On behalf of the Cystic Fibrosis Foundation, we write to provide input on the proposed framework for reforming the National Institutes of Health (NIH). We appreciate that the Chair recognizes the critical role that the NIH plays in furthering scientific discovery and ensuring that the United States remains at the forefront of basic science and biomedical research innovation; though there are legitimate areas for improvement in current NIH structure, operations, and policies, we urge the Chair and the Committee to be extremely cautious about the extent and nature of the reforms proposed in the new framework to avoid hindering or diminishing the function of the NIH as it continues to serve as the foundation of the American research system.
Cystic fibrosis is a rare genetic disease that affects nearly 40,000 people of every racial and ethnic group in the United States. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene result in a buildup of thick mucus in multiple organ systems, leading to lung damage, life-threatening infections, and other complications. To facilitate the development of CF therapeutics — and, ultimately, a cure for CF — the CF Foundation is engaged in virtually every element of the research and development process, from preclinical discovery and identification of new therapeutics to conducting clinical trials and post-marketing surveillance. This includes significant support for a breadth of basic and translational research to better elucidate the biology of CF and the complications that arise from the disease, much of which is split between academic institutions and the CF Foundation Therapeutics Laboratory.
In addition to this involvement, CF Foundation has an extensive history of collaboration with the NIH. The CF Foundation has augmented research and core funding at multiple NIH-funded P30 academic Centers, supplemented NIH funding for the development of new technologies and model systems, provided training and experimental support by way of the CFFT lab to NIH funded investigators, implemented biorepositories and bio-banking efforts world-wide in support of CF research, and established a funding mechanism for meritorious NIH applications hindered by budget constraints. The CF Foundation has also sponsored, planned, and co-hosted research workshops on topics such as CF research needs in the era of highly-effective modulator therapies, advancing gene editing technologies for the treatment of CF lung disease, and CF-related diabetes. With this experience in mind, we offer the following feedback for the House Energy and Commerce Committee and Rep. McMorris-Rodgers as they refine their recommendations for structural and policy reforms of the NIH, especially in relation to NIH mission and leadership, funding, and grants.
Structural Reform: In certain circumstances, the structural reforms proposed within this framework — most of which entail consolidating multiple current Institutes and Centers (ICs) into larger, umbrella ICs — may be appropriate. Merging ICs with similar or complementary missions and research focuses may lead to valuable collaboration. However, it is critical to ensure that reorganization drives collaboration without resulting in diminished funding for important research or detrimental competition for funding between divisions within the newly-established ICs. This concern is particularly salient if funding to umbrella ICs fails to keep pace with the separate funding levels that would have been anticipated for the original constituent ICs.
Term Limits for IC Directors: Though periodic changes in leadership are valuable for bringing new perspectives to organizations such as the NIH, overly restrictive term limits have the potential to undermine the authority of IC directors, prevent IC directors from building the experience and knowledge required to help ICs operate at their fullest potential, and lead to the overall destabilization of ICs more broadly. Furthermore, with IC leadership functioning as a revolving door, NIH employees, collaborators, and grantees would be subject to constantly shifting goalposts and priorities, which would be especially difficult for newly-combined ICs with multiple disparate research focuses. For that reason, we recommend against establishing the five-year term limit described in this framework.
Congressional Role in NIH Review: The NIH is not, and should not be, exempt from review as it relates to performance, mission, objectives, and programs. However, it is critical that the NIH be able to operate free from undue political influence; the establishment of a congressionally-mandated NIH review commission empowered to make actionable recommendations jeopardizes the apolitical, non-partisan nature and reputation of the NIH. For that reason, we recommend against the formation of a congressionally-mandated NIH review commission.
Limitations on Grants for Investigators with Multiple NIH Engagements and Supporting Opportunities for Early-Stage Investigators (ESIs): The increasing trend towards awarding more grants and allocating a greater proportion of NIH funding to principal investigators with multiple active NIH engagements has the potential to impede the careers of ESIs. With that in mind, we agree that it is critical to ensure that funding is not disproportionately allocated to research groups with significant existing NIH resources and we support the proposed focus on providing grants and awards only to primary investigators that do not have more than three ongoing concurrent NIH engagements. At the same time, it is important to pursue additional other policies to continue to bolster and support ESIs, including increased opportunities for funding, training, and mentorship.
Indirect Costs: Given concerns about the abuse of the indirect cost system across the country, and the lack of visibility for either the NIH or investigators with regard to where that funding goes, we support increased transparency on indirect dollars as suggested by the reform framework, including making indirect F&A costs (e.g., fixed capital costs, administrative overhead, and labor costs) publicly available and searchable. We further believe that it would be reasonable to pursue options for reigning in indirect costs, including either outright caps on indirect costs or capping indirect costs at a graduated rate.
Community Review Oversight Boards: We are seriously concerned about the composition, authority, and data access of the proposed community review oversight boards. Reviewing and approving protocols, ensuring proper compliance with regulations and guidelines impacting the surrounding community, and creating processes for regular community access to information — the proposed duties of community review oversight boards, many of which are redundant with those of existing research oversight mechanisms, such as institutional review boards — is an enormous amount of power and responsibility. The reform proposal does not acknowledge this, nor does it address major questions about community review oversight boards, each of which would have significant ramifications for the scientific community and research infrastructure. These include board membership and the level of expertise required to serve; the extent of the power of the board, and whether or not it would have the authority to override other bodies involved in the research sanctioning and oversight process; and which individuals, both on the board and within the community, would be granted access to specific data, given potential confidentiality concerns. We therefore oppose the establishment of community review oversight boards as articulated by this framework.
We look forward to serving as a resource for the House Energy and Commerce Committee and Rep. McMorris-Rodgers as they continue to develop a framework for NIH reform.