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 that many of these individuals and their families experience when they interact with the federal government on new approaches for these conditions. Too often, they are unaware when drugs are under review, or are confused about why outside experts or patients are not consulted. The individuals suffering from these conditions and their families need greater clarity about the process for evaluating and approving such drugs, and they ought to be included and informed every step of the way.
It is critical that treatments that do exist for those with rare conditions are accessible and affordable. We must continue to protect these individuals from discrimination in insurance coverage and work to bring down costs. We have to ensure that incentives designed to spur the development and accessibility of treatments that the rare disease community desperately needs are not abused.
Senator Hatch, how can we focus on sharing this message with our colleagues and constituents?
SENATOR HATCH: We must continue to urge FDA to fully implement its relatively new authority. Every one of us in this body represent constituents who are battling rare diseases, and I urge FDA to ensure this flexibility is applied in reviewing all candidate therapies.
I will continue to work closely with my Senate colleagues to ensure that FDA uses the tools, authorities and resources required to provide patients and physicians with new treatment options. I have also contacted the FDA frequently during the past year to encourage the agency to listen to the voices of patients during the agency’s evaluation process.
And when the Senate considers the nominee for FDA Commissioner, I will continue to stress the importance of incorporating a balanced and flexible approach when weighing risks, benefits and outcomes, especially when dealing with small patient populations and such rapidly progressing prognoses.
Patients with limited or no treatment options are depending on FDA to utilize the flexibility outlined in FDASIA. This law, which provides full and fair review of new drug therapies in a timely manner, gives hope to patients suffering from life-threatening diseases and their families.
Senator Klobuchar, how can we move forward into the next user fee agreement?
SENATOR KLOBUCHAR: Affordability and accessibility remain paramount. We should also be thinking about the burden of these conditions and the critical role of the voice of the patient.
As you stated, more than 7,000 rare diseases exist, and the vast majority have no treatments. This is an extraordinary burden borne everyday by Americans in every single state across the country. As we seek to continue making progress, including monitoring implementation of the advances in the bipartisan 21st Century Cures Act, we must ensure that rare disease treatments receive sufficient attention.
We also must encourage federal agencies to better incorporate the patient voice in their decision-making process. All too often, as we rightly focus on evidence-based medicine, we can lose sight of the human experience of these diseases and different therapies. What may seem simple in a lab may be overwhelming or difficult when applied to real patients in real-life situations – all the more so when children are involved. The FDA and all agencies should ensure that they have appropriate processes to seek and incorporate this vital input.
I’d like to thank the senior Senator from Utah for his time today to discuss these issues that are very important to both of us. We look forward to engaging with our colleagues on these issues further.
SENATOR HATCH: Thank you to my friend, the senior Senator from Minnesota, for her time here with me today. This is just the start of our conversation for this Congress. There is so much left for us to do, and I am certain that we will succeed as long as we stay together.