Industrializing rare disease therapy discovery and development

Sean Ekins; Nature Biotechnology 35, 117–118 (2017) doi:10.1038/nbt.3787 Published online 08 February 2017

There would also need to be close interactions between these scientists, clinicians, rare disease patients, and their families and the many rare disease foundations that could perhaps help fund uch a concentrated effort. To build the scale that could handle orders of magnitude more diseases would require the use of computational, automation, and informatics tools to help identify, create, and manufacture treatments and facilitate the collaborations necessary to create a pipeline to bring them to the clinic. Centralizing efforts on rare diseases would also need to bring with it the relevant expertise necessary to go from the preclinical to clinical stages. Treatment discovery in the pharmaceutical and biotech industry has been suggested as needing disruption. Industrialization of rare disease treatment development may lead the way to a more sustainable future that will have considerable support if it ultimately drives down the cost of rare disease treatments.