Notice Number: NOT-TR-17-004

Key Dates
Release Date: October 24, 2016

Issued by
National Center for Advancing Translational Sciences (NCATS)

Purpose

The purpose of this Notice is make modifications for PAR-15-172 " Collaborative Innovation Award, Clinical and Translational Science Award (CTSA) Program (U01)" for the March 9, 2017, and subsequent due dates, and expand the list of high priority translational research opportunities to novel clinical trials of drugs that target shared molecular etiologies underlying multiple diseases.

These changes do not apply to applications submitted for the November 10, 2016, due date.

Part 2. Section I. Funding Opportunity Description

Current Language:

Specific Objectives

Some examples of such opportunities include but are not limited to:

• Development and implementation of innovative community engagement methods and technologies that demonstrably increase the efficiency and effectiveness of intervention development and deployment, and measurement of their effects on improving health outcomes.  Community engagement should be defined broadly to include local and distributed, physical and virtual communities.  Research aimed at engaging minority, vulnerable, or other understudied populations in translational research is one area of particular interest.
• Improving the consent process,  for example simplifying and reducing the length of the consent forms without sacrificing content; ensuring that the consent form is commensurate with potential participants’ needs and is culturally sensitive; making the consent process more user-friendly through the use of visual aids or recorded video; in multi-site research, providing similar information across investigators and sites.
• Educating and training the translational research workforce through collaborative initiatives across multiple CTSA hubs that leverage local strengths, using innovative features such as shared, online resources, individualized training with focus on competencies rather than degrees, training in multi-disciplinary team science, regulatory science, entrepreneurship and experiential learning experiences, for example through externships in industry, regulatory agencies, or nonprofit organizations.
• Developing mechanisms for CTSA-catalyzed networks to identify and fast-track particularly promising translational research projects, thereby reducing the time required for intervention development or demonstration of unfeasibility.
• Adapting technology and methodology that are shown to be successful in other domains to address challenges in clinical and translational research.
• Fostering innovative multi-disciplinary collaborations that bring together new types of teams to solve particular clinical and translational research problems.
• Innovative clinical research studies or trials that use mobile technology and web- or phone- based communication to obtain information such as patient reported outcomes and remote biological measures. 
• Innovative clinical research designs, such as adaptive designs, serial “n of 1” design, or other approaches to small populations, which could overcome some of the challenges in evaluating interventions.
• Innovative approaches to the implementation of precision medicine
• Innovative methodologies that address translational research roadblocks specific to pediatric, geriatric, or other populations throughout the lifespan - e.g., new ways to develop outcome measures or validate biomarkers relevant to those populations, and enhancing lifestage-specific investigator training. 

Revised Language:
The italicized text is added to the end of this section.

Specific Objectives

Some examples of such opportunities include but are not limited to:

• Development and implementation of innovative community engagement methods and technologies that demonstrably increase the efficiency and effectiveness of intervention development and deployment, and measurement of their effects on improving health outcomes.  Community engagement should be defined broadly to include local and distributed, physical and virtual communities.  Research aimed at engaging minority, vulnerable, or other understudied populations in translational research is one area of particular interest.
• Improving the consent process,  for example simplifying and reducing the length of the consent forms without sacrificing content; ensuring that the consent form is commensurate with potential participants’ needs and is culturally sensitive; making the consent process more user-friendly through the use of visual aids or recorded video; in multi-site research, providing similar information across investigators and sites.
• Educating and training the translational research workforce through collaborative initiatives across multiple CTSA hubs that leverage local strengths, using innovative features such as shared, online resources, individualized training with focus on competencies rather than degrees, training in multi-disciplinary team science, regulatory science, entrepreneurship and experiential learning experiences, for example through externships in industry, regulatory agencies, or nonprofit organizations.
• Developing mechanisms for CTSA-catalyzed networks to identify and fast-track particularly promising translational research projects, thereby reducing the time required for intervention development or demonstration of unfeasibility.
• Adapting technology and methodology that are shown to be successful in other domains to address challenges in clinical and translational research.
• Fostering innovative multi-disciplinary collaborations that bring together new types of teams to solve particular clinical and translational research problems.
• Innovative clinical research studies or trials that use mobile technology and web- or phone- based communication to obtain information such as patient reported outcomes and remote biological measures. 
• Innovative clinical research designs, such as adaptive designs, serial “n of 1” design, or other approaches to small populations, which could overcome some of the challenges in evaluating interventions.
• Innovative approaches to the implementation of precision medicine
• Innovative methodologies that address translational research roadblocks specific to pediatric, geriatric, or other populations throughout the lifespan - e.g., new ways to develop outcome measures or validate biomarkers relevant to those populations, and enhancing lifestage-specific investigator training. 

PAR-15-172 supports applications for innovative collaborative investigations (involving three or more CTSA sites) into improvements of the methods of translational research, at any step in the translational spectrum (T1-T4). It is anticipated that the combined effort of three or more CTSA hubs in flexible networks will substantially enhance the effectiveness of the CTSA consortium to address high priority translational research questions. PAR-15-172 therefore aims to support innovative and collaborative experimental translational research projects carried out in the CTSA consortium. Below is an additional high priority translational research area suitable for this FOA:

Clinical trials of drugs targeting shared molecular etiologies underlying multiple diseases. Genomically driven “basket” trials are increasingly used for drugs that target molecular defects common to different cancer types. NCATS is interested in supporting clinical trials in which this approach is applied to diseases other than cancer. The essential feature of such trials is that patient enrollment is based on the underlying molecular etiology, rather than clinical features of disease. To maximize the translational impact of the CTSA program, NCATS encourages applications that involve laboratory scientists with expertise in disease biology/molecular pathways collaborating with clinicians. NCATS will give priority to applications for trials with rare diseases; examples of shared molecular etiologies in rare disease include but are not limited to: protein misfolding, premature termination codons, defects in RNA splicing, epigenetic dysregulation, or defects in molecular signaling pathways (e.g. MTOR, RAS, TGF-beta). Drugs used in the proposed trials must have an unequivocal mechanism of action that targets a shared molecular etiology. Leveraging partnerships with patient groups and industry is encouraged. Trials of oncology or infectious disease drugs will not be accepted, as this approach is established in those areas.

All other aspects of this FOA remain the same.

Inquiries

Please direct all inquiries to:

Philip J. Brooks, Ph.D.
National Center for Advancing Translational Sciences (NCATS)
Telephone: 301 443-0513
Email: [email protected]