21st Century Cures Act (2016) Regulatory

U.S. federal law that, among many provisions, formally established Patient Experience Data (PED) as part of FDA drug review and required FDA to issue the PFDD Guidance Series. The legal foundation for much of the current FDA patient engagement infrastructure. Pub. L. 114-255; FDA PFDD Guidance Series

Adaptive trial Research

Study design that allows modifications based on interim results — changing dose, adding arms, or stopping early. More flexible than fixed designs.

Adverse event (AE) / Serious adverse event (SAE) Research

An unfavorable medical occurrence in a study participant, whether or not caused by the intervention. SAEs (life-threatening, hospitalization, disability) trigger reporting to IRBs, sponsors, and regulators.

AHRQ — Agency for Healthcare Research and Quality Org

Federal agency funding comparative effectiveness research and evidence-based guidelines. Increasingly emphasizes patient engagement in research priority-setting.

Aim / Specific Aim Research

The discrete research questions a grant proposes to answer. Specific Aims are the heart of an NIH application; PWLE input has highest leverage when given before aims are set.

All of Us Research Program NIH

NIH’s million-person precision medicine cohort with substantial participant engagement infrastructure including Participant Ambassadors and Tribal Collaboration Working Group. allofus.nih.gov

Allocation concealment Research

Methods preventing researchers and participants from knowing in advance which intervention a participant will receive. Distinct from blinding, which applies after assignment.

AMCP — Academy of Managed Care Pharmacy Org

Professional organization that develops formulary evaluation guidelines increasingly incorporating patient-reported outcomes and PWLE perspectives in drug coverage decisions.

Arnstein’s Ladder of Citizen Participation Framework

Foundational 1969 framework describing participation from “manipulation” through “consultation” to “citizen control.” Still referenced in modern engagement frameworks. Arnstein 1969, J Am Inst Planners 35(4):216-224

Authorship (ICMJE criteria) Concept

International Committee of Medical Journal Editors criteria for who qualifies as a paper’s author: substantial contribution to design, drafting, final approval, and accountability. PWLE who contribute substantively increasingly meet these criteria but are often acknowledged rather than authored — if you’ve shaped the work, you can ask about authorship. ICMJE Recommendations, current edition; icmje.org

Benefit-risk assessment Regulatory

FDA/EMA framework weighing a treatment’s potential benefits against its risks. Increasingly incorporates patient preference information, especially in device approvals and rare-disease drug reviews. FDA Benefit-Risk Assessment Guidance, 2023

Benefits impact of compensation Concept

Being paid for advisory work can reduce or end means-tested benefits. Under SSI, honoraria are generally counted as earned income, and SSI eligibility usually carries Medicaid with it; under SSDI, earnings above the Substantial Gainful Activity threshold can trigger a continuing disability review. This is why some PWLE decline payment and never say why, and why “we’ll pay you” is not automatically good news. It is also why reimbursement and compensation are not interchangeable. Talk to a benefits counselor or a Work Incentives Planning and Assistance (WIPA) program before you accept — not after. Nothing in this entry is financial or legal advice. SSA; Congressional Research Service R44948

Bias (selection / measurement / confounding) Research

Systematic errors that distort study results. Selection = who gets into the study; measurement = how outcomes are captured; confounding = an unmeasured variable affecting both intervention and outcome.

Biomarker Research

A measurable biological indicator (lab test, imaging, genetic marker) used to track disease state or treatment response. Patient input often surfaces whether a biomarker tracks something patients actually care about.

Blinded / Masked trial Research

Study where participants don’t know which treatment they’re receiving. “Double-blind” means researchers also don’t know.

Burden Concept

The time, effort, and complexity demands placed on study participants or engaged PWLE. Good engagement design minimizes unnecessary burden.

Camden Coalition — Camden Coalition of Healthcare Providers Org

Nonprofit organization improving care for people with complex health needs. Pioneer in patient engagement and co-design methodologies for healthcare transformation.

Capacity building Concept

Training and support that prepares PWLE to engage effectively. Distinct from co-learning, which is bidirectional.

Caregiver / Care partner Role

A family member, friend, or informal supporter whose lived experience comes through caring for someone with a condition. Often engaged alongside PWLE.

Caregiver-reported outcome / ObsRO Research

Outcome measures reported by someone other than the patient — a family member, care partner, or clinician — used when the patient cannot self-report. Distinct from PRO. FDA PFDD Guidance 3, 2022

Carman Framework (2013) Framework

Widely cited framework defining patient engagement as a continuum across direct care, governance, and policy domains. Carman et al., Health Affairs 2013;32(2):223-231

Case report form (CRF) / Electronic CRF (eCRF) Research

The standardized form (paper or electronic) used to collect data from each participant in a study. PWLE review of CRFs often surfaces unnecessarily burdensome data collection.

CBPR — Community-Based Participatory Research Concept

Research approach involving community members as equal partners throughout — from question formulation to dissemination.

CDER — Center for Drug Evaluation and Research Regulatory

FDA division that regulates prescription and over-the-counter drugs. Houses the Patient-Focused Drug Development program.

CDMRP — Congressionally Directed Medical Research Programs Org

DOD-managed research funding with strong consumer (patient) advocate involvement in proposal review. The Consumer Reviewer program is widely considered the gold standard for PWLE in research funding decisions. cdmrp.health.mil

CDRH — Center for Devices and Radiological Health Regulatory

FDA division regulating medical devices. Increasingly incorporates patient perspectives through patient preference studies.

Central sensitization Research

A state in which the central nervous system amplifies pain signaling, so that pain persists, spreads, or is triggered by things that should not hurt; sometimes described as wind-up. Implicated in conditions including fibromyalgia and CRPS. Part of why “there is nothing on the scan” is not the same sentence as “there is nothing wrong.”

