PCORI Funding Announcement: Healthy Aging: Optimizing

Cycle 3 2021 Funding Cycle

PCORI Funding Announcement: Healthy Aging: Optimizing Physical and Mental

Functioning Across the Aging Continuum Published September 7, 2021

This PCORI Funding Announcement (PFA) applies to the funding cycle that closes January 11, 2022, at 5 pm ET. Submission Instructions, templates, and other resources are available at https://www.pcori.org/funding-opportunities/announcement/healthy-aging-optimizing-physical-mental-functioning-across-aging-continuum-cycle-3-2021.

PCORI Cycle 3 2021: Healthy Aging Funding Announcement

About PCORI

PCORI was authorized by federal law in 2010 and reauthorized for an additional 10 years in 2019 as a

nonprofit, nongovernmental organization. PCORI’s purpose, as defined by our authorizing legislation, is

to help patients, caregivers, clinicians, policy makers, and other healthcare system stakeholders make

better-informed health decisions by “advancing the quality and relevance of evidence about how to

prevent, diagnose, treat, monitor, and manage diseases, disorders, and other health conditions” and by

promoting the dissemination and uptake of this evidence.

PCORI is committed to transparency and a rigorous stakeholder-driven process that emphasizes patient

engagement. PCORI uses a variety of forums and public comment periods to obtain public input to

enhance its work. PCORI helps people make informed healthcare decisions and improves healthcare

delivery and outcomes by producing and promoting high-integrity, evidence-based information that

comes from research guided by patients and other stakeholders.

Patient-Centered Outcomes Research Institute 1828 L St., NW, Suite 900 Washington, DC 20036 Phone: 202-827-7700 Fax: 202-355-9558 Email: [email protected]

Follow us on Twitter: @PCORI

mailto:[email protected]


Important Considerations Related to COVID-19 and Research Studies The significant global impact of the COVID-19 pandemic has markedly affected healthcare delivery and

research. Substantial uncertainties exist about the nature and duration of its impact on research,

including intervention delivery and the collection, analysis, and the interpretation of study data.

Research staff may face conflicting local and institutional policies to promote safety and the provision of

care for those afflicted with COVID-19. They may also face personal risks of exposure, illness, and

incapacity related to the pandemic. PCORI considers the safety and well-being of study participants,

research staff, and stakeholders to be paramount and advocates that safety be the foundational

principle guiding research decisions.

In light of the risks and uncertainties of COVID-19 on population health, health care, and research,

PCORI requests applications to this PFA to include an explicit assessment of potential risks and risk

management plans/contingencies for the proposed research as it may be affected by COVID-19. In

addition to risk assessment and management related to the planning and conduct of the research itself,

applicants should also consider provisions in their leadership and staffing plan to assure study continuity

in the event of personnel absences due to quarantine, illness, or the provision of clinical care.


Key Dates Online System Opens: September 7, 2021 Town Hall: September 13, 2021, 12 pm ET Letter of Intent (LOI) Deadline: October 5, 2021, by 5 pm (ET) LOI Status Notification: November 2, 2021 Application Deadline: January 11, 2022, by 5 pm (ET) Merit Review: April 2022 Awards Announced: July 2022 Earliest Project Start Date: November 2022

Maximum Project Budget (Direct Costs)

$5 million

Maximum Research Project Period

5 years

Funds Available Up To

$50 million

Review Criteria 1. Potential for the study to fill critical gaps in evidence 2. Potential for the study findings to be adopted into clinical practice and improve delivery of

care 3. Scientific merit (research design, analysis, and outcomes) 4. Investigator(s) and environment 5. Patient-centeredness 6. Patient and stakeholder engagement

Contact Us Programmatic Inquires: [email protected], phone (202-627-1884), or online (http://www.pcori.org/PFA/inquiry). Administrative, Financial, or Technical Inquiries: [email protected] or phone (202-627-1885). PCORI will respond within two business days. However, we cannot guarantee that all questions will be addressed two business days prior to a LOI or application deadline. Applicants must plan accordingly; it is the applicant’s responsibility to submit on time.


http://www.pcori.org/PFA/inquiry



Table of Contents

Table of Contents ................................................................................................................... 5

I. Introduction ..................................................................................................................... 1

Summary of Program .......................................................................................................................... 1

Topic Background................................................................................................................................ 1

Evidence Gaps ..................................................................................................................................... 3

Maintain Function and Independence................................................................................................ 4

Facilitate Chronic Disease Management ............................................................................................ 4

Support Individuals with Significant Functional Impairment ............................................................. 4

Reduce Caregiving Burden and Improve Quality of Life ..................................................................... 5

Research Questions ............................................................................................................................ 5

Specific Requirements for This Funding Announcement ................................................................... 6

Funds Available and Duration of Studies ............................................................................................ 8

II. General Requirements for PCORI Research ................................................................. 9

Research Priorities .............................................................................................................................. 9

Categories of Non-responsiveness ..................................................................................................... 9

Principles for Consideration of Full Range of Outcomes Data in PCORI Funded Research .............. 10

Coverage of Intervention Costs ........................................................................................................ 11

Avoiding Redundancy ....................................................................................................................... 11

Methodological Considerations ........................................................................................................ 12

Patient-Centered Outcome Measures .............................................................................................. 12

Leveraging Existing Resources, Including PCORnet .......................................................................... 12

Patient and Stakeholder Engagement .............................................................................................. 13

Populations Studied and Recruited .................................................................................................. 14

Protection of Human Subjects .......................................................................................................... 15

Required Education of Key Personnel on the Protection of Human Subject Participants ............... 15

III. LOI Review ......................................................................................................................15

IV. Merit Review ...................................................................................................................16

Preliminary Review ........................................................................................................................... 16

In-Person Review .............................................................................................................................. 20

Post-Panel Review ............................................................................................................................. 20


Summary Statements and Funding Recommendations ................................................................... 20

V. PCORI Policies that Govern Awardees Related to Data Access, Privacy, and Public Reporting ........................................................................................................................21

Registering Research Projects ........................................................................................................... 21

PCORI Public Access Policy ................................................................................................................ 21

Standards for Privacy of Individually Identifiable Health Information ............................................. 21

Data Management and Data-Sharing Plan ....................................................................................... 22

Peer Review and Release of Research Findings ................................................................................ 22

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PCORI Cycle 3 2021 Healthy Aging Funding Announcement

I. Introduction

The Patient-Centered Outcomes Research Institute (PCORI) funds patient-centered outcomes research

(PCOR), a type of comparative clinical effectiveness research (CER) that focuses on outcomes that

matter to patients, their caregivers, and their families. PCORI-funded studies must include the

perspectives of patients and other healthcare stakeholders.

The public entrusts PCORI to fund research that matters to patients, their caregivers, and other

stakeholders (defined as clinicians and clinician societies, hospitals and health systems, payers

[insurance], purchasers [business], industry, researchers, policy makers, and training institutions). By

emphasizing the role of diverse research teams that include varying perspectives, PCORI seeks to change

how research is conducted. PCORI distinguishes itself by supporting studies in which patients,

caregivers, practicing clinicians, and the broader stakeholder community are actively engaged in

generating research questions, reviewing research applications, conducting research, disseminating

research findings, promoting the implementation of those findings, and using the results to understand

and address patient and other stakeholder needs.

Summary of Program

This Targeted PFA (tPFA) on healthy aging seeks to fund high-quality CER studies that aim to optimize

the physical and mental functioning of community-dwelling older adults and their caregivers across the

aging continuum.

