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OBJECTIVE: To provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials to external controls from individual patient-level real-world data (IPD-RWD). In addition, to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.
METHODS: A systematic literature review (until March 1st 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively to methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and four European HTA organizations (2015-2023).
RESULTS: Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data to IPD-RWD-based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data; and analytical comparative modelling methods. Seven guidelines also focused on research design, RWD quality and transparency aspects, and four of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n=15) and HTA (n=35) assessment reports were often based on aggregate data and lacked transparency due to the few details provided.
CONCLUSIONS: Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision-making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.

UNASSIGNED: This paper summarizes the results from a forum of healthcare experts, academia representatives, and public agency officials from emerging and established market countries on Value-Based Healthcare (VBHC) and Health Technology Assessment (HTA). Presentations from experts provided insights into current developments and challenges, followed by interactive roundtable discussions. Emerging markets have unique healthcare systems, patient populations, resource constraints and needs.
UNASSIGNED: Each roundtable explored specific topics including the role of HTA and Real-world evidence (RWE) in healthcare decision-making, challenges in biosimilar value assessment and incorporating non-price criteria reflecting context-related specifications of emerging markets such as the multifaceted nature of value in healthcare decision-making, emphasizing stakeholder perspectives and system complexities.
UNASSIGNED: RWE emerged as important in understanding biosimilar value recognition and decision-making processes, with insights into its applications and challenges. Recommendations were provided for utilizing Multi-Criteria Decision Analysis (MCDA) in pharmaceutical procurement, particularly for off-patent medicines, underscoring the importance of comprehensive evaluation frameworks and adherence to value-based principles. Overall findings suggest avenues for collaboration between industry, academia, and public agencies to address implementation barriers and promote equitable, efficient, and high-quality healthcare systems in emerging markets through public-private partnerships, joint capacity building and training initiatives, and knowledge transfers.

Previous reviews on the cost of illness (COI) of Parkinson\'s disease (PD) have often focused on health-care costs due to PD, underestimating its effects on other sectors. This systematic review determines the COI of PD from a societal perspective. The protocol was registered in PROSPERO (ID: CRD42023428937). Embase, Medline, and EconLit were searched up to October 12, 2023, for studies determining the COI of PD from a societal perspective. From 2812 abstracts, 17 studies were included. The COI of PD averaged €20,911.37 per patient per year, increasing to almost €100,000 in the most severely affected patients. Health-care costs accounted for 46.1% of total costs, followed by productivity loss (37.4%) and costs to patient and family (16.4%). The COI of PD strongly varied between different geographical regions, with costs in North America 3.6 times higher compared to Asia. This study is the first to identify the relative importance of different cost items. Most important were reduced employment, government benefits, informal care, medication, nursing homes, and hospital admission. There was strong variety in the cost items that were included, with 55.2% of cost items measured in fewer than half of articles. Our review shows that PD-COI is high and appears in various cost sectors, with strong variety in the cost items included in different studies. Therefore, a guideline for the measurement of COI in PD should be developed to harmonize this. This article provides a first step toward the development of such a tool by identifying which cost items are most relevant. © 2024 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

OBJECTIVE: Colonoscopy-based surveillance to prevent colorectal cancer (CRC) causes substantial burden for patients and healthcare. Stool tests may help to reduce surveillance colonoscopies, by limiting colonoscopies to individuals at increased risk of AN.
METHODS: This cross-sectional observational study included individuals aged 50-75 with surveillance indication. Before bowel preparation, participants collected samples for a multitarget stool DNA (mt-sDNA) test and two fecal immunochemical tests (FITs). Test accuracies were calculated for all surveillance indications. Only for the post-polypectomy indication, most common and associated with a relatively low CRC risk, long-term impact of stool-based surveillance was evaluated with the ASCCA model. Stool-based strategies were simulated to tune each tests\' positivity threshold to obtain strategies at least as effective as colonoscopy surveillance.
RESULTS: 3453 individuals had results for all stool tests and colonoscopy. 2226 had previous polypectomy, 1003 previous CRC and 224 familial risk. Areas under the receiver operating characteristic curve for AN were 0.72 (95% CI; 0.69-0.75) for the mt-sDNA test, 0.61 (95% CI; 0.58-0.64) for the FIT OC-Sensor and 0.59 (95% CI; 0.56-0.61) for the FIT FOB-Gold. Stool-based post-polypectomy surveillance strategies at least as effective as colonoscopy surveillance, reduced the number of colonoscopies by 15-41% and required 5.6-9.5 stool tests over the lifetime of a person. Mt-sDNA-based surveillance was more costly than colonoscopy surveillance, whereas FIT-based surveillance saved costs.
CONCLUSIONS: This study shows that stool-based post-polypectomy surveillance strategies can be safe and cost-effective, with potential to reduce the number of colonoscopies by up to 41%.

UNASSIGNED: The major drivers of carbon dioxide (CO2eq) emissions of external beam radiation therapy (EBRT) are not well known and limit our ability to initiate mitigation strategies.
UNASSIGNED: We describe the carbon footprint of four typical centers. We explore direct EBRT associated factors such as the impact of fractionation and use of MRI-LINAC, as well as indirect factors (e.g. patient rides). Treatment strategy related CO2eq emissions are included in a health technology assessment analysis that takes into account CO2eq emissions.
UNASSIGNED: A typical EBRT treatment emits from 185 kgCO2eq to 2066 kgCO2eq. CO2eq emissions are mostly driven by (i) accelerator acquisition and maintenance (37.8 %), (ii) patients and workers rides (32.7 %), (iii) drugs and medical devices (7.3 %), (iv) direct energy consumption (6.1 %), and (v) building and bunker construction (5.6 %) with a substantial heterogeneity among centers. Hypofractionation has a strong impact to mitigate emissions. MRI-LINAC is associated with a substantial increase in CO2eq emissions per fraction and requires ultra hypofractionation in 5 fractions to achieve a similar carbon footprint compared to 20 fractions treatment schemes. The expected limited small increase in toxicities due to hypofractionation (when existing) are in the same range as avoided detrimental effects to future people\'s health thanks to CO2eq mitigation.
UNASSIGNED: Carbon footprint of EBRT is not neglectable and could be mitigated. When safely feasible, hypofractionation is one of the main factors to decrease this impact. Taking into account CO2eq emissions has a substantial impact on the health technology assessment of EBRT, favoring hypofractionated regimens.

Vaccine-preventable diseases continue to generate a substantial burden on health, healthcare systems, and societies, which is projected to increase with population ageing. There is a need to better understand the full value of adult immunisation programmes corresponding to the broader value of vaccine frameworks that are recommended for evidence-based decision-making. This review aims to summarise and map evidence for the value of selected adult immunisation programmes (seasonal influenza, pneumococcal disease, RSV, and HZ) in ten diverse countries. We conducted a structured literature review of evidence published from 2017 to 2023. An existing framework was used to structure the assessment, developing matrices demonstrating the elements of value evidenced for each vaccine and country of focus. Our analysis showed substantial evidence base on the value of adult immunisation programmes, but the availability of evidence varied by value element and by vaccine. The impact on the quality of life of the vaccinated individual was the most evidenced value element. Mortality benefits for vaccinated individuals and cost-offsets to healthcare systems were also well-evidenced. The availability of evidence for \'broader\' societal value elements (such as transmission value, carer productivity and impact on social equity, and antimicrobial resistance prevention) varied. No evidence was identified relating to the broader value elements of macroeconomic effects, value to other interventions, or effects on the quality of life of caregivers. Robust evidence exists to show that adult immunisation programmes generate substantial value for population health and health systems, yet some elements of broader value remain underrepresented in the academic literature. Without such evidence, the full value of immunisation programmes is underestimated, risking suboptimal policy decisions.

