Glossary of Patient Engagement Terms

02/13/2019

The definitions of these terms, which includes Family Caregiver, Meaningful Engagement, and Patient-Focused Drug Development, stem largely from our patient engagement work, including our ‘Patient-Focused Drug Development – Recommended Language for Use in Guidance Document Development’ Paper with Genetic Alliance, as well as our Value Rubric, and Representativeness Rubric.

family caregiver: someone caring for those with a medical condition(s). Their care recipients are dependent on the health care system after the diagnosis of a medical condition or disability. A caregiver’s views on health issues, such as the benefit and risk of new treatments, will vary depending on the severity of the condition and personal circumstances of those they care for. ¹^,²

family caregiving organization: a 501(c)(3) organization that has a mission invested in improving the lives of family caregivers and their care recipients. It engages in programs, such as research, education, advocacy, and service to family caregivers. It takes a holistic view of the caregivers it represents and seeks universal support from stakeholders for its mission and programs. ¹^,²

meaningful engagement: direct relationships and partnerships that are bidirectional, reciprocal, and continuous. Communications are open, honest, and clear. Engagement goals, participants, methods, desired impacts, and actual impacts are clearly outlined and transparent.¹

online patient communities: Internet-based platforms that unite patients who have been diagnosed with a disease. They offer support, networking and/or information sharing.³^,⁴ Platforms may also help patients record health information and/or become involved in research. For researchers, online patient communities can be a useful resource for identifying patients to recruit as advisers or to participate in a study (e.g., qualitative study on patient experience, clinical trial participant, or surveys of opinions/experiences). Online communities are operated by patient organizations, provider groups, and others including for-profit organizations. For-profit organizations should not be confused with patient organizations, as they typically collect data but do not advocate for a specific constituency and utilize patient data primarily for commercial purposes.

patient: someone having or at-risk of having a medical condition(s), whether or not they currently receive medicines or vaccines to prevent or treat a disease. They are dependent on the health care system after the diagnosis of a medical condition or disability. A patient relies on the health care system to feel better and to have a longer, healthier, and more robust life. An individual patient’s views on health issues, such as the benefit and risk of new treatments, will vary depending on the severity of his or her condition and personal circumstances.²

patient advocacy organization: a 501(c)(3) organization that has a mission to combat a particular disease, disability, or group of diseases and disabilities, or to improve and protect the health of a particular group of people. It engages in programs, such as research, education, advocacy, and service to individuals and communities. It takes a holistic view of the conditions for the patients it represents and seeks universal support from stakeholders for its mission and programs. While a patient advocacy organization may advocate for patient access to care, they do not have prescribing authority; formulary control, responsibility, or decision-making authority; or make drug purchases.²

patient centered: Any process, program or decision focused on patients in which patients play an active role as meaningfully engaged participants, and the central focus is on optimizing use of patient-provided information. Patient centered means doing things WITH –not FOR or TO –patients.⁶

patient-centered core outcome set (PC-COS): An agreed upon, standardized set of outcomes (often with related measures, and/or endpoints identified) that should be gathered from patients and reported at a minimum in research in specific areas of health or health care. They include, but are not limited to how patients feel and function, mortality, biomarkers as surrogate outcomes, etc. The outcome-concept identification and prioritization are guided by patient contributions as partners, recognizing their unique experiences, values, and expertise.⁵

patient-centered core impact set (PC-CIS): An agreed upon, standardized set of impacts on a patient’s daily life that should be gathered from patients and reported at a minimum in research in specific areas of health or health care. The impact-concept identification and prioritization are guided by patient contributions as partners, recognizing their unique experiences, values, and expertise. The term “impacts” includes health outcomes, such as what would be found in a core outcome set, and any other patient experience information (e.g., financial- or caregiver-related burdens) patients might report as important to them.

patient-centered health care: care that is respectful of and responsive to individual patient preferences, needs, and values in context of their own social worlds. Patient centeredness is created by engaging, informing, and actively listening to people with chronic conditions at every point of contact –from the research bench to the bedside and everywhere in between.¹^,⁷^,⁸

patient-centered outcome (PCO): The outcomes reported by patients as important to them in the way they experience a disease or treatment for that disease.⁶

patient-centered value assessment (VA): Patients are engaged, heard, understood, and respected throughout the entire VA process, and their input is incorporated and guides decision-making.

patient community: broadly encompasses individual patients, family caregivers, and the organizations that represent them. The patient community is heterogeneous and brings to the value discussion different perspectives that have been informed by their experiences, trajectory or stage of disease, level of expertise, and many other factors.¹

Patient engagement: Refers to “the active, meaningful, authentic and collaborative interaction between patients and other stakeholders across all aspects of the health ecosystem, where decision-making with regard to an activity or process is guided by patients’ contributions as partners, recognizing their unique experiences, values and expertise.” ¹⁴ This definition is also applicable across stakeholders and engagement activities (e.g., participation in an interview, focus group).