Clinical equipoise Concept

The ethical principle that a randomized trial is justified only when there’s genuine uncertainty about which treatment is better. PWLE may legitimately challenge whether “genuine uncertainty” exists.

Clinical Research Coordinator (CRC) Role

Staff member responsible for day-to-day study operations — recruitment, scheduling, data collection, participant communication. Often the most direct point of contact for participants.

ClinicalTrials.gov Research

U.S. registry of clinical trials. Most U.S. trials must register and post results, including a patient-friendly summary — review of these summaries before posting is high-impact, low-burden PWLE work. FDAAA 801 Final Rule (42 CFR 11)

CMS — Centers for Medicare & Medicaid Services Org

Federal agency administering Medicare and Medicaid. Coverage decisions increasingly informed by patient-reported outcomes and comparative effectiveness research.

Co-Investigator (Co-I) / Patient Co-Investigator Role

Partner with significant intellectual contribution, named on the grant with budgeted effort. Patient Co-I status is meaningful engagement; ask whether you’re being offered Co-I or just “advisor.”

Co-learning Concept

PCORI engagement principle: researchers and PWLE teach each other, expertise flows both ways. Distinct from “capacity building,” which is unidirectional. PCORI Engagement Principles

Co-production / Co-design Concept

Model where PWLE and professionals share decision-making power from the start of a project through completion. Co-design specifically refers to shared creation of tools or interventions.

Cohort Research

A group of people sharing a common characteristic, followed over time. Cohort studies are observational, not experimental.

COI — Conflict of Interest Concept

Financial or personal relationships that could bias judgment. Personal experience with the condition is generally not a COI — it’s the qualification — but financial relationships with sponsors usually need disclosure.

Common Fund programs NIH

NIH Director’s strategic initiatives crossing institute boundaries. Many include strong community engagement components (CEAL, INCLUDE, RECOVER). commonfund.nih.gov

Common Rule (45 CFR 46) Regulatory

Federal regulations governing human subjects research at all U.S. institutions receiving federal funding. Defines what counts as human subjects research, what triggers IRB review, what informed consent requires, and protections for vulnerable populations. The 2018 Revised Common Rule updated several provisions including broad consent for biospecimens. 45 CFR 46; HHS Office for Human Research Protections (OHRP)

Community Advisory Board (CAB) Role

Standing group broader than a Patient Advisory Council — includes PWLE, caregivers, and community members. Common in community-based participatory research.

Community Engagement Studio (CE Studio) Framework

A structured consultative session in which a research team brings a plan to a panel of community members with relevant lived experience and takes questions and recommendations before the design is fixed. Developed by the Meharry-Vanderbilt Community-Engaged Research Core in 2009 and now used across CTSA sites and beyond. Disease-agnostic and deliberately one-off, which is both its strength and its limit: it front-loads input, but it is a consultation, not a seat. Its value depends entirely on whether it happens before the decisions are locked. Joosten YA et al. Acad Med. 2015; Meharry-Vanderbilt CERC

Compensation / Honoraria Concept

Payment for PWLE time and expertise. A growing norm in ethical engagement; unpaid engagement is increasingly viewed as extractive. See also Fair Market Value, Compensation Framework, and Vendor status.

Compensation Framework (PCORI) Concept

PCORI’s structured guidance for compensating patient and partner contributions to research, recognizing engagement as expertise that should be paid. Distinct from but complementary to the National Health Council’s Fair Market Value calculator (rate-setting); the PCORI framework focuses on appropriate compensation by role and contribution type. PCORI Compensation Framework, current edition

Confidentiality / De-identification Research

Protections for participant data. De-identified data has had direct identifiers removed; limited datasets retain some indirect identifiers; fully identified data carries the strongest protections.

Consent (informed / re-consent / broad) Research

Process ensuring participants understand a study before agreeing. Re-consent = renewed agreement when the study changes; broad consent = agreement to future unspecified uses. PWLE review of consent forms is high-impact engagement work.

Consumer Reviewer (CDMRP model) Role

Patient or family member trained and credentialed by DOD CDMRP to serve as a voting member on grant review panels. Widely considered the gold-standard model for PWLE in research funding decisions.

Crossover trial Research

Study where participants receive multiple treatments in sequence, often with a “washout” period between treatments.

CTTI Patient Groups & Clinical Trials Report (2015) Framework

Landmark report establishing recommendations for pharmaceutical-patient organization partnerships in clinical research. Defines principles for ethical, meaningful collaboration.

CTTI — Clinical Trials Transformation Initiative Org

Public-private partnership developing evidence-based recommendations to improve clinical trial design and conduct. Emphasizes patient engagement and diversity.

Data and Safety Monitoring Plan (DSMP) Research

Required for clinical trials — describes how the study will monitor participant safety and data integrity. Smaller trials may have a DSMP without a full DSMB.

Data Safety Monitoring Board (DSMB) Role

Independent body monitoring trial safety; can recommend stopping a study early. Increasingly includes a patient/community representative — a high-trust role with real authority.

Data sharing plan Research

NIH-required plan describing how research data will be shared after study completion. PWLE input on data sharing plans is welcomed and often missing. NIH Data Management and Sharing Policy, 2023

Decentralized clinical trial (DCT) Research

A trial in which some or all activity happens away from a traditional site — at home, locally, or remotely, using telehealth, wearables, and local labs. Can cut burden dramatically for people who cannot travel. Can also move work onto participants and their care partners without anyone naming it as work. Both things are true, and both are worth saying out loud in a design meeting.

