PCORI's Pragmatic Clinical Studies funding announcements to date: Spring 2014 | Fall 2014 | Winter 2015 | Spring 2015 | Cycle 2 2016 | Cycle 3 2016 | Cycle 1 2017 | Cycle 2 2017 | Cycle 3 2017 | Cycle 1 2018

Staff and the SOC used Tier 4 review criteria to review questions recommended by the workgroup and assigned these questions to the suggested-topic list in Pragmatic Clinical Studies PFAs. These questions may also appear as suggested topics or areas of special interest in other funding announcements.

 

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Cancer

Bladder Cancer | View topic brief

• Compare the effectiveness of treatments (including various intravesical agents or bladder-preserving alternatives to cystectomy) in patients with intermediate or high-risk non-muscle invasive bladder cancer (NMIBC) who have failed first-line induction intravesical therapy with BCG or other agents. Proposed studies should be large-scale (preferably an RCT) and should include patient-centered outcomes such as mortality, progression to MIBC, side effects, and quality of life.

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Dental Health

Dental Caries

• Compare the effectiveness of the various alternative delivery models (e.g., primary care, schools, mobile vans) versus the dental office in preventing dental caries in children in medically underserved areas. | View the topic brief

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Mental and Behavioral Health

Anxiety in Children, Adolescents, and Young Adults

• Compare the effectiveness of two or more evidence-based approaches to the treatment of anxiety in children, adolescents, and young adults (through age 25). PCORI is interested in studies which expand the evidence to support feasible, acceptable, and broadly available treatments for patients in primary care and the community. PCORI is interested in studies which examine comparisons of different approaches to treatment initiation, sequencing, monitoring, maintenance, and/or relapse prevention following an initial effective course of treatment. | View the topic brief

Mental Health and Primary Care

• Compare the effectiveness of care models that integrate mental and behavioral health care--including substance abuse treatment--into the primary care provided by community health centers and other relevant settings, with the goal of reducing disparities in care (i.e., access to mental and behavioral health services and the diagnosis and treatment of mental and behavioral health conditions) and improving health outcomes among underserved populations, including racial and ethnic minorities.

Remote Delivery of Mental Healthcare

• Compare the effectiveness of different remote delivery approaches to evidence-based, non-pharmacological treatments for depression and anxiety conditions. PCORI is particularly interested in research that compares the effectiveness of different levels of intensity of monitoring, guidance and/or feedback, and of different types of professionals (e.g., technicians, clinicians, mental health clinicians) providing such monitoring, guidance, and/or feedback for Internet-based nonpharmacological treatment. Specific subgroups of interest include patients with differing severities of the target condition(s), as well as patients at risk of reduced access to care (e.g., rural populations, low-income individuals, and racial and ethnic minorities).

Substance Abuse

• Compare the effectiveness of multicomponent interventions taking place in clinics and/or community-based settings to reduce initiation of tobacco use and promote cessation of tobacco among racial/ethnic minorities, low-income populations, and/or rural populations. | View the topic brief
• Compare the effectiveness of SBIRT for adolescent alcohol abuse in different settings (school-based versus primary care based), using different delivery modes (in-person, remote, computer-based), and/or providers (physician, mental health specialist, nurse, staff worker, peer). | View the topic brief

Suicide

• Compare the effectiveness of evidence-based screening and primary prevention approaches, including different modes and settings (e.g., universal screening versus targeting at-risk individuals; virtual versus face-to-face screening; and within primary-care setting versus school-based) at minimizing suicidality among adolescents. | View the topic brief

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Muscular and Skeletal Disorders

Osteoarthritis (OA) | View the topic brief

• Compare methods for deciding when to undergo OA surgery; use outcomes such as patient satisfaction, functional status, clinical status, and quality of life.
• Compare the effectiveness of strategies for engaging early-stage OA patients to adopt behaviors that can prevent OA progression and disability.
• Compare different nonsurgical therapies (e.g., pharmacotherapy, injections, physical therapy or exercise, weight loss alone and in combination with other therapies, and complementary medicine alternatives) to prevent OA progression and disability. The studies should seek to identify heterogeneity of treatment response among important subgroups of patients.

