveness of specialized treatments highlight systemic shortcomings.
Purpose of the Study:
- To analyze the role of academic researchers in orphan medicine development.
- To identify challenges in the development, regulation, and accessibility of rare disease therapies.
- To propose strategies for improving academic engagement and medicine affordability.
Main Methods:
- Literature review and analysis of academic contributions to orphan medicine development.
- Examination of current industry-driven development models and academic-industry interactions.
- Evaluation of existing regulatory and market access frameworks.
Main Results:
- Academic contributions are concentrated in early research, lacking continuity.
- Limited academic expertise exists in regulatory affairs, market access, pricing, and licensing.
- Industry-driven development often sidelines academia, leading to reactive roles.
Conclusions:
- Sustained academic engagement across all development stages is vital.
- Integrating regulatory and market access training is essential for researchers.
- Independent academic-industry collaboration and socially responsible frameworks are recommended to enhance rare disease therapy access and affordability.
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