Decision aid / Decision-support tool Concept

Structured resource helping patients and clinicians make decisions together by presenting options, outcomes, and tradeoffs in plain language. PWLE are increasingly involved in co-designing them. International Patient Decision Aid Standards (IPDAS)

DEIA in clinical trials Regulatory

Diversity, Equity, Inclusion, and Accessibility — FDA now requires diversity action plans for many trial submissions. Engagement quality increasingly tied to whether engaged PWLE reflect the affected population. FDA Diversity Action Plans Guidance, 2024

Disparities research / Health equity research Research

Research focused on differences in health outcomes across populations and the social, economic, and structural causes of those differences. NIMHD is the lead NIH institute.

Dissemination and Implementation (D&I) science Research

The study of how research findings actually get used in practice. PWLE engagement is core to most D&I frameworks.

Effectiveness vs. efficacy Research

Efficacy = how well a treatment works under ideal conditions (typical Phase III). Effectiveness = how well it works in real-world clinical practice. Pragmatic trials test effectiveness.

EL-PFDD — Externally Led Patient-Focused Drug Development Regulatory

Patient-organization-led version of an FDA PFDD meeting. Often the most accessible direct route for a disease community to put patient experience data on the FDA record.

EMA — European Medicines Agency Regulatory

European drug regulator. Has established patient and consumer working parties and requires patient involvement in drug evaluation.

Endpoint (primary / secondary / exploratory) Research

The outcomes a study measures. Primary = determines trial success/failure. PWLE review of endpoints is one of the highest-leverage engagement activities — does the primary endpoint reflect what patients actually care about?

Engagement Plan Research

A required component of PCORI applications and an increasingly common requirement in NIH and industry-sponsored research. Describes who will be engaged, in what roles, at what stages, with what compensation, and how engagement quality will be assessed. PCORI awardees submit an Updated Engagement Plan within the project’s first six months — a key moment for PWLE input. PCORI Foundational Expectations; PCORI Updated Engagement Plan template

Engagement vs. Involvement vs. Participation Concept

NIHR distinction: Participation = being a study subject. Involvement = research with/by PWLE. Engagement = sharing research with the public. U.S. usage often collapses these. NIHR briefing notes for researchers

ePRO — Electronic Patient-Reported Outcome Research

PRO data collected electronically — apps, web platforms, wearables, in-clinic tablets. Raises specific engagement questions: digital access equity, recall windows, alert fatigue, and data ownership.

Equity in research Concept

The principle that research benefits and burdens should be fairly distributed, and that historically excluded groups should be meaningfully included in design and conduct, not just enrolled.

EUPATI Guidance Documents (four-part series) Framework

Four EUPATI guidance documents covering patient involvement in: (1) industry-led R&D, (2) ethics committees, (3) regulatory authorities, and (4) HTA bodies. Together they form one of the most comprehensive published frameworks for patient involvement across the medicines lifecycle. > EUPATI Guidance Documents (2018), patientacademy.eu.*

EUPATI — European Patients’ Academy on Therapeutic Innovation Org

Pan-European initiative providing education and training for patient advocates. Originator of the term “Patient Expert.” patientacademy.eu

Exclusion criteria Research

Rules determining who cannot participate in a study. Often where PWLE review surfaces problems — exclusions that don’t reflect real-world patient populations.

Experiential knowledge / Lived experience expertise Concept

The category of knowledge PWLE bring — distinct from clinical or scientific training, but no less rigorous as evidence about what living with a condition entails. Useful term when pushing back against “anecdotal” framings. Borkman, Soc Service Rev 1976;50(3):445-456

Expert by Experience Role

Chiefly UK usage, common in mental health and social care: a person whose expertise comes from living the condition rather than studying it. Near-equivalent to PWLE. The phrase does work that “patient” does not — it names the knowledge as expertise, and puts it in the job title.

Fair Market Value (FMV) — for PWLE compensation Concept

Methodology for setting PWLE compensation aligned with industry-standard professional rates, addressing both undercompensation (extractive) and excessive payment (perceived bias). See also PCORI Compensation Framework. NHC Fair-Market Value Calculator

FDA Patient Preference Information Guidance (2026) Regulatory

FDA final guidance on collecting and using patient preference information in medical device regulatory decisions across the total product life cycle. Supersedes the 2016 PPI Guidance. > FDA, “Incorporating Voluntary Patient Preference > Information over the Total Product Life Cycle,” March 30, 2026.*

FDA PDUFA VI Patient Engagement Framework (2017) Regulatory

FDA commitment to enhance patient engagement in drug development and regulatory decision-making. Established structured approach to collecting patient experience data.

FDA — Food and Drug Administration Regulatory

U.S. agency regulating drugs, devices, and biologics. Has established Patient-Focused Drug Development and other patient engagement initiatives.

Feasibility study / Pilot study Research

Small early-stage study testing whether a larger trial is logistically possible — recruitment rates, retention, data collection. Not designed to test treatment effectiveness.

FOA / NOFO — Funding Opportunity / Notice of Funding Opportunity NIH

NIH’s official announcements of available funding with detailed requirements. NOFO is the newer term. The NOFO is the source of truth on whether engagement is required.

FQHC — Federally Qualified Health Center Org

Community health centers serving underserved populations. Required to have patient representation on governing boards — one of the longest-standing patient governance requirements in U.S. healthcare.

Governance inclusion Concept

Formal representation of PWLE on decision-making bodies — boards, steering committees, oversight panels — with voting power, not just advisory status.

GRIPP2 — Reporting Involvement of Patients and the Public Framework

Reporting checklist for patient and public involvement in research. The standard for documenting how PWLE were engaged and what changed because of it. Staniszewska et al., BMJ 2017;358:j3453

HEAL Initiative — Helping to End Addiction Long-term NIH

NIH cross-institute initiative on pain and addiction research with strong community and lived-experience engagement requirements. Especially relevant for chronic pain advocacy. heal.nih.gov

Health equity Concept

The principle that everyone has a fair opportunity to attain their full health potential. Engagement frameworks increasingly require attention to who is — and isn’t — represented.