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Neurological Disorders

Insomnia

• Compare the benefits and harms of pharmacologic, psychological, or combination treatments for treating different types of insomnia (sleep onset vs. sleep maintenance insomnia) on sleep and patient-centered outcomes including next-day function, mood, and quality of life.

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Non-Disease Specific

Care Transitions

• Compare the effectiveness of diverse models of comprehensive support services (e.g., incorporation of wraparound services, alternative providers, and technology) for infants and their families/caregivers following discharge from neonatal intensive care unit. | View the topic brief

Medication Management

• Compare the benefits and risks of different models of integrating pharmacists into the care transitions team in order to reduce adverse drug events, improve patient-centered outcomes, and lower preventable emergency department visits and re-hospitalizations post hospital discharge among patients with multiple chronic co-morbidities. | View the topic brief

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Nutritional and Metabolic Disorders

Diabetes

• Compare the effectiveness of clinical interventions to reduce nontraumatic lower-extremity amputations in racial or ethnic minorities and low-income populations with diabetes. Compare the effectiveness of expert, protocol-driven, team-based care to established guideline-based care to reduce the risk of nontraumatic lower-extremity amputations for racial or ethnic minorities and low-income populations with diabetes. Examples of team-based care could include acute-care teams with patient teams; staff working as a team (triage staff, etc.); use of standing orders, electronic medical records (EMRs), or hardcopy templates and reminders; knowledge and use of community resources; EMR registry functionality; empanelment; group visits; and/or novel outpatient-based interventions with rapid response from outpatient setting. | View the topic brief

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Reproductive and Perinatal Health

Preterm Birth and Low Birth Weight

• Compare the effectiveness of multicomponent systems interventions--such as evidence-based models of perinatal care--aimed at improving outcomes like preterm birth and low birth weight for mothers and babies at risk for health disparities. | View the topic brief

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Respiratory Disease

Community-Acquired Pneumonia

• What is the comparative effectiveness and safety of alternative FDA-approved antibiotic regimens in the empiric outpatient treatment of adults with community-acquired pneumonia? | View the topic brief
  • Alternative regimens may include:
    • Across-class comparisons of FDA-approved antibiotic regimens that are recommended for use or are commonly used in community-acquired pneumonia.
    • Varying duration of treatment, such as 3 or 5 days, or discontinuation at resolution of symptoms of infection.
  • The proposed studies should be of sufficient sample size to ensure the resulting evidentiary information is readily generalizable to the broader population. In addition, applications should address effectiveness in distinct subpopulations (e.g., patients with chronic conditions, immunosuppression, and the elderly).

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Trauma/Injury

Hip Fracture

• Compare the effectiveness of different surgical treatments in elderly patients with hip fracture in terms of functional and other patient-centered outcomes. | View the topic brief

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Topics No Longer Under Active Consideration

A topic may be removed from active consideration if, for example, research in that area is already under way, the topic doesn’t align with our current research strategy, or it doesn’t meet criteria now in place. These questions may be returned to consideration at a later date.

Cancer

Breast Ductal Carcinoma in Situ (DCIS) | View the topic brief

• Compare the effectiveness of standard treatment options for DCIS (lumpectomy with or without radiation therapy; mastectomy; hormonal therapy post-surgery) with non-standard options (hormonal therapy alone; active surveillance) on progression to invasive cancer, recurrence of DCIS, DCIS progression without invasive cancer, quality of life, satisfaction with treatment choice at study completion, decisional conflicts, and other patient-relevant outcomes such as self-image, sexual activity, or change in marital status.
• Compare different approaches to informed decision making about management of DCIS. Outcomes to include decisional conflicts, treatments received (e.g., mastectomy, lumpectomy, contralateral mastectomy), satisfaction with decision, match of the chosen treatment with the woman’s strength of preference for having an intact breast, and aforementioned clinical outcomes.

