During a House Energy Committee hearing last week, Rep. Gus Bilirakis (R-FL) asked Health and Human Services Secretary Robert F. Kennedy Jr. about rare disease cures and drug development.
00:00He now recognizes the gentleman from Florida, Representative Bill Arrakis, for five minutes of questioning.
00:05Thank you, Mr. Chairman. I appreciate it very much.
00:08Thanks for holding this hearing, and I want to thank you, Mr. Secretary, for your service to our country, and thanks for being here today.
00:17Secretary Kennedy, one of the areas I've been passionate about is the need for the U.S. to continue leading the world and developing innovative, life-saving movements.
00:27And I know you are, too. So this is particularly important and challenging in the area of rare disease and ultra-rare disease,
00:37where cures and treatments can be much harder because of the economic and regulatory hurdles to developing treatments for these very small populations.
00:48One important tool in promoting this drug development is the FDA's Rare Pediatric Disease Priority Review Voucher Program,
00:58which has led to the approval of over 50 treatments for 39 different rare pediatric diseases,
01:0636 of which had no FDA approval treatments before the program began.
01:11I was very pleased to see the President's FDA budget allocate funding for this Priority Review Voucher Program,
01:19and I have bipartisan legislation that would fully reauthorize the program for years to come.
01:25So the question is, Mr. Secretary, will you commit to working with me to ensure this critical PRV incentive is retained for American innovators
01:36to develop new treatments for children with rare diseases?
01:41And can you elaborate on any additional initiatives that HHS foresees implementing to further accelerate the approval of life-saving treatments?
01:52Yeah, I mean, this is another priority for us.
01:55I've met two weeks ago with CEOs and investors from the biologic space, which are developing rare disease drugs.
02:06I was down in South Carolina about a month ago with Senator Tim Scott inaugurating an innovative program
02:17to provide sickle cell disease to every person on Medicaid in South Carolina.
02:25There's 356,000 black Americans who have sickle cell.
02:29It's an agonizing disease.
02:31It's just one of those diseases that we're talking about.
02:35In the big, beautiful bill, there's a provision that will relax some of the regulatory impediments
02:43or getting approval from FDA for rare diseases.
02:48And I urge you to take a look at that and support it.
02:54And Marty McCary now, the director of FDA, is on a listening tour around the country meeting with the innovators
03:02who are developing rare disease innovations.
03:05We are going to try to fast-track that approval.
03:08We're using AI to fast-track it.
03:11We're using an expansion of the compassionate use exemptions to expand it.
03:18It's something that we talk about every day at HHF.
03:21Very good. Thank you.
03:22I have a question.
03:23And 95 percent, the rare diseases, 95 percent did not have treatments, as you know, or cures.
03:30So I really, thanks for making that a priority.
03:34I look forward to working with you.
03:35Another question, I'm also encouraged to hear about the announcement last week to launch the Commissioner's National Priority Voucher Program,
03:43which aims to increase drug development in the areas the FDA deems to be in support of use national interests, which is so important.
03:53We saw this mechanism work successfully with the Operation Warp Speed and other efforts that the Trump administration has done to get red tape and bureaucracy out of the way
04:07and instead focus on accelerating gold standard science to develop cures.
04:12So the question is, I know I don't have a lot of time, can you speak further on the need for the national priority voucher program
04:20and how HHS will broadly ensure U.S. leadership in innovative treatments and that those treatments are developed here in America?
04:31We are already, we're using that program now extensively.
04:36We're very excited about it because it crashes through the red tape.
04:41It may, it facilitates a very rapid approval of these drugs.
04:44We've already applied it to a number of new drugs and it's something that we're going to, as I said, use extensively.
04:53So I appreciate your support on that and I'm happy to work with you on it.
04:59Well, I really appreciate it very much.
05:00I don't have a lot of time, so I'll probably yield back.
05:03But thank you again for thinking outside the box.
