Session Topic: Clinical Trials Portfolio Analysis and NIAMS Goals for Clinical Research
The NIAMS is committed to improving the health of patients with rheumatic, musculoskeletal, and skin diseases by supporting investigator-initiated clinical trials that lead to better clinical outcomes. The NIAMS' goal is to identify and fund trials that are timely, informative and will lead to improvements in clinical practice for the prevention, diagnosis, and treatment of diseases and disorders within the Institute’s mission areas.
NIAMS expanded its clinical trials portfolio during the late 1990s, when Congress began doubling the NIH budget. Evaluation of studies funded during that period revealed that an initial period of support to allow investigators to complete the required planning and administrative procedures would make the studies more efficient.
In response, NIAMS recently instituted a two-phase clinical trials award mechanism. Investigators are expected to apply for and receive a NIAMS Clinical Trial Planning Grant (U34) prior to applying for the UM1/U01 to implement a trial, except when planning and administrative tasks for the trial have been completed through a previous U34, R34 or other means. The Institute also established a standing study section to evaluate its clinical trials-related applications.
While these and other changes should improve the management of costs associated with trials and could improve the efficiency of the trials through more thorough planning phases, it is unclear whether they will improve the impact that NIAMS-funded studies have on patient care and public health. This retreat session is a part of the ongoing discussion about strategies to improve the return on the NIAMS’ clinical trial investments.
The NIAMS portfolio analysis and the following points will provide a framework for the retreat session, next steps and subsequent discussions:
- What is the distribution of trials by disease area and type of intervention? Are there over- or under-represented areas in the NIAMS portfolio? In the aggregate, is the NIAMS investing in a portfolio that enhances the knowledge base to treat, diagnose, and prevent arthritis and musculoskeletal and skin conditions?
- How do we define the potential impact of ongoing or new studies? What are the most meaningful measures of success or predictive performance indicators? Are there models we can adopt that would enable us to identify areas of high priority for future research (e.g., panel of experts)?
- Based on the preliminary evaluation of the FY2012 Pilot and Feasibility Clinical Research Grants applications and awards, are we providing the best opportunities for proof of concept studies that could lead to future clinical trials that make an impact in the field? What changes should be made to the program?
- How should NIAMS work with organizations to identify priorities or to organize potential trials? What advice should NIAMS give the leadership of such organizations? How could these partnerships expand into co-funded studies?
- What is the Institute’s role in disseminating results within the research community? Among practitioners? To the general public? As many of the studies we funded in the late 1990s are finishing, we are ideally positioned to think about how we can disseminate results.