Health literacy Concept

A person’s capacity to obtain, process, and understand basic health information and services. Distinct from plain language: plain language describes the writing, health literacy describes the reader’s capacity. AHRQ Health Literacy Universal Precautions Toolkit

HIPAA — Health Insurance Portability and Accountability Act Regulatory

U.S. federal law (1996) protecting the privacy and security of personal health information. Affects PWLE in two ways: as participants whose data is protected, and as engaged partners who may need HIPAA training before handling data, sitting in on protocol discussions involving identifiable information, or accessing study records. 45 CFR Parts 160 and 164; HHS Office for Civil Rights

HTA — Health Technology Assessment Concept

Systematic evaluation of health technologies’ clinical and economic value. Increasingly incorporates patient perspectives and preferences.

HTAi Patient Involvement Roadmap Framework

Guide for implementing patient and citizen involvement in health technology assessment. Provides practical steps from planning to evaluation.

HTAi — Health Technology Assessment International Org

Global organization promoting HTA methods and policy. Has active patient and citizen involvement interest group.

Human Research Protection Training (CITI / OHRP) Research

Required training in research ethics and participant protection for anyone formally engaged on a research team. Most institutions use the CITI Program (citiprogram.org); OHRP’s Human Research Protection Foundational Training is also accepted. PWLE serving as Co-Investigators, IRB members, or in some advisory roles must complete it; renewal is typically every 3 years. Some institutions also offer a Community Partner Training designed specifically for community/PWLE collaborators — ask whether this option is available at the institution you’re working with. NIH Policy 3014-103; CITI Program HSR series; OHRP Human Research Protection Foundational Training

IAP2 Spectrum of Public Participation Framework

Framework defining five engagement levels: Inform, Consult, Involve, Collaborate, Empower. Widely adapted for patient engagement in healthcare and research.

ICER Patient Engagement Guide Framework

Framework for incorporating patient perspectives in health technology assessment. Defines patient council role and structured input methods throughout review process.

ICER — Institute for Clinical and Economic Review Org

Independent organization conducting health technology assessments to inform U.S. coverage decisions. Has established patient engagement protocols for all reviews.

ICH — International Council for Harmonisation Regulatory

Organization harmonizing drug development guidelines globally. E6 guideline increasingly emphasizes patient-centered approaches in clinical trials.

Inclusion criteria Research

Rules determining who can participate in a study. PWLE review often identifies criteria that unnecessarily limit real-world populations.

Institutional Review Board (IRB) Role

Committee reviewing research protocols for ethical standards. Federal regulations require at least one non-scientist member; IRB service gives PWLE direct influence over which studies move forward. 45 CFR 46 (Common Rule)

Intent-to-treat (ITT) / Per-protocol analysis Research

Two ways to analyze trial data. ITT analyzes everyone randomized — closer to real-world effectiveness. Per-protocol analyzes only those completing treatment as planned.

INVOLVE / NIHR Org

UK national infrastructure supporting public involvement in NHS, public health, and social care research. Pioneer in PPI with mandatory requirements for funding.

IOM Patient-Centered Research Priorities Report (2012) Framework

Institute of Medicine report that laid groundwork for PCORI. Emphasized need for patients to set research agendas and participate throughout research lifecycle.

ISPOR Patient Engagement Good Practices Report (2018) Framework

Task force report establishing standards for patient engagement in health economics and outcomes research. Defines meaningful engagement principles and evaluation methods.

ISPOR — International Society for Pharmacoeconomics and Outcomes Research Org

Professional organization developing methodological standards for patient engagement in health economics and outcomes research.

Just-in-Time (JIT) — NIH NIH

NIH stage just before grant award, when the agency requests specific compliance documentation (human subjects training, IRB approval, just-in-time budget details). PWLE serving as Key Personnel may be asked to provide CITI completion records and other documentation here.

K series — Career Development Awards NIH

Mentored grants supporting individual investigators. PWLE rarely interact directly with K awards, but may serve as community mentors or advisory contacts for K awardees doing community-engaged research.

Key Personnel (NIH grant terminology) Role

Individuals who contribute to scientific development or execution of an NIH-funded project in a substantive, measurable way. Named on the grant with a defined effort percentage. Different from “Other Significant Contributors” (also named, but without measurable effort). Patient Co-Investigators, when budgeted with effort, are Key Personnel. NIH Grants Policy Statement, current edition

Knowledge Translation (KT) Concept

The dynamic and iterative process of synthesizing, disseminating, exchanging, and applying research findings to improve health. Distinct from one-way “dissemination”: KT involves bidirectional exchange between researchers and end users (including PWLE) throughout the research lifecycle. Common term in Canadian and international research. Canadian Institutes of Health Research (CIHR) Knowledge Translation framework

Lay summary / Plain language summary Concept

Non-technical summary of a study or its results, written for general audiences. Increasingly required by funders and journals; reviewing them before publication is high-impact, low-burden PWLE work.

LBBC — Living Beyond Breast Cancer Org

Patient advocacy organization providing education and support for breast cancer survivors. Active in research advocacy and FDA drug approval processes.

Letter of Support / Letter of Intent Concept

Documents PWLE organizations are asked to produce or sign for grants, FDA dockets, or campaigns. Roles range from signing pre-written letters (low engagement) to drafting substantive positions (high engagement).

LLS — The Leukemia & Lymphoma Society Org

Patient advocacy organization funding blood cancer research. Has established peer review panels with patient advocate involvement in research funding decisions.

