Charge to the NINDS Advisory Panel for Clinical Research

Background on NINDS Strategic Planning

In 1999, the first National Institute of Neurological Disorders and Stroke (NINDS) strategic plan, "Neuroscience in the New Millennium" set broad scientific goals across the breadth of the NINDS mission. Building on this foundation, NINDS launched a new strategic planning effort in early 2007 that will combine forward-looking "blue sky" goals with focused, practical strategies to pursue the NINDS mission more effectively. The challenge for the NINDS is to balance basic, translational, clinical and disease-related research; seize immediate opportunities while nurturing the long-term advancement of neuroscience; and encourage research in opportune areas while also allowing for unexpected discoveries.

The NINDS has begun by developing blue sky goals and grand challenges in basic and clinical neuroscience for the next fifteen years. The Institute is now exploring how best to focus and prioritize its basic, translational, clinical and disease-focused research activities in order to realize this vision and pursue its mission more effectively. The NINDS has formed internal working groups and external advisory panels for each of these four themes. The advisory panels will be apprised of current NINDS activities and help the Institute set priorities for the next five to ten years. The NINDS will integrate the blue sky goals with the recommendations of the four panels and release a report in early 2009.

Purpose of the Advisory Panel for Clinical Research

The Advisory Panel for Clinical Research will review current NINDS investments and opportunities in clinical research (including but not limited to clinical trials) and offer general recommendations to the NINDS regarding future investments in this area and the best strategies to pursue them. The Panel and an internal NINDS working group will both work in parallel towards developing a series of questions about NINDS-supported clinical research programs , and data collected in response to these questions will drive the Panel's final recommendations. As a starting point, the panel should consider the following overarching questions:

• Has NINDS clinical research taken full advantage of the available scientific knowledge and human resources to advance the treatment of patients with neurological disorders? How might the Institute do better?
• Has NINDS' clinical research been as effective as it could be, given the opportunities and resources available?
• How can the NINDS more effectively improve the pace of clinical research advances and should it do so?
• What should the NINDS continue to do, and what should it do differently? In which new clinical research activities and opportunities should the NINDS invest?
• If you could transform the NINDS clinical research enterprise, how would you do so?

To address these overarching questions and others which they may wish to pose, Panel members are encouraged to pursue an incisive exploration of the Institute's activities in the area of clinical research, develop targeted/specific questions about its clinical portfolio, grant opportunities, and current review processes, and then provide specific advice on a broad range of issues, including but not limited to those relating to research priorities, environment, and process.

Examples of the types of specific questions that will drive the data collection, analysis, and recommendations related to the Institute's clinical research planning are provided below. However, this list is not all-inclusive, and Panel members are free to modify and expand this list through the course of their conference calls and in-person discussions. Given unlimited time and resources, the Institute would like to have all these questions answered; however, considering the time and budget constraints, we are relying on the panel members to tell us which amongst these (or any others) are realistically addressable and how they ought to be prioritized.

• Is NINDS clinical research addressing those questions that will have the greatest impact on reducing the burden of neurologic illness?
• Is the current balance of epidemiological, natural history studies and clinical trials supported by the NINDS appropriate?
• Is there an appropriate ratio of investigator-initiated research to studies that the NINDS could initiate and manage proactively? Should the institute plan and implement a clinical study from the "top down?" If yes, in what situations should this occur?
• When is it appropriate for the NINDS to fund research consortia or networks outside of a specific clinical trial? What is the appropriate balance of network vs. independent investigator run trials?
• When is it appropriate to engage the NINDS in a preliminary discussion of a specific clinical research application prior to submission?
• Is the NINDS taking full advantage of collaborative opportunities with other Institutes?
• Does the Institute's current clinical research portfolio reflect its commitment to its primary mission of reducing the burden of neurological disease, or is the NINDS taking on clinical research in this area that is outside of its core mission?
• Is the Institute doing all it should to ensure success of its clinical trials in the changing research environment (e.g., rising costs, pros and cons of relying on foreign sites, variations in IRBs, variations in contract practices)?
• Should the Institute be pushing forward technologies such as electronic medical records?
• What facilities and shared resources should the NINDS support to advance clinical research, e.g., brain banks, neuroimaging respositories, and bio-specimen repositories?
• What strategies should be considered for managing genome-wide association study resources across a broad range of neurologic disorders?
• What is the appropriate balance between resource commitment for research on optimizing the use of currently available treatments vs. research focused on developing new treatments?
• Is it possible to perform multiple inexpensive trials to answer the plethora of questions in clinical practice of neurology, neurosurgery and neuroradiology, and related disciplines? If it is possible, should the NINDS fund such studies?
• How should the NINDS best match the cost of trials with likely impact; should the Institute set different budget limits for trials of different types?

The Panel should focus on strategies and opportunities that cut across diseases, rather than disease-specific issues. The Panel should also think creatively and broadly but also pragmatically, considering ways that NINDS can best leverage available resources.

Advisory Panel Composition

The Advisory Panel for Clinical Research will consist of ~15- 20 members from the research community, the National Advisory Neurological Disorders and Stroke Council and other NIH Institutes or Centers.

Advisory Panel Activities

• The Advisory Panel for Clinical Research will work closely with NINDS program and policy staff members to identify data or other background materials that will help the Panel formulate its final recommendations to the Institute. The NINDS staff will serve as the Panel's liaisons to the Institute, providing data and any other support requested by the Panel.
• Panel discussions will occur via regular conference calls and at two one and a half-day meetings in the Washington, D.C. area. The first face-to-face meeting will take place in May 2008. The second will involve a final review of the data and discussion of recommendations, and is expected to occur in late 2008.
• The final product of the Panel activities will be a summary report and set of recommendations to be delivered to the NINDS in early 2009. The primary audience for this report will be the NINDS leadership, although the report will be made public. NINDS planning and communications staff will prepare a separate report for the general public that integrates the conclusions of the Advisory Panel for Clinical Research with those from three other strategic planning advisory panels.

Review all NINDS Strategic Planning Modules