NIAMS Clinical Trials
The purpose of this session is to explore and analyze approaches that NIAMS can use to more effectively identify and manage high-quality clinical trials.
Background/Relevance to NIAMS Mission
Clinical research is a significant NIAMS investment aimed at answering critical questions about a particular disease or disease process. It is essential for identifying promising new therapeutics with the potential to improve health and clinical care. The majority of NIAMS-supported clinical trials are small (i.e., their sample sizes range from "a few" to "a few hundred" participants), early phase studies and that would not be done in the private sector. Most are investigator-initiated, are supported by grants and reviewed by the Center for Scientific Review (CSR) Study Sections. Currently, there is limited NIAMS involvement in the identification and prioritization of clinical trials before submission. Increasingly, NIH Institutes are establishing external advisory groups, centers, and networks to assist them in setting priorities for supporting clinical trials. Such groups provide a platform for collaborations and identifying scientific and research design trends. Examples include TrialNeti, the Wellstone Muscular Dystrophy Research Centersii, the Autoimmunity Centers of Excellenceiii, and the Rare Disease Clinical Research Networkiv. During the session, we will discuss the advantages and disadvantages of some of these approaches to improve the quality and outcomes of NIH-supported clinical trials.
The randomized control trial (RCT) is accepted as the most convincing and reliable study design and is the approach most commonly used in NIAMS-supported clinical trials. Increasingly, other designs are being developed to overcome the shortcomings of the RCTs, in particular for Phase I/II trials and for trials in rare diseases. Several designs in the context of RCTs are also underway, to maximize recruitment and offer all participants exposure to the experimental agent. However, the lack of consensus about criteria for the selection of the best study design and outcomes often cause significant delays in the initiation of studies or result in studies that are methodologically flawed. We will discuss some of these issues and potential approaches the NIAMS can undertake to advance this aspect of clinical trials research.
Clinical trials are monitored more closely than other types of research grants. Each trial with more than minimal risk is monitored by either a Data and Safety Monitoring Board (DSMB) or a Safety Officer. The boards and safety officers monitor data quality and participant safety and provide guidance to investigators about strategies to improve study progress. In addition, NIAMS staff and a clinical research support contractor may provide oversight and support activities to review implementation procedures and remove recruitment barriers. All of these monitoring methods, however, are implemented after peer review or after a study has been ongoing for many months; therefore, they do not serve as the best solution for proactive resolution of design flaws and study progress issues that can increase costs and diminish the quality of a study and the usefulness of its results.
Expected Session Outcomes
During the discussion, we hope to identify ways to enhance clinical trials research opportunities through exploring:
- Approaches to identify key clinical trials opportunities.
- Approaches to identify key stakeholders and to enhance NIAMS interactions with academic and private sector clinical trial scientific communities.
- The potential value and drawbacks of an NIAMS-convened workshop, roundtable, or advisory board on clinical trials with participation by expert advisors outside the NIH.
- Various models for priority setting.
- The potential value and drawbacks associated with the various grant and contract mechanisms to support clinical trials.
- A framework of activities to enhance the post-award administration of NIAMS-supported clinical trials.
- What can the NIAMS do to ensure that it has an effective process to solicit from the community the best ideas for high-priority clinical trials?
- What are the goals of the NIAMS clinical trials portfolio?
- How can staff evaluate the impact of NIAMS-supported clinical trials? What are the most meaningful or predictive performance indicators?
- What can the NIAMS do to ensure the productivity and success of a clinical trial?
- Are certain funding mechanisms more effective than others?
- How do other NIH Institutes identify, select, and prioritize their clinical trials?
iType 1 Diabetes TrialNet, http://www.diabetestrialnet.org. Accessed February 19, 2009.
iiSenator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers, http://www.wellstonemdcenters.nih.gov/. Accessed February 3, 2009.
iiiThe Autoimmunity Centers of Excellence, http://www.autoimmunitycenters.org. Accessed February 18, 2009.
ivThe Rare Diseases Clinical Research Network (RDCRN), http://rarediseasesnetwork.epi.usf.edu/. Accessed February 18, 2009. [url changed May 25, 2011]