by Emil D. Kakkis
October 8, 2009
“The beginning of treatment requires knowing that you are sick…”
In July, we got a great gift that we were not expecting. After some travel for the foundation in May and June, I went away with family on vacation. But others were at work, and it was a good thing. Following a talk I gave at the Rare Disease Summit May 14, 2009 hosted by NORD that highlighted our key issues with the regulatory process, Francis Collins (before becoming NIH director) talked with Senator Brownback (R, Kansas) regarding rare and neglected disease regulation that lead to an amendment sponsored by Senator Brownback (R, Kansas) and Senator Brown (D, Ohio) to the 2010 FDA appropriation bill.
The amendment directs the FDA to assemble a team to evaluate various aspects of the regulatory process for rare and neglected diseases and provide a report to the Commissioner. This is a great gift to our efforts this year and we were happy to discover this amendment had been submitted and approved by unanimous consent. The amendment went to the Conference Committee in which the differences between the House and Senate Appropriations bills are ironed out. The House bill did not have the same amendment and the Conference Committee must decide what stays and what goes out of the bill to reach a consensus bill. The Committee met last week, and the Brownback/Brown amendment was retained in the bill. The language was strengthened to say that the FDA “shall” not just “may”, put these review groups together to evaluate their policies around rare diseases.
This amendment is a first step toward establishing what the problems are and what changes are needed at the FDA for rare and neglected diseases. This works well with our goal to create science-based changes to public policy around rare diseases.
If the group is able to better define the problems, we can set the Agency and regulatory process down a path of improvement. To be able to CureTheProcess, we have to detail how the process is sick, and getting a group to clearly define that within the FDA, is a step toward treatment and recovery. The amendment is saying, “…call a doctor”. We would hope to help with the diagnosis.
The passage of the Brownback/Brown amendment is an important step forward and an important first success for our CureTheProcess campaign. We look forward to working with FDA and others on scientifically sound improvements to regulatory policy.
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