Priority 5: Translating Findings Into Interventions Through Clinical and Epidemiologic Studies

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Whereas the priority of Studying Molecular and Cellular Mechanisms of Health and Disease in Humans and Understanding the Resulting Phenotypes and Endotypes focuses on basic and preclinical research, this priority addresses the translation of discoveries into patient care or public health efforts. In accordance with FY 2024 recommendations from the NAMSAC Clinical Trials Working Group, NIAMS will emphasize studies with notable potential to advance clinical management and the development of guidelines related to diseases within the NIAMS mission that are not likely to be funded by industry. 

Consistent with the institute’s Conducting Research to Address Health Disparities priority, NIAMS-funded clinical and epidemiologic research should include efforts to identify and reduce health disparities and provide all Americans with access to clinical and epidemiologic studies and health care whenever possible. When designing clinical trials, investigators are encouraged to think beyond the traditional randomized clinical trial design that measures average outcomes between two or more groups of participants and instead consider designs that will answer questions regarding who, within the relevant population, would likely benefit more from (or be harmed by) an intervention.

Examples of efforts under this topic include the following.

Strategy 5.1: Creating New Resources for Clinical Research and Patient Care

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• Developing and validating tools such as patient-reported outcome measures that are responsive to change and effectively capture health-related quality of life measures among people with diseases covered by the institute’s mission. This could include:
  • Developing and validating tools to measure patient experiences, including the impact of pain and itch on daily activities and social functioning as well as the impact of social stigma due to factors such as pain or fatigue.
• Developing and validating measures of functional ability such as wearable devices that accurately assess how a person performs activities of daily living.
• Developing and testing device-based interventions for pain that can provide long-lasting and non-opioid-based relief.
• Adapting and improving methodologies and technologies that enable decentralized clinical trials.
• Advancing digital endpoints and digital health technologies to support clinical trials, advance therapeutic development, and improve patient care.
• Improving tools to enable at-home monitoring and biospecimen collection.
• Identifying best practices for measuring patient experience data to inform patient perspectives on unmet medical needs and the value of different therapeutic options.
• Designing and disseminating effective education materials for prospective study participants and their caregivers to increase engagement in research.
• Refining strategies to improve community engagement and community-based participatory research in arthritis, musculoskeletal, and skin studies to enhance the quality, outcomes, and value of the research questions being answered.
• Combining demographic, behavioral, clinical, and molecular data to redefine arthritis, musculoskeletal, and skin phenotypes and conditions. These new definitions could be used when designing clinical trials and selecting treatments.

Strategy 5.2: Improving Health for All

• Developing and testing innovative precision medicine approaches that integrate patients’ biology (including age and sex), social and environmental context, and behaviors. This could include:
  • Designing proactive interventions to prevent disease in people at risk for diseases and conditions relevant to the NIAMS mission.
• Determining effective care strategies and the optimal sequence of therapies in diseases of interest to NIAMS. This could include:
  • Combining medications and physical and rehabilitation interventions.
  • Integrating protocols that identify and treat co-occurring mental health conditions such as depression into treatment regimens.
• Understanding the impact of HIV infection or antiretroviral therapy on diseases of interest to NIAMS among people living with HIV. This could include:
  • Studying the prevalence, risk assessment, or management of these conditions.
• Identifying strategies, including the use of patient-reported information, to incorporate shared decision-making into clinical care for people who have diseases covered by the NIAMS mission.
• Developing and testing primary and secondary prevention strategies based on disease mechanisms in people who are at increased risk for conditions studied by NIAMS due to genetic, environmental, and other biologic markers. Care should be taken to include participants of a range of ages from diverse backgrounds to ensure the interventions are appropriate for everyone.
• Identifying cost-effective interventions to improve access to treatments and prevention strategies and improve high-value care.
• Developing and testing approaches for integrating sustainable evidence-based prevention strategies and treatments into health care and community settings.