Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Objectives: This study quantified the public value (PV) of the criteria and sub-criteria in the current drug reimbursement systems in South Korea and examined sociodemographic factors that associated with PV.

Methods: The Analytic Hierarchy Process (AHP) was used to quantify the PVs of criteria and sub-criteria. We developed a questionnaire to generate pairwise comparison matrices among criteria and sub-criteria. From 27 March to 1 April 2023, we recruited 1,000 study participants using a quota sampling method stratified by age, sex, and region based on Korean census data.

Results: The PVs for the criteria were highest for clinical usefulness (28.5%), followed by cost-effectiveness (27.1%), budget impact (24.3%), and reimbursement in other countries (20.1%). The sociodemographic characteristics of the participants had a significant impact on the PVs of the criteria. Willingness to pay additional premiums for national health insurance was negatively associated with PV for clinical usefulness and cost-effectiveness and positively associated with PV for reimbursement in other countries.

Conclusions: The public prioritized clinical usefulness and cost-effectiveness as the main criteria. However, the PVs of the criteria were divergent and associated with sociodemographic factors. Divergent public interests require an evidence-informed deliberative process for reimbursement decisions.

Introduction: The Human Immunodeficiency Virus (HIV) is one of the greatest public health challenges still facing communities worldwide, and until this moment, no vaccine is available for its prevention. In Brazil, the Rio de Janeiro State has stood out regarding the prevalence of this disease. As a result, an important state to consider the Willingness to Pay (WTP) for a hypothetical HIV vaccine to help with future pricing.

Methods: A cross-sectional study was conducted to assess the acceptability and WTP of individuals from Rio de Janeiro State for a hypothetical HIV vaccine with a 70% efficacy.

Results: 600 individuals were interviewed and the acceptability for this hypothetical vaccine was 77.2%. In addition, 452 participants were eligible for the WTP analysis and would accept a WTP US$79.37 (400 BRL) for this vaccine, a higher value than that found in another study (200 BRL) conducted in the Northern region of Brazil under the same methodological conditions.

Conclusion: Economic studies such as WTP can contribute to discussions regarding the prices and specifications for future vaccines, particularly for a HIV vaccine in countries such as Brazil with over 5,000 municipalities spread across regions with diverse characteristics and challenges in terms of socioeconomic, epidemiological and cultural differences.

Objectives: To develop a robust algorithm to accurately calculate 'daily complete dose counts' for inhaled medicines, used in percent adherence calculations, from electronically-captured nebulizer data within the CFHealthHub Learning Health System.

Methods: A multi-center, cross-sectional study involved participants and clinicians reviewing real-world inhaled medicine usage records and triangulating them with objective nebulizer data to establish a consensus on 'daily complete dose counts.' An algorithm, which used only objective nebulizer data, was then developed using a derivation dataset and evaluated using internal validation dataset. The agreement and accuracy between the algorithm-derived and consensus-derived 'daily complete dose counts' was examined, with the consensus-derived count as the reference standard.

Results: Twelve people with CF participated. The algorithm derived a 'daily complete dose count' by screening out 'invalid' doses (those <60s in duration or run in cleaning mode), combining all doses starting within 120s of each other, and then screening out all doses with duration < 480s which were interrupted by power supply failure. The kappa co-efficient was 0.85 (0.71-0.91) in the derivation and 0.86 (0.77-0.94) in the validation dataset.

Conclusions: The algorithm demonstrated strong agreement with the participant-clinician consensus, enhancing confidence in CFHealthHub data. Publishingdata processing methods can encourage trust in digital endpoints and serve as an exemplar for other projects.

Introduction: Sensor-based digital health technology (DHT) has emerged as a promising means to assess patient functioning within and outside clinical trials. Sensor-based functional outcomes (SBFOs) provide valuable insights that complement other measures of how a patient feels or functions to enhance understanding of the patient experience to inform medical product development.

Areas covered: This perspective paper provides recommendations for defining SBFOs, discusses the core evidence required to support SBFOs to inform decision-making, and considers future directions for the field.

