The 21st Century Cures Act: Addressing unmet needs in children with rare disease

21st Century Cures Act and children
Among its other provisions, the Cures Act would advance implementation of the 2013 National Pediatric Research Network Act, boosting therapeutic development for rare childhood diseases.

Medical solutions often require countless hours of investigation, months of testing and monitoring, years of post-trial and market analysis and billions of dollars of investment — with no certainty of success.

Last year, after years of groundwork, the U.S. House of Representatives passed the 21st Century Cures Act. A companion measure is being developed in the Senate, and stakeholders are optimistic that agreement on a package — even a slimmed down bill — could happen this year.

While Congress has addressed research and medical product regulatory needs before, the Cures Act has been unique in its comprehensive approach, looking at all elements of the research spectrum — from basic discovery science to translational research to regulatory review. It would upgrade the National Institutes of Health’s research capabilities and update the Food and Drug Administration’s approval policies to get new drugs and devices to the clinic sooner.

The Cures Act has been developed in a highly open and transparent process with the engagement of hundreds of stakeholders, including patients and families. Its passage was largely bipartisan, a bit of a rarity in recent years, and most have praised the measure. Much of the delay in Senate passage has been related to how to pay for the package, particularly the sizeable bump in NIH funding ($1.75 billion for the next five years).

While the bill would go a long way toward addressing unmet medical needs, some stakeholders have expressed concerns over certain provisions they say could rush costly and unproven products to market. For example, the Cures Act broadens the definition of data that the FDA can use for an approval; rather than relying mainly on clinical trial data, the FDA can now consider data from observational studies. The FDA would receive an annual $110 million for recruiting scientific experts, who would help the agency handle a rising number of approval requests and revise its regulations to create greater flexibility. To encourage investment in therapeutic research and development, companies would be allowed six months of market exclusivity if they discover new uses for their FDA-approved drugs.

Boosting therapeutic development in pediatrics

One provision of the Cures Act is very important for children. As a founding member of the Coalition for Pediatric Medical Research, Boston Children’s Hospital has played a key role in ensuring that the Cures Act addresses the dire need for therapeutic development for rare pediatric diseases. The Coalition is comprised of major research hospitals from across the country and has been the main driver behind the 2013 National Pediatric Research Network Act (NPRNA).

The NPRNA authorizes the NIH to establish a network of multi-institution consortia focused on pediatric research. Each consortium would consist of a principal or hub institution and multiple partners. Applicants would propose a research agenda, from basic discovery through translation and clinical research, and the NIH would select awards through a peer review process. Consortia could be established along geographic lines or nationally around the core scientific agenda, leaving much discretion to the research community.

But while the NPRNA has been law for nearly three years, it has yet to be implemented by the NIH as intended by Congress. To push implementation forward, the Coalition has worked with leaders in both the House and Senate to make some changes to the existing law. This small but important provision is part of the House-passed Cures Act.

As Congress returns for a brief session before adjourning for the elections, Boston Children’s and our colleague institutions will continue pushing aggressively for the NRPNA enhancement provision to be included in any final package. Strengthening federal support for research with a pediatric focus is the stepping stone necessary to find cures for rare diseases in children.