Meaningful engagement Concept

Engagement that has real influence on decisions, is resourced appropriately, and is evaluated — as opposed to tokenistic or performative involvement.

Merit Review Role

The process by which a funder decides what gets funded — and the main door through which PWLE enter research governance. PCORI’s model seats patients and other stakeholders on review panels alongside scientists, where lived experience is a scoring criterion rather than a courtesy; reviewers are trained and compensated. Compare Study Section (NIH), where PWLE presence is rarer and less formalized, and Consumer Reviewer (CDMRP model), which is the longest-running version of the idea. If you want to influence what gets studied rather than how, this is the room.

MID / MCID — Minimal (Clinically) Important Difference Research

The smallest change in a measured outcome that patients perceive as meaningful. “Statistically significant” is not the same as “matters to patients”; PWLE input on MID values is increasingly used in protocol design. FDA PFDD Guidance 4, 2023

MRCT Patient Engagement Framework and Toolkit Framework

Comprehensive framework and operational toolkit for patient engagement in clinical research. Defines principles, practices, and metrics for meaningful patient partnership throughout the research lifecycle. > Multi-Regional Clinical Trials Center, “Achieving > Meaningful Patient Engagement” toolkit.*

MRCT — Multi-Regional Clinical Trials Center Org

Brigham/Harvard center developing widely-referenced frameworks and toolkits for patient engagement in clinical research.

NAACCR — North American Association of Central Cancer Registries Org

Organization maintaining cancer surveillance data. Increasingly engages cancer survivors and patient advocates in registry governance and research priority-setting.

NCATS — National Center for Advancing Translational Sciences NIH

NIH center funding the Clinical and Translational Science Awards (CTSA) program. CTSA hubs increasingly required to demonstrate community and patient engagement.

NCCN — National Comprehensive Cancer Network Org

Alliance of cancer centers developing treatment guidelines. Has established patient advocate review panels for guideline development and updates.

NCI — National Cancer Institute NIH

NIH institute funding cancer research. Has Office of Advocacy Relations and requires patient advocate involvement in many grant review processes.

NExTRAC ENGAGE Report — Engaging the Public as Partners in Clinical Research Framework

November 2025 NIH report from the Novel and Exceptional Technology and Research Advisory Committee’s ENGAGE Working Group, providing a roadmap for incorporating public voices across all phases of NIH-funded clinical research. Endorsed by the NIH Director in December 2025; implementation began in 2026. The first agency-wide NIH framework for clinical research engagement, establishing vision, framework, and recommendations for meaningful, bi-directional public engagement — including funding, training, organizational support, and infrastructure. The NIH-side parallel to PCORI’s Foundational Expectations. NExTRAC ENGAGE Report, November 2025; partnersinresearch.nih.gov; NIH Director’s Statement, December 2025

NHC Patient Engagement Rubric Framework

National Health Council’s rubric, distinct from PCORI’s. Designed for broader stakeholder applicability — useful for patient organizations, payers, and industry, not just research funders. Frequently used in PFDD and HTA contexts.

NHC — National Health Council Org

Coalition of patient organizations publishing widely-used resources including the FMV calculator and the NHC Patient Engagement Rubric.

NIH Pragmatic Trials Collaboratory NIH

Network of large pragmatic clinical trials embedded in healthcare systems. Has a dedicated Patient and Stakeholder Engagement Core. rethinkingclinicaltrials.org

NIH — National Institutes of Health Org

Primary federal agency funding U.S. medical research. Increasingly emphasizes patient engagement across institutes; specific funding mechanisms are listed individually below (R01, U24, etc.). See also NExTRAC ENGAGE Report for the NIH-wide engagement framework.

NIHR — National Institute for Health and Care Research Org

UK’s largest funder of health research. Pioneer in patient and public involvement with mandatory PPI/PPIE requirements.

NINDS — National Institute of Neurological Disorders and Stroke NIH

NIH institute funding neurological research. Has consumer review panels and patient-focused research initiatives for conditions like epilepsy and chronic pain.

Nothing About Us Without Us Framework

The motto of the disability rights movement, and the ancestor of every patient engagement framework that followed. James Charlton first heard it in South Africa in 1993, from Michael Masutha and William Rowland of Disabled People South Africa, who had it from an Eastern European activist at an international conference; his 1998 book made it the movement’s name for itself. It became the theme of the UN’s 2004 International Day of Persons with Disabilities and underwrites the Convention on the Rights of Persons with Disabilities. Note what it actually demands. Not consultation. Control. Charlton JI. Nothing About Us Without Us: Disability Oppression and Empowerment. University of California Press, 1998

NPAF — National Patient Advocate Foundation Org

Organization providing case management and advocacy for patients. Trains patient advocates and engages in healthcare policy advocacy at state and federal levels.

Observational study Research

Research that observes people without intervening. Includes cohort studies, case-control studies, and registries.

Onboarding (of PWLE / patient partners) Concept

Structured process for introducing a new PWLE to a research team, study, or advisory body. Best practices specify onboarding should include study background, role expectations, institutional requirements (training, vendor status), introductions, and time for relationship-building. Whether a research team has a real onboarding process is one of the strongest signals of how seriously they take engagement. PCORI Best Practices in Engaging Stakeholders

Open-label trial Research

Study where participants and researchers know what treatment is being given. No placebo or blinding.

P series — Program Project / Center Grants NIH

Multi-project center grants supporting a coordinated research program. Centers often have community advisory boards and patient engagement cores.

P01 — Research Program Project Grant NIH

Multi-project program grants, typically several related research projects sharing core resources. Often have advisory committees that may include patient representatives.

P30 — Center Core Grant NIH

Funds shared core resources for institutions with multiple NIH-funded investigators (Cancer Centers, Diabetes Centers). May include community outreach and engagement cores.

Patient Advisory Council (PAC) / Patient and Family Advisory Council (PFAC) Role

Standing groups providing input on research, programs, or institutional priorities. Before joining: ask what decisions the group influences, how input is documented, and whether members are compensated.

Patient Advocate Role

Someone who speaks and acts on behalf of patients — their own or a broader community — in policy, clinical, or organizational settings.

Patient engagement Concept

Umbrella term for meaningful involvement of PWLE in decisions that affect them — across research, clinical care, policy, and organizational governance.

Patient Engagement Core / Community Engagement Core Role

Structural component within multi-project research consortia (U24, U54, P30, UG3/UH3) responsible for embedding PWLE in governance, design, recruitment, and dissemination. Different consortia name them differently — Community Engagement Core, PACE, Patient Advocacy Core, Stakeholder Engagement Core.

Patient experience data (PED) Regulatory

FDA’s regulatory term, formalized by the 21st Century Cures Act, for data on patients’ experiences with their condition and treatments. Encompasses qualitative input (PFDD testimony) and quantitative measures (PROs, preference studies). 21st Century Cures Act §3001; FDA PFDD Guidance Series

Patient Expert (EUPATI usage) Role

EUPATI’s term for a PWLE who has completed formal training in research and drug development. Sits between “patient advocate” (broader) and “patient representative” (representational).

Patient journey / Patient journey mapping Concept

Structured method for documenting a person’s experience with a condition over time — from first symptoms through diagnosis, treatment, and ongoing management. Heavily used in PFDD meetings.

Patient Navigator Role

A person who helps someone move through a health system — explaining options, connecting services, following up over time. Originated in cancer care and increasingly built into trial designs as the intervention being tested rather than a service wrapped around it. Distinguish from Patient Advocate (speaks for interests) and Patient Partner (shapes the research).

Patient Partner Role

PWLE who collaborates as an equal team member on research, policy, or quality improvement work — not a study subject or advisory voice, but a co-producer.

Patient preference information (PPI — FDA usage) Regulatory

Assessments of what tradeoffs patients are willing to make between treatment attributes (efficacy vs. side effects). Note acronym collision: PPI also means Patient and Public Involvement in UK/EU contexts. FDA “Incorporating Voluntary Patient Preference Information,” 2026

Patient registry / Disease registry Research

Organized collection of standardized data on people with a particular condition, maintained over time. Often the most common entry point for advocacy organizations into research. AHRQ Registries User’s Guide, 4th ed.

Patient Representative Role

PWLE formally appointed to speak for a patient constituency on a committee or panel. Implies a representational duty beyond personal experience.

Patient-centered / Patient-centric Concept

Organized around patient needs, values, and preferences. “Centric” often implies more structural organizational commitment than “centered.”

Patient-Centered Outcomes Research

Outcomes that reflect what matters to patients themselves — quality of life, function, symptom burden, ability to do daily activities — rather than clinical or laboratory measurements alone. Distinct from clinical outcomes (lab values, imaging, biomarkers). Reviewing whether a study’s primary endpoint is a patient-centered outcome is one of the highest-leverage protocol review activities for PWLE. PCORI; Carman et al. Health Affairs 2013

PCOR / PCORI — Patient-Centered Outcomes Research / Institute Org

PCOR = research comparing options using outcomes patients actually care about. PCORI = independent U.S. organization funding PCOR with required patient and partner engagement; since 2024, applicants must address PCORI’s Foundational Expectations for Partnerships in Research (which superseded the 2014 Engagement Rubric).

PCORI Engagement Rubric Framework

Assessment tool measuring quality of patient engagement across the research lifecycle. Gold standard framework used widely beyond PCORI-funded research. Evaluates meaningful involvement principles.

PCORI Foundational Expectations for Partnerships in Research Framework

PCORI’s six required engagement building blocks (2024): (1) Representative Involvement; (2) Early & Ongoing Engagement; (3) Dedicated Funds for Engagement & Partner Compensation; (4) Build Capacity to Work as a Team; (5) Meaningful Inclusion of Partners in Decision Making; (6) Ongoing Review & Assessment of Engagement. Replaces the 2014 PCORI Engagement Rubric. pcori.org Foundational Expectations, 2024

PCORI Methodology Standards for Patient Engagement Framework

Rigorous standards defining how to conduct patient-centered outcomes research with meaningful patient engagement. Sets expectations for research quality and engagement evaluation.

PEQG — Patient Engagement Quality Guidance Framework

Peer-reviewed quality framework from PFMD, developed across industry, academia, and patient organizations. Often cited alongside PCORI frameworks in industry contexts. PFMD; BMJ Innovations 2018

PFDD — Patient-Focused Drug Development Regulatory

FDA initiative to systematically gather patient perspectives on their condition and treatment options to inform drug development and regulatory decisions. See also EL-PFDD.

PFMD — Patient Focused Medicines Development Org

Multi-stakeholder coalition (industry, patients, academia) developing tools and frameworks for patient engagement throughout medicines development. patientfocusedmedicine.org

Phases of clinical trials (I / II / III / IV) Research

Sequential stages: Phase I (safety, 15-30 people), II (efficacy, 30-100), III (comparison, 100-3,000), IV (post-market surveillance). Engagement opportunities and risks differ by phase.

Placebo-controlled trial Research

Study where some participants receive an inactive treatment to compare against the active treatment. Ethical only when no effective treatment exists.

Plain language Concept

Communication written for comprehension by a general audience — no jargon, short sentences, active voice. Often required for patient-facing materials.

