Our Government has an immense capability to help advance treatments and cures for rare diseases through incentives for industry, research grants, and improving regulatory conditions; however, our politicians often lack the public support to challenge the status quo and make good changes to the system.
Our democratic system was created to be responsive to the people; however you could argue that it was also specifically designed with checks and balances to only allow incremental change.  With an urgent need for treatment, families affected by a rare disease only become frustrated with this process.  Ask any patient advocate in D.C. and they will warn you about the difficultly or even impossibility of moving a bill through Congress.  Most advocates will spend most of their careers trying to prevent the loss of services, benefits, or funding and rarely have the opportunity to make real steps forward to improve access to treatments.
In fact, there has been no major legislative initiative to accelerate treatments for rare diseases since the passage of the Orphan Drug Act in 1983 – more than 25 years ago.  Science has made great progress over this time; however policy has not followed pace – until now.
Congress is listening and they want to help.  The Senate’s H.E.L.P. (Heath Education Labor & Pensions) Committee and Appropriation Subcommittee on Agriculture, FDA and Rural Development both held Congressional Hearings in the last month on how to increase the development of new treatments for rare diseases.  A Rare and Neglected Disease Congressional Caucus is also forming to address the needs of patients.  There is even new funding for rare diseases in the NIH and FDA budgets and new requirements for guidances for the FDA to improve the regulatory process.
While it is great to hear about all of the very exciting programs that have been initiated in the last year, there is no time to rest.  Too many children will still wake up tomorrow without treatment.  We must use this momentum to ensure our progress translates into real treatments.
Now is the time when we need your help.  You must become an advocate and take an active role in the political process.  Organizations like The Kakkis EveryLife Foundation, NORD and others are only powerful when their members and supporters take action.  Congress must hear from you – the patients, parents, grandparents, doctors, care takers, academics, and scientists.
I urge you to contact your Members of Congress – write letters, make phone calls, and meet with staffers in the district office to tell them how important advancing new treatments is to you.  Our Foundation has launched its political action center on its CureTheProcess website to allow you to easily contact your Members of Congress, and learn more about current legislation.
You have the power to make a difference.  Click here now and help turn science into medicine.
Contributed by Julia Jenkins, Kakkis EveryLife Foundation