Thank you, Senator Hatch. I'm so proud to be the co-chair of the Rare Disease Congressional Caucus with you, and I share your concerns. I think there must be improvements that are made. I continue to be inspired by the families across my state, and your state, and our country that work so hard to make it easier for their kids to simply have access to drugs to treat their illnesses. Unfortunately, we haven't yet achieved all we do for these families.
I heard time and time again about the emotional roller coaster that many of them have experienced when they interact with the federal government on new approaches for these rare disease conditions. Too often they are unware when drugs are under review or confused about why experts or patients are not even consulted. The individual suffering from these conditions and their families need greater clarity about the process for evaluating and approving these drugs, and they ought to be included and informed every step of the way. It's critical that treatments exist for those with rare conditions and that they be accessible and affordable, and that we continue to protect these individuals from discrimination and insurance coverage and work to bring down costs. We have to ensure that incentives designed to spur the development and accessibility of treatment that the rare disease community needs and are not used. Senator Hatch, the bill you passed helped so many. How do you focus on sharing this message with our colleagues and our constituents?
Well, that is going to be very important and really an opportunity to make sure that this works for patients with rare diseases and their families. We know that affordability and accessibility remain paramount. We should also think about the burden that these conditions play and the critical role of the voice of the patient. As you associated, Senator Hatch, more than 7,000 rare diseases exist and the vast majority have no treatments. This is an extraordinary burden borne every day by Americans in every single state across the country.
As we seek to make progress, including monitoring implementation of the advances in the bipartisan 21st Century CURES Act, we must make sure rare diseases receive sufficient attention. Federal agencies need to incorporate the voice in the decision-making process as I mentioned earlier. All too often, as we rightly focus on evidence-based medicine, we can lose sight of different therapies. Some of these can be difficult for real patients in real-life situations--all the more so when children are involved. The FDA all agencies should ensure they have appropriate processes to seek this vital input and again, the user fee agreement will be opportunity for us to make this case.
I'd like to thank you, Senator Hatch, for your time to discuss these issues that are important to both of us. We look forward to engaging our colleagues on these issues as we look forward with the implementation of the CURES Act and the user free agreement.