RFI – Supporting Development of Therapeutics for Rare and Neglected Diseases

The National Center for Advancing Translational Sciences (NCATS) is conducting market research to determine capabilities of existing organizations that execute in vivo pharmacology studies and animal model support for the Therapeutics for Rare and Neglected Diseases (TRND) program.

Currently, most of TRND’s in vivo pharmacology studies are conducted using a one-off approach with customized arrangements being made for each study, more likely than not, dictated by where the animal model resides. This is often met by lack of available human and other resources to conduct a well powered study to support drug development efforts in a reasonable time frame.

The purpose of this RFI is to obtain extensive feedback on how a capable organization can effectively and efficiently coordinate resources needed to execute any given study or set of studies (potentially including animal model refinement/optimization or validation) that may become the subject of a TRND Request for Proposal (RFP).

Potential tasks include:

• Develop, optimize or use existing validated animal models to conduct in vivo pharmacology studies for rare disorders and neglected diseases for projects accepted into TRND’s portfolio. These models are not able to be pre-determined as needs change depending on the disease, hence, access to both small (e.g., mice, rat) and large animal models (e.g., dog, cat monkey, pig) will be needed.
• Characterize and validate animal models that require development or further optimization/ refining prior to use for studies involving a therapeutic under development. Therapies can cover a wide range of modalities; small molecules, and biologics (ex. antibody, proteins, cell and gene therapy).
• Conduct necessary in vivo studies in validated models to support initial programmatic requirement of efficacy verification and further product development to involve studies such as:
• Conduct, if necessary, in vitro pharmacology studies to test therapeutics in existing, refined or newly developed ex-vivo or cell-based models of the disease.

Further details are available via Solicitation Number: HHS-NIH-NCATS-RFI-17-001. Responses are due by January 10, 2017.