PPI — Patient and Public Involvement (UK/EU) Concept

Research conducted with or by patients rather than to, about, or for them. Standard in UK/EU frameworks. Note collision: PPI in U.S. FDA contexts means Patient Preference Information.

PPIE — Patient and Public Involvement and Engagement Concept

Current UK terminology, replacing PPI alone. Standard in NIHR funding language since the late 2010s.

Pragmatic trial Research

Study designed to test how treatments work in real-world clinical practice, not idealized research conditions. Often more relevant to PWLE experience than explanatory trials.

Principal Investigator (PI) / Multiple PI (MPI) Role

Lead scientist responsible for the research project. NIH allows multiple PIs of equal standing. PWLE-as-MPI is rare and a strong signal of meaningful engagement when it happens.

PRO — Patient-Reported Outcome Research

Health status measurement that comes directly from the patient without clinician interpretation. Reviewing PRO instruments is a major PWLE engagement activity — look for relevance, recall window, response burden, accessibility.

PROMIS — Patient-Reported Outcomes Measurement Information System Research

NIH-developed system of standardized PRO measures across physical, mental, and social health domains. Increasingly used as the default PRO instrument set in NIH-funded research. healthmeasures.net; NIH Common Fund

Protocol Research

Detailed plan for a clinical trial or research study. Increasingly, PWLE review protocols before finalization. Frequent amendments often signal design problems better up-front input could have prevented.

Protocol amendment Research

A formal change to an approved protocol. Requires re-review by the IRB and may require participant re-consent. Frequent amendments often signal design problems that better up-front PWLE input could have prevented.

PWLE — Person with Lived Experience Concept

Someone with direct personal experience of a health condition, treatment system, or circumstance being studied. Preferred over “patient” in many engagement contexts because it acknowledges expertise outside formal medical encounters.

Quality Improvement (QI) vs. Research Concept

QI = activities to improve care within a specific setting (typically does not require IRB review). Research = systematic investigation designed to produce generalizable knowledge (requires IRB review). The distinction matters because activities labeled QI face fewer protections for participants — and the line between QI and research can be contested. PWLE invited to participate in “QI projects” should ask whether participants would be informed and protected as in research. OHRP guidance on QI vs. Research; 45 CFR 46.102

R series — Research Project Grants NIH

Investigator-initiated research grants. The most common NIH mechanism. Engagement is generally not required but increasingly expected, especially for clinical and translational research.

R01 — Research Project Grant NIH

NIH’s flagship research grant — 4-5 years, larger budgets, single investigator’s research program. Engagement is usually optional; ask what role PWLE played in shaping the original application.

R03 — Small Research Grant NIH

Small (2-year, limited budget) grants for pilot studies, secondary data analysis, or methodology development.

R13 / U13 — Conferences NIH

Funds conferences and scientific meetings. Conferences increasingly require diverse participation, including patient/community voices.

R21 — Exploratory/Developmental Research Grant NIH

Shorter (2 years) and smaller than R01, for early-stage or higher-risk projects. Often where new patient engagement methods get piloted before scaling.

R34 — Clinical Trial Planning Grant NIH

Supports planning a clinical trial — protocol development, feasibility, partnership building. If invited onto an R34, you’re being engaged before the protocol is locked — a high-leverage moment.

R61/R33 — Phased Research Awards NIH

Two-phase grants: R61 milestone-driven planning phase, R33 implementation phase. Common in NIMH and other institutes.

Randomization Research

Process of assigning participants to study arms by chance, to avoid systematic differences between groups. The defining feature of an RCT.

RCT — Randomized Controlled Trial Research

Study where participants are randomly assigned to receive either the experimental treatment or a control. The “gold standard” for testing treatments.

Real-World Evidence (RWE) / Real-World Data (RWD) Research

RWD = data on patient health collected outside trials (EHRs, claims, registries, wearables). RWE = clinical evidence derived from RWD. Increasingly important in regulatory and coverage decisions.

Reasonable accommodation Concept

The adjustments that let a disabled person actually take part — captions, a scheduled break, materials sent in advance, a remote option, a room a wheelchair fits in, a meeting that isn’t at 8 a.m. Required of most US employers and public entities under the ADA; the obligation toward research partners is far less settled, which is exactly why it belongs in the Terms of Reference before you agree to join. Ask early. Asking mid-meeting rarely works.

Reciprocal relationships Concept

PCORI engagement principle: the relationship between researchers and PWLE involves mutual exchange — both contribute and both benefit. A check against extractive engagement.

Recruitment / Retention Research

Recruitment = enrolling participants. Retention = keeping them through completion. Both are areas where PWLE input commonly improves study performance — patients understand barriers researchers may not see.

Recruitment Materials Review Research

PWLE review of flyers, advertisements, social media posts, and other materials used to recruit study participants. One of the most concrete and impactful PWLE engagement activities — materials approved by IRBs are often clinically accurate but read awkwardly to patients; PWLE input typically improves clarity, tone, and inclusivity. PCORI Best Practices in Engaging Stakeholders; FDA PFDD Guidance 1

Reimbursement vs. compensation Concept

Not the same thing, and the difference can matter more than the amount. Reimbursement returns money you already spent — travel, parking, a hotel, a carer’s time. Compensation pays you for your time and expertise. Reimbursement is generally not income; compensation generally is, which is why the distinction bears on taxes, vendor paperwork, and benefits. A team offering only reimbursement is not paying you. A team that calls compensation “reimbursement” may be doing you a favor or may be avoiding a budget line — ask which.

Research Core / Core Facility Role

Shared resource within a multi-component grant (U24, P30, U54). If a grant has a Patient Engagement or Community Engagement Core, that’s where formal PWLE work usually lives.

Return of results Concept

What participants get back when a study ends: individual results, aggregate findings, or — most commonly — nothing at all. Rarely planned, rarely budgeted, and almost never asked about until someone in the room asks. One of the most reliable places for a PWLE to raise a question the team has not considered.

Rigor and Reproducibility Research

NIH-required language in grant applications since 2016, addressing the scientific premise, experimental design, biological variables (sex, age), and authentication of key resources. Appears as a dedicated section in most NIH applications. PWLE on review panels see it constantly; questions about whether outcomes matter to patients fit naturally into the “scientific premise” section. NIH NOT-OD-15-103 and successors; nih.gov/grants/policy/rigor.htm

Sample size / Power calculation Research

Statistical determination of how many participants a study needs to detect a meaningful difference between groups. Underpowered studies cannot reliably detect even real effects.

SBIR / STTR — Small Business Innovation / Technology Transfer Research NIH

Funding for small businesses developing health technologies. Compensation expectations differ from academic engagement — closer to industry consulting rates.

SDOH — Social Determinants of Health Concept

Economic, social, and environmental factors that affect health outcomes. Engagement work increasingly addresses how these create barriers to participation.

Service user / Consumer Role

Service user is standard UK usage for a person who uses health or social care services; Consumer is the Australian equivalent and appears throughout Australian engagement policy. Neither travels comfortably into US usage, but both are everywhere in the international literature. That the field carries PWLE, patient, service user, consumer, Expert by Experience, patient partner, and patient representative all at once is itself the finding: nobody has settled what to call us, because nobody has settled what we are for.

Single IRB (sIRB) Regulatory

Federal requirement (since 2018) that multi-site NIH-funded research use a single IRB of record rather than each site’s own IRB. Speeds approvals; can also concentrate decision-making authority away from local communities. PWLE serving on a Single IRB make decisions affecting many sites simultaneously. NIH Single IRB Policy, NOT-OD-16-094

Stakeholder Concept

Any party with an interest in a project’s outcomes. Some advocates distinguish PWLE from other stakeholders because their stake is personal, not professional.

Steering Committee Role

Governance body with decision-making authority over a study or consortium. Distinct from an advisory board, which advises but does not decide. Membership with a vote = meaningful governance; advisory without vote = consultation.

Study Section / Scientific Review Group Role

Peer review panel scoring NIH grant applications. Most are entirely scientific; some — especially in CDMRP and PCORI — include consumer/patient reviewers.

Surrogate endpoint Research

A measured outcome used as a stand-in for a clinically meaningful one (tumor shrinkage as a surrogate for survival). PWLE protocol review often surfaces concerns about over-reliance on surrogates.

Synodos Community Engagement Model Framework

Patient-driven research initiative for sarcoma using community-based participatory research principles. Demonstrates patient leadership in setting research priorities and conducting studies.

T and F series — Training Grants and Fellowships NIH

Support graduate students, postdocs, and early-career trainees. PWLE may serve on training program advisory boards or as community partners for trainee projects.

Tokenism Concept

Including PWLE in form but not substance — inviting input without the power, timing, or resources to shape outcomes. A pattern engagement frameworks actively work against.

ToR — Terms of Reference Concept

Document defining a group’s purpose, membership, decision rights, and operations. Critical for clarifying PWLE role and authority before joining.

Trustworthiness (vs. trust) Concept

An institution’s responsibility to be worthy of trust, rather than expecting communities to extend trust. A reframing increasingly used in community-engaged research, especially with historically marginalized populations. National Academies; community-engaged research literature

U series — Cooperative Agreements NIH

Like R grants, but with substantial NIH staff involvement and often consortium structure. Many U-series mechanisms require formal patient/community engagement components.

U01 — Research Project Cooperative Agreement NIH

Like an R01 but with substantial NIH program staff involvement. Often used for clinical trials networks and multi-site studies; frequently has formal engagement structures.

U24 — Resource-Related Research Project Cooperative Agreement NIH

Funds research resources and infrastructure — registries, data coordinating centers, shared facilities. Commonly includes a Patient Engagement or Community Engagement Core; one of the mechanisms most likely to have formal PWLE governance.

U54 — Specialized Center Cooperative Agreement NIH

Large multi-project center grants, common in health disparities and rare disease research. Almost always have a Community Engagement Core or equivalent.

UG3/UH3 — Phased Innovation Awards NIH

Two-stage cooperative agreements: UG3 planning phase, UH3 full implementation. Used for HEAL Initiative and Pragmatic Trials Collaboratory. UG3 is high-leverage — engagement structures get designed there.

USASP — United States Association for the Study of Pain Org

National interdisciplinary organization advancing pain research, education, and policy, with growing emphasis on partnership with people with lived experience.

VA PRG — Veterans Affairs Patient Research Group Org

Network of veteran patient-partners who review VA research proposals and provide veteran perspectives on study design, recruitment, and dissemination. Model for peer-led patient engagement.

Vendor status (at a university) Concept

The administrative process required at most universities and academic medical centers before they can pay an outside collaborator or consultant — including PWLE engaged for compensated work. Typically requires a W-9 form, banking/ACH information, sometimes a conflict-of-interest disclosure, and registration in the institution’s procurement system. Setup can take days to weeks; ask early in the engagement so payment isn’t delayed. Once set up at one institution, you typically have to repeat the process at each new institution that pays you. Institution-specific; ask the project’s grant administrator or sponsored programs office for the exact process

WHO Patient Engagement Framework Framework

World Health Organization framework for patient and family engagement in health care. Establishes global principles and implementation strategies for health system transformation.

WHO — World Health Organization Org

UN agency for international public health. Has issued frameworks for patient and family engagement in healthcare.