Development and Testing of a Chronic-Disease Patient Experience Mapping Toolbox.

BACKGROUND: Stakeholders increasingly expect research and care delivery to be guided by and to optimize patient experiences. However, standardized tools to engage patients to gather high-quality data about their experiences, priorities, and desired outcomes are not publicly available. The objective of this study was to develop and test a Toolbox with a disease-agnostic interview guide template and accompanying resources to assist researchers in engaging patients living with chronic disease in a dialogue about their experiences. METHODS: Guided by a multidisciplinary workgroup, a targeted literature review (PubMed) was conducted, followed by group discussions to identify/thematically organize patient experience concepts, development of a conceptual model, and drafting of an interview guide template and patient-facing visual. Materials were tested/refined via cognitive (n = 5) and pilot (n = 30) interviews conducted virtually with US patients diagnosed with chronic/potentially disabling conditions from December 2020 to April 2021. Patient-facing tools were reviewed by health literacy experts for applicability/accessibility. English-speaking adults who self-reported receiving a chronic condition diagnosis at least 6 months prior participated in a 60-90 min interview. RESULTS: Patient experience concepts were organized thematically under three domains: (1) life before a diagnosis, (2) experiences getting a diagnosis, and (3) experiences living with a diagnosis. A plain language consent sheet template, interview guide template, and patient experience conceptual model were developed and revised based on input from interviewees, interviewers, and the workgroup. CONCLUSIONS: A disease-agnostic patient-engagement Toolbox was developed and tested to capture patient experience data. These materials can be customized based on study objectives and leveraged by various stakeholders to identify opportunities to enhance the patient centricity of healthcare delivery and research.

A Foundation for Patient-Centered Core Impact Sets: Key Learnings from Past and Existing Approaches.

Despite growing commitment to patient centricity, challenges persist in consistently identifying the impacts of disease and/or treatment that patients report as most important to them, especially across myriad potential downstream uses. Patient-centered core impact sets (PC-CIS), disease-specific lists of impacts that patients report as most important, are proposed as a solution. But, PC-CIS is a new concept, currently in the pilot stage with patient advocacy groups. We conducted an environmental scan to explore PC-CIS conceptual overlap with past/existing efforts [e.g., core outcome sets (COS)] and to inform general feasibility for further development and operationalization. With guidance and advice from an expert advisory committee, we conducted a search of the literature and relevant websites. Identified resources were reviewed for alignment with the PC-CIS definition, and key insights were gleaned. We identified 51 existing resources and five key insights: (1) no existing efforts identified meet the definition of PC-CIS as we have specified it in terms of patient centricity, (2) existing COS-development efforts are a valuable source of foundational resources for PC-CIS, (3) existing health-outcome taxonomies can be augmented with patient-prioritized impacts to create a comprehensive impact taxonomy, (4) current approaches/methods can inadvertently exclude patient priorities from core lists/sets and will need to be modified to protect the patient voice, and (5) there is need for clarity and transparency on how patients were engaged in individual past/existing efforts. PC-CIS is conceptually unique from past/existing efforts in its explicit emphasis on patient leadership and being patient driven. However, PC-CIS development can leverage many resources from the past/existing related work.

Developing Patient-Centered Real-World Evidence: Emerging Methods Recommendations From a Consensus Process.

OBJECTIVES: The Joint ISPOR-ISPE Special Task Force on Real-World Evidence included patient/stakeholder engagement as a recommended good procedural practice when designing, conducting, and disseminating real-world evidence (RWE). However, there are no guidelines describing how patient experience data (PED) can be applied when designing real-world data (RWD) studies. This article describes development of consensus recommendations to guide researchers in applying PED to develop patient-centered RWE. METHODS: A multidisciplinary advisory board, identified through recommendations of collaborators, was established to guide development of recommendations. Semistructured interviews were conducted to identify how experienced RWD researchers (n = 15) would apply PED when designing a hypothetical RWD study. Transcripts were analyzed and emerging themes developed into preliminary methods recommendations. An eDelphi survey (n = 26) was conducted to refine/develop consensus on the draft recommendations. RESULTS: We identified 13 recommendations for incorporating PED throughout the design, conduct, and translation of RWE. The recommendations encompass themes related to the development of a patient-centered research question, designing a study, disseminating RWE, and general considerations. For example, consider how patient input can inform population/subgroups, comparators, and study period. Researchers can leverage existing information describing PED and may be able to apply those insights to studies relying on traditional RWD sources and/or patient registries. CONCLUSIONS: Applying these emerging recommendations may improve the patient centricity of RWE through improved relevance of RWE to patient communities of interest and foster greater multidisciplinary participation and transparency in RWD research. As researchers gather experience by applying the methods recommendations, further refinement of these consensus recommendations may lead to "best practices."

Enhancing Patient Centricity of Real-World Data Research: An Exploratory Analysis Using the Patient Experience Mapping Toolbox.

OBJECTIVES: There is an increasing expectation that medical product development and assessment be guided by patient input captured through patient engagement. Recently published consensus guidelines describe how qualitative patient experience data (PED) can guide the design, conduct, and translation of real-world research that reflects patients' lived experience. The objective of this exploratory analysis is to examine how researchers could leverage PED captured through the Patient Experience Mapping Toolbox (PEMT) to guide real-world data (RWD) research designs. METHODS: This exploratory analysis included a thematic analysis of interview transcripts collected while pilot testing the PEMT followed by a qualitative analysis of the emerging themes aligned with stages listed in the patient-centered real-world evidence, Real-World Research Design Framework. RESULTS: PED collected using the PEMT include information about symptomology, interactions with the healthcare system, information-seeking behavior, misdiagnoses, lifestyle changes, treatments, side effects, and comorbidities. This information can be leveraged at key study design decisions, including (1) identifying study cohorts and subgroups, (2) identifying exposures, (3) informing covariates and potential confounders; and (4) refining study periods. Additionally, participants described where they seek information about treatments and diseases, which should inform dissemination strategies. CONCLUSIONS: We identified opportunities for PED collected using the PEMT to inform RWD study designs. The PED described in this exploratory analysis stem from pilot testing of the PEMT across a variety of conditions. In the next phase of development in this area, researchers should evaluate how data collected using the PEMT can be applied to RWD research for a specific disease.

Patient-Centered Core Impact Sets: What They are and Why We Need Them.

A quote attributed to Mark Twain states, "What gets us into trouble is not what we don't know. It's what we know for sure that just ain't so." The growing focus on patient centricity has revealed a misalignment between what patients report as important to them about their disease and/or treatment, and the data collected in research and care. Decisions across healthcare are made using an evidence base most stakeholders acknowledge is inadequate. Patients might report that what is important to them are everyday life impacts, concepts that can be very different from the more typical clinical outcomes we often track. In this paper, we encourage expanding current thinking to all "impacts," not only health outcomes, but also the other equally (and sometimes more important) concerns patients report as important to them. We propose that a patient-centered core impact set be developed for each disease or condition of interest, and/or subpopulation of patients. A patient-centered core impact set begins with gathering from patients and caregivers an inventory of all impacts disease and treatments have on a patient's (and carers' and families') life. Then, through a formal prioritization process, a core set of impacts is derived, inclusive of but extending beyond relevant health outcomes. We offer several recommendations on how to move the goal of a patient-centered core impact set forward through collaboration, leadership, and establishment of a patient-centered core impact set development blueprint with supporting tools.