NCCAM Priority Setting — Framework and Other Considerations

Introduction

Since its creation as an independent Center, the National Center for Complementary and Alternative Medicine (NCCAM) has twice developed strategic plans (2000-2004; 2005-2009) to guide the implementation of its complex research mission. As NCCAM now embarks on its third strategic planning process, it is particularly important to consider how the Center should seek to maximize the impact of its activities on the public and the public good across the broad and diverse field of complementary and alternative medicine (CAM) research (see "NCCAM's Mandate and Mission"). This paper provides background information germane to the general topic of research priorities for the field of CAM research. It also presents several issues that are intended to stimulate discussion and stakeholder input into developing a framework for NCCAM's future priority-setting processes.

Background

Two overarching research priorities of NCCAM's first decade have been to address 1) the relative paucity of foundational scientific information on the biological properties, safety, and efficacy of most CAM modalities and 2) the need for large clinical trials testing the efficacy and safety of CAM interventions frequently used by the public. These themes have driven the types of research programs and activities established by the Center.

The need for better foundational information has been addressed primarily through approaches aimed at fostering and supporting a robust stream of highly meritorious, peer-reviewed, traditional, investigator-initiated, research project grants across the broad range of topics relevant to the Center's mission. Within this context, NCCAM periodically has identified more specific research priorities and goals through various Funding Opportunity Announcements, Requests for Applications, Program Announcements, and Web site listings. In general, these initiatives have embodied an investigator-initiated, research project grant approach. Recent examples include Funding Opportunity Announcements "Mechanisms of Immune Modulation," "Translational Tools for Clinical Studies of CAM Interventions," and "CAM Approaches in the Management of HIV Disease and Its Complications."

NCCAM has used various targeted approaches to support the conduct of large, often multicenter, clinical trials. These have been modeled on similar activities of other National Institutes of Health (NIH) institutes and centers (ICs). Most have been funded through specifically solicited grants or cooperative agreements and have been carried out in collaboration with NIH IC partners. While these studies have attracted very substantial public and scientific attention, interest, and sometimes criticism over the years, they have accounted for only 15 to 20 percent of the Center's overall research investment.

In all cases, priorities have been guided by extensive expert input obtained through scientific workshops and forums, networking with investigators and other stakeholders, the advice and guidance of the Center's National Advisory Council, and reports such as those issued in 2002 by a White House Commission[1] or in 2005 by the Institute of Medicine.[2]

Together, these approaches have resulted in the funding of more than 2,200 individual research projects and the publication of more than 3,300 scientific articles in peer-reviewed journals. This work has contributed enormously to the body of scientific information available concerning the biological effects, efficacy, safety, and potential promise of many CAM interventions employed for many different indications. It also has nurtured the growth of research capacity as the CAM research community established collaborations and as fundamental research tools and approaches were explored and developed.

Shaping the Investigator-Initiated, Research Grant Portfolio

A relatively broad, nontargeted, investigator-initiated, research project grant approach was quite appropriate when NCCAM was a new entity, and the overarching goal was to develop foundational evidence and multidisciplinary research capacity where little existed. In looking forward, it is important to take stock of the current state of foundational knowledge about CAM and CAM practices, much of it a direct result of this body of work. Some illustrative examples of promising leads include insight gained into the:

Given these and other such opportunities of promise and potential, at this juncture in its history NCCAM suggests that the public interest would be best served by a greater degree of strategic shaping of the investigator-initiated, research project grant stream. The goal would be to better focus investment of available and finite resources on areas of research and development that offer the greatest potential of health benefit for the public or advancement of the general state of health and biomedical research.

The Need for Specific, Targeted Research and Development Initiatives

NCCAM's "unsolicited," investigator-initiated, research project grant stream is increasingly robust. The Center has the lowest funding success rate of all NIH ICs, and an increasing proportion of NCCAM's research grant portfolio is devoted to R01 (investigator-initiated) research grants reviewed by NIH's Center for Scientific Review. However, this approach cannot be relied on to ensure that major gaps in knowledge are filled, or that development of a definitive evidence base regarding specific, important, and promising research leads or opportunities are pursued with goal-oriented clarity, timeliness, and efficiency.

This matter is not unique to NCCAM or CAM research. In fact, most NIH ICs, particularly those with substantial clinical responsibilities, devote a significant fraction of their overall extramural research portfolio to targeted research and development activities. Naturally, there is a tension between the resources available for "unsolicited," investigator-initiated research project grants on the one hand, and targeted, solicited programs on the other.

Criteria/Framework for Priority Setting

At this juncture in its history, NCCAM suggests that the public and the field of CAM research will be optimally served by:

In order to pursue this approach, it is important to first consider carefully the framework, criteria, and processes that NCCAM would use to establish priorities. This framework would guide shaping of both the general investigator-initiated research project grant stream and the identification and pursuit of areas of research activity that require specific, targeted research and development program approaches.

With respect to a potential framework for setting research priorities that are in alignment with the Center's mission and legislative mandates, four factors seem to be of paramount importance. These factors represent independent criteria for high-priority programs, initiatives, or projects, and they are to be considered and weighed in relative and cumulative importance on a case-by-case basis.

For current purposes, the scope of each of the four factors is elucidated by a series of questions.

  1. Scientific Promise: Is there a reasonable body of evidence indicating that the proposed research project or program has the potential to make important contributions to available options for treatment of troubling or prevalent health conditions or symptoms, approaches to pursuit of better health and well-being, or currently available research methods or technology?
  2. Extent and Nature of Practice and Use: Is the CAM intervention in widespread use by practitioners and/or the general public? Does the research address an urgent public health concern regarding efficacy, safety, or lack thereof? Will the research project or program provide important and needed information about the frequency and nature of use of CAM by practitioners or the general public?
  3. Amenability to Rigorous Scientific Inquiry: Is the research or development question amenable to rigorous scientific investigation, given available methods, tools, and technologies? Will the project or program develop better and needed research tools and technologies? Are potential approaches feasible and scientifically plausible, and do they lend themselves to rigorous quality control?
  4. Potential to Change Health Practices: Is there substantial likelihood that the results of the project or program will lead to changes in the health or health care practices of consumers or health care providers?

This framework is primarily intended to assist NCCAM and its National Advisory Council in:

In practice, NCCAM staff would continue to rely heavily on information and expert input obtained through scientific workshops and forums; networking with investigators, practitioners, and other stakeholders; surveys and other interactions with the public; and the advice and guidance of its National Advisory Council. Finally, the framework is not intended to be used to exclude the submission or eventual funding of individual investigator-initiated research project grants.

INVITATION: PUBLIC COMMENT

NCCAM invites comment from its stakeholder community and other interested parties on this summary document. Of particular interest are perspectives on:


[1] White House Commission on Complementary and Alternative Medicine Policy. Final Report. Washington, DC: White House Commission on Complementary and Alternative Medicine Policy; 2002. NIH publication no. 03-5411. Available at: http://www.whccamp.hhs.gov.

[2] Institute of Medicine. Complementary and Alternative Medicine in the United States. Washington, DC: National Academies Press; 2005.