Medicine Development and Access for Rare Diseases: Can We Do Better?

WHY IT MATTERS

If you or a loved one has a rare disease, this research highlights why medicines for your condition may be expensive or hard to get — and pushes for systemic changes that could improve your access to treatment options.

Scientists and doctors are saying that the system for developing and selling medicines for rare diseases needs improvement. New discoveries in genetics have helped create targeted treatments, but these medicines are often very expensive and hard to access. The article calls on researchers, doctors, and other groups to work together to make rare disease medicines more affordable and available to patients who need them.

Medicine Development and Access for Rare Diseases: Can We Do Better? Abstract: Recent advances in molecular biology and genomics have significantly enhanced our understanding of rare diseases. While enabling the development of highly targeted therapies, it also leads to complexity in the development, regulation, and accessibility of orphan medicines. Unmet need and great promise of new medicines, combined with high prices and uncertain effectiveness, highlight the shortcomings of the system, particularly evident for highly specialized treatment options, such as advanced therapy medicinal products and RNA-based treatments. While all stakeholders in this field must take responsibility, academic researchers and clinicians have a vital role which must be strengthened to improve access to and affordability of medicines. Regarding academia-driven orphan medicine developmen Authors: Hollak et al. Journal: Journal of inherited metabolic disease MeSH: Rare Diseases, Humans, Orphan Drug Production, Drug Development, Health Services Accessibility, Drug Industry