Particle Beam Therapy

• Compare the use of particle beam radiation therapy with other forms of radiation therapies in patients with lung, breast, or prostate cancer. Short- and long-term outcomes of interest might include tumor site–specific toxicities, severity of adverse effects, cancer-specific and overall mortality, quality of life, and functional outcomes.

Pulmonary Nodules

• Compare different protocols for managing people with lung nodules. Differences in protocols might include the types of imaging technologies, frequency of follow-up, indications for invasive diagnostic procedures, and biomarkers.

Cardiovascular Health

Cardiovascular Disease (CVD)

• Compare the effectiveness of multi-disciplinary, systems-focused, and data-driven interventions to improve efficiency, effectiveness, and reliability of care to reduce CVD disparities in underserved populations. Targets for reducing disparities include improvements in hypertension control, treatment for hyperlipidemia, smoking cessation, and/or appropriate use of aspirin. The studies should examine which components of the interventions are critical for achieving risk reduction and provide details on patient-centered outcomes.
• Compare the effectiveness of various interventions to support self-management of hypertension, hyperlipidemia, tobacco addiction, and/or appropriate use of aspirin in underserved populations. The studies should examine which components of the interventions are critical for achieving risk reduction and provide details on patient- centered outcomes.

Carotid Artery Disease

• Compare the effectiveness of aggressive medical treatments with invasive procedures, in patients with asymptomatic carotid artery disease in terms of stroke rates and other patient-centered outcomes. | View the topic brief

Gastrointestinal Disorders

Crohn’s Disease | View the topic brief

• Compare the use of one TNF-alpha inhibitor with another, or with other biologics— administered for either induction or maintenance of remission in adults and children diagnosed with Crohn’s disease—in such outcomes as patient-reported outcomes, steroid reduction, Crohn’s Disease Activity Index (CDAI), pediatric CDAI, and mucosal healing.
• Compare the use of a TNF-alpha inhibitor and a thiopurine with the TNF-alpha inhibitor alone in maintenance of remission in adults and children, considering such outcomes as patient-reported outcomes, steroid reduction, CDAI, pediatric CDAI, and mucosal healing.

Kidney Disease

End-Stage Renal Disease

• Benefits and harms of continuous ambulatory peritoneal dialysis compared with hemodialysis (daily or intermittent home, or conventional in-center) in patients with end-stage renal disease and in important patient subgroups. (e.g., by age, race, ethnicity, cardiovascular risk, other comorbidities) | View the topic brief

Mental and Behavioral Health

Autism Spectrum Disorder

• Perform a large-scale, multi-center, randomized control trial or well-designed observational study with long-term follow-up comparing the effectiveness of applied behavioral analysis (in children 2 to 5 years old) with other accepted treatments for alleviating externalizing and internalizing behavior and improving social skills, parent-child interactions, family well-being, and other patient-relevant outcomes (e.g., changes in core and associated symptoms). Studies should be sufficiently large to permit rigorous analysis of HTE related to provider, parent, family, child, intervention, and other characteristics.

Bipolar Disorder | View the topic brief

• Compare clinical outcomes when children and adolescents presenting with symptoms of bipolar disorder are diagnosed by clinical judgment alone or by standard DSM diagnostic criteria.
• Compare the outcomes of long-term treatment (more than two years) with different treatment strategies (e.g., antipsychotic medication classes and types; monotherapy or combinations; psychotherapy; assertive community treatment; adjunct therapies such as antidepressants, anxiolytics, and stimulants). Outcomes should include but are not limited to symptoms and socio-developmental, adaptive behavioral, and adverse effects (including substance abuse).

