Proposal for capturing patient experience through extended value frameworks of health technologies.

BACKGROUND: Inclusion of patient experience (PEx) in health technology assessment (HTA) has become increasingly important; however, no harmonized approach exists to help manufacturers or decision makers ensure PEx considerations are fair, consistent, and thorough within global HTA frameworks. OBJECTIVE: To develop a proposal for including PEx in the HTA frameworks of health technologies. METHODS: A systematic literature review (SLR) on existing value frameworks (VFs) was conducted to capture how PEx-related value judgment is currently considered. Guided by the results of the SLR, a research group including HTA experts and patient representatives used an iterative process to develop potential value domains to capture PEx, in accordance with international guidelines. Subsequently, a panel of international payer experts was used to challenge the proposed PEx domains and provide recommendations for implementation. RESULTS: The SLR found 61 VFs and multi-criteria decision analyses (MCDAs) that considered PEx; however, PEx-related value elements were often referred to superficially, without clear definitions. Five potential PEx domains, with proposed measures for each, were developed and refined using expert feedback: (1) responsiveness to patient's individual needs, (2) improved health literacy and empowerment, (3) patient and caregiver reported outcomes, (4) household's financial burden, and (5) improved access for vulnerable patient populations. A flexible approach for framework implementation was proposed. CONCLUSIONS: Proposed PEx domains could be implemented at multiple levels of healthcare decision making to formalize consideration of PEx in the assessment of value, either through the extension of existing VFs or to create new PEx-focused VFs and more holistic decision making tools. DISCLOSURES: This study was funded and sponsored by UCB Pharma. The funding agreement ensured the authors' independence in designing the study, interpreting the data, writing, and publishing the report. Charokopou, Mountain, and Szegvari are employed by UCB Pharma. Inotai, Jakab, and Kalo are employed by Syreon Research Institute, which received funding from UCB Pharma for this research. Brixner has received fees from AbbVie, Elevar, Millcreek Outcomes Group, Novartis, Sanofi, UCB Pharma, and Xcenda. Campbell has received grants and contracts from the PhRMA Foundation and the Institute for Clinical and Economic Review. During a sabbatical leave, Campbell collaborated with Syreon Research Institute on research projects that included funding from UCB Pharma. Hawkins has received consultancy fees from UCB Pharma. Kristensen has received speakers bureau fees from Pfizer, AbbVie, Amgen, UCB Pharma, Celgene, Bristol-Myers Squibb, MSD, Novartis, Eli Lilly, and Janssen Pharmaceuticals and consultancy fees from UCB Pharma.

Development and psychometric evaluation of the assessment of self-injection questionnaire: an adaptation of the self-injection assessment questionnaire.

BACKGROUND: Patient-reported outcome (PRO) instruments provide robust and effective means of evaluating patients' treatment experience; however, none adequately cover experience using self-injection devices with enhanced features, such as an electromechanical autoinjector (e-Device). The aim of this study was to develop a PRO instrument that accurately assesses patient experience of using an e-Device and to evaluate its psychometric properties. METHODS: A mixed-methods approach was taken; two parallel, targeted literature reviews were conducted to identify relevant concepts and existing self-injection PRO instruments that could be adapted. Patient feedback obtained from two focus groups was used to inform initial instrument development. The pilot instrument was then administered in a multicenter, open-label, phase 3 clinical study in which patients self-injected certolizumab pegol using an e-Device, to gather evidence of its psychometric qualities. Exit interviews were conducted with a sub-sample of patients enrolled in the study to confirm the appropriateness and clarity of the items included and cognitively debrief the instrument. Confirmatory factor analysis (CFA) was conducted on all items, and each domain's internal consistency was measured using Cronbach's ɑ. RESULTS: The literature searches identified several e-Device-specific concepts related to device features, device function, side effects/reactions/pain, confidence, and interference/convenience in daily life. Seven existing PRO instruments were identified. The Self-Injection Assessment Questionnaire (SIAQ), containing pre- and post-injection questionnaire modules, was selected as most suitable and adapted using feedback from 19 patients in the two focus groups to form the pilot Assessment of Self-Injection (ASI) questionnaire. CFA resulted in some changes to the grouping of items in the post-injection module domains following psychometric evaluation of the ASI. Internal consistency was satisfactory for all pre- and post-injection domains (ɑ > 0.8). Cognitive debriefing results from 12 patient exit interviews confirmed the ASI's appropriateness and clarity. CONCLUSIONS: The ASI was developed iteratively with patient input and was evaluated in its intended clinical context of use. Psychometric analyses indicated promising cross-sectional results; the ASI was well understood and considered relevant by patients self-injecting using the e-Device, suggesting that it could be used in real-world settings to aid with clinical decision making. TRIAL REGISTRATION: NCT03357471.

