Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases (U44 Clinical Trial Required) | RFA TR 20 032

Opportunity Information: Apply for RFA TR 20 032

This NIH funding opportunity (RFA-TR-20-032) supports small businesses that want to run "basket" clinical trials in rare diseases, meaning a single clinical trial framework that tests a drug across multiple distinct rare diseases that share a common molecular cause or pathway. The central idea is to speed therapy development by focusing on shared biology rather than treating each rare disease as an entirely separate program. A project under this announcement is expected to be an actual clinical trial (the title specifies "Clinical Trial Required"), and the award mechanism is a cooperative agreement (U44), which generally means the NIH will have substantial scientific or programmatic involvement during the project compared with a standard grant.

The scientific emphasis is on drugs that target a shared molecular etiology across more than one rare disease. In practical terms, applicants should be able to justify, with strong mechanistic and/or human data, why one therapeutic approach is likely to affect a common driver across multiple conditions. Basket trials are meant to make clinical development more efficient in settings where patient populations are very small, and where dividing efforts into multiple separate trials could be slow, expensive, or infeasible. By enrolling patients based on molecular features that cut across diagnoses, the approach aims to generate clearer signals of activity and safety, while also making better use of limited rare-disease patient pools.

To reduce startup time and take advantage of existing infrastructure and data, the NIH states a preference for applications that focus on diseases already being studied within the Rare Disease Clinical Research Network (RDCRN). The FOA also favors applications that include collaborations with RDCRN clinical investigators. This preference signals that proposals aligned with RDCRN priorities, cohorts, registries, natural history data, experienced sites, and established investigator networks may be viewed as more feasible and better positioned to launch and execute a complex multi-disease trial efficiently.

Eligibility is limited to small businesses (the listed eligible applicant type), consistent with the U44 mechanism. Foreign institutions are not eligible to apply, and non-U.S. components of U.S. organizations are not eligible. However, the FOA notes that "foreign components" may be allowed as defined in the NIH Grants Policy Statement, which typically refers to discrete elements of a project that are performed outside the U.S. under specific conditions and with NIH approval, rather than the applicant organization itself being foreign. Applicants considering any international involvement would need to align with NIH policy and clearly justify why that component is necessary.

Administratively, the opportunity is issued by the National Institutes of Health, categorized under health, and associated with CFDA 93.350. It is a discretionary funding opportunity using a cooperative agreement instrument type. The posting indicates an original closing date of November 12, 2020, and a creation date of September 15, 2020, which is important for interpreting the opportunity as time-bound and potentially no longer open unless reissued or extended under a new announcement.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases (U44 Clinical Trial Required)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.350.
• This funding opportunity was created on 2020-09-15.
• Applicants must submit their applications by 2020-11-12. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Eligible applicants include: Small businesses.

Apply for RFA TR 20 032

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