Expert commentary: The clinical outcome assessment (COA) development process provides an important starting point for developing patient-centered SBFOs; however, given the infancy of the field, SBFO development may benefit from a hybrid approach to evidence generation by merging exploratory data analysis with patient engagement in measure development. Effective SBFO development requires combining unique expertise in patient engagement, measurement and regulatory science, and digital health and analytics. Challenges specific to SBFO development include identifying concepts of interest, ensuring measurement of meaningful aspects of health, and identifying thresholds for meaningful change. SBFOs are complementary to other COAs and, as part of an integrated evidence strategy, offer great promise in fostering a holistic understanding of patient experience and treatment benefits, particularly in real-world settings.

Introduction: Over the last decade increasing examples indicate opportunities to measure patient functioning and its relevance for clinical and regulatory decision making via endpoints collected through digital health technologies. More recently, we have seen such measures support primary study endpoints and enable smaller trials. The field is advancing fast: validation requirements have been proposed in the literature and regulators are releasing new guidances to review these endpoints. Pharmaceutical companies are embracing collaborations to develop them and working with academia and patient organizations in their development. However, the road to validation and regulatory acceptance is lengthy. The full value of digital endpoints cannot be unlocked until better collaboration and modular evidence frameworks are developed enabling re-use of evidence and repurposing of digital endpoints.

Areas covered: This paper proposes a solution by presenting a novel modular evidence framework -the Digital Evidence Ecosystem and Protocols (DEEP)- enabling repurposing of measurement solutions, re-use of evidence, application of standards and also facilitates collaboration with health technology assessment bodies.

Expert opinion: The integration of digital endpoints in healthcare, essential for personalized and remote care, requires harmonization and transparency. The proposed novel stack model offers a modular approach, fostering collaboration and expediting the adoption in patient care.

Introduction: Randomized controlled trials (RCTs) are important sources of evidence that strongly influence guidelines for patient management, including for elevated blood pressure in adults.

Areas covered: Critical questions regarding the interpretation of hypertension trial results have recently increased, especially for concerns over methodology. In particular, investigator adherence to the protocol and patient adherence to investigational drugs are often far from optimal. These issues may be ignored or underreported because physicians' behavior during trials is often not monitored and patients' medication adherence is neither measured adequately nor reported or analyzed in the final report or in the publication. This situation may lead to misinterpretations of study results and misevaluations of the safety and efficacy profile of new drugs. In this short review, the problem of measuring, reporting, and analyzing drug adherence in RCTs is discussed and illustrated with several examples in the field of hypertension.

Expert opinion: The main conclusion is that drug adherence should always be measured in clinical trials, possibly with more than one method. In addition, prespecified analyses of adherence data should be included in the statistical plan of all trials to improve their overall quality.

Introduction: Digital measures and digital health-care delivery have been rarely implemented in lower-and-middle-income countries (LMICs), contributing to worsening global disparities and inequities. Sustainable ways to implement and use digital approaches will help to improve time to access, management, and quality of life in persons with epilepsy, goals that remain unreachable in under-resourced communities. As under-resourced environments differ in human and economic resources, no one approach will be appropriate to all LMICs.

Areas covered: Digital health and tools to monitor and measure digital endpoints and metrics of quality of life will need to be developed or adapted to the specific needs of under-resourced areas. Portable technologies may partially address the urban-rural divide. Careful delineation of stakeholders and their engagement and alignment in all efforts is critically important if these initiatives are to be successfully sustained. Privacy issues, neglected in many regions globally, must be purposefully addressed.

Expert opinion: Epilepsy care in under-resourced environments has been limited by the lack of relevant technologies for diagnosis and treatment. Digital biomarkers, and investigative technological advances, may finally make it feasible to sustainably improve care delivery and ultimately quality of life including personalized epilepsy care.

Introduction: As digital health expands, reliance on digital endpoints is rapidly increasing to improve diagnostic accuracy and management in the healthcare field. Digital endpoints are beneficial to monitor how patient's clinical information is processed outside of a clinical setting.

Areas covered: Implications of cardiac digital endpoints play a role in allowing patients to track their clinical data outside of a clinical setting. Advances in cardiac digital endpoints involve advanced devices and implants, trackers, and artificial intelligence. We will explore further digital endpoints within cardiology and threats as well as security concerns for policies to focus on the maintenance of safe patient health data analysis, transmission, and processing.

Expert opinion: As digital endpoints evolve and expand, policymakers must ensure there is adequate cybersecurity surrounding them. We believe guidelines should be in place to make sure data is accessed solely on a secure connection and access to digital literacy for patients should be readily available.