What is the Future of NIH funded Clinical Research in IBD?

Transcription

1 IBD Clinical Trials Workshop What is the Future of NIH funded Clinical Research in IBD? Stephen P. James, M.D. January 13-14, 2017

2 Future?

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4 FY 2015 NIH Awards for IBD related research 338 Awards $128 Million Major sources (no. awards): NIDDK: 59% NIAID: 13% NCI: 8% NIGMS: 4% OD 6% 12 0thers of the 27 IC s 10%

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6 Each with a different: mission & priorities budget funding strategy NIGMS International Center

7 Division of Digestive Diseases & Nutrition Mission: Support basic, translational and clinical research Disease Areas Alimentary GI tract Liver Exocrine Pancreas Clinical Obesity Research Nutrition Research

8 NIDDK DDN Funding Mechanisms Research project grants Research project grant (R01) Pilot/feasibility (R21) Program grants (P01) Large projects (R24, RC2) Cooperative agreements (U01s) IBD Genetics Consortium MERIT-UC trial Protect-UC trial Intestinal stem cell consortium Small business grants R41, R42, R43, R44 Centers (P30, infrastructure) Conferences/workshops (R13) Career development Post doc PhD (K01) Physician basic/clinical (K08, K23) Faculty Transition (K99/R00) Fellowships Individual F s Institutional T32 NIDDK DNA, biospecimen, data repository

9 Pathogenesis of IBD Host genes, epigenetics CNS, Autonomic NS, ENS Environment Adaptive immunity IBD Nutrient metabolism Innate immunity Barrier, repair Microbiome

10 Major theme areas for NIDDK support Basic science Epithelial biology Transport/barrier Motility, neurobiology Immunity and Inflammation Stem cells/repair Microbiome/hostmicrobiome interaction Genetics Nutrition, metabolomics of IBD Research Patient-based Basic science applied to humans/samples Human genetics Biomarkers Clinical trials P & F Cohort studies RCT s Epidemiology Health care delivery/qi

11 Pre-Clinical Translational Research

12 DDN Human subjects research Approximately ½ of funded applications Approximately ½ of award $

13 NIDDK Clinical Trials Generally meant to complement industry trials Early stage, proof of concept Trials that industry is unlikely to sponsor Emphasis on leveraging trial to further fundamental knowledge about disease: ancillary studies, biospecimens Partnership with Foundations, Industry permitted: clinical trial agreements, CRADA

14 NIH: Broad definition of clinical trial NIH definition: A research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes. Definition needs further refinement and clarification

15 Policy on FOAs for Clinical Trials Not OD Policy Statement NIH will require that all applications involving one or more clinical trials be submitted through a Funding Opportunity Announcement (FOA) specifically designed for clinical trials. This means that the NIH will no longer accept clinical trial applications through "parent" FOA announcements or through other FOAs that are not specifically designed to accept clinical trials. The purpose of this policy is to improve our ability to identify proposed clinical trials, ensure that key pieces of trial-specific information are submitted with each application, and uniformly apply trial-specific review criteria 1. Background Over the past few years, the NIH began taking steps to enhance its management and oversight of clinical trials. One recommendation NIH is implementing is the use of clinical trials-specific FOAs. Some NIH Institutes and Centers already require clinical trials to be submitted in this manner, and this will now be implemented across the NIH. The NIH recognizes that there is great breadth and diversity to the types and topics for clinical trials, and the Institutes and Centers will retain the flexibility to determine how best to design funding opportunities for clinical trials for their communities. Scope and Applicability This FOA policy will apply to all applications involving one or more clinical trials (see definition), including applications that involve a combination of studies that are clinical trials as well as studies that are not. These latter hybrid FOAs include applications that will involve a combination of trial and non trial aims. These and other types of trial applications will also be submitted to clinical trial-specific FOAs if the combination studies involve a NIH defined clinical trial. Effective Date The target effective date for the NIH Policy on Funding Opportunity Announcement (FOA) for Clinical Trials is September 27, All applications with receipt dates on or after the effective date with plans to conduct clinical trials must be submitted in response to a clinical trial-specific FOA. After that date, applications planning a clinical trial that are submitted to a non-clinical trial FOA will be returned without review.

16 NIH Clinical Trials Application forms still under development Main impact likely to be on single or two center clinical trials using an R01 application. Requirements for multi-center clinical trials will not change for IC specific PARs and RFAs.

17 NIDDK U34/U01 NIDDK does not accept R01 applications for multi-center clinical trials U34 planning grant: PAR U01 multi-center clinical center cooperative agreement PAR

18 NIDDK: Multi-center clinical trial application additional requirements Attachments: 1. Clinical protocol, approved by DSMB 2. Statistical Analysis Plan 3. Data and Safety Monitoring Plan 4. Milestone plan 5. Planning documents: Manual of operations, case report forms, database, regulatory documents, etc.

19 IBD examples Protect UC MERIT UC

20 NIDDK Exploratory Clinical Trials for PAR Small Business (R44) Extensive clinical trial application requirements modeled on existing U34/U01 process

21 R21s PA : Pilot and Feasibility Clinical and Translational Research in Digestive Diseases and Nutrition PA : Secondary Analyses in Obesity, Diabetes and Digestive and Kidney Diseases

22 Policy on Good Clinical Practice Training for NIH Awardees Involved in NIH-funded Clinical Trials Notice Number: NOT-OD Policy Statement This policy establishes the expectation that all NIH-funded investigators and staff who are involved in the conduct, oversight, or management of clinical trials should be trained in Good Clinical Practice (GCP), consistent with principles of the International Conference on Harmonization (ICH) E6 (R2). 1 Effective Date This policy is effective as of January 1, 2017

23 Single IRB for multi-site research NOT OD This Notice informs the research community that NIH is extending the effective date of the NIH Policy on the Use of a Single Institutional Review Board for Multi-Site Research to September 25, 2017.

24 NIDDK DDN Staff Stephen James, Director Jessica Lee, Deputy Director Alimentary and pancreatic diseases Frank Hamilton, Jose Serrano, Dana Andersen, Jill Carrington, Patricia Greenwell, Peter Perrin Genetics, genomics, microbiology, microbiome, metabolomics Bob Karp, Peter Perrin, Padma Maruvada Liver and hepatobiliary diseases Jay Hoofnagle, Ed Doo, Averell Sherker, Bonnie Burgess-Beusse, Jose Serrano Obesity prevention and treatment Sue Yanovski, Mary Evans, Stavroula Osganian, Bob Kuczmarski Nutrition Christopher Lynch, Padma Maruvada, Mary Evans Epidemiology, clinical trials Aynur Unalp-Arida Clinical trial support Rebecca Torrance, Sherry Hall, Rebekah Van Raaphorst Small business programs Chris Densmore Career, T and F programs David Saslowsky, Chis Densmore Program Analysts Lauren Meskill, Dee Gamliel

25 NIDDK Central support NIDDK DEA NIDDK Review, John Connaughton NIDDK Grants Management, Bob Pike NIDDK OD Office of obesity research Office of Nutrition research Biostatistics Regulatory affairs Office of Science Policy Office of Communications and Public Liaison Central Repository Office of Technology Transfer Budget office

26 Questions?