Topic Background

By 2060, the number of Americans over age 65 is projected to reach around 95 million, making up

almost a quarter of the US population.1 The number of people of color among the older adult population

is projected to increase from 12.3 million in 2018 (23 percent of older adults) to 27.7 million in 2040 (34

percent of older adults).2 Approximately 85 percent of older adults have at least one chronic condition,

and 60 percent have at least two.3 Chronic conditions can affect any older adult, but they excessively

burden people of color who, for structural and other reasons, have less access to quality health care and

lower rates of healthcare utilization.4,5,6,7

Most older adults, including those with multiple chronic conditions, prefer to stay in their homes and

1 Apóstolo J, R. Cooke, E. Bobrowicz-Campos, et al. Effectiveness of interventions to prevent pre-frailty and frailty progression in older adults: a

systematic review. JBI Evid Synth. 2018;16(1):140-232. 2 Ristolainen H, S. Kannasoja, E. Tiilikainen, et al. Effects of “participatory group-based care management” on wellbeing of older people living

alone: a randomized controlled trial. Arch Gerontol Geriatr. 2020;89:104095. 3 Liu CJ, H. Xu, N. R. Keith, et al. Promoting ADL independence in vulnerable, community-dwelling older adults: a pilot RCT comparing 3-Step

Workout for Life versus resistance exercise. Clin Interv Aging. 2017;12:1141-1149. 4 Johnson KS. Racial and ethnic disparities in palliative care. J Palliat Med. 2013;16(11):1329-1334. 5 Liss DT, Baker DW. Understanding current racial/ethnic disparities in colorectal cancer screening in the United States: the contribution of

socioeconomic status and access to care. Am J Prev Med. 2014;46(3):228-236. 6 Rooks RN, E. M. Simonsick, L. M. Klesges, et al. Racial disparities in health care access and cardiovascular disease indicators in Black and White

older adults in the Health ABC Study. J Aging Health. 2008;20(6):599-614. 7 Weinstock RS, J. A. Teresi, R. Goland, et al. Glycemic control and health disparities in older ethnically diverse underserved adults with

diabetes: five-year results from the Informatics for Diabetes Education and Telemedicine (IDEATel) study. Diabetes Care. 2011;34(2):274-279.

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communities for as long as they can, a concept referred to as “aging in place.”8 Caregivers—those family

members or close friends who serve as the primary caregiver to an older adult—play a critical role in

helping older adults age in place, by directly addressing their care needs and helping them navigate a

fragmented healthcare system.9 As the needs of older adults become more complex, the caregiver role

also intensifies, often resulting in detrimental impact on caregiver well-being.

As it stands, the current US healthcare system is not well equipped to address the complex care needs of

a rapidly growing and diverse older adult population and their caregivers.10 Regardless of how a future

healthcare system plans to address these needs, it is vital to maintain the physical and mental

functioning of older adults living in the community at levels that are optimal for each phase of the aging

continuum, and caregivers’ well-being must likewise be a priority. Thus, there is an urgent need to

implement an effective set of interventions directed at both older adults and their caregivers. Such

interventions could also target the arrest of condition progression and should ensure that the needs of

all older adults, regardless of background, are equally addressed. Recognizing this urgency, the National

Academy of Medicine has identified “actualizing better health and healthcare for older adults” to be one

of five vital directions for health and healthcare in 2021.11

PCORI has received significant input from diverse stakeholders, including patient and caregiver

advocates, clinicians, payers, policy makers, and other research funders in the healthy aging field. They

emphasized the need for real-world CER across the aging continuum that could inform evidence-based

clinical practice. For them, population-based interventions that focus on optimizing the physical and

mental functioning of older adults and that address the needs of caregivers are a priority. Stakeholders

also emphasized the need for effective interventions that can advance health equity—particularly by

examining how to best integrate community-based services and social determinants of health (e.g.,

transportation, nutrition, housing) into primary and/or specialty care, to comprehensively address the

healthcare needs of older adults and their caregivers. Many stakeholders have also expressed the need

to identify factors, as part of the research process, that would facilitate the real-world implementation

of interventions after the research studies are complete.

8 AARP. 2018 Home and Community Preferences Survey: A National Survey of Adults Age 18-plus. AARP Research; 2018. Accessed on June 20,

2021. https://www.aarp.org/content/dam/aarp/research/surveys_statistics/liv-com/2018/home-community-preferences-survey.doi.10.26419-2Fres.00231.001.pdf 9 AARP, National Alliance for Caregiving. Caregiving in the United States 2020. AARP; 2020. 10 Rowe J, L. Berkman, L. Fried, et al. Preparing for Better Health and Health Care for an Aging Population, in Vital Directions for Health and

Health Care Series. National Academy of Medicine; 2016. 11 “Supporting Older Patients With Chronic Conditions”, in “Talking With Your Older Patient.” National Institute on Aging. 2017. Accessed on

June 25, 2021. https://www.nia.nih.gov/health/supporting-older-patients-chronic-conditions#:~:text=Approximately%2085%20percent%20of%20older,for%20Disease%20Control%20and%20Prevention

https://www.aarp.org/content/dam/aarp/research/surveys_statistics/liv-com/2018/home-community-preferences-survey.doi.10.26419-2Fres.00231.001.pdf

https://www.aarp.org/content/dam/aarp/research/surveys_statistics/liv-com/2018/home-community-preferences-survey.doi.10.26419-2Fres.00231.001.pdf

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Figure 1. The Aging Continuum

Adapted from Tkatch et al. 201612

As Figure 1 shows, the different phases of the aging continuum include, on the one end, the healthiest

older adults who have no chronic conditions, followed by those who may have one or two well-managed

conditions; their main goal is to maintain function and independence. Next on the continuum are older

adults with chronic or multiple chronic conditions that are sub-optimally managed; their primary health

focus is to manage their condition(s) and slow or reverse progression. This group is followed by those

adults with significant functional impairment (physical and/or cognitive); these individuals are at higher

risk for adverse events, require more support, and are likely to have more healthcare utilization and

expenditures. Finally, at the other end of the continuum are seriously ill older adults who may need end-

of-life care. Applications responding to this tPFA may focus on populations in different phases of the

aging continuum except for those at the two extremes: the healthiest of older adults with no chronic

health condition and the seriously ill who may require end-of-life care.

To ensure that older adults can age in place at any phase of the continuum, there is a need to develop a

broader, population-based, health-promoting approach comprised of interventions that target

optimization of functioning for all older adults delivered in home- and community-based settings in

addition to interventions delivered in clinical settings. To facilitate a more comprehensive focus on

addressing the medical as well as social needs of community-dwelling older adults and their caregivers,

applicants are hence encouraged to take a broader approach to intervention delivery —one that is not

limited to clinical settings alone.

Evidence Gaps

Here, we describe the evidence gaps in this tPFA’s four domains of interest: (a) Maintain function and

independence, (b) Facilitate chronic disease management, (c) Support individuals with significant

functional impairment, and (d) Reduce caregiving burden and improve quality of life.

12 Tkatch R, S. Musich, S. MacLeod, et al. Population health management for older adults: review of interventions for promoting successful

aging across the health continuum. Gerontol Geriatr Med. 2016;2.

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Maintain Function and Independence

Common interventions for maintaining function and independence include physical activity, nutrition,

occupational therapy, home adaptation, and social support, delivered either alone or in combination.13

Systematic reviews have identified the need for future research on head-to-head comparisons of

different individual and multicomponent interventions for promoting physical and cognitive functioning

among older adults across the aging continuum.12 Multiple stakeholders also highlighted the need for

evidence-based approaches to mitigate the negative impact of social isolation on the functioning of

older adults in the United States; social isolation is increasingly recognized as a public health risk factor

with an impact on premature death comparable to that of other risk factors such as smoking, obesity,

and physical inactivity.10

Facilitate Chronic Disease Management

Stakeholders pinpointed management of multiple chronic conditions to be one of the primary issues

facing their members. They noted that many interventions for managing chronic conditions focus on a

single condition and do not address other comorbid conditions or the specialized needs of older adults

and their caregivers. This singularity of focus has led to adverse polypharmacy, excessive treatment

burden, and fragmented care. Literature reviews also found that multicomponent care coordination

approaches targeting specified combinations of chronic diseases, as opposed to a single disease in the

context of comorbid conditions, were more effective; head-to-head comparisons of such models was

indicated.14 There is a need for CER to evaluate different approaches to leveraging technology, including

telehealth, to facilitate care coordination and chronic disease self-management among older adults,

especially those with limited access to care.15, 16 Similarly lacking are evaluations of person-centered

models that ensure older adults with multiple chronic conditions and their caregivers are engaged in

shared decision making and in development of care plans that respond to their goals and preferences.17

Support Individuals with Significant Functional Impairment

Older adults with significant functional impairments need a suite of care and support services that

address both their medical and social needs and help them to continue living in the community.

