MDF staff regularly speaks with potential funders and passes the information along to the research community via this space to help increase the breadth and depth of funded research in myotonic dystrophy. In the case of the National Institutes of Health (NIH), annual funding strategies, including pay lines, can be found on the Institute webpages (search “NIAMS,” “NINDS,” “NHLBI,” etc. and “Funding Strategy”).
We learned about current funding policies and funding opportunity announcements in a recent meeting with the National Heart, Lung and Blood Institute (NHLBI). Currently, there are very few federally funded projects directed at cardiovascular and respiratory involvement of DM. Because of NIH’s focus on funding the best science, increased funding for burdensome heart and respiratory system involvement in DM would require an increase in the number and quality of research grant applications.
Is there an opportunity for DM funding from NHLBI? First, NHLBI is part of the interagency Muscular Dystrophy Coordinating Committee and has emphasized its interest in applications in muscular dystrophies. Second, its recent pay line, at the 15th percentile, is better than most other Institutes funding DM. Third, for Early Stage Investigators (ESI), the “bump” in the R01 pay line is very substantial, to 25th percentile in the 2016 fiscal year. So, there is an extraordinary opportunity for ESIs to obtain funding for an understudied niche, basic, translational and clinical cardiorespiratory research for DM.
Finally, clinical trial development grants also present an interesting opportunity at NHLBI. The currently active NHLBI initiative, NHLBI Clinical Trial Pilot Studies (R34), provides funding for up to 3 years/$450,000 total direct costs to support studies sufficient to the design of interventional clinical trials in their mission, which includes key DM issues—heart, lung and sleep disorders. Activities supported by this initiative include:
- Perform studies to determine the appropriate study population, intervention, or outcome.
- Collect information necessary to estimate available populations, attrition rate, or response rate.
- Refine the intervention.
- Test the feasibility of an outcome or intervention in the field.
- Determine whether adequate adherence to a treatment is achievable.
- Standardize and validate survey instruments.
- Standardize and test the effectiveness of training tools.
- Adapt and test a survey instrument or protocol for a population that differs culturally from the population for which the instrument was originally designed.
The duration and budget allowed by this initiative represent an important opportunity to develop interventional clinical trials for DM.
Contact Jonathan Kaltman, M.D., for more information.