Background The scarcity of resources restricts healthcare financing decisions, affecting the population\'s health. Health technology assessment (HTA) balances restricted budgets with the best possible health outcomes. We aim to characterize the current status of HTA in Algeria and describe the future directions for HTA implementation according to the priorities set by local stakeholders. Methods Stakeholders from the public and private sectors responded to a policy survey about the current and preferred future status of HTA implementation in Algeria. The survey was administered during an online workshop and used a widely accepted international scorecard covering eight domains: capacity building, HTA financing, process and organizational structure, scope of HTA implementation, decision criteria, standardization of methodology, use of local data, and international collaboration. After that, one-on-one interviews with another local expert were conducted to validate and modify the draft recommendations. The interviewees were representatives from government agencies, hospitals, and pharmaceutical companies. Results Thirty-one experts filled out the HTA scorecard survey; most of them were from the public sector (74%). They highlighted that project-based HTA workshops or short courses were the most common form of HTA education in Algeria and recommended the establishment of postgraduate HTA training programs in the future to build sustainable capacities. They reported a lack of funding for HTA research and critical appraisal and recommended an increased public budget for HTA and the introduction of submission fees by manufacturers. There was consensus about the need for local HTA evidence generation in the future. Most of the experts advocated an explicit soft decision threshold. The interviewees further recommended using multi-criteria decision analysis in the short term. The application of quality indicators was believed to improve the reliability of the HTA process. Conclusion The results of our policy research delineate the gap between the current and preferred future status of HTA in Algeria based on insights from multiple stakeholders. The need to improve the educational HTA programs in Algeria, use local data in policy decisions, and increase funding for HTA were the most advocated recommendations.

Cost-effectiveness analyses (CEA) are important in healthcare decision-making and resource allocation; however, expanding the scope of CEAs beyond the traditional clinicoeconomic concepts to also include value elements such as health equity has attracted much interest in recent years. This umbrella review aimed to synthesize evidence on how equity concepts have been considered in modified types of CEAs. Publicly available articles in MEDLINE were searched on January 25, 2024, to identify systematic reviews (SLRs) published in English since 2013 that incorporate health equity considerations in CEAs. Title/abstract, full-text article screening and data extraction were conducted by a single reviewer and validated by a second reviewer. Results were qualitatively synthesized to identify common themes. Eight SLRs were included. Distributional CEAs (DCEA), equity-based weighting, extended CEA (ECEA), mathematical programming and multi-criteria decision analysis (MCDA) were the most discussed approaches. A lack of consensus on the best approach for incorporating health equity into CEAs was highlighted, as these approaches are not currently consistently used in decision-making. Important limitations included scarcity of robust data to inform health equity indices, bias associated with commonly used health outcome metrics and the challenge of accounting for additional contextual factors such as fairness and opportunity costs. Proposals to expand CEAs to address equity issues come with challenges due to data unavailability, methods complexity, and decision-makers unfamiliarity with these approaches. Our review indicates that extended and distributional CEAs can support decision-making by capturing the impact of inequity on the clinical and cost-effectiveness assessment of treatments, although future modeling should account for additional contextual factors such as fairness and opportunity costs. Recommendations for actions moving forward include standardization of data collection for outcomes related to equity and familiarity with methodologies to account for the complexities of integrating health equity considerations in CEAs.

There is global interest in institutionalizing Health Technology Assessment (HTA) to inform resource allocation decisions. However, institutionalization of HTA remains limited particularly in low- and lower-middle-income countries. We conducted this scoping review to synthesize evidence on factors that influence the institutionalization of HTA at the macro (national)-level across countries globally. We searched for relevant literature in six databases namely PubMed, Embase, CINAHL, Scopus, EconLit, and Google Scholar. We conducted the last search on December 31, 2021. We identified 77 articles that described factors that influence institutionalization of HTA across 135 high-, middle-, and low-income countries. We analyzed these articles thematically. We identified five sets of factors that influence the institutionalization of HTA across countries of different income levels. These factors include: (1) organizational resources such as organizational structures, and skilled human, financial, and information resources; (2) legal frameworks, policies, and guidelines for HTA; (3) learning and advocacy for HTA; (4) stakeholder-related factors such as stakeholders\' interests, awareness, and understanding; and (5) collaborative support for HTA through international networks and non-governmental and multi-lateral organizations. Countries seeking to institutionalize HTA should map the availability of the factors identified in this review. Developing these factors wherever necessary can influence a country\'s capacity to institutionalize the conduct and use of HTA.