What research does PCORI fund?

PCORI funds patient-centered research to improve healthcare decision making by patients, clinicians and caregivers. PCORI funds comparative clinical effectiveness research—determining which clinical options or approaches work best for patients and their caregivers given their particular concerns and characteristics.

PCORI funds patient-centered research that compares outcomes. More details can be found in Research We Support.

Research proposals submitted in response to PCORI Funding Announcements (PFAs) are evaluated against PCORI's review criteria. These criteria are consistent with patient-centeredness and are specifically designed to include a diverse set of perspectives in decision making.

What research does PCORI NOT fund?

PCORI discourages proposals in the following categories and will deem them nonresponsive:

  • Development of instruments, such as new surveys or scales.
  • Development, testing and validation of new decision aids and tools or clinical prognostication tools.
  • Pilot studies intended to inform larger efforts.
  • Comparison of patient characteristics rather than clinical strategy options.
  • Studies designed to test the efficacy of unproven interventions.

Consistent with PCORI’s authorizing law, PCORI does not fund research for which findings will include the following:

  • Coverage recommendations.
  • Payment or policy recommendations.
  • Creation of clinical practice guidelines or clinical pathways.
  • Establishment of efficacy for a new clinical strategy.
  • Pharmacodynamics.
  • Study of the natural history of disease.
  • Basic science or the study of biological mechanisms.
  • Formal cost-effectiveness analysis conducted of alternative approaches to providing care.
  • Direct comparisons of the costs of care between two or more alternative approaches to providing care.
  • Research relying on cost simulation modeling to develop estimates of total costs of care beyond the scope and time horizon of the study.

Each funding opportunity may have additional non-responsiveness criteria unique to that solicitation.

Will PCORI consider CER studies where one of the interventions being compared is a medical product being used off-label in clinical practices?

In some instances, off-label use of Food and Drug Administration (FDA)-approved medical products, such as drugs or devices, is a realistic choice in clinical practice and appropriately represents a viable alternative therapy in need of evaluation. Clinicians can use approved medical products outside of their FDA-labeled approved indications and do so when approved interventions are either unavailable or unsatisfactory. With understudied conditions and populations, such as rare diseases or pediatric populations, use of medical products off-label is not uncommon.

In instances where off-label use of a medical product is proposed for CER, a specific rationale and justification to support its inclusion should be clearly documented in the application. As part of the justification for the importance of the study, investigators should describe how widely and often the off-label interventions are currently used in clinical practice for treatment of the condition being studied. Securing an investigational drug or device use approval may be required and should be addressed in proposed study plans. Please note, PCORI does not support research with a primary intent to secure FDA approval for expanded indication(s).

Does PCORI fund research focused on developing and validating an instrument to measure behavior change? 

Possibly. PCORI will not fund research whose principal aim is to develop and validate an instrument but will consider studies for which instrument development and/or validation is an ancillary benefit. 

For questions about the nature of your research, please email us at [email protected]  to schedule a call with program staff. 

Does PCORI have funding quotas—e.g., geographic, by tax area, by population group or by institution?

PCORI tracks awardees by many variables, including geographic region, therapeutic area and populations studied, but there are no specific quotas for the research PCORI funds. PCORI does seek to fund studies strategically, however, based on content. 

Does PCORI make available information on research projects it has funded? 

Yes. To learn about research projects that PCORI has funded, please see PCORI's portfolio of funded studies and other projects. They can be searched by state or year funded.

Does PCORI provide feedback on the suitability of a proposed project and guidance on determining the appropriate PFA to which to submit an application?

Yes. For questions about the nature of your research, please email us at [email protected] to schedule a call with program staff.

How are PCORI’s Methodology Standards incorporated into the application and review process?

All proposals must adhere to all relevant PCORI Methodology Standards. The standards were approved by our Board of Governors in November 2012, published in December 2012, and periodically updated. We recognize that a variety of study designs and analytic methods can produce valid new knowledge. Regardless of the particular methods employed, proposals are expected to use rigorous methodology. Standards are for research proposals and submitted as a checklist as a part of the application submission.

How does Patient Centered Outcomes Research (PCOR) relate to comparative effectiveness research (CER), and does PCORI fund CER?

PCOR is a broad category of research with an emphasis on answering questions important to patients and other healthcare stakeholders. Some, but not all, PCOR is also CER. PCORI does fund patient-centered CER, although not all CER is patient-centered. PCORI’s definition of patient-centered is research that addresses questions important to patients and other healthcare stakeholders, and studies outcomes meaningful to patients and other stakeholders.

PCOR can be used by patients and their caregivers to communicate and make informed healthcare decisions, allowing their voices to be heard in assessing the value of healthcare options. This research:

  • Assesses the benefits and harms of preventive, diagnostic, therapeutic, palliative or health-delivery system features to inform decision making, highlighting the choices that matter to people.
  • Is inclusive of an individual’s preferences, autonomy and needs, focusing on outcomes that people notice and care about, such as survival, functioning, symptoms and health-related quality of life.
  • Incorporates a wide variety of settings and diversity of participants to address individual differences as well as barriers to implementation and dissemination.
  • Directly compares clinical interventions that are generally available in clinical settings.
  • Obtains the perspectives of stakeholders to address the burdens to individuals, availability of services and requirements for technology and personnel.

How does the research PCORI funds differ from Community-Based Participatory Research (CBPR)?

The main difference is the focus of CBPR on social action, which is not a requirement for patient-centered CER funded by PCORI. Both are similar in the inclusion of end users in the production of evidence.

If an applicant proposes the establishment of a rare disease registry, would this meet requirements for PCORI funding, and under which PFA would it be funded?

Possibly. PCORI might fund a rare disease registry if it is a component of a research study aimed to answer comparative research questions. In this case, the proposal would most likely fit under the Broad PFA.

If an applicant receives a National Institutes of Health (NIH) K Award, is he or she still qualified to receive PCORI funding?

Yes. The level of effort stated in the K Award must remain consistent, but awardees are not restricted in the remaining effort or salary level. Also, PCORI is not a federal funding agency, and K awardees may supplement the NIH contribution to their salary based on the NIH salary scale (as long as the funds are not federal).

What populations of interest does PCORI want to address with its research?

Our PFAs have always noted the importance of including diverse populations in studies with respect to age, gender, race, ethnicity, geography or clinical status. We have developed the following, more detailed list of “hard-to-reach” or lesser-studied populations to guide our research and engagement efforts:

  • Underrepresented racial and ethnic groups.
  • Low-income groups.
  • Women.
  • Children (ages 0 to 17 years).
  • Older adults (ages 65 years and older).
  • Residents of rural areas.
  • Individuals with special healthcare needs, including individuals with disabilities.
  • Individuals with multiple chronic diseases.
  • Individuals with rare diseases.
  • Individuals whose genetic makeup affects their medical outcomes.
  • Patients with low health literacy or numeracy or limited English proficiency.
  • Gender and sexual minorities.
  • Veterans and members of the US Armed Forces and their families.

What resources does PCORI make available for applicants to determine the level of patient and stakeholder engagement required in an application?

Engagement can take on many forms, so we ask teams to come up with their own appropriate blueprints for partnerships. Due to many researchers not having experience engaging patients and other stakeholders in the planning, conduct and dissemination of research, we provide information about engagement on our “The Value of Engagement” page, as well as Engagement Resources.

In what types of research studies of interventions is PCORI interested?

PCORI is interested in studies of interventions that may include:

  • Specific drugs, devices and procedures.
  • Other types of alternatives, such as medical and assistive devices and technologies.
  • Behavior change, including the use of behavioral or financial incentives (the latter for patients only).
  • Organizational models and policies within and across healthcare systems (e.g., patient-centered medical homes, clinical protocols such as standing orders, clinical pathways).
  • Communication and/or dissemination strategies.

Please note that some opportunities may call out specific interventions of interest. For more information on what PCORI is interested in funding, please review our Funding Opportunities webpage section which lists all open funding announcements as well as upcoming and past opportunities.

Can multiple individuals from an applying organization access the Letter of Intent (LOI?)

Yes. The principal investigator (PI) may assign up to two PI Designees to access and edit the LOI. Only one person may work on the application in PCORI Online at a time. 

How are applicants notified after a LOI is submitted?

Applicants will receive an email confirmation when the LOI is successfully submitted via PCORI Online. PCORI staff screen LOIs for programmatic fit and administrative noncompliance. Approximately five weeks after the LOI deadline, PCORI will notify applicants via email whether or not they have been invited to submit an application. PCORI encourages prospective applicants to contact us if they have any questions prior to the deadline.

Additionally, one-time funding announcements may have special guidelines regarding LOI submission and screening. Applicants should carefully read the related PFA to learn about and follow all guidelines.

Does PCORI allow for a placebo to be an active comparator?

PCORI considers placebo to be a reasonable comparator if active treatments/interventions/screenings are not available, or if there is a compelling reason not to use an active treatment/comparator in the study design. The choice of placebo as a comparator must be well-justified.

Can an applicant propose "usual care" as an active comparator?

Yes. If an applicant proposes usual care as a rational and important comparator in the proposed study, then it must be described in detail, coherent as a clinical alternative, and properly justified as a legitimate comparator (e.g., usual care is guidelines-based). The choice of usual care must represent current clinical and ethical standards of care. It must also be accompanied by an explanation of how the care given in the usual care group will be measured in each patient and how appropriate inferences will be drawn from its inclusion. Usual care must be described as mentioned previously to ensure that it accounts for geographic and temporal variations and that it has wide interpretability, applicability, and reproducibility.

Can an applicant propose a vitamin or a dietary supplement as a comparator in a trial?

Yes, however, there are important considerations for trials that propose a vitamin or dietary supplement as a comparator.

Rigorous and generalizable trials require the use of well-characterized interventions to ensure wide interpretability and reproducibility. As such, investigators should ensure there is consistent high quality and purity of the product and its identity, strength, origin source, and composition. Monitoring of the delivered dose by chemical, biomarker, or other approved testing is encouraged. At the same time, real-world applicability and the ability to implement and disseminate study findings is critical and should be considered in designing the study and selecting the interventions.

PCORI expects the efficacy or effectiveness of each comparator to be known. If the efficacy or evidence base is insufficient, then data need to be provided to document that the comparator is used widely.

Evidence must be provided in the application on the safety of the proposed product(s) at the proposed dosage and timing for the proposed indication and population. Additionally, the proposed trial should include appropriate safety monitoring and evaluation. This includes, but is not limited to, the possibility of the vitamin/dietary supplement or its components having adverse impact(s) upon comorbid clinical conditions or treatments (such as medications) as well as any other safety monitoring/reporting required by the study’s DSMB.

An IND may be required for a clinical investigation evaluating the vitamin/dietary supplement’s ability to diagnose, cure, mitigate, treat, or prevent a disease. It may also be necessary for investigators to establish a legal agreement with the company (or companies) who produce the proposed product(s) to assure appropriate safety monitoring and quality standards listed above are met.

Can an applicant propose a vitamin or dietary supplement as a comparator in an observational study?

Yes. PCORI is interested in funding applications with rigorous study designs, including well-designed observational studies, where efforts to minimize confounding are well-documented. However, there are important considerations for studies that propose a vitamin or dietary supplement as a comparator.

Use of a vitamin or dietary supplement in an observational study should be naturalistic. That is, use of the vitamin or dietary supplement should be unassigned or voluntary.

Rigorous and generalizable studies require the use of well-characterized comparators to ensure wide interpretability. As such, investigators should ensure that the study team characterizes and assures the product quality, identity, strength, origin source, and composition. Monitoring of the delivered dose by chemical, biomarker, or other approved testing should be considered. At the same time, real-world applicability and the ability to disseminate study findings is critical and should be considered in designing the study and selecting the comparators.

PCORI expects the efficacy or effectiveness of each comparator to be known. If the efficacy or evidence base is insufficient, then data need to be provided to document that the comparator is used widely.

My LOI’s evidence-based intervention has been shown to be efficacious in the general population but lacks evidence for efficacy in a specific subpopulation or setting. Can I propose a study to adapt and test this intervention?

Applications may propose studies that tailor and test interventions that have documented efficacy or effectiveness in similar situations, with adaptations appropriate for the population or setting of interest. In such cases, the efficacy must be well documented (e.g., with prior research or with a systematic review) and a strong rationale provided for why the intervention would be expected to be efficacious in the proposed new setting(s) and/or population(s).

If an intervention is to be adapted based upon stakeholder engagement or other methods, PCORI expects most of the proposed time and budget to be directed to conducting the comparative clinical effectiveness research study rather than adapting the intervention. While there is no stated maximum, PCORI anticipates adaptation efforts should be less than 20% of the proposed project period and total budget.

How does PCORI define widespread use?

Per PCORI’s funding announcements, PCORI expects the efficacy or effectiveness of each intervention to be known. If the efficacy base is insufficient, then data must be provided to document that the intervention is used widely. Acceptable ways to demonstrate widespread use include, but are not limited to:

  • Observational data that demonstrates the intervention is in common use at multiple institutions/in multiple regions of the U.S. (e.g., claims data, EHR data, registry data, PCORnet);
  • Multiple case series from different institutions in the literature published by investigators that are not planned key personnel within the proposed research;
  • Evidence of coverage by insurance payors for the indication proposed in the application;
  • Society or guideline recommendations for use of the intervention for the indication proposed in the application

Can I propose a Phase 2 or Phase 3 clinical trial for PCORI funding?

To be considered responsive to PCORI’s research award funding opportunities, applicants are expected to describe research that compares two or more alternatives, each of which has established efficacy and/or is in widespread use. Projects aiming to develop new interventions that lack evidence of efficacy, effectiveness, or widespread use will be considered out of scope.

Phase 2 and Phase 3 clinical trials typically aim to evaluate the safety and efficacy of a new treatment; as such, they are beyond the scope of PCORI-funded research.

Of note, PCORI requires FDA approval for any medical products being studied. Study of off-label indications from FDA-approved labeling may need an Investigational New Drug or Device (IND) approval or exemption. Additionally, PCORI does not support research with a primary intent to secure FDA approval for expanded indication(s).


Posted: September 1, 2023; Updated: April 17, 2024

What's Happening at PCORI?

The Patient-Centered Outcomes Research Institute sends weekly emails about opportunities to apply for funding, newly funded research studies and engagement projects, results of our funded research, webinars, and other new information posted on our site.

Subscribe to PCORI Emails

Image

Hand pointing to email icon