The National Heart, Lung, and Blood Institute (NHLBI) has a legacy of conducting and funding clinical trials that benefit public health through improved prevention, diagnosis, and treatment of heart, lung, blood, and sleep disorders. Currently, the traditional large randomized controlled trial predominates among NHLBI-funded applications. However, for trials conducted in limited populations, traditional clinical trial designs may not achieve adequate statistical power, so innovative approaches to statistical design and analysis plans may be needed. Scientific areas in which innovative approaches might be required include studies of rare diseases and/or therapeutics in which sample size limitations provide a challenge to the statistical power of traditional randomized clinical trial designs. These design limitations are particularly relevant to many blood disorder and therapeutics trials. The purpose of a new dual pilot Funding Opportunity Announcement entitled Catalyzing Innovation in Late Phase Clinical Trial Design and Statistical Analysis Plans (Catalyzing Innovation) is to support investigators who are planning late-phase single-site or multisite investigator-initiated clinical trials that address critical clinical questions within the mission of NHLBI and require nontraditional clinical trial designs with the opportunity for statistical innovation and/or novelty. Investigators will be afforded 2 pathways through which to access up to 12 months of expert consultative services in innovative clinical trial design analysis. This program aims to target underrepresented areas in NHLBI’s current clinical trials portfolio that include less common disorders and thus represent a singular opportunity for clinical investigators in blood disorders and therapeutics.

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Contribution: D.M.D. is the sole author of the article and the speaker in the audio version of this Blood Advances Talk.

Conflict-of-interest disclosure: The author declares no competing financial interests.

Correspondence: Donna M. DiMichele, National Heart, Lung, and Blood Institute, National Institutes of Health, 6701 Rockledge Dr, Rockledge Two, Room 9132, Bethesda, MD 20892; e-mail: