tch, Klobuchar Discuss Rare Diseases in Colloquy the cochairs of the Senate Rare Disease Caucus, participated in a colloquyto discuss how best to incorporate the patient voice into broader policy conversations regarding the treatment of rare disease
PoliticalNews.me - Mar 11,2017 - Hatch, Klobuchar Discuss Rare Diseases in Colloquy
Washington, D.C.—Senators Orrin Hatch, R-Utah, and Amy Klobuchar, D-Minn., the co-chairs of the Senate Rare Disease Caucus, participated in a colloquy this afternoon to discuss how best to incorporate the patient voice into broader policy conversations regarding the treatment of rare diseases. The colloquy served as an opportunity to showcase the bipartisan support for developing innovations that will treat and cure our children, parents, siblings, and neighbors suffering from rare diseases. Senator Hatch continues to lead congressional efforts to bring safe and effective cures to the rare disease community.
Video Via YouTube
The full colloquy, as prepared for delivery, is below:
SENATOR HATCH: Mr. President, I would like to engage Senator Klobuchar in a colloquy to commemorate Rare Disease Day in order to discuss issues facing patients and their families who have been diagnosed with these types of conditions. As Co-Chairs of the Rare Disease Caucus, Senator Klobuchar and I have worked hard to bring more hope to patients and their families who are coping with rare diseases on a daily basis.
Today, one in twenty individuals worldwide is living with one of the more than 7,000 rare diseases, ninety-five percent of which do not have an effective treatment. While the incentives provided in the Orphan Drug Act, first championed by myself in 1983, has led to the approval of nearly 600 orphan drugs, much more must be done.
Many patients living with rare diseases rely on the FDA to evaluate and approve treatment options for their conditions. That is why it is so important for FDA to use its authority to accelerate the evaluation and approval of drugs for treating rare diseases, and for Congress to ensure that proper incentives exist for research to discover and make treatments and cures available for this community.
To address this issue, Congress passed the FDA Safety and Improvement Act (FDASIA) of 2012, which refined and strengthened the tools available to FDA to accelerate the evaluation and approval of new drugs targeting unmet medical needs for rare conditions. I have been paying close attention to how this new authority translates into advances for patients suffering from conditions such as Duchenne Muscular Dystrophy (DMD), Atypical Hemolytic Uremic Syndrome, Bertrand-N-glycanase deficiency, and other rare diseases.
In light of these changes over the past few years, I ask my friend from Minnesota whether the current approval process is achieving its goals of safety and efficacy without hampering the development of new therapies?
SENATOR KLOBUCHAR: Thank you Senator Hatch for beginning this colloquy. As Co-Chair of the Rare Disease Congressional Caucus, I share my colleague’s concerns. I continue to be inspired by the network of families who have worked so hard to make it easier for their children to have access to drugs that treat their illnesses. Unfortunately, we have not yet achieved all that we could for these families and the clinicians who work in this field.
I have heard time and again about the emotional roller coaster