Value Lies in the Eye of the Patients: The Why, What, and How of Patient-Reported Outcomes Measures

Abstract Patient-reported outcomes (PROs) are any report of the status of a patient's health condition that comes directly from the patient (or in some cases from a caregiver or surrogate responder), without interpretation by a practitioner or anyone else. PROs are increasingly used as a valuable source of data in different domains of health care, including research, clinical practice, health care management, and decision making on the regulation, coverage, and reimbursement of new technologies. Several factors must be considered when selecting which PRO measure to use to ensure their appropriate use and interpretation as well as their relevance for decision makers. The increasing availability of PRO data, its integration with other data sources, and the improvements in data analytics offer a valuable opportunity to place the patient at the center of any health care process. However, several issues need to be addressed, including interoperability, data governance, security, privacy, and ethics, to realize an effective, integrated, standardized, real-time assessment of PROs in the health care systems

New Emerging Biomedical Technologies for Home-care and Telemedicine Applications: the Sensorwear Project

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Development features and study characteristics of mobile health apps in the management of chronic conditions:A systematic review of randomised trials

COVID-19 pandemic challenges have accelerated the reliance on digital health fuelling the expanded incorporation of mobile apps into healthcare services, particularly for the management of long-term conditions such as chronic diseases (CDs). However, the impact of health apps on outcomes for CD remains unclear, potentially owing to both the poor adoption of formal development standards in the design process and the methodological quality of studies. A systematic search of randomised trials was performed on Medline, ScienceDirect, the Cochrane Library and Scopus to provide a comprehensive outlook and review the impact of health apps on CD. We identified 69 studies on diabetes (n = 29), cardiovascular diseases (n = 13), chronic respiratory diseases (n = 13), cancer (n = 10) or their combinations (n = 4). The apps rarely adopted developmental factors in the design stage, with only around one-third of studies reporting user or healthcare professional engagement. Apps differed significantly in content, with a median of eight behaviour change techniques adopted, most frequently pertaining to the 'Feedback and monitoring' (91%) and 'Shaping knowledge' (72%) categories. As for the study methodologies, all studies adopted a traditional randomised control trial (RCT) design, with relatively short follow-ups and limited sample sizes. Findings were not significant for the majority of studies across all CD, with most RCTs revealing a high risk of bias. To support the adoption of apps for CD management, this review reinforces the need for more robust development and appropriate study characteristics to sustain evidence generation and elucidate whether study results reflect the true benefits of apps or a biased estimate due to unsuitable designs

Surrogate endpoints in trials-a call for better reporting

Better reporting of RCTs with primary surrogate endpoints

Recommendations for developing a lifecycle, multidimensional assessment framework for mobile medical apps

Digital health and mobile medical apps (MMAs) have shown great promise in transforming health care, but their adoption in clinical care has been unsatisfactory, and regulatory guidance and coverage decisions have been lacking or incomplete. A multidimensional assessment framework for regulatory, policymaking, health technology assessment, and coverage purposes based on the MMA lifecycle is needed. A targeted review of relevant policy documents from international sources was conducted to map current MMA assessment frameworks, to formulate 10 recommendations, subsequently shared amongst an expert panel of key stakeholders. Recommendations go beyond economic dimensions such as cost and economic evaluation and also include MMA development and update, classification and evidentiary requirements, performance and maintenance monitoring, usability testing, clinical evidence requirements, safety and security, equity considerations, organizational assessment, and additional outcome domains (patient empowerment and environmental impact). The COVID‐19 pandemic greatly expanded the use of MMAs, but temporary policies governing their use and oversight need consolidation through well‐developed frameworks to support decision‐makers, producers and introduction into clinical care processes, especially in light of the strong international, cross‐border character of MMAs, the new EU medical device and health technology assessment regulations, and the Next Generation EU funding earmarked for health digitalization

Real-World Data for the Evaluation of Transarterial Radioembolization versus Sorafenib in Hepatocellular Carcinoma: A Cost-Effectiveness Analysis

Abstract Objectives To perform a cost-effectiveness analysis comparing the use of transarterial radioembolization (TARE) with that of sorafenib in the treatment of patients with intermediate or advanced hepatocellular carcinoma (HCC) according to the Barcelona Clinic Liver Cancer staging system. Methods Patient-level data were consecutively recorded and collected at three oncology centers in Italy. A propensity score matching was performed to compare patients with similar clinical characteristics who underwent TARE or sorafenib treatment. Clinical data from the matched cohorts were used to populate a Markov model to project, on a lifetime horizon, life years, quality-adjusted life years, and economic outcomes associated with TARE and sorafenib for both intermediate and advanced HCC stages. Results Starting from data covering 389 and 241 patients who underwent TARE and sorafenib treatment, respectively, the propensity score matching yielded a total of 308 matched patients. For intermediate-stage patients, the model estimated for TARE versus sorafenib an incremental cost-utility ratio of €3,302/QALY (incremental cost-effectiveness ratio of €1,865 per life year gained), whereas for patients in advanced stage TARE dominated (lower costs and greater health improvements) compared with sorafenib. Conclusions From an Italian health care service perspective, TARE could be a cost-effective strategy in comparison with sorafenib for patients with intermediate or advanced HCC. The results from forthcoming randomized controlled trials comparing TARE with sorafenib will be able to confirm or reject the validity of this preliminary evaluation. In the meantime, decision makers can use these results to control and coordinate the diffusion of the technology

Distinguishing features in the assessment of mHealth apps

Introduction: The unparalleled surge in digital health adoption during the COVID-19 pandemic has emphasized the potential of mHealth apps. However, the quality of available evidence is generally low, and regulatory frameworks have focused on apps with medical purposes only, overlooking apps with significant interactions with patients that may require stronger oversight. Areas covered: To support this expanded evidence generation process, we identified the reasons that distinguish mHealth apps compared to medical devices at large and that should differentially feature their assessment. mHealth apps are characterized by the iterative nature of the corresponding interventions, frequent user interactions with a non-linear relationship between technology usage, engagement and outcomes, significant organizational implications, as well as challenges associated with genericization, their broad diagnostic potential, and price setting. Expert Opinion: The renewed reliance experienced during the pandemic and the unprecedented injection of resources through recovery instruments can further boost the development of apps. Only robust evidence of the benefits of mHealth apps will persuade health-care professionals and beneficiaries to systematically deploy them. Regulatory bodies will need to question their current approaches by adopting comprehensive evaluation processes that adequately consider the specific features of mHealth apps

Distinguishing features in the assessment of mHealth apps

Introduction: The unparalleled surge in digital health adoption during the COVID-19 pandemic has emphasized the potential of mHealth apps. However, the quality of available evidence is generally low, and regulatory frameworks have focused on apps with medical purposes only, overlooking apps with significant interactions with patients that may require stronger oversight. Areas covered: To support this expanded evidence generation process, we identified the reasons that distinguish mHealth apps compared to medical devices at large and that should differentially feature their assessment. mHealth apps are characterized by the iterative nature of the corresponding interventions, frequent user interactions with a non-linear relationship between technology usage, engagement and outcomes, significant organizational implications, as well as challenges associated with genericization, their broad diagnostic potential, and price setting. Expert Opinion: The renewed reliance experienced during the pandemic and the unprecedented injection of resources through recovery instruments can further boost the development of apps. Only robust evidence of the benefits of mHealth apps will persuade health-care professionals and beneficiaries to systematically deploy them. Regulatory bodies will need to question their current approaches by adopting comprehensive evaluation processes that adequately consider the specific features of mHealth apps