Description
Welcome and General Introductions
Dr. Julie Panepinto, Director for the Division of Blood Diseases and Resources (DBDR), and Executive Secretary of the Sickle Cell Disease Advisory Committee (SCDAC), opened the meeting by introducing the new chair of the SCDAC, Dr. Titilope Fasipe. Dr. Panepinto explained the reason for the meeting, which was to provide NHLBI Sickle Cell Disease (SCD) and Cystic Fibrosis (CF) portfolio updates in response to the request made by the SCDAC members to NHLBI.
Dr. Fasipe thanked the outgoing members whose term ended on June 30, 2024, and welcomed the candidates who are in process of becoming members. She provided time for SCDAC members to introduce themselves followed by Dr. Panepinto who introduced the NHLBI team from the Portfolio Analysis and Evaluation Branch (OPAE), who prepared the analysis on NHLBI SCD and CF portfolios to be presented and discussed.
NHLBI SCD and CF Portfolio Updates
Dr. Lindsay Scott from OPAE presented the analysis of the SCD portfolio, as well as a portfolio comparison between SCD and CF at NHLBI alone, and across ICs at the NIH. The interest for the analysis stemmed from two peer reviewed publications1,2 which argued that the NIH provided more research funds per affected individual with CF than for SCD. The analyses presented were structured in an additive way comparing the funding trends between the two over a sixteen year time frame from 2008 to 2023. Among the findings, it was noted that the overall funding for both diseases is similar, and that the NHLBI has increased its investment in SCD since the beginning of the reported timeframe. However, the number of applications has remained flat for both diseases despite SCD having more than double the patients than CF. The analyses were made using the Centers for Disease Control (CDC) estimates of US population affected by CF (35,000) and by SCD (100,000).
General Discussion
Dr. Fasipe opened up the session for questions and comments where the SCDAC committee members took the opportunity to thank the NHLBI for putting together the rich data presentation. The conclusion from the analysis was that there is a robust and equivalent investment to SCD compared to CF. Dr. Panepinto commented on the difficulty to measure and evaluate what the right value is to invest per affected individual on any particular disease, posing the question “how much is enough?” Instead, she challenged the members to encourage their peers to submit more grant proposals to NHLBI to help increase the number of awards toward SCD research. The consensus was that the flat number of grant applications for SCD was likely due to the reduced number of investigators applying for NIH funding. In contrast to SCD where most of the research funds are coming from the NIH, there are numerous funding opportunities and an enormous lobbying and investment for CF coming from the CF Foundation (non-NIH), especially in the clinical trials/drug development space. This emphasized the importance for SCD researchers to submit grant applications as they rely heavily on NIH funding support.
The topic on the type of applications most commonly received/awarded and the conformation of study sections and inclusion of hematology expertise on them was discussed. Dr. Traci Mondoro explained that although some awards such as the K and SBIR awards are reviewed by NHLBI study sections, other applications such as R01s and Fs are managed by the Center for Scientific Review (CSR) and do not belong to any one particular institute. As a result, Dr. Panepinto made a plea to the members to accept when they are asked to participate as a reviewer in study sections. She also briefly touched on how study sections are assembled and warned that some requirements and rules when forming a study section could make some candidates ineligible to participate. A study section requires a balance based on geography, gender, as well as other demographic factors, and rules to ensure a diverse and unbiased representation. The idea for a SCD study section was brought up, specifically for K awards and the need to resolve the fact that there are not enough SCD experts to evaluate them, as well as the need to address the recusal rules since there are many collaborations between SCD researchers due to the small research community, which further reduces the availability of investigators who can participate as reviewers. Dr. Mondoro emphasized the importance for trainees who are thinking to apply for K awards to contact the corresponding Program Officer at DBDR to discuss their particular interests, and what kind of expertise would be valuable for their ideal review.
Members asked whether or not the comparative analysis between SCD and CF included training grants such as K awards. The OPAE team responded that they were included in all of the applications or award cohorts reviewed; however, the main analysis was focused on Research Program Grants (RPGs), which do not include K awards. This topic could be a follow-on upon request from the SCDAC and DBDR leadership. Dr. Fasipe asked how NHLBI/DBDR staff reacted after seeing the data presented in terms of the level of satisfaction in the results, and whether or not SCD will remain a priority. Dr. Panepinto alluded to her previous response stating there is much more to be done to continue improving the lives of people with SCD, “we are not nearly where we should be in advancing the quality of life, there is a lot more to do and sickle cell disease will remain a priority.”
Topics for next SCDAC meeting
The goal moving forward is to have two meetings per year, one virtual and the other in person. The next SCDAC meeting will be scheduled at the end of January 2025 and will be virtual. Members were asked to suggest topics to be discussed, and the following topics were recommended:
- Discuss the importance of funding clinical trials as it pertains to SCD as well as information on the proportion of funding clinical trials v. basic science
- Perform similar analysis to what was presented, but related to research topics of awarded grants. For example, transfusion versus neurologic, pain, gene therapy, etc. to identify research gaps and be able to direct new investigators to fill those gaps
- Determine how to increase applications for minority supplements submitted for SCD grants
- Investigators and clinicians are going to industry, potentially due to the lack of funding, or burnout from clinical responsibilities. Need to identify ways to attract, develop and retain physician scientists and researchers with great potential in the academic space while at the same time encourage minorities in research
- Identify ways in which patient and advocacy can be integrated into the SCDAC. Dr. Mondoro and Dr. Panepinto addressed the question of how community members can contribute in the SCDAC meetings and made clear that the DBDR wants to balance the meeting by providing a space to the community. The importance of Community Based Participatory Research (CBPR) was discussed including community advocate and warriors. Research cannot be done without the community as the community helps guide researchers and keep them anchored in the reality they are trying to address. Ideas recommended to improve community involvement included the following:
- Schedule time outside the SCDAC meeting to discuss ideas, identify priorities from the community and debrief with the community members on the topics to be discussed before the SCDAC meeting
- Identify current progress and disparities within NHLBI, and how to disseminate that information to the community
- Use other NHLBI sponsored venues like the Annual SCD Research meeting that takes place in August as a space where the community voice can be heard
- The NHLBI has the Cure Sickle Cell Initiative, which has an integrated space for the community, and it’s important for this advisory board to be aware of and understand it’s mission
- Awareness of research findings from the NHLBI funded research that are of interest to the community and how to disseminate research findings of interest to the community.
Understanding the various ways that NHLBI interacts within the SCD community may help create one vision.
The meeting was adjourned.
Titilope Fasipe, MD, PhD
Julie A. Panepinto, MD, MSPH