OPINION:
Every day since Robert F. Kennedy Jr. (henceforth “RFK2”) was nominated and then confirmed as the secretary of health and human services, highly credentialed, experienced and committed public health experts have had a collective meltdown. I refuse to be one of them.
It’s the dawning of a new era, and Americans are filled with hope that their new leaders on both ends of Pennsylvania Avenue in Washington have the expertise and dedication to fix the most stubbornly persistent problems with the nation’s health care. The president’s focus on tackling the root causes of chronic diseases promises to “make America healthy again,” while new leadership at the Health and Human Services Department, as well as the Food and Drug Administration, the National Institutes of Health and the Centers for Medicare & Medicaid Services, are bringing their ideas to overhaul these federal agencies for the benefit of patients.
While Congress and the Trump administration lay the groundwork for wide-reaching reforms, they must keep the 30 million Americans with rare diseases at the forefront of any policy conversations. Alarmingly, 95% of rare diseases lack FDA-approved treatments or cures, a major reason the economic burdens (direct medical costs plus factors such as lost earnings and decreased productivity) exceed $1 trillion annually. Most important, rare diseases are deeply personal, with countless parents and loved ones lying awake at night wondering whether their children will get the care they need to live long and ultimately healthy lives.
Rare diseases cannot be ignored. To make a real difference, policymakers must extend the Rare Pediatric Disease Designation and Priority Review Voucher program, which expired in December, and formally establish a Rare Disease Center of Excellence at the FDA.
Housed under the Office of Orphan Products Development, the Priority Review Voucher program has been a critical incentive for pharmaceutical manufacturers to research and develop cutting-edge treatments for rare pediatric diseases. Allowing companies that earn approval for a pediatric rare disease treatment the possibility of a timely priority review for a subsequent and different product (or even selling the voucher to another manufacturer) can help defray some of the major investments in drugs for small populations that would otherwise make little financial sense in the ultra-competitive pharmaceutical market.
So far, the voucher program has been a huge benefit for the country’s most vulnerable patients. From 2012 through 2024, the program spurred the development of 53 innovative treatments for 39 pediatric rare diseases. Unfortunately, Congress seriously disappointed the entire rare disease community, including the families of children holding out hope for a cure, when it failed to reauthorize this vital program in December. In the spirit of the Trump administration’s commitment to restoring life, liberty and the pursuit of happiness in American public policy, the program must be reauthorized immediately to give more children a chance to grow up, live healthy lives and pursue their dreams.
In addition, Congress and the administration have a once-in-a-generation opportunity to overhaul the FDA, which is a commitment expressed by the Trump administration. As part of that process, they should strengthen rare disease policy coordination, as well as the drug review process, by establishing a Rare Disease Center of Excellence for which rare disease groups and bipartisan leaders have long advocated.
To date, the FDA has been fragmented, with experts scattered throughout dozens of offices, many of whom have provided conflicting guidance, have duplicated work with wasteful results, or have little to no experience in rare diseases. Worse, this broken system has led to serious delays in the review process, frustrating and even bankrupting innovative companies, increasing the costs and preventing the development of promising cures, and depriving patients of treatment opportunities when time is of the essence. Despite recognizing the lack of coordination at the FDA regarding rare diseases, the agency has taken only modest steps to improve these processes. Although the newly designated Rare Disease Innovation Hub announced last summer is a step in the right direction, it lacks the staff, budget and centralized authority held by more impactful governmental agencies such as the FDA’s Oncology Center of Excellence and the CMS Innovation Center.
What’s perhaps most important, the Hub lacks true decision-making power. Its charge is to help answer questions and provide guidance early in drug development. In other words, it does nothing to ensure that the FDA teams reviewing applications for rare disease treatments are fully coordinated and consistent in their approval decisions across applications. It does not even ensure that the most relevant experts in physiology, pharmacology, small-trial design and other highly technical areas are included in the review.
Worse yet, no single decision-maker at the hub has the authority to address, correct and/or prevent these problems. The result is inconsistent, unreliable and conflicting guidance and decisions on the part of the FDA. So, although it is optically a major milestone shining a light on this critical area of need for 30 million Americans, the Rare Disease Innovation Hub is not set up for tangible progress or meaningful success.
To make sure rare disease candidates are appropriately understood and reviewed against a uniform set of criteria, the FDA must create a Center of Excellence for Rare Diseases under single leadership akin to and with the same mandate as the similar centers it has already established to streamline efforts to improve cancer care and digital health. Instead of “rule by committee,” this person must be accountable and have the responsibility and authority, similar to the director of the Oncology Center of Excellence for cancer treatments, to make decisions about rare disease candidates. This will help remove barriers, drive innovation and ultimately help patients who currently lack treatment or cure for their rare disease.
The Trump administration should work with Congress to advance these two rare disease priorities to help make America healthy again. With their success, millions of patients and their loved ones can turn their hopes into healing.
• Peter J. Pitts, a former Food and Drug Administration associate commissioner, is president of the Center for Medicine in the Public Interest and a visiting professor at the University of Paris School of Medicine.
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