Priorities for Strategy 3.3
Updated: January 2019
Test interventions for effectiveness in community practice settings
Effectiveness research aims to generate information for patients, clinicians, and policy makers regarding which interventions are most effective, for which patients, under given circumstances in real-world settings. Effectiveness research is most useful for informing practice or policy decisions when it addresses a condition that has substantial public health significance in terms of prevalence and burden; when the practical benefit of the intervention can be justified in terms of its estimated effect size compared with already available approaches; when it is conducted in diverse, representative populations; when it tests interventions that are potentially scalable and could be disseminated into current practice; and, when it assesses a broad array of stakeholder-relevant outcomes.
Given the resources necessary to launch large-scale trials and conduct intervention research in community/practice settings, studies must be justified in terms of potential impact, use innovative platforms and designs, and advance treatment personalization. To be responsive to this objective, large-scale effectiveness studies should be designed to detect substantial gains and justify the potential impact of the proposed intervention/services models on practice and public health. In addition, applications to test the impact of interventions on clinical endpoints without assessing whether the intervention engages the targets presumed to underlie clinical benefit, or to test preventive-, therapeutic-, or services-interventions with limited potential for broad implementation in community practice settings will not be considered responsive. Finally, applications to test on-patent medications without demonstrated superiority over off-patent medications or applications that involve adaptions of currently available treatments for new subpopulations without strong empirical justification will not be considered responsive.
- Develop and test bundled intervention components (each validated individually in prior research) that have the greatest impact on patients’ lives and functioning.
Priority areas include:
- Performing effectiveness studies in community practice settings that systematically examine heterogeneity in representative patient populations, of diverse racial/ethnic/gender groups and across ages – including standardized collection of core data to promote data integration, sharing, and pooled analysis – in the service of identifying mediators/moderators and informing more prescriptive approaches.
- Developing and testing theory-based, scalable preventive/early interventions that are designed to intervene proximally on shared modifiable risk and protective factors (e.g., target mechanisms relevant to self-regulation), with the goal of changing children’s life trajectories and reducing distal risk for multiple mental health disorders (e.g., depression, anxiety, conduct disorder). NIMH encourages culturally and developmentally sensitive approaches (e.g., interventions that target sensitive periods or key transitions) that can be sustainably implemented to prevent or forestall a broad array of mental illnesses.
- Testing modular or stepped-care approaches for matching intervention intensity and components to illness severity and specific functional impairments.
- Refining and testing approaches for augmenting, combining, or sequencing psychosocial and/or pharmacological interventions to optimize effectiveness and safety while minimizing unnecessary or off-label use of psychotropic medications among children and adolescents (e.g., stepped care or combination strategies to reduce unnecessary polypharmacy, approaches to minimize dose or duration of pharmacotherapy).
- Developing and testing individualized strategies targeted to specific functional impairments or developmental stages or to those individuals most at risk for relapse/recurrence to maximize the chances of complete recovery and sustained remission.
- Developing strategies for managing chronic disorders over time, including mental and clinical comorbidities, and for keeping people well following acute episodes.
- Together with key stakeholders, including patient, provider, payer, and other research funding groups, conduct efficient pragmatic trials that employ new tools to rapidly identify, engage, assess, and follow participants in the context of routine care.
Priority areas include:
- Conducting trials that leverage practice-based research and research investments, such as comparative effectiveness networks funded via the Patient-Centered Outcomes Research Initiative (PCORI) and the NIMH-sponsored Mental Health Research Network (MHRN) or that utilize other existing infrastructure to increase the efficiency and diversity of participant recruitment and to facilitate the collection of mediator/moderator measures, longer-term follow-up data, and broader, stakeholder-relevant outcomes.
- Conducting effectiveness studies that go beyond examining the effect of interventions on symptomatic or functional outcomes and addressing questions regarding how patient-, provider-, community-, and organizational-level factors impact clinical outcomes, as well as the implementation and scalability of research-generated interventions.
- Conducting effectiveness trials that incorporate measures of putative mechanisms to confirm whether mechanisms of action identified in efficacy studies are operative when evidence-based interventions are transported to community practice settings.
- Conducting deployment-focused effectiveness trials across diverse settings (e.g., rural, urban, and non-specialty settings including primary care, schools) in order to identify setting characteristics (e.g., workforce capacity, case mix) that impact the delivery of research-supported interventions and to test strategies that can be used to support sustained effectiveness and quality of implementation in settings with limited resources.
- Enhance the practical relevance of effectiveness research, taking into account how patient-, provider-, and organizational-level factors impact the outcomes of interventions in practice settings.
Priority areas include:
- Developing and testing adaptations of existing approaches for new indications (i.e., to address mental illnesses for which efficacious interventions are lacking) and to interrogate hypothesized therapeutic change mechanisms, consistent with NIMH’s experimental therapeutics emphasis.
- Developing and testing adaptations or augmentations of evidence-based interventions, when research suggests that a moderator or negative prognostic factor can be targeted to improve response substantially and personalize treatment for a readily identifiable refractory subgroup.
- Conducting deployment-focused research to improve the fit of evidence-based interventions and optimize their clinical impact for use in representative community/practice settings, especially studies that systematically take into account patient-, provider-, and organizational-level factors that impact the transportability of interventions.
- Testing the utility of novel applications of technology that could generalize across indications, target populations, and operating platforms, for facilitating the delivery of research supported strategies in a manner that both enhances the reach of evidence-based interventions and boosts their therapeutic value over and beyond standard delivery approaches (see the NAMHC Workgroup on Behavioral and Social Science Research Report: Opportunities and Challenges of Developing Information Technologies on Behavioral and Social Science Clinical Research and the Notice: NIMH High-Priority Areas for Research on Digital Health Technology to Advance Assessment, Detection, Prevention, Treatment, and Delivery of Services for Mental Health Conditions (NOT-MH-18-031)).