Caregiver Engagement and Support

• Compare the effectiveness of strategies that incorporate involvement and/or support of patients and their families or other caregivers in care for patients with chronic mental illness (e.g., bipolar disorder, major depression, anxiety disorders, schizophrenia). Studies comparing different delivery system designs and measuring patient and caregiver outcomes (e.g., health-related quality of life, symptom relief, caregiver stress) are of particular interest. | View the topic brief

Substance Abuse

• Compare different combinations of treatment options for people with opioid substance abuse, focusing on long-term outcomes. Treatment options might include medication- assisted treatments, psychosocial therapies, and complementary medicine alternatives.

Neurological Disorders

Migraine | View the topic brief

• Compare pharmacologic and non-pharmacologic strategies to prevent the transition from episodic to chronic migraine.
• Compare pharmacologic and non-pharmacologic strategies for treating individual headache episodes on the incidence of medication-overuse headache in patients with high-frequency episodic or chronic migraine.

Multiple Sclerosis

• Compare management options for modifying disease progression. These might include FDA-approved disease-modifying agents; behavioral interventions, including exercise and physical therapy; and complementary medicine alternatives. | View the topic brief

Non-Disease Specific

Insurance Design

• Compare specific features of health insurance on access to care, use of care, and other outcomes that are especially important to patients. Examples of specific insurance design features that may be considered include: utilization management approaches, consumer-directed care benefits, prior authorization programs, value-based insurance designs, and alternative patient cost- sharing schemes. Studies should take into account the preferences and needs of chronically ill patients and account for variations in care settings and providers’ payer mix, where applicable.

Medication Management

• Compare the effectiveness of various strategies for enhancing medication adherence. Studies should take into account the needs of patients with chronic conditions who are prescribed medications for short- and/or long-term indications.

Pain

Chronic Back Pain

• Compare the effect of different combinations of multimodal approaches to patients with episodic back pain (including self-care with or without over-the-counter medications; movement-based therapies, such as exercise and yoga; manipulation and/or mobilization; complementary medicine alternatives; and cognitive-behavioral therapies) on the transition from episodic acute back pain to chronic back pain, symptom relief, patient satisfaction, quality of life, and functional outcomes. Specific outcomes might also include reduction in pain-medication use, reduction in patient visits for low-back pain, increase in quality of life, increased time between low-back-pain episodes, and decreased severity of episodes (i.e., decreased pain and increased function).

Chronic Pain

• Compare the effectiveness of pharmacist- or nurse-led interventions, or health information technology-based interventions, to enhance primary-care physician management of patients suffering from chronic, noncancer pain. Outcomes of primary interest include the impact of such evidence-based strategies on improving patient functioning, patient quality of life, and reducing opioid dependence.

Musculoskeletal Pain

• Compare the effectiveness of alternative models of coordinated pain management (such as specialized, integrated pain centers, coordinated care models that emphasize cross-provider data- and communication-sharing, or those that include risk triage systems with care coordination) for treatment of chronic nonspecific, musculoskeletal pain on improving functional status and other patient-centered outcomes.

Reproductive and Perinatal Health

Pelvic Organ Prolapse

• Compare the relative benefits and harms of different types of current FDA-approved transvaginal mesh (e.g. permanent vs. absorbable) or biological grafts in repairing pelvic organ prolapse in terms of infections, de novo urinary or fecal incontinence, bowel injury, pain, sexual function, quality of life, and other patient-centered outcomes.
• Compare the relative benefits and harms of different surgical techniques in repairing pelvic organ prolapse by compartment (i.e., anterior, posterior, multi-compartment) using currently FDA-approved transvaginal mesh in terms of the outcomes mentioned above.

Trauma/Injury

Traumatic Brain Injury

• Compare the effectiveness of multidisciplinary rehabilitation programs (e.g., community integrated rehabilitation: neurobehavioral; residential community; comprehensive holistic and home-based services) for moderate to severe traumatic brain injury in non-military or veteran adults. | View the topic brief

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Posted: May 4, 2015; Updated: December 12, 2017