We have been working all year long with our partners and the FDA on the concepts and strategies for improving the regulation and development of rare diseases treatments. The FDA wants to get it right and does appreciate the needs, but has been strapped due to short budgets for drugs reviews which are now two-thirds paid for by corporate user fees. But rare disease treatments are exempt from these fees, and the base support FDA from Congressional appropriations has not kept pace.
So, to get specialized, they need the funds to create a separate review division. That is where we come in. We have been pressing Congress in a tight budget year to provide a $10 million line item to create a rare disease review division. If we get the funds, FDA will make it happen.
Why do this?
Well, if we want a reviewer to say “yes” to a treatment, they have to know the disease and the treatments well enough to be able to make the right call easily. Without that knowledge it gets hard and “no” is easier to say. We all want effective and safe drugs but figuring that out is harder for some rare diseases. So, I want the best people we can get and have them focus on these rare diseases exclusively. They will then provide the right guidance and make the right decisions. It makes sense. The right people and the right process.
We are close to achieving one of our goals. Call Senators Kohl and Brownback and make the review of complicated rare diseases specialized in a new review division.
Call now and express your support by asking the Senators to “submit a $10million request for a new rare disease division at the FDA.”
Senator Herb Kohl: (202) 224-5653
Senator Sam Brownback: (202) 224-6521
Emil D. Kakkis, M.D., Ph.D.
Kakkis EveryLife Foundation