Search

Osteogenesis imperfecta (OI) is a multisystem disorder most often caused by pathogenic variants in genes that encode type I collagen. Type I collagen is abundant not only in bone but also in multiple tissues including skin, tendons, cornea, blood vessels and heart. Thus, OI can be expected to affect cardiovascular system, and there are numerous reports of cardiovascular disease (CVD) in people with OI. However, there is no consensus on how CVD in OI should be assessed or managed. To fill this gap, a multidisciplinary group was convened to develop clinical guidance. The work included a systematic review of the available literature and, using a modified Delphi approach, the development of a series of statements summarizing current knowledge. Fourteen clinical recommendations were developed to guide clinicians, patients, and stakeholders about an approach for CVD in adults with OI. This paper describes how the work was conducted and provides the background and rationale for each recommendation. Furthermore, we highlight knowledge gaps and suggest research priorities for the future study of CVD in OI., (© The Author(s) [2024]. Published by Oxford University Press on behalf of the American Society for Bone and Mineral Research.)

Objectives: Quality-adjusted life years (QALYs) have been challenged as a measure of benefit for people with disabilities, particularly for those in low-utility health states or with irreversible disability. This study examined the impact of a QALY-based assessment on the price for a hypothetical treatment for Duchenne muscular dystrophy (DMD), a progressive, genetic neuromuscular disease., Methods: A previously published, 5-state model, which analyzed treatments for early ambulatory (EA) DMD patients, was replicated, validated, and adapted to include early nonambulatory (ENA) DMD patients. The model was used to assess a QALY-based threshold price (maximum cost-effective price) for a hypothetical treatment for 13-year-old ENA and 5-year-old EA patients (initial health states with lower and higher utility, respectively). All inputs were replicated including willingness-to-pay thresholds of $50 000 to $200 000/QALY., Results: In contrast to EA patients, ENA patients had a 98% modeled decline in QALY-based threshold price at a willingness-to-pay of $150 000/QALY or higher, despite equal treatment benefit (delayed progression/death). At $100 000/QALY or lower, net nontreatment costs exceeded health benefits, implying any treatment for ENA patients would not be considered cost-effective, even at $0 price, including an indefinite pause in disease progression., Conclusions: For certain severe, disabling conditions, traditional approaches are likely to conclude that treatments are not cost-effective at any price once a patient progresses to a disabled health state with low utility value. These findings elucidate theoretical/ethical concerns regarding potential discriminatory properties of traditional QALY assessments for people with disabilities, particularly those who have lost ambulation or have other physical limitations., Competing Interests: Author Disclosures Author disclosure forms can be accessed below in the Supplemental Material section., (Copyright © 2024. Published by Elsevier Inc.)

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. We describe the development of a conceptual, yet practical, framework for measuring the impact of engagement to achieve this. The framework depicts how metrics can be used to capture and assess the inputs, outputs, and value of patient engagement across the medicines lifecycle. Although conceived in the context of systems and processes within one company, Novartis, the framework was co-created with patient advisors and designed to be both patient-relevant and adaptable for any pharmaceutical organization. The adoption and evolution of the framework will help to demonstrate the value-to patients, healthcare systems, and businesses-of integrating patient engagement into core activities across the medicines lifecycle. We encourage the pharmaceutical industry to apply impact measurement to build a robust evidence base, through measuring, publishing, and communicating the value of patient engagement., (© 2024. The Author(s).)

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., (© 2024. The Author(s).)

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., Setting: The study focused on the use of the DVPRS within the Department of Defense (DoD) and Veterans Health Administration (VA) healthcare settings., Subjects: The DVPRS was evaluated based on published studies and information provided by measure developers. The assessment included content validity, reliability, construct validity, and ability to detect change. Patient-centeredness and patient engagement were assessed across multiple domains., Methods: Two independent reviewers assessed the DVPRS using a tool/checklist/questionnaire, and any rating discrepancies were resolved through consensus. The assessment included an evaluation of psychometric properties and patient-centeredness based on established criteria., Results: The DVPRS lacked sufficient evidence of content validity, with no patient involvement in its development. Construct validity was not assessed adequately, and confirmatory factor analysis was not performed. Patient-centeredness and patient engagement were also limited, with only a few domains showing meaningful evidence of patient partnership., Conclusions: The DVPRS as a PROM for pain assessment in the military population falls short in terms of content validity, construct validity, and patient-centeredness. It requires further development and validation, including meaningful patient engagement, to meet current standards and best practices for PROMs., (© The Author(s) 2023. Published by Oxford University Press on behalf of the American Academy of Pain Medicine. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.)

Letter to the Editor We are writing regarding the Innovations in Pharmacy commentary entitled, "Evidentiary Standards for Patient-Centered Core Impact Value Claims."(1) We thank Dr. Langley for commenting on the National Health Council's work on patient-centered core impact sets (PC-CIS), an initiative spearheaded by the nonprofit organization and its membership with multi-stakeholder representation and input.(2-4) While we have tried to be clear and transparent about the intent of PC-CIS, the commentary made it apparent to us we need to (and will) do more to be explicit about what a PC-CIS is and is not, and its possible downstream uses. We believe the PC-CIS concept was misrepresented in the commentary and want to provide clarification for readers so they can consider the merits of the initiative for themselves., (© Individual authors.)

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., (© 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)

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.", (Copyright © 2022. Published by Elsevier Inc.)