Measuring and Demonstrating the Value of Patient Engagement Across the Medicines Lifecycle: A Patient Engagement Impact Measurement Framework.

As the pharmaceutical industry advances towards more patient-focused product development, it is well recognized that meaningful patient engagement is required for the authentic patient voice to inform research and regulatory decisions. However, for this to happen systematically and consistently across the industry, there is still a need to evaluate and communicate the value of patient engagement to all stakeholders. Evaluating engagement also informs process improvement, elevating the value further.

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.

Co-creation of the Global Patient Experience Data Navigator: a multi-stakeholder initiative to ensure the patient voice is represented in health decision-making.

Background: Putting patients' needs and priorities at the forefront of healthcare initiatives and medical product development is critical to achieve outcomes that matter most to patients. This relies on the integration of early, meaningful patient engagement (PE) to learn what is important to patients, and collection of representative patient experience data (PXD). The increased number of PE/PXD efforts across global regulatory, health technology assessment, and healthcare systems is an important step forward to deliver improved health outcomes for patients.

Patient-centeredness and psychometric properties of the Defense and Veterans Pain Rating Scale 2.0 (DVPRS).

Objective: This study aims to assess the patient-centeredness and psychometric properties of the Defense and Veterans Pain Rating Scale 2.0 (DVPRS) as a patient-reported outcome measure (PROM) for pain assessment in a military population.

Design: A critical evaluation of the DVPRS was conducted, considering its fit-for-purpose as a PROM and its patient-centeredness using the National Health Council's Rubric to Capture the Patient Voice.

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.

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.

Stakeholder perception of pharmaceutical value: A multicriteria decision analysis pilot case study for value assessment in the United States.

Recent attention to value frameworks has highlighted limitations of current conventional value and health technology assessment (V/HTA) methods (eg, cost-effectiveness). Multicriteria decision analysis (MCDA) has potential as a supplemental tool to incorporate additional value criteria into conventional value assessment. To conduct a pilot study to illustrate the impact of an MCDA approach on the value perceptions of hypothetical treatment profiles from a multistakeholder panel.

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.

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.

The national economic burden of rare disease in the United States in 2019.

Background: To provide a comprehensive assessment of the total economic burden of rare diseases (RD) in the United States (U.S.) in 2019.

Development of an international template to support patient submissions in Health Technology Assessments.

Objectives: To develop an international template to support patient submissions in Health Technology Assessments (HTAs). This was to be based on the experience and feedback from the implementation and use of the Scottish Medicines Consortium's (SMC) Summary Information for Patient Groups (SIP).

Methods: To gather feedback on the SMC experience, web-based surveys were conducted with pharmaceutical companies and patient groups familiar with the SMC SIP.

Health Preference Research in Europe: A Review of Its Use in Marketing Authorization, Reimbursement, and Pricing Decisions-Report of the ISPOR Stated Preference Research Special Interest Group.

Objective: This study examines European decision makers' consideration and use of quantitative preference data.

Methods: The study reviewed quantitative preference data usage in 31 European countries to support marketing authorization, reimbursement, or pricing decisions. Use was defined as: agency guidance on preference data use, sponsor submission of preference data, or decision-maker collection of preference data.

Defining Patient Engagement in Research: Results of a Systematic Review and Analysis: Report of the ISPOR Patient-Centered Special Interest Group.

Objectives: Lack of clarity on the definition of "patient engagement" has been highlighted as a barrier to fully implementing patient engagement in research. This study identified themes within existing definitions related to patient engagement and proposes a consensus definition of "patient engagement in research."

Methods: A systematic review was conducted to identify definitions of patient engagement and related terms in published literature (2006-2018).

Patient-Community Perspectives on Real-World Evidence: Enhancing Engagement, Understanding, and Trust.