Stakeholder discussions underscored the need for evaluating different multicomponent, long-term

services and supports (LTSS) for older adults with significant functional impairment. The opportunities to

conduct head-to-head trials of LTSS programs are substantial, given their widespread use and funding by

Medicaid. Yet the evidence base for such services is limited and can hamper policy makers’ decision

making on which services are effective and ready for expansion. There is a need for both collaborative

models between primary and specialty care as well as models that integrate primary and/or specialty

13 Zubala A, S. MacGillivray, H. Frost, et al. Promotion of physical activity interventions for community dwelling older adults: a systematic

review of reviews. PLoS One. 2017;12(7):e0180902. 14 Kastner M, R. Cardoso, Y. Lai, et al. Effectiveness of interventions for managing multiple high-burden chronic diseases in older adults: a

systematic review and meta-analysis. CMAJ. 2018;190(34):e1004. 15 Mallow J, S. Davis, J. Herczyk, N., et al. Dose of telehealth to improve community-based care for adults living with multiple chronic

conditions: a systematic review. Ehealth Telecommun Syst Netw. 2021;10:20-39. 16 Kilbourne AM, D. Hynes, T. O’Toole, et al. A research agenda for care coordination for chronic conditions: aligning implementation,

technology, and policy strategies. Transl Behav Med. 2018;8(3):515-521. 17A. Mohandas, J. Reifsnyder, M. Jacobs and T. Fox Current and future directions in frailty research. Popul Health Manag. 2011;14(6):277-283.

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care providers with home- and community-based long-term care services, to provide comprehensive

care to community-dwelling frail, older adults.1,17 Stakeholders also identified a need for evaluating

single and multicomponent nonpharmacologic interventions to maintain functioning and quality of life

of older adults with Alzheimer’s and related dementias. Several nonpharmacologic interventions (e.g.,

diet/nutrition, music, art, exercise, cognitive retraining) have been found to be efficacious but are in

need for larger scale evaluations and tailoring to address the needs of underserved populations.

Reduce Caregiving Burden and Improve Quality of Life

Meeting the needs of caregivers of older adults will not be a one-size-fits-all approach. Requirements of

caregivers vary greatly across the trajectory of caregiving, depending on the illness (type and/or

severity) of the care recipient.9,18 Caregivers themselves also represent individuals from all ages and

backgrounds, with one in every five Americans fulfilling a caregiving role in a given year.9,19,20,21,22,23,24,25,26

As the nation develops the first national family caregiving strategy as a result of the RAISE Family

Caregivers Act, there is a critical need for evidence to inform which programs most effectively support

caregivers.27 Stakeholder discussions highlighted the need for identifying effective training and

education interventions to support caregivers to perform their role, including complex medical tasks

that increasingly fall on them to manage at home, and evaluating psychosocial interventions to support

caregiver coping and mental and physical well-being. Also insufficient is research that identifies the

impact of tailored interventions that recognize multicultural and age differences, with caregivers

spanning generations creating a heterogeneous informal caregiver role.

Research Questions

PCORI seeks to fund rigorous, high-quality, and impactful studies that address the following research

questions that align with the four domains of interest in this tPFA.

1. What are the most effective approaches to maintaining function and independence among older

adults living in the community across different phases of the aging continuum?

18 T. A. Harvath, J. M. Mongoven, J. T. Bidwell, et al. Research priorities in family caregiving: process and outcomes of a conference on family-

centered care across the trajectory of serious illness. Gerontologist. 2020;60(suppl 1):S5-S13. 19 M. Leng, Y. Zhao, H. Xiao, et al. Internet-based supportive interventions for family caregivers of people with dementia: systematic review

and meta-analysis. J Med Internet Res. 2020;22(9):e19468. 20 D. A. Lindeman, K. K. Kim, C. Gladstone , et al. Technology and caregiving: emerging interventions and directions for research. Gerontologist.

2020;60(suppl 1):S41-S49. 21 Schulz R, Eden J, eds. Families Caring for an Aging America: A Report of the National Academies of Sciences, Engineering, Medicine. National

Academy of Sciences; 2016. 22 M. Y. Nilsson, S. Andersson, L. Magnusson , et al. Ambient assisted living technology-mediated interventions for older people and their informal carers in the context of healthy ageing: a scoping review. Health Sci Rep. 2021;4(1):e225. 23 C. J. Treanor, O. Santin, G. Prue, H. , et al. Psychosocial interventions for informal caregivers of people living with cancer. Cochrane Database Syst Rev. 2019;6: CD009912. 24 E. Gonzalez-Fraile, J. Ballesteros, J. R. Rueda et al. Remotely delivered information, training and support for informal caregivers of people with dementia. Cochrane Database Syst Rev. 2021;1:CD006440. 25 Butler M, Gaugler, JE, Talley KMC, et al. Care Interventions for People Living With Dementia and Their Caregivers. Agency for Healthcare

Research and Quality; 2020. 26 US Preventive Services Task Force. Screening for cognitive impairment in older adults: US Preventive Services Task Force recommendation

statement. JAMA. 2020;323(8):757-763. 27 Administration for Community Living Family Caregiving Advisory Council. Final Recommendations. Administration for Community Living

Family Caregiving Advisory Council; 2020.

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2. What are the most effective approaches to facilitating management of chronic/multiple chronic

conditions among older adults living in the community?

3. What are the most effective approaches to supporting older adults in the community who have

significant functional (physical and/or cognitive) impairment maintain their quality of life?

4. What are the most effective approaches to reducing caregiver burden and improving quality of life

of caregivers caring for older adults across different phases of the aging continuum?

Specific Requirements for This Funding Announcement

Applicants responding to the tPFA should strive to meet the following requirements:

General

• Applicants should focus on a comparative effectiveness question that is important to older

adults and their caregivers in one or more of the following domains: (a) Maintain function and

independence, (b) Facilitate chronic disease management, (c) Support individuals with

significant functional impairment, and (d) Reduce caregiving burden and improve quality of life.

• Applicants should demonstrate sufficient engagement with patients, caregivers, clinician or

healthcare organizations, payers or purchasers, community organizations, and other relevant

stakeholders to determine if the study is answering a critical question.

• Applicants are strongly encouraged to propose individual or cluster randomized controlled trials

(RCTs); however, PCORI will also consider well-specified natural experiments to be responsive.

Applicants should also pay special attention to issues of intervention implementation with an

aim of facilitating widespread uptake of findings after completion of the study. To that end,

PCORI encourages hybrid type I or hybrid type II designs, mixed-methods approaches, or RCTs

with a qualitative component.

Population

• PCORI anticipates that most proposed studies will focus on Medicare-eligible older adults;

however, studies enrolling younger populations will be considered with appropriate

justification. This exception acknowledges that some populations may experience the phases of

aging at chronologically earlier ages and that some diseases that are a significant burden to

older adults begin at earlier ages.

• PCORI encourages studies focusing on the needs of underserved, underrepresented, or

historically excluded populations who would benefit from health equity approaches (e.g., 1)

rural-dwelling older adults, 2) individuals with low income, 3) racial/ethnic minorities, 4)

individuals with low health literacy and/or limited English proficiency, or 5) those living in

unstable circumstances). In addition, studies that provide knowledge about an intervention’s

impact on multiple subgroups (i.e., studies that are adequately powered for evaluating

heterogeneity of treatment effects) are encouraged.

• In terms of the caregiver population, a caregiver is a family member or close friend serving as

the primary caregiver to an older adult. These individuals typically provide care informally

without compensation. PCORI strongly recommends including caregivers for the first and second

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domains (Maintain function and independence, Facilitate chronic disease management), and

caregiver inclusion is required for the third domain (Support individuals with significant

functional impairment). Ideally, caregivers should be enrolled in all studies; however, we

acknowledge that some types of interventions (e.g., physical activity, nutrition) may not be

accompanied by high caregiver burden. That said, for studies proposing not to include caregivers

for domains one and two, applicants should justify such exclusion in the Letter of Intent (LOI)

and subsequent full application if their LOI is approved.

Interventions

• Applicants should propose interventions and comparators that have evidence of prior efficacy or

are in widespread use within appropriate settings and populations. Justification of prior efficacy

or widespread use could include, but is not limited to, findings from pilot studies, clinical trials,

literature reviews, clinical practice guidelines, and standardized usual care protocols. If usual

care is included as a comparator, it must be well justified, well defined, and sufficiently

measured. Please see additional guidance below under the section of General Requirements for

PCORI Research.

• In addition to intervening individually on older adults or their caregivers, this tPFA encourages

studies that evaluate interventions that focus on the older adult-family caregiver unit and are

powered for both individual- and family-level outcomes as appropriate. Studies focusing on

multiple family caregivers will be considered responsive. Furthermore, the second and third

domains (Facilitate chronic disease management; Support individuals with significant functional

impairment) may also evaluate interventions that include formal caregivers, in addition to

caregivers as defined above.

Outcomes

Applications should propose well-justified outcomes that are clinically meaningful and considered

important by patients and/or their caregivers and upon which impact by proposed interventions can be

assessed during the duration of the CER study. Examples of possible outcomes include, but are not

limited to:

• Physical function measures (activities of daily living, instrumental activities of daily living,

mobility)

• Mental health measures (depression, anxiety, social isolation, cognitive function)

• Older adult and caregiver quality of life and caregiver stress/burden

• Delay to institutionalization and healthcare utilization

• Additional clinical measures (e.g., blood pressure control, antipsychotic use)

(Note: Not all of these example outcomes may be relevant to every study.)

Investigators may be asked to harmonize outcomes, where possible, across multiple studies funded as

part of this tPFA. For more information on cost-related outcomes, please review the Principles for

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Consideration of Full Range of Outcomes Data in PCORI Funded Research section below.

Timing

• Studies should include well-justified follow-up periods to generate evidence for dissemination

and implementation of proposed interventions after study completion.

Settings

• This tPFA focuses on interventions that may be delivered in outpatient clinical, home, or

community-based settings. Study settings can include primary and specialty care clinics; homes

of older adults or their caregivers; and community settings such as assisted living, senior

housing, retirement facilities, and adult daycare centers. Interventions may be delivered across

multiple settings. Multisite studies (e.g., across multiple sites within a health system, preferably

in diverse geographies; across multiple health care systems; across multiple communities) are

required in order to support widespread implementation of effective interventions. A study with

interventions exclusively delivered in institutional and in-patient settings (e.g., nursing homes,

hospitals, mental health facilities) will be considered nonresponsive.

• Foreign organizations and nondomestic components of organizations based in the United States

may apply for PCORI funding if there is demonstrable benefit to the US healthcare system and if

the essential scientific needs will be met by conducting the study outside the United States or

having the study conducted by non-US research organizations. The proposed study must

demonstrate meaningful effort and involvement of US organizations and investigators with

pertinent expertise and experience to contribute to the project. US efforts in the area of patient-

centered research must be clearly shown, the engagement plans must include US patients and

stakeholders, and the research must be relevant to the US healthcare system

(https://www.pcori.org/funding-opportunities/what-who-we-fund). However, PCORI’s

programmatic research priority for this tPFA is to identify the utility of interventional impacts on

US older adult populations—and particularly on Medicare- and Medicaid-eligible/covered

populations. Therefore, PCORI expects all sites of research in this PFA to be based in the United

States. Exceptions could be made for foreign institutions’ participation, contingent upon a clear

benefit or applicability to the US system that is demonstrated in the application.

Funds Available and Duration of Studies

PCORI has allotted up to $50 million under this tPFA to fund high-quality CER studies that respond to the

research questions of interest. The proposed budget for studies under this initiative may be up to $5

million in direct costs, as appropriate.

Even though the maximum allowable project period is five years (60 months), not all studies may require

five years to complete. PCORI encourages studies with rigorous, well-justified design and analysis plans

that can be completed in a compact timeframe. An appropriate timeframe for the research question and

study design should be chosen in consultation with stakeholders.

PCORI seeks efficient studies, such as those that take advantage of large populations already under

observation; registries; research cooperatives and networks; and the supportive involvement of delivery

https://www.pcori.org/funding-opportunities/what-who-we-fund

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systems or health plans to enhance recruitment, data collection, and coverage of intervention-related

costs.

II. General Requirements for PCORI Research

This section includes language that is specific to PCORI’s requirements for programmatic responsiveness

under this funding announcement. Applicants should use this section as guidance when preparing their

applications. For information related to administrative and technical requirements for LOI and

application submission, please consult the PCORI Submission Instructions.

Research Priorities

To be considered responsive, applications must:

• Describe comparators. Regardless of the approach being studied, all proposed research projects

must compare at least two alternatives. If the 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). 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 above to ensure that it

accounts for geographic and temporal variations, and it has wide interpretability, applicability,

and reproducibility.

• Describe research that compares two or more alternatives, each of which has established efficacy.

PCORI expects the efficacy or effectiveness of each intervention to be known. If the efficacy or

evidence base is insufficient, then data need to be provided to document that the intervention

is used widely. The application must provide information about the efficacy of the interventions

that will be compared; pilot data might be appropriate. Projects aiming to develop new

interventions that lack evidence of efficacy or effectiveness will be considered out of scope.

• Describe research that studies the benefits and harms of interventions and strategies delivered in

real-world settings. PCORI is interested in studies that provide practical information that can

help patients and other stakeholders make informed decisions about their health care and

health outcomes.

• Describe consultation with patients and other stakeholders about how the study is answering a

critical question. Explain the pertinent evidence gaps and why the project questions represent

decisional dilemmas for patients, caregivers, clinicians, policy makers, and other healthcare

system stakeholders. Describe why project outcomes are especially relevant and meaningful

endpoints to patients and other stakeholders.

Categories of Non-responsiveness

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

https://www.pcori.org/sites/default/files/PCORI-2021-Cycle-3-Aging-Submission-Instructions.pdf

10


• Instrument development, such as new surveys, scales, etc.

• Developing, testing, and validating new decision aids and tools, or clinical prognostication tools

• Pilot studies intended to inform larger efforts

• Comparing patient characteristics rather than clinical strategy options

• For the purpose of the Healthy Aging PFA, studies that evaluate the role of community health

workers or patient navigators as their primary focus will be considered nonresponsive. However,

studies that integrate community health workers or patient navigators as part of multi-

component health system interventions are allowed.

