Economies of scale for rare disease R&D
What is it about?
This small correspondence throws out the idea of what if we could find economies of scale for rare disease R&D then perhaps we could tackle many more of these 7000 diseases.
Why is it important?
It describes how a rare disease gene therapy was brought to clinical trials for several million dollars and how Small business grants from the NIH can also be used for ultra rare diseases. The return on investment may not only be the FDA priority review voucher but the ability to show that treatments for diseases can be developed cost effectively. Providing a model that can be used elsewhere. Perhaps most important, it may offer hope to rare disease families that developing treatments may not be out of reach.
The following have contributed to this page: Dr Sean Ekins