Background: Healthcare stakeholders have pronounced both enthusiasm and apprehension over the expanding use of real-world evidence (RWE). The patient community-those who benefit from new treatments but are vulnerable to potential safety risks and whose routine medical encounters are used to generate RWE-has been less vocal. Understanding patient perspectives on the use of RWE to guide clinical decision making and inform regulatory decisions and value assessments is imperative.

Patient-Reported Outcome Measures in the Food and Drug Administration Pilot Compendium: Meeting Today's Standards for Patient Engagement in Development?

Background: In 2016, the Food and Drug Administration (FDA) released a Pilot Clinical Outcome Assessment Compendium (COA Compendium) intended to foster patient-focused drug development (PFDD). However, it is unclear whether patient perspectives were solicited during development or validation of the included patient-reported outcome (PRO) measures.

Objective: To examine the pedigree of a sample of measures included in the COA Compendium.

Increasing uptake of comparative effectiveness and patient-centered outcomes research among stakeholders: insights from conference discussion.

The goal of comparative effectiveness research (CER) and patient-centered outcomes research (PCOR) is to improve health outcomes by providing stakeholders with evidence directly relevant to decision making. In January 2017, the Pharmaceutical Research and Manufacturers Association Foundation, alongside the Academy for Managed Care Pharmacy, organized a conference aimed at engaging experts and opinion leaders representing clinicians, patients and payers to identify and discuss barriers and strategies to enhancing uptake and use of CER/PCOR. This report summarizes the conference discussion in the following sections: preconference survey; summary of barriers and strategies to the uptake of CER/PCOR identified by conference attendees; and future perspectives on the field.

PEER-REVIEWED JOURNAL EDITORS' VIEWS ON REAL-WORLD EVIDENCE.

Objectives: Peer-review publication is a critical step to the translation and dissemination of research results into clinical practice guidelines, health technology assessment (HTA) and payment policies, and clinical care. The objective of this study was to examine current views of journal editors regarding: (i) The value of real-world evidence (RWE) and how it compares with other types of studies; (ii) Education and/or resources journal editors provide to their peer reviewers or perceive as needed for authors, reviewers, and editors related to RWE.

Methods: Journal editors' views on the value of RWE and editorial procedures for RWE manuscripts were obtained through telephone interviews, a survey, and in-person, roundtable discussion.

Evaluation of Healthcare Interventions and Big Data: Review of Associated Data Issues.

Although the analysis of 'big data' holds tremendous potential to improve patient care, there remain significant challenges before it can be realized. Accuracy and completeness of data, linkage of disparate data sources, and access to data are areas that require particular focus. This article discusses these areas and shares strategies to promote progress.

Value to Whom? The Patient Voice in the Value Discussion.

Background: Professional societies and other organizations have recently taken a visible role trying to define treatment value via value frameworks and assessments, providing payer or provider recommendations, and potentially impacting patient access. Patient perspectives routinely differ from those of other stakeholders. Yet, it is not always apparent that patients were engaged in value framework development or assessment.

National Trends in ADHD Diagnosis and Treatment: Comparison of Youth and Adult Office-Based Visits.

Objectives: The study objective was to assess national trends in the diagnosis of attention-deficit hyperactivity disorder (ADHD) in outpatient visits by comparing adults and youths. Also examined were recent stimulant prescribing patterns for ADHD visits by youths and adults.

Methods: Databases from the 1999-2010 National Ambulatory Medical Care Survey and National Hospital Ambulatory Medical Care Survey were used in this cross-sectional study to analyze outpatient visit data of youths (ages two to 17 years; unweighted N=112,404) and adults (ages 18-64; unweighted N=426,209).

FDAMA Section 114: Why the Renewed Interest?

The FDA regulates the use of information by biopharmaceutical companies in their promotional activities. Section 114 of the Food and Drug Administration Modernization Act of 1997 (FDAMA) was specifically designed to allow companies to more readily disseminate health care economic information (HCEI) to those who need it for formulary decision making. However, very little HCEI has been distributed promotionally under this provision over the past 17 years.