A Targeted Literature Review Examining Biologic Therapy Compliance and Persistence in Chronic Inflammatory Diseases to Identify the Associated Unmet Needs, Driving Factors, and Consequences.

Chronic inflammatory diseases (CIDs) represent a substantial clinical and economic burden to patients, providers, payers and society overall. Biologics, such as tumor necrosis factor inhibitors (TNFi), have emerged as effective treatment options for patients with CIDs. However, the therapeutic potential of biologics is not always achieved in clinical practice, with results from studies examining the use of biologics in real-world settings suggesting lower levels of treatment effectiveness compared with clinical trial results. Using a targeted approach, this literature review demonstrates that compliance and persistence with biologic therapy is suboptimal and that this has implications for both clinical outcomes and treatment costs. The review identified a variety of predictors of treatment compliance and persistence, including increased age, female gender, presence of comorbidities, increased disease activity, longer disease duration, smoking, increased body mass index, higher biologic treatment dose, higher treatment cost and lower health-related quality-of-life scores. Patients often cited factors associated with medication delivery as a reason for non-compliance and non-persistence, and device-related improvements to treatment delivery were associated with higher rates of compliance and persistence. The articles identified in this review provide insights that have the potential to help guide the development of new solutions to improve disease management and optimize treatment regimens. This has the potential to benefit patients' health by improving clinical outcomes and to reduce the burden to society by limiting the economic impact of patients' disease. FUNDING: UCB Pharma.

The Parker Model: Applying a Qualitative Three-Step Approach to Optimally Utilize Input from Stakeholders When Introducing New Device Technologies in the Management of Chronic Rheumatic Diseases.

BACKGROUND AND OBJECTIVE: Qualitative methods such as semi-structured interviews and focus-groups are used to evaluate the applicability and relevance of device technologies in clinical practice, but when used alone, often lack generalizability. This study aimed to assess the face validity and feasibility of using a composite, three-step qualitative method (the Parker Model), to inform the development and implementation of ava®, an electromechanical device (e-Device) for subcutaneous self-administration of the biologic, certolizumab pegol (CZP), used to treat rheumatic diseases. METHODS: The Parker Model combines concept mapping (CM), participatory design (PD), and stakeholder evaluation (SE). CM, a structured group process, was used to identify patients' opinions and concerns regarding the e-Device. Patients used this information in iterative PD sessions to create personal e-Device prototypes in cooperation with a designer and a healthcare professional. SE was performed based on semi-structured group and individual interviews with patients and disease-management stakeholders. RESULTS: The study recruited 14 patients, two doctors, two nurses, one medical secretary, and four other public servants. Three CM workshops revealed four key considerations: technical usability, physical design, concerns, and enthusiasm. Four personalized prototypes were developed during PD sessions. SE confirmed that the identified considerations were pivotal for the implementation and adaptation of the e-Device. CONCLUSIONS: This study is the first to apply a composite, qualitative research model when introducing an e-Device for the treatment and management of rheumatic disease. Results show that input from patients and other stakeholders using the Parker Model can add value to the development and implementation of an e-Device.