Amy Klobuchar: “Well, that is going to be very important and really an opportunity to make sure that this works for patients with rare…”

“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 stated, Senator Hatch, more than 7,000 rare diseases exist, and the vast majority have no treatment. This is an extraordinary burden borne every day 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's voice in their decisionmaking process. As I mentioned earlier, all too often as we rightly focus on evidence-based medicine, we can lose sight of the human experience of these and different therapies. What may seem simple in a lab may be overwhelming or difficult when applied to 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. The user fee agreement will be an opportunity for us to make this case. I would like to thank Senator Hatch again for his time to discuss these issues that are very important to both of us.…”