Goal 3: Advance Translational Research

A Pipeline for Investigator- Initiated Translational Science

How might the NHLBI effectively encourage and support its investigators to collaborate with strategic partners to pursue the early translation of their HLBS discoveries into new diagnostics and therapeutics?

 

The critical challenges to effective early translation of discovery science experienced by the investigator community include:

 

1. The need for translational skills development, training and guidance

 

2. Need for support during all pre-IND phases of translational science

 

3.The lack of appropriate mechanisms and scientific review to facilitate reproducible pre-clinical research, including animal and humanized models, medical chemistry, and pharmacological toxicology

 

4.The similar lack of appropriate mechanisms and scientific review tailored to early phase or adaptive design clinical trials

 

Competitive, peer-reviewed investigator- initiated translational research might require centralized and coordinated NHLBI-supported Resources for efficiency and expert conduct.

Tags (Keywords associated with the idea)

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Compelling Question (CQ)

Details on the impact of addressing this CQ or CC :

During the past several decades there have been revolutionary changes in technology that have enabled an unprecedented understanding of basic biologic and pathobiologic phenomena. However, there has not been a concomitant increase in novel technologies to prevent, diagnose, and treat disease. While NHLBI funds thousands of R01 grants that support mechanistic research, only a small percentage moves the basic discoveries into the pre-clinical space, a critical step in fulfilling NHLBI’s mission. An analysis of the Division of Blood Diseases applications for the FY 2012, showed that while 32% (174/541) of the unsolicited applications (RO1, R21, PO1) had at least one early translational component, only 15% went beyond proof of concept and only 3% included early phase clinical trials. Patent filings, a main gateway to translation, are another way to estimate the translation of NHLBI discovery science. A survey of 90 NHLBI investigators, randomly selected across all three Divisions, found 76% of the investigators had no patent activity over a 10 year period and 12% accounted for 82% of all the patent activity. These data suggest that there may be barriers to investigators being able to realize the translational potential of promising discoveries beyond proof of concept. NHLBI needs an overarching, coordinated, and efficient plan to encourage and support investigators in their independent pursuit of translational research.

 

The creation of an integrated, facilitative programmatic pathway at NHLBI that would leverage existing NIH resources to ease the major barriers to translation of scientific discovery in the investigator community by:

•Providing skills development training and guidance for investigators wishing to do translational research

•Coordinating the support of all pre-IND phases of translational science

Facilitating reproducible pre-clinical research in animals, including animal and humanized models, medical chemistry, pharmacological toxicology

•Providing support for early phase or adaptive design clinical trials

•Ensuring scientific review that is available and tailored to all phases of investigator-initiated translational science

•Establishing a robust system for ongoing portfolio analysis and program evaluation as well as metrics for evaluating programmatic outcomes

 

The creation of an integrated, facilitative programmatic pathway at NHLBI that would leverage existing NIH resources to ease the major barriers to translation of scientific discovery in the investigator community by:

•Providing skills development training and guidance for investigators wishing to do translational research

•Coordinating the support of all pre-IND phases of translational science

Facilitating reproducible pre-clinical research in animals, including animal and humanized models, medical chemistry, pharmacological toxicology

•Providing support for early phase or adaptive design clinical trials

•Ensuring scientific review that is available and tailored to all phases of investigator-initiated translational science

•Establishing a robust system for ongoing portfolio analysis and program evaluation as well as metrics for evaluating programmatic outcomes

Feasibility and challenges of addressing this CQ or CC :

The overwhelming response to NHLBI translational initiatives suggests a critical mass of investigators willing and able to explore the translational potential of their discoveries. The barriers to the continuous receipt of translational applications outside of the RFA mechanism have been identified and the proposed model for a competitive peer-reviewed process that spans the early translational pipeline has already been successfully implemented in at least one other NIH Institute. Adopting this model, the pipeline, as envisioned, would mostly consist of facilitative PAs and Review would not require a major up front expenditure of NHLBI targeted funding.

Name of idea submitter and other team members who worked on this idea : NHLBI Staff

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Idea No. 88