Consistent with PCORI's authorizing law,28 PCORI does not fund research whose findings will include:

• 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

Further, consistent with past funding announcements, PCORI will consider an application nonresponsive if the proposed research does the following:

• Conducts a formal cost-effectiveness analysis of alternative approaches to providing care

• Directly compares the costs of care between two or more alternative approaches to providing care, or relies on modeling to develop estimates of “total costs of care” designed to enable such comparisons

Principles for Consideration of Full Range of Outcomes Data in PCORI Funded Research

PCORI’s authorizing law was amended by reauthorization legislation in 2019 to include a new mandate

to consider, as appropriate, the full range of clinical and patient-centered outcomes data relevant to

patients and stakeholders. The reauthorizing language clarifies that, in addition to the relevant health

outcomes and clinical effectiveness, relevant outcomes included within PCORI-funded projects may

include the potential cost burdens and economic impacts of the utilization of medical treatments, items,

and services when relevant to patients and caregivers or to other stakeholders. The parameters for

appropriately including such outcomes are further described below and in the accompanying FAQs.

PCORI’s intention is that PCORI-funded research will, when germane, capture such cost burdens and

28 Available at http://www.pcori.org/sites/default/files/PCORI_Authorizing_Legislation.pdf/.

http://www.pcori.org/sites/default/files/PCORI_Authorizing_Legislation.pdf

https://www.congress.gov/bill/116th-congress/house-bill/1865

11


economic impacts to provide the full range of outcomes data relevant to decision makers.

Specifically, applications responding to this PFA may include the following:

• Data collection on cost burdens or economic impacts associated with interventions that

are relevant to patients and caregivers. Examples of elements of cost burden and economic

impacts important to patients and caregivers include patient time in hospital, caregiver time

away from work, cost and time for transport, childcare and eldercare costs, and medical out-of-

pocket costs.

• Data collection on cost burdens and economic impacts relevant to other stakeholders, when

these outcomes have a near-term or longer-term impact on patients, such as cost of

treatment/intervention, costs associated with impacts of treatment on healthcare utilization,

costs of a new intervention (program costs), and employer burden.

PCORI-funded studies have often included impacts of healthcare utilization, and data that capture the

costs of these impacts will now be considered responsive. However, proposed research may not

measure economic impacts as the primary outcome of a proposed study. Proposals that have economic

measures as the primary outcome will be considered nonresponsive.

For further information, please reference our cost-effectiveness analysis FAQs. PCORI has a continued interest in studies addressing questions about conditions that lead to high costs

to individuals or society. This interest is reflected in our review criterion on the condition’s impact on

the health of individuals and populations. Thus, as addressed in the cost-effectiveness analysis FAQs,

PCORI is interested in studies that do the following:

• Examine the effect of costs on patients, such as patients’ out-of-pocket costs, hardship, or lost

opportunity, or costs as a determinant of, or barrier to, access to care.

• Address cost-related issues, such as the resources needed to replicate or disseminate a

successful intervention.

• Evaluate interventions to reduce health system waste or increase health system efficiency.

In March 2021, PCORI’s Board of Governors approved Principles for the Consideration of the Full Range

of Outcomes. These Principles will inform both PCORI’s expectations for applicants and the

corresponding review evaluation of applications submitted in response to this PFA.

Coverage of Intervention Costs

In general, PCORI will not cover costs for study interventions that constitute the procedures, treatments,

interventions, or other standard clinical care (“patient care”) that are being proposed for comparison in

the research project (“patient care costs”).

Avoiding Redundancy

PCORI encourages potential applicants to review funded research at pcori.org. We intend to balance our

https://help.pcori.org/hc/en-us/sections/200565804-Cost-Effectiveness-Analysis

https://www.pcori.org/sites/default/files/PCORI-Principles-for-Consideration-of-Full-Range-of-Outcomes-Data-in-PCORI-Funded-Research.pdf

https://www.pcori.org/sites/default/files/PCORI-Principles-for-Consideration-of-Full-Range-of-Outcomes-Data-in-PCORI-Funded-Research.pdf

https://pcori.sharepoint.com/science/PFA%20Development/2017%20Cycle%203/Broad%20PFAs%20&%20Specific%20Templates/pcori.org

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funded portfolio to achieve synergy and avoid redundancy where possible.

Methodological Considerations

The PCORI Methodology Standards represent minimal requirements for the design, conduct, analysis,

and reporting of scientifically valid, patient-centered outcomes research. Regardless of study design,

applications must adhere to all relevant PCORI Methodology Standards, and all deviations need to be

justified. Applicants should address additional best practices—including relevant guidelines for

conducting clinical trials developed by other organizations—in the application for PCORI funding. For

this PFA, applicants should pay special attention to PCORI’s methodological standards for complex

interventions, mixed methods research, and qualitative methods as appropriate.

Patient-Centered Outcome Measures

PCORI encourages investigators to design their research using validated outcome measures. Include

preliminary data that support using the proposed measures in the study population. We encourage

investigators to consider those measures described in the Patient-Reported Outcomes Measurement

Information System29 (PROMIS). Likewise, PCORI encourages the use of core outcome sets, such as those

developed by the Core Outcomes Measures in Effectiveness Trials Initiative to facilitate cross-study

analysis. See http://www.comet-initiative.org/.

Leveraging Existing Resources, Including PCORnet

PCORI encourages applicants to consider the potential merits of using the clinical research

infrastructure and data resources of PCORnet®, the National Patient Centered Clinical Research

Network. PCORnet® is designed to improve the nation’s capacity to conduct efficient large-scale clinical

research, by drawing upon a network of clinical research networks capturing the healthcare experiences

of millions of Americans. PCORnet enables connections to patients, clinicians, researchers, and health

systems across the country interested in supporting research to improve health care and health

outcomes.

The network currently includes clinical research networks (CRNs) and a coordinating center. PCORnet

infrastructure resources include access to electronic health record (EHR) data from over 60 million

patients across the United States, encompassing a diverse range of care settings including hospitals,

primary care practices, outpatient specialty care practices, emergency and urgent care centers, federally

qualified health centers and community clinics. PCORnet can be a source of research-ready clinical sites

to enroll participants for feasibility testing of recruitment or for participation in a full-scale trial; it

can also be used to provide preparatory data in support of clinical trial design through its large

longitudinal data sets that capture clinical outcomes and details of specific procedures, treatments,

disease severity, and comorbid illnesses.

PCORnet offers:

• Clinical research networks that are able to participate as clinical sites in randomized research

trials

29 Available at http://www.nihpromis.org/.

https://www.pcori.org/research-results/about-our-research/research-methodology/pcori-methodology-standards

http://www.pcori.org/research-results/research-methodology/pcori-methodology-report

http://www.nihpromis.org/

http://www.nihpromis.org/

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• Actively engaged patients, clinicians, and health systems

• Preexisting, standardized, curated, and research-ready clinical data to inform clinical trial design,

conduct, and operations

• A readiness among network members to collaborate and a willingness to share data in pursuit of

patient-centered research aims

Outside of participation as clinical sites, PCORnet data resources may inform aspects of clinical

trial design, feasibility assessment, effect sizes, and potential study power. Examples include, but are not

limited to, the following:

• Providing background to the research question or feasibility of study

• Documenting the importance of the research question

• Estimating the size of the potentially eligible population

• Determining the range of current treatment practices and sequencing

• Assessing the duration of continuous treatment, care, and follow-up

Applicants interested in using PCORnet resources are encouraged to contact the PCORnet Front Door, to

explore opportunities for collaboration.

Patient and Stakeholder Engagement

In PCORI-funded research, patients and other healthcare stakeholders are viewed as partners who

leverage their lived experience and/or professional expertise to influence research to be more patient

centered, relevant, and useful. When developing an engagement strategy, PCORI encourages applicants

to consider the time and resources needed to identify, confirm, and prepare stakeholders for

collaborating; the infrastructure needed to manage stakeholder engagement activities; and the specific

decision points that will draw on the expertise of stakeholder partners. Research partners must include

representatives of the populations most impacted by the condition or issue addressed by the study.

Applicants’ use of multiple approaches that are along a continuum of engagement from input to shared

leadership are allowable and encouraged.30 For example, study teams may find it useful to solicit input

from a large group of stakeholders using quick-turnaround methods (e.g., focus groups, surveys, crowd-

sourcing, virtual or in-person roundtables and community forums) in addition to engaging stakeholders

via ongoing consultative groups (e.g., advisory committees, working groups), collaborative

arrangements, and leadership positions (e.g., co-investigators, multidisciplinary steering committees)

that are sustained over the course of the study.

Applicants should provide an overview of their engagement approach that should include (1) a proposed

list of patient and other healthcare research partners (include names and affiliations, if available), the

perspectives they will represent, and justification for their inclusion; (2) the goals for working with

30 Forsythe LP, Carman KL, Szydlowski V, et al. Patient engagement in research: early findings from the Patient-Centered Outcomes Research

Institute. Health Aff (Millwood). 2019;38:359-367. https://doi.org/10.1377/hlthaff.2018.05067

https://pcornet.org/front-door/

https://www.pcori.org/about-us/our-programs/engagement/public-and-patient-engagement/pcoris-stakeholders

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stakeholders, which may include affecting the acceptability, feasibility, rigor, and/or relevance of the

study; and (3) a description of how the team will collaborate with and/or gather input from stakeholders

at key decision points throughout the study. Funded awardees are required to submit a more detailed

engagement plan six months after contract execution.

Populations Studied and Recruited

PCORI seeks to fund research that includes diverse populations with respect to age, gender, race,

ethnicity, geography, or clinical status, so that possible differences in outcomes may be examined in

defined subpopulations. PCORI recognizes that some proposed studies might represent important PCOR

opportunities, even in the absence of a broadly diverse study population. However, the burden is on the

applicant to justify the study’s importance in the absence of diversity; to discuss which subgroups are

most important; and to discuss how the subgroups will be analyzed, including whether or not the study

will be powered to examine the question of effectiveness in subgroups.

PCORI is particularly interested in including previously understudied populations for whom effectiveness

information is especially needed, such as hard-to-reach populations or patients with multiple conditions.

Thus, comparisons should examine the impact of the strategies in various subpopulations, with

attention to the possibility that the strategy’s effects might differ across subpopulations. PCORI has

developed the following list of populations of interest to guide our efforts in research and engagement.

(Note that the Addressing Disparities Priority Area requires that proposed research focus on at least one

of the groups indicated by an asterisk below.)

• Racial and ethnic minority groups*

• Low-income groups*

• Women

• Older adults (age 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, numeracy, or limited English proficiency*

• Gender and sexual minorities*

• Veterans and members of the Armed Forces and their families

Regardless of the population studied, investigators are expected to provide evidence-based estimates

regarding the representativeness of the potential pool of participants from which recruitment will occur;

the target sample size; and recruitment and retention rates, reflecting the study’s inclusion and

exclusion criteria as well as factors that may impact the final sample size (e.g., loss to follow-up).

https://www.pcori.org/sites/default/files/PCORI-Updated-Engagement-Plan-Template.pdf

15


Protection of Human Subjects

PCORI follows the Federal Regulation for the Protection of Human Subjects (45 CFR part 46), including

the Common Rule. For more detailed information, please see Section 5, titled “Human Subjects

Research Policy,” in the Supplemental Grant Application Instructions for All Competing Applications and

Progress Reports,31 which is issued by the U.S. Department of Health and Human Services (HHS). In

referencing the HHS Supplemental Grant Application Instructions, note that PCORI does not require that

applicants comply with sections of that policy that refer to requirements for federal-wide assurance and

the inclusion of women, minorities, and children in the proposed studies. Instead, PCORI expects

applicants to address diversity in study participants in the research plan, through a focus on

subpopulations, as described in the above section on Populations Studied and Recruited. Awardees

must also comply with appropriate state, local, and institutional regulations and guidelines pertaining to

the use of human subjects in research.

PCORI requires awardees to ensure that there is a Data and Safety Monitoring Plan, which may include

the need to appoint a Data and Safety Monitoring Board, as provided in the PCORI Policy on Data and

Safety Monitoring Plans for PCORI-Funded Research.32

PCORI merit reviewers will examine plans for protection of human subjects in all applications and may

provide comments regarding the plans (see How To Evaluate Human Subjects Protections33). Reviewers’

comments on human subject research are not reflected in the overall application score, but PCORI staff

might use them during potential funding negotiations. Final determinations about the adequacy of

human subject protections rest with the Institutional Review Board or international equivalent that have

jurisdiction for the study.

The Awardee Institution, whether domestic or foreign, bears ultimate responsibility for safeguarding the

rights and welfare of human subjects in PCORI-supported activities.

Required Education of Key Personnel on the Protection of Human Subject Participants

PCORI requires that all applicants adhere to the National Institutes of Health (NIH) policy on education in

the protection of human subject participants in the conduct of research. This applies to all individuals

listed as key personnel in the application. The policy and FAQs are available on the NIH website.34

III. LOI Review

Applying for funding for this PFA is a two-stage process. An LOI must be submitted, and an applicant

must be invited to submit an application.

LOIs are evaluated based on the following:

• Importance and relevance of the topics to PCORI priorities, as evidenced by critical gaps

identified by clinical guidelines developers and recent systematic reviews

31 See http://grants.nih.gov/sites/default/files/supplementalinstructions.docx 32 See http://www.pcori.org/sites/default/files/PCORI-Policy-Data-Safety-Monitoring-Plans.pdf 33 See http://www.pcori.org/sites/default/files/PCORI-Checklist-for-Evaluating-Human-Subjects-Protections.pdf/. 34 See http://grants.nih.gov/grants/guide/notice-files/NOT-OD-08-054.html.

http://www.hhs.gov/ohrp/policy/ohrpregulations.pdf

http://grants.nih.gov/sites/default/files/supplementalinstructions.docx


http://www.pcori.org/sites/default/files/PCORI-Policy-Data-Safety-Monitoring-Plans.pdf

http://www.pcori.org/sites/default/files/PCORI-Policy-Data-Safety-Monitoring-Plans.pdf

http://www.pcori.org/sites/default/files/PCORI-Checklist-for-Evaluating-Human-Subjects-Protections.pdf

http://grants.nih.gov/grants/guide/notice-files/NOT-OD-08-054.html


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• Clarity and credibility of applicants’ responses to the LOI questions

• The investigators’ prior relevant experience

• Programmatic fit and balance, considering whether the LOI overlaps with previously funded

studies or concurrent LOIs and/or applications to a significant degree or, conversely, whether

the application fills a gap in PCORI’s funded portfolio with certain characteristics, including

disease category, topics, priority populations, methodologies, and other variables

Only applicants whose LOIs are deemed most responsive to this PFA will be invited to submit a full

application. A minimum of two PCORI staff members review the LOIs, which are not scored during

review.

The LOI Template provides guidance on responding to each item. Please refer to the Submission

Instructions for information on how to submit an LOI via PCORI Online.

IV. Merit Review

PCORI’s merit review process is designed to support the following goals:

• Identify applications that have the strongest potential to help patients, caregivers, clinicians,

policy makers, and other healthcare system stakeholders make informed decisions to improve

patient outcomes.

• Implement a transparent, fair, objective, and consistent process to identify these applications.

• Elicit high-quality feedback that reflects a diversity of perspectives to ensure that the PCORI-

funded research reflects the interests and views of patients and other stakeholders and those

who care for them, and that it meets the criteria for scientific rigor.

• Fund projects that fill important evidence gaps and have strong implementation potential.

• Regularly evaluate and continually improve the merit review process and policies in support of

PCORI's mission.

PCORI merit review is a multiphase process that includes the review panel’s preliminary review of full

applications and an in-person panel discussion of a subset of applications (identified by PCORI’s Program

staff and based on the preliminary review and program priorities). After merit review, key steps include:

post-panel review of application by PCORI staff; the Selection Committee’s recommendation of

applications for funding; and, finally, Board award approval.

Preliminary Review

PCORI conducts rigorous merit review of the full applications it receives. Note that PCORI may eliminate

applications from the review process for administrative or scientific reasons (e.g., non-responsiveness).

An application may be administratively withdrawn if it is incomplete; submitted past the stated due date

and time; or does not meet the formatting criteria outlined in the Submission Instructions, in the PCORI

templates, and in PCORI Online. An application may be scientifically withdrawn if it is not responsive to

the guidelines described in this PFA, describes research that is not comparative, includes a cost-



https://pcori.force.com/engagement


17


effectiveness analysis, or otherwise does not meet PCORI programmatic requirements.

PCORI Merit Review Officers (MROs) recruit each review panel based on the number of invited LOIs and

topic areas represented by the invited LOIs. MROs recruit the panel chair, scientist reviewers who are

subject matter experts, patient representatives, and representatives of other stakeholder groups. All

panel members receive training during the review cycle to ensure that they understand the

programmatic and organizational goals of review.

Below are PCORI’s merit review criteria. PCORI’s merit review panels use these criteria during the

preliminary and in-person review phases to evaluate and score all submitted applications, and to ensure

consistency and fairness in how applications are evaluated.

Criterion 1. Potential for the study to fill critical gaps in evidence:

The application should address the following questions:

• Does the application convincingly describe the clinical burden?

• Does the application identify a critical gap in current knowledge as noted in systematic reviews,

guideline development efforts, or previous research prioritizations?

• Does the application identify a critical gap in current knowledge, evidenced by inconsistency in

clinical practice and decision making?

• Would research findings from the study have the potential to fill these evidence gaps?

Criterion 2. Potential for the study findings to be adopted into clinical practice and improve delivery of

care

The application should describe how evidence generated from this study could be adopted into clinical

practice and delivery of care by others. The application should also address the following questions:

• Does the application identify who will make the decision (i.e., the decision maker) or use (i.e.,

the end-user) the study findings (not the intervention) this study produces, such as local and

national stakeholders?

• Does the application identify potential end-users of study findings—such as local and national

stakeholders—and describe strategies to engage these end-users?

• Does the application provide information that supports a demand for this kind of a study from

end-users?

• Would this study’s research findings have the potential to inform decision making for key

stakeholders? If so, provide an example. How likely is it that positive findings could be

reproduced by others, resulting in improvements in practice and patient outcomes? Identify the

potential barriers that could hinder adoption of the intervention by others.

• Does the application describe a plan for how study findings will be disseminated beyond

publication in peer-reviewed journals and at national conferences? Does the application

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describe a clear path for future efforts to facilitate widespread uptake of research findings after

completion of the study?

Criterion 3. Scientific merit (research design, analysis, and outcomes)

The application should show sufficient technical merit in the research design to ensure that the study

goals will be met. The application should also address the following questions:

• Does the application describe a clear conceptual framework anchored in background literature

which informs the design, key variables, and relationship between interventions and outcomes

being tested?

• Does the Research Plan describe rigorous methods that demonstrate adherence to the PCORI

Methodology Standards?

• Is the overall study design justified?

o If a hybrid study design is proposed, are the chosen implementation strategies

appropriate?

o Are the proposed activities likely to result in successful uptake of study findings?

• Are the patient population and study setting appropriate for the proposed research question?

• Does the application provide justification that the outcome measures are validated and

appropriate for the population?

• Are each of the comparators (e.g., active intervention arm and comparator arm) described

clearly and well-justified? If “usual care” is one of the arms, is it adequately justified, and will it

be sufficiently measured?

• Are the sample sizes and power estimates appropriate? Is the study design (e.g., cluster

randomized design, randomized controlled trial, or observational study) accounted for and is the

anticipated effect size adequately justified?

• Is the study plan feasible? Is the project timeline realistic, including specific scientific and

engagement milestones? Is the strategy for recruiting participants feasible? Are assumptions

about participant attrition realistic, and are plans to address patient or site attrition adequate?

Criterion 4. Investigator(s) and environment

The application should demonstrate the appropriateness (e.g., qualifications and experience) of the

investigator(s)/team and the environment’s capacity (e.g., resources, facilities, and equipment) to

support the proposed project. Assessment of this criterion should not focus on the institution’s

reputation, but rather on the breadth and depth of its available personnel and resources. The

application should also address the following questions:

• How well-qualified are the PIs, collaborators, and other researchers to conduct the proposed

activities? Is there evidence of sufficient clinical or statistical expertise (if applicable)?

• Does the investigator or co-investigator have demonstrated experience conducting projects of a

19


similar size, scope, and complexity?

• If the project is collaborative or dual-PI, do the investigators have complementary and

integrated expertise? Are the leadership, governance, and organizational structures appropriate

for the project?

o (Dual-PI Option Only) Does the Leadership Plan adequately describe and justify PI roles

and areas of responsibility?

• Is the level of effort for each team member appropriate for successfully conducting the

proposed work?

• Does the application describe adequate availability of and access to facilities and resources

(including patient populations, samples, and collaborative arrangements) to carry out the

proposed research?

• Is the institutional support appropriate for the proposed research?

Criterion 5. Patient-centeredness

The application should demonstrate that the study focuses on improving patient-centered outcomes

and employs a patient-centered research design –that is a design informed or endorsed by patients.

(NOTE: The study can be patient-centered even if the end-user is not the patient, as long as patients will

benefit from the information.) The application should also address the following questions:

• Does the application include a thorough description about which outcomes (both benefits and

harms) are important to patients, and are those outcomes included in the study plan?

• Does the application provide information that indicates that closing the evidence gap is

important to patients and other stakeholders?

• Are the interventions being compared in the study available to patients now, and are they the

best options for comparison (including whether they would be chosen by patients and their

healthcare providers for managing the condition being studied)?

Criterion 6. Patient and stakeholder engagement

The application should demonstrate the engagement of relevant patients and other stakeholders (e.g.,

patients, caregivers, clinicians, policy makers, hospital and health system representatives, payers

[insurance], purchasers [business], industry, researchers, training institutions) in the conduct of the

study. Quality of engagement will be evaluated based on scope, form, and frequency of patient and

stakeholder involvement throughout the research process.

The application should also address the following questions:

• Does the application provide a well-justified description of how the research team incorporates

stakeholder involvement? Does the study include the right individuals (e.g., patients, caregivers,

clinicians, policy makers, hospital and health system representatives, payers, purchasers,

industry, researchers, and training institutions) to ensure that the projects will be carried out

successfully?

20


• Does the application show evidence of active engagement among scientists, patients, and other

stakeholders throughout the research process (e.g., formulating questions, identifying

outcomes, monitoring the study, disseminating, and implementing)? Is the frequency and level

of patient and stakeholder involvement sufficient to support the study goals?

• Is the proposed engagement approach appropriate and tailored to the study?

• Are the roles and the decision-making authority of all study partners described clearly?

• Are the organizational structure and resources appropriate to engage patients and stakeholders

throughout the project?

In-Person Review

During preliminary review, all administratively and scientifically compliant applications are evaluated

and scored by panels of external reviewers based on PCORI’s merit review criteria, including evaluation

of adherence to the PCORI Methodology Standards. After preliminary review, PCORI program staff

members evaluate panel scores and critiques to identify a subset of applications for merit reviewers to

discuss at the in-person review meeting. Not all submitted applications move forward to in-person

review.

During the in-person review, merit reviewers meet to discuss applications and to clarify further the

merits of the proposed research. They also identify areas for improvement. Each application is re-scored

based on the content of discussion. The Panel Chair and PCORI MRO lead the in-person panel meeting

and ensure that all applications receive a fair and thorough review according to the standards outlined

in the PFA.

Post-Panel Review

After the in-person meeting, PCORI program staff evaluate final merit review panel scores and

comments, identify duplication or synergy among funded projects, and consider the fit of applications

within the programmatic vision. Program staff members then recommend projects to a Selection

Committee, which includes members of the Board. The Selection Committee considers

recommendations and works with staff to identify a slate of applications for possible funding based on

merit review scores, programmatic balance and fit, and PCORI’s strategic priorities. This slate is then

proposed to the Board for consideration and approval.

In addition, PCORI evaluates applicant risk before issuing a PCORI award. Factors considered include

financial stability, quality of management systems, audit findings, and past performance on PCORI

awards (e.g., compliance with PCORI reporting requirements, conformance to PCORI terms and

conditions on previous awards, and timely achievement of milestones). Based on the risk assessment,

PCORI may impose special terms and conditions on awardees or withhold contract issuance until such

business risks are mitigated. PCORI will not award new contracts to current awardees with overdue

reports (progress, interim, final, etc.) until the overdue reports have been submitted to PCORI.

Summary Statements and Funding Recommendations

Summary statements are provided to applicants approximately two weeks before funding decisions are

announced. If an application progresses to in-person discussion, the applicant will receive a summary

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statement which will include:

• In-person panel discussion notes

• Final average overall score

• Preliminary reviewer critiques

• Application quartile, to help applicants understand how they did relative to other discussed

applications, as appropriate

Summary statements for applications that do not progress to in-person discussion include only the

preliminary reviewer critiques.

Funding recommendations are made by identifying meritorious applications that fit the programmatic

needs and that satisfactorily address the merit review criteria while adhering to the PCORI Methodology

Standards. Programs also consider the funds allotted for the current funding announcement when

deciding which applications to recommend to the Board for approval. Applicants to this current cycle’s

PFA will receive summary statements and notification of the funding status of their application no later

than July 2022.

V. PCORI Policies that Govern Awardees Related to Data Access, Privacy, and Public Reporting

Applicants should be aware that all PCORI awardees are required to comply with the following

requirements:

Registering Research Projects

PIs are required to use the naming convention “PCORI-PCORI application number” (i.e., PCORI-XXXX-

XXXXX). Clinical trials must be registered before enrollment of the first patient. All trials that meet the

definition on the NIH database35 (see Data Element Definitions) are required to register, if funded.

Funded clinical trials or observational outcomes studies must be registered at ClinicalTrials.gov.

Funded evidence-synthesis studies must be registered at PROSPERO.36 Funded patient registries must be

registered at https://patientregistry.ahrq.gov/.

PCORI Public Access Policy

PCORI requires all awardees to adhere strictly to PCORI’s publication policies, which will be shared with

awardees within the research contract.

Standards for Privacy of Individually Identifiable Health Information

On August 14, 2002, the Department of Health and Human Services issued a final modification to the

Standards for Privacy of Individually Identifiable Health Information, the “Privacy Rule.” The Privacy Rule

is a federal regulation under the Health Insurance Portability and Accountability Act (HIPAA) of 1996

35 Available at https://prsinfo.clinicaltrials.gov/. 36 Available at http://www.crd.york.ac.uk/prospero/.

http://prsinfo.clinicaltrials.gov/

http://prsinfo.clinicaltrials.gov/definitions.html

http://www.clinicaltrials.gov/

http://www.crd.york.ac.uk/prospero/

https://patientregistry.ahrq.gov/

22


that governs the protection of individually identifiable health information and is administered and

enforced by the Department of HHS Office for Civil Rights.

Decisions about the applicability and implementation of the Privacy Rule reside with the researcher and

his or her institution. The Office for Civil Rights37 provides information on the Privacy Rule, including a

complete Regulation Text and a set of decision tools related to “Am I a covered entity?” Information on

the impact of the HIPAA Privacy Rule on NIH processes involving the review, funding and progress

monitoring of grants, cooperative agreements, and research contracts is available from NIH.38

Data Management and Data-Sharing Plan

PCORI is committed to publishing and disseminating all information and materials developed using

PCORI funding, in accordance with its authorizing legislation. All recipients of PCORI contracts must

agree to these principles and take steps to facilitate data availability.

PCORI encourages openness in research and making research data available for purposes of replication

and reproducibility. As such, if an award is made, the awardee will be expected to adhere to PCORI’s

Policy for Data Management and Data Sharing. The Policy articulates PCORI’s requirement that certain

Awardees make the underlying data and data documentation (e.g., study protocol, metadata, and

analytic code) from their PCORI-funded research projects available to third-party requestors.

A full data management and data sharing plan is not required at the time of application. If an award is

made -- specifically for the Pragmatic Clinical Studies (PCS) and the targeted PFA studies -- the Awardee

is required to develop and maintain such a plan, which is described in detail in the PCORI Methodology

Standards for Data Integrity and Rigorous Analyses, specifically Standard IR-7. This plan must be

appropriate for the nature of the research project and the types of research project data, and consistent

with applicable privacy, confidentiality, and other legal requirements. The Policy includes details about

what data certain Awardees will be expected to deposit into a PCORI-designated data repository and

when that data would be available for third-party requests.

For research awards funded under Broad funding announcement (Assessment of Options, Improving

Healthcare Systems, Addressing Disparities, Communication and Dissemination Research, Improving

Methods), the Policy calls for Awardees to maintain the Full Data Package for seven (7) years. PCORI

may, in selective cases, notify the researcher of its intent to provide funds for the deposition of the Full

Data Package in a PCORI-designated repository in circumstances where PCORI requests such deposition.

The information here is meant for informational purposes only and does not attempt to be an

exhaustive representation of the Policy for Data Management and Data Sharing. Please refer to the

Policy in its entirety for additional information.

Peer Review and Release of Research Findings

PCORI has a legislative mandate to ensure the scientific integrity of the primary research it supports and

to make study findings widely available and useful to patients, clinicians, and the general public within a

specific timeframe. Accordingly, the PCORI Board of Governors (Board) adopted the Process for Peer

37 Available at http://www.hhs.gov/ocr/. 38 Available at http://grants.nih.gov/grants/guide/notice-files/NOT-OD-03-025.html.

http://www.hhs.gov/ocr

http://grants.nih.gov/grants/guide/notice-files/NOT-OD-03-025.html

https://www.pcori.org/blog/pcoris-new-policy-data-management-and-data-sharing-step-forward-open-science






http://www.pcori.org/sites/default/files/PCORI-Peer-Review-and-Release-of-Findings-Process.pdf

23


Review of Primary Research and Public Release of Research Findings.39

In summary, Awardee Institutions are required to submit to PCORI for peer review a draft final research

report that provides the methodological details, describes the main study results, and interprets the

findings in clinical or other decisional contexts. After Awardee Institutions have responded to reviewers’

comments to PCORI’s satisfaction, the report will be accepted and considered final. PCORI will then

prepare two 500-word standardized abstracts summarizing the study results (as detailed below), which

the Awardee Institution will review and approve.

No later than 90 days after the draft final research report is accepted, PCORI will post the following

materials on its website: (1) a 500-word abstract for medical professionals; (2) a 500-word standardized

abstract summarizing the study results for patients and the general public; (3) a link to the study record

on ClinicalTrials.gov (as applicable); and (4) ancillary information, including conflict of interest

disclosures. The final research report, along with anonymized reviewer comments, will be made publicly

available on the PCORI website no later than 12 months after its acceptance, except by prior mutual

agreement with the Awardee Institution.

39 See http://www.pcori.org/sites/default/files/PCORI-Peer-Review-and-Release-of-Findings-Process.pdf.

http://www.pcori.org/sites/default/files/PCORI-Peer-Review-and-Release-of-Findings-Process.pdf

https://clinicaltrials.gov/

Documents

PCORI Funding Announcement: Healthy Aging: Optimizing