Concept: NCCIH Clinical Trials Initiative for Intervention Development and Testing

Project Concept Review

Council Date: September 20, 2019

Program Officer: Wendy Weber, N.D., Ph.D., M.P.H.

Background

The National Center for Complementary and Integrative Health (NCCIH) is committed to the rigorous evaluation of the clinical effects of complementary and integrative approaches. Program staff previously presented to Council concepts for the development of initiatives to support clinical research in the areas of mind and body approaches and natural products in 2014 and 2015. Both of these presentations discussed the importance of early-stage clinical research to inform the design of fully powered efficacy, effectiveness, or pragmatic trials. As a result of the previous concepts, NCCIH issued several funding opportunity announcements (FOAs) to support the pipeline of clinical research studies from early-stage to multisite efficacy trials for natural products and mind and body interventions.

NCCIH has chosen to develop and issue specific funding opportunities for clinical trials, rather than allowing these studies to be submitted through the generic National Institutes of Health (NIH) parent R01 funding opportunity. This allows NCCIH to develop high-priority areas for clinical trials and develop specific review criteria to allow reviewers to score applications based on the critical elements of clinical trial applications. NCCIH has also used cooperative agreement mechanisms to support larger scale studies, as this allows for closer monitoring and NIH staff involvement in these awards, which typically are large investments.

Purpose of Proposed Program

The purpose of this proposed initiative is to encourage clinical trial applications along the full pipeline of intervention development and testing for efficacy and effectiveness. Clinical trials of complementary and integrative health interventions in areas that are high priority for NCCIH will be encouraged. An emphasis will be placed on the preliminary data needed at each stage of the research to justify the design aspects of the trial and demonstrate the feasibility of the intervention in the patient population to be studied before efficacy or effectiveness studies are conducted. When these preliminary data do not exist, investigators will be encouraged to use funding opportunities for early-stage clinical research.

NCCIH would like to use a cooperative agreement mechanism for some of the FOAs in this initiative to allow for NCCIH staff involvement and more careful follow up and monitoring of these larger scale studies. In some cases, NCCIH will use a phased award mechanism to allow for a planning period to ensure that all elements of trial startup are complete before funds are committed to the implementation phase of a large clinical trial.

Objectives

The objectives to be met by this concept include but are not limited to the following:

• Natural Product Clinical Trials
  • Support early-stage trials to understand the pharmacokinetics and pharmacodynamics of natural products.
  • Support early-stage trials to demonstrate the natural product has a reproducible impact on a biological signature relevant to a clinical condition.
  • Conduct studies to determine the appropriate dose and formulation of a product to maximize impact on the biological signature.
  • Conduct fully powered efficacy trials of natural products when the pilot data are promising. Efficacy trials will be designed to maximize generalizability and objectivity with a multisite design and an independent clinical coordinating center.
• Mind and Body Clinical Trials
  • Support early-stage trials to understand the feasibility and acceptability of the intervention.
  • Conduct studies to determine the appropriate dose and delivery methods of the intervention to maximize feasibility and acceptability.
  • Conduct studies to examine what components of an intervention are necessary and should be included in future efficacy or effectiveness trials.
  • Conduct fully powered efficacy, effectiveness, or pragmatic trials of mind and body interventions when the pilot data are promising. Fully powered trials will be designed to maximize generalizability and objectivity with a multisite design and an independent clinical coordinating center.

The funding opportunities developed from this initiative may use cooperative agreement mechanisms.