Patient experience: Information about patients’ personal knowledge living with a disease or condition. It includes perspectives, needs and priorities related to, but not limited to,

Symptoms of their condition and its natural history
Impact of the conditions on their functioning and quality of life – Experience with treatments
Input on which outcomes are important to them
Patient preferences for outcomes and treatments
Relative importance of any issue as defined by patients²²

patient-focused drug development: the meaningful engagement of patients in the research and development of therapeutic products and the various important roles patients can play in improving the processes, from study endpoint selection that reflects outcomes meaningful to patients, recruitment and retention in clinical trials, and more effective post-marketing safety surveillance.⁹^,¹⁰

patient-generated health data: health-related data created, recorded, or gathered by or from patients (or family members or other caregivers) to help address a health concern.¹^,¹¹

patient information: broadly means all information regarding a patient from any source, such as patient-provided information as well as other medical information about a patient, including but not limited to: test results (e.g., genetic, pathology, imaging), identifying information, family history, provider opinion, and any other information in a patient’s medical records.¹^,¹⁵

patient-informed drug development: generally, refers to drug development that uses PPIn to guide or inform decisions but may or may not include patient involvement.¹^,²

patient journey: The “patient journey” is a metaphor that describes the experiences of a patient (and their care partners and families) with an illness and its treatment, depicting the time prior to, during, and after diagnosis as akin to a voyage or expedition. The term “patient experience” is often more acceptable to patients.

patient need: a desire or requirement expressed by a patient related to their health.¹^,¹⁶

patient perspective information: a subset of patient-provided information; information regarding the attitude or the point of view of the patient, including anecdotal comments in correspondence to FDA or testimony at Advisory Committee Panel meetings ¹⁷ or Patient-Focused Drug Development meetings¹⁸ patient opinions expressed publicly including through social media, patient responses to qualitative, ad hoc surveys, quantitative measurements of patient-reported outcomes, and more.¹^,¹⁶^,¹⁹

patient preference information: a subset of PPIn; qualitative or quantitative assessments of the relative desirability, or acceptability of, attributes that differ among alternatives (e.g., alternative therapeutic strategies).¹⁹Attributes of a drug or biologic include characteristics such as effectiveness, safety, route of administration, dosing regimens, duration of effect, duration of use, and other product features about which patients express preferences.¹

patient-provided information (PPIn): describes information a patient contributes directly at any point during the product lifecycle. The term broadly encompasses the entirety of information that can be collected from an interaction with a patient. The focus should be the patients view on their disease(s)/condition(s), desired attributes for treatments, experiences with treatments, benefit- risk preferences, and desired goals and outcomes. It should not be primarily focused on any one product.¹^,²

patient-reported information: a subset of PPIn; information that is reported directly by a patient without amendment or interpretation by a clinician, researcher, or any other entity¹^,¹²

patient-reported outcome (PRO): a subset of PPIn; an outcome measure based on a report that comes directly from the patient (e.g., study subject) about the status of the patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else. A PRO can be measured by self-report or by interview provided that the interviewer records only the patient’s responses.¹^,⁶^,¹³

patient risk tolerance: a qualitative term reflecting the extent to which a patient will accept the risk(s) of an adverse effect from a treatment for a probable benefit; risk tolerance will vary among patients, which affects individual patient decisions as to whether risks are acceptable.¹^,²⁰

patient voice: The “patient voice” is a metaphor that captures the belief that the health ecosystem must do a better job of listening to what patients (individual and patient communities) express about their experiences, needs, values, goals, priorities, preferences, and expectations related to having a disease and its management starting with their journey to diagnosis, symptoms, burden, and impacts, and perceptions regarding treatment-related benefits, risks, burden, tradeoffs, unmet need and access.

representativeness: a sufficient number of and types of people are included in the engagement activity to ensure that those engaged can speak on behalf of the target population. It refers to “who” and “how many” individuals to include in an interaction in order to, as closely as possible, engage with individuals that represent the broader, target patient population.²¹

How to cite this report:

National Health Council Patient Engagement Glossary of Terms. Washington DC: National Health Council; 2019. Available from: https://nationalhealthcouncil.org/glossary-of-patient-engagement-terms/

References:

¹See National Health Council. The Patient Voice in Value: The National Health Council Patient-Centered Value Model Rubric, https://www.nationalhealthcouncil.org/wp-content/uploads/2019/12/Value-Rubric.pdf.

²National Health Council, Genetic Alliance. Dialogue/Advancing Meaningful Patient Engagement in Research Development, and Review of Drugs. (https://www.nationalhealthcouncil.org/wp-content/uploads/2019/12/PatientEngagement-WhitePaper.pdf)

³van der Eijk M, Faber MJ, Aarts JW, Kremer JA, Munneke M, Bloem BR. Using Online Health Communities to Deliver Patient-Centered Care to People With Chronic Conditions. J Med Internet Res. 2013;15(6). doi:10.2196/jmir.2476

⁴Solberg LB. The Benefits of Online Health Communities. AMA Journal of Ethics. 2014;16(4):270-274. doi:10.1001/virtualmentor.2014.16.4.stas1-1404

⁵Adapted from: COMET Initiative. Core Outcome Measures in Effectiveness Trials. Available from: http://www.comet-initiative.org/

⁶National Health Council. The National Health Council Rubric to Capture the Patient Voice: A Guide to Incorporating the Patient Voice into the Health Ecosystem. June 2019. Washington, DC. Available from: https://www.nationalhealthcouncil.org/Patient-Engagement-Rubric

⁷Institute of Medicine (IOM). 2001. Crossing the Quality Chasm. Crossing the Quality Chasm: A New Health System for the 21st Century. Washington, D.C: National Academy Press

⁸Epstein RM, Street RL. The Values and Value of Patient-Centered Care. Annals of Family Medicine. 2011;9(2):100- 103. doi:10.1370/afm.1239 8

⁹See Clinical Trials Transformation Initiative, CTTI Recommendations: Effective Engagement with Patient Groups Around Clinical Trials, http://www.ctti-clinicaltrials.org/files/PatientGroups/PGCTrecs.pdf (last visited February 16, 2016).

¹⁰National Health Council, Genetic Alliance. Patient-Focused Drug Development – Recommended Language for Use in Guidance Document Development , (https://www.nationalhealthcouncil.org/wp-content/uploads/2019/12/NHC-GA%20Feb201…

¹¹See patient-generated health data, HealthIT.gov website, Consumer eHealth, https://www.healthit.gov/topic/scientific-initiatives/patient-generated-health-data (last visited April 8, 2016).

¹²The Voice of the Patient: A Series of Reports from FDA’s Patient-Focused Drug Development Initiative, http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm368342.htm (last visited February 16, 2016)

¹³See the guidance for industry, Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims (http://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/…).

¹⁴ISPOR Patient-Centered SIG. Defining Patient Centeredness and Engagement in HEOR: Proposed Definition and Stakeholder Response. https://www.ispor.org/docs/defaultsource/presentations/1388.pdf?sfvrsn=c…Published May 21, 2018. Accessed April 15, 2019.

¹⁵National Health Council, Genetic Alliance. Integrating the Patient into the Drug Development Process: Developing FDA Guidance, (https://www.nationalhealthcouncil.org/sites/default/files/NHC-GA-Patient-Engagement.pdf).

¹⁶See FasterCures, Enhancing Integration of Patient Perspective Data in the Drug Development Process, Proposal for PDUFA VI, (https://www.nationalhealthcouncil.org/sites/default/files/FasterCures-PDUFA-Comment-Letter-FDA.pdf).

¹⁷See FDA website, About the Patient Representative Program, (https://www.fda.gov/patients/learn-about-fda-patient-engagement/about-fda-patient-representative-program) (last visited February 16, 2016).

¹⁸See Patient-Focused Drug Development: Disease Area Meetings Planned for Fiscal Years 2013-2017, http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm (last visited February 16, 2016).

¹⁹Draft Guidance for Industry, Food and Drug Administration Staff; and Other Stakeholders; Patient Preference Information – Submission, Review in PMAs, HDE Applications, and De Novo Requests, and Inclusion in Device Labeling. For the most recent version of a guidance, check the FDA Drug guidance Web page at https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs (last visited February 16, 2016).

²⁰See CDRH guidance for industry, Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions, http://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm506679.pdf (last visited November 7, 2016).

²¹National Health Council. Tackling Representativeness: A Roadmap and Rubric. Washington, DC. Available from: (https://www.nationalhealthcouncil.org/sites/default/files/Representativeness%20in%20Patient%20Engagement.pdf)

²² Derived from: 21st Century Cures Act.; 2016. Title III: Section 3002(c). Accessed August 2, 2021. https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf