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Background: The design of health benefits package (HBP), and its associated payment and pricing system, is central to the performance of government-funded health insurance programmes. We evaluated the impact of revision in HBP within India's Pradhan Mantri Jan Arogya Yojana (PM-JAY) on provider behaviour, manifesting in terms of utilisation of services. Methods: We analysed the data on 1.35 million hospitalisation claims submitted by all the 886 (222 government and 664 private) empanelled hospitals in state of Punjab, from August 2019 to December 2022, to assess the change in utilisation from HBP 1.0 to HBP 2.0. The packages were stratified based on the nature of revision introduced in HBP 2.0, i.e., change in nomenclature, construct, price, or a combination of these. Data from National Health System Cost Database on cost of each of the packages was used to determine the cost-price differential for each package during HBP 1.0 and 2.0 respectively. A dose-response relationship was also evaluated, based on the multiplicity of revision type undertaken, or based on extent of price correction done. Change in the number of monthly claims, and the number of monthly claims per package was computed for each package category using an appropriate seasonal autoregressive integrated moving average (SARIMA) time series model. Findings: Overall, we found that the HBP revision led to a positive impact on utilisation of services. While changes in HBP nomenclature and construct had a positive effect, incorporating price corrections further accentuated the impact. The pricing reforms highly impacted those packages which were originally significantly under-priced. However, we did not find statistically significant dose-response relationship based on extent of price correction. Thirdly, the overall impact of HBP revision was similar in public and private hospitals. Interpretation: Our paper demonstrates the significant positive impact of PM-JAY HBP revisions on utilisation. HBP revisions need to be undertaken with the anticipation of its long-term intended effects. Funding: Deutsche Gesellschaft für Internationale Zusammenarbeit (GIZ).

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INTRODUCTION: In a 5-year follow-up study, we investigated the enduring effects of cognitive training on older adults with mild cognitive impairment (MCI). METHODS: A randomized controlled single-blind trial involved 145 older adults with MCI, assigned to cognitive training (MEMO+), an active control psychosocial intervention, or a no-contact condition. Five-year effects were measured on immediate and delayed memory recall, the Montreal Cognitive Assessment screening test (MoCA), self-reported strategy use, and daily living difficulties. RESULTS: At follow-up, participants who received cognitive training showed a smaller decline in delayed memory and maintained MoCA scores, contrasting with greater declines in the control groups. Cognitive training participants outperformed controls in both delayed memory and MoCA scores at the 5-year time point. No significant group differences were observed in self-reported strategy use or difficulties in daily living. DISCUSSION: Cognitive training provides long-term benefits by mitigating memory decline and slowing clinical symptom progression in older adults with MCI. Highlights: Cognitive training reduced the 5-year memory decline of persons with MCI.Cognitive training also reduced decline on the Montreal Cognitive Assessment (MoCA).No intervention effect was found on strategy use or activities of daily living.

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OBJECTIVE: To assess whether the adult Expanded Food and Nutrition Education Program (EFNEP) is a cost-effective intervention that generates sustained improvement in biomarkers of chronic disease risk. DESIGN: A longitudinal quasi-experimental design with 2 parallel arms (untreated comparison vs EFNEP) and 4 waves of data collection (pretest, posttest, 6 months, and 12 months after completion). SETTING: Eligible adult EFNEP community settings in Colorado, Florida, Maryland, and Washington. PARTICIPANTS: Free-living adults (n = 500) aged 18-50 years, with income ≤ 185% of the Federal Poverty Line. INTERVENTION(S): Adult EFNEP delivered using an evidence-based curriculum, Eating Smart • Being Active. MAIN OUTCOME MEASURE(S): Chronic disease biomarkers (body mass index, blood pressure, and HbA1c), food and physical activity behaviors, dietary intake, health status, and demographics will be measured using objective biometric indicators, the Adult EFNEP Questionnaire, a 24-hour dietary recall, a health questionnaire, and demographic forms. ANALYSIS: Linear mixed models will be used to assess whether adult EFNEP has a significant (P < 0.01) impact on 3 chronic disease biomarkers. The program's estimated impact on chronic disease biomarkers will be incorporated into a cost-benefit analysis framework to assess the economic value generated by adult EFNEP through chronic disease risk reduction.

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Protected areas such as national parks constitute an increasing land mass globally, but these areas are under increasing threat from climate change events such as drought, flooding, and bushfires. The recent Yosemite National Park fires in California provide an example of this issue. After any such disaster, authorities will need to restore those protected areas to their former state at significant costs within any public funding cycle. To corroborate that request, clear economic assessments of total costs and benefits will be required. However, in previous studies of these issues a complete set of government cost and/or benefit data may not be provided, skewing assessment results accordingly. Using South Australia's Kangaroo Island protected areas-which were significantly destroyed by bushfire in 2019-20-as a case study with a unique set of State government cost data we calculate a set of analyses via economic methods. Despite significant restoration costs the study found the discounted net present value of returning tourists to the Island is 3.15 over ten years for park tourism and regional economic impacts, providing an internal rate of return of 22%. The rebuild work is also expected to support around 430 full time equivalent (FTE) jobs during construction, with a return to full tourism supporting another 744 FTEs across relevant sectors (e.g. accommodation, retail) of the Kangaroo Island economy. This robust assessment makes it far easier for protected area managers to argue their funding case.

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Due diligence is a fundamental component of ensuring a sustainable and legal wildlife trade that is also supportive of the livelihoods and businesses that depend on the trade. This is particularly true with species listed on the Convention on International Trade in Endangered Species (CITES) that are considered threatened or may become threatened by trade. Undertaking due diligence exercises requires access to information on which to base such decisions; however, the extent to which information is available is unclear. We used the trade in tropical pitcher plants (Nepenthes) for horticultural purposes as a case study to determine the extent to which information is available. A systematic survey of online trade was conducted for species described from 1996 to 2016. For the species found in trade, these were cross-referenced with the CITES trade database, and inquiries were made to the relevant CITES Management Authorities and National Focal Points Access and Benefit Sharing (ABS). Of 83 newly described species, 61% were offered for sale online in 2018. Despite all Nepenthes species being listed on CITES, only 23% (n = 19) of the species being sold online were reported in trade on the CITES Trade Database, and only 3 were from the countries of origin. Thirty-two of these species had no international trade recorded according to the database. Management authorities of CITES for the countries of origin confirmed trade had been permitted for 5 of 32 species. Lack of CITES records may be explained by trade under "Nepenthes spp." or as exempt parts and derivatives. However, permits to collect and commercialize are likely to be required as part of the Nagoya Protocol on ABS from the Convention on Biological Diversity. The ABS National Focal Points were contacted to determine whether collection or commercialization permits had been issued for the remaining species. Only 2 of 7 focal points replied, and both stated no permits had been issued. Lack of traceability information or response related to the issuance of collection and commercialization permits is concerning and hinders the due diligence of businesses and consumers wanting to ensure their trade is legal, sustainable, and ethical.

Definición de la legalidad de especies recién catalogadas en CITES en la horticultura comercial de plantas de jarra tropicales (Nepenthes) Resumen La diligencia debida es un componente fundamental para garantizar un comercio de vida silvestre legal y sostenible que también apoye los medios de subsistencia y las empresas que dependen del comercio. Esto es especialmente cierto en el caso de las especies incluidas en la Convención sobre el Comercio Internacional de Especies Amenazadas de Fauna y Flora Silvestres (CITES) que se consideran amenazadas o pueden verse amenazadas por el comercio. La realización de ejercicios de diligencia debida requiere acceso a información con la cual fundamentar tales decisiones; sin embargo, no está claro hasta qué punto se dispone de información. Usamos como estudio de caso el comercio de plantas de jarra tropicales (Nepenthes) con fines hortícolas para determinar cuánta información hay disponible. Realizamos un estudio sistemático del comercio en línea de las especies descritas entre 1996 y 2016. Para las especies encontradas en el comercio, hicimos referencias cruzadas con la base de datos de comercio CITES y consultamos a las Autoridades Administrativas CITES pertinentes y a los Puntos Focales Nacionales de Acceso y Distribución de Beneficios. De las 83 especies con descripción reciente, el 61% se pusieron a la venta en línea en 2018. A pesar de que todas las especies de Nepenthes están catalogadas en CITES, sólo el 23% (n = 19) de las especies que se vendían en línea figuraban en la base de datos sobre comercio CITES, y sólo tres procedían de los países de origen. Treinta y dos de estas especies no tenían comercio internacional registrado según la base de datos. Las autoridades de gestión de CITES de los países de origen confirmaron que se permitió el comercio de 5 de las 32 especies. La falta de registros CITES puede explicarse por el comercio de «Nepenthes spp¼ o como partes y derivados exentos. Sin embargo, es probable que se exijan permisos de recolección y comercialización en el marco del Protocolo de Nagoya sobre Acceso y Participación en los Beneficios (APB) del Convenio sobre la Diversidad Biológica. Contactamos a los Puntos Focales Nacionales de APB para determinar si se habían expedido permisos de recolección o comercialización para las especies restantes. Sólo dos de los siete puntos focales respondieron y ambos afirmaron que no se había expedido ningún permiso. La falta de información de rastreo o de respuesta en relación con la expedición de permisos de recolección y comercialización es preocupante y obstaculiza la diligencia debida de las empresas y los consumidores que desean asegurarse de que su comercio es legal, sostenible y ético.

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Background: Endometriosis affects 1 in 10 women, many of whom have surgery for persistent pain. Recurrence of symptoms following an operation is common. Although hormonal treatment can reduce this risk, there is uncertainty about the best option. Objectives: To evaluate the clinical and cost-effectiveness of long-acting progestogen therapy compared with the combined oral contraceptive pill in preventing recurrence of endometriosis-related pain and quality of life. Design: A multicentre, open, randomised trial with parallel economic evaluation. The final design was informed by a pilot study, qualitative exploration of women's lived experience of endometriosis and a pretrial economic model. Setting: Thirty-four United Kingdom hospitals. Participants: Women of reproductive age undergoing conservative surgery for endometriosis. Interventions: Long-acting progestogen reversible contraceptive (either 150 mg depot medroxyprogesterone acetate or 52 mg levonorgestrel-releasing intrauterine system) or combined oral contraceptive pill (30 µg ethinylestradiol, 150 µg levonorgestrel). Main outcome measures: The primary outcome was the pain domain of the Endometriosis Health Profile-30 questionnaire at 36 months post randomisation. The economic evaluation estimated the cost per quality-adjusted life-years gained. Results: Four hundred and five women were randomised to receive either long-acting reversible contraceptive (N = 205) or combined oral contraceptive pill (N = 200). Pain scores improved in both groups (24 and 23 points on average) compared with preoperative values but there was no difference between the two (adjusted mean difference: -0.8, 95% confidence interval -5.7 to 4.2; p = 0.76). The long-acting reversible contraceptive group underwent fewer surgical procedures or second-line treatments compared with the combined oral contraceptive group (73 vs. 97; hazard ratio 0.67, 95% confidence interval 0.44 to 1.00). The mean adjusted quality-adjusted life-year difference between two arms was 0.043 (95% confidence interval -0.069 to 0.152) in favour of the combined oral contraceptive pill, although this cost an additional £533 (95% confidence interval 52 to 983) per woman. Limitations: Limitations include the absence of a no-treatment group and the fact that many women changed treatments over the 3 years of follow-up. Use of telephone follow-up to collect primary outcome data in those who failed to return questionnaires resulted in missing data for secondary outcomes. The COVID pandemic may have affected rates of further surgical treatment. Conclusions: At 36 months, women allocated to either intervention had comparable levels of pain, with both groups showing around a 40% improvement from presurgical levels. Although the combined oral contraceptive was cost-effective at a threshold of £20,000 per quality-adjusted life-year, the difference between the two was marginal and lower rates of repeat surgery might make long-acting reversible contraceptives preferable to some women. Future work: Future research needs to focus on evaluating newer hormonal preparations, a more holistic approach to symptom suppression and identification of biomarkers to diagnose endometriosis and its recurrence. Trial registration: This trial is registered as ISRCTN97865475. https://doi.org/10.1186/ISRCTN97865475. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 11/114/01) and is published in full in Health Technology Assessment; Vol. 28, No. 55. See the NIHR Funding and Awards website for further award information. The NIHR recognises that people have diverse gender identities, and in this report, the word 'woman' is used to describe patients or individuals whose sex assigned at birth was female, whether they identify as female, male or non-binary.

Endometriosis is a condition where cells similar to ones that line the womb are found elsewhere in the body. Endometriosis affects 1 in 10 women, many of whom have surgery for persistent pain. Unfortunately, symptoms often return and some women will need repeat operations. Hormonal contraceptives can prevent the return of endometriosis-related pain: either long-acting reversible contraceptives (injections or a coil, fitted inside the womb) or the combined oral contraceptive pill (often called 'the pill'). We do not know which is the best option. The aim of this trial was to find out which of these two hormone treