Open Call: Improving Methods for Conducting Patient-Centered Clinical Effectiveness Research (US)

Priority Areas

• Methods Related to Ethical and Human Subjects Protections (HSP) Issues in Patient-Centered CER: PCORI is interested in funding projects to address important ethical and HSP issues related to the conduct of patient-centered comparative clinical effectiveness research. The following topics are priorities for this area:
  • Measures Recruitment and informed consent processes for participation in clinical research in the context of randomized controlled trials (RCTs), cluster RCTs, pragmatic trials, adaptive or platform trials, decentralized clinical trials, observational studies (including those conducted in learning health care system settings), and natural experiments. Applications examining alterations of consent are welcome
  • Assessing the concept of minimal risk in clinical research (including clinical trials)
  • Review and monitoring activities, including protocol adherence and adjudication of study outcomes.
• Methods to Improve Study Design: PCORI is interested in funding projects that foster improvements in clinical research study designs to address patient-centered CER questions. The following topics are priorities:
  • Methods to improve the design and conduct of Sequential Multiple Assignment Randomized Trials (SMART)
  • Methods to improve the design and conduct of decentralized clinical trials (DCTs)
  • Methods to manage adaptation of treatment strategies while minimizing threats to internal validity in studies of complex interventions
  • Methods to improve the design and conduct of patient-centered CER studies in circumstances limiting the use of RCTs
  • Methods to improve the design and conduct of patient-centered.
• Methods to Support Data Research Networks: PCORI is interested in funding projects that offer generalizable and scalable insights into multisite patient-centered CER using data research networks. Applications that address Data Research Networks must address one or more of the following priorities for this area:
  • Methods to improve distributed analyses in data research networks
  • Methods to improve data quality in data research networks
  • Methods to promote interoperability of data science methods and data across data research networks
  • Methods to improve geocoding and address challenges of geospatial data integration.
• Methods to Improve Use of Artificial Intelligence and Machine Learning in Clinical Research: PCORI is interested in funding methodological research that seeks to improve how massive amounts of data from a variety of electronic health data sources1 can be appropriately analyzed and integrated into clinical care and health care delivery systems and/or used to facilitate population health models and social determinants of health (SDoH) analyses to help support patient-centered CER. The following topics are priorities:
  • Approaches to modeling longitudinal electronic health data, patient-generated health data, or other sources of real-world health-related data
  • Approaches to improving the integration and analysis of rare event or outcome data
  • Approaches to improving multi-modal data streams (e.g., extracting and incorporating information from images, audio/video, patient reported/generated data from sensor/wearable/personal devices). Methods to assess and improve data quality (e.g., data provenance, bias and fairness, validity and reproducibility of datasets for model
    training)
  • Methods for causal inference machine learning to estimate heterogeneous treatment effects
  • Developing experimental methods, tests, or other approaches to assess the validity and/or safety of AI/ML models (e.g., large language models)
  • Approaches to assessing robustness and resilience of algorithms to withstand changes in patient demographics, data sources, and clinical contexts
  • Approaches to increasing the transparency of algorithms and their outputs (e.g., co-designing approaches with engaged research partners (e.g., patients, families, etc.), methods for increasing the explainability of ‘black box’ approaches, etc.)
  • Approaches that support innovations in the identification, recruitment, and retention of study participants.

Funding Information

• Funds Available up to approximately: $12 million
• Maximum Project Budget: $750,000
• Maximum Research Project Period: 3 years

Eligibility Criteria

• In general, applications for the conduct of research and management of funding may be submitted by appropriate academic research, private sector research, or study-conducting entities. This may include, among others, agencies and instrumentalities of the federal government, nonprofit and for-profit research organizations, and colleges and universities.
• Foreign organizations and nondomestic components of U.S. organizations must provide a thorough and thoughtful justification for the research’s ability to benefit the U.S. healthcare system and that the engagement plans include U.S. patients and stakeholders and are relevant to the U.S. healthcare system.

Ineligibility Criteria

• Individuals are not permitted to apply.

Review Criteria

• Study identifies critical methodological gap(s) in patient-centered CER
• Potential for the study to improve patient-centered CER methods
• Scientific merit (research design, analysis, and outcomes)
• Investigator(s) and environment
• Patient centeredness
• Patient and stakeholder engagement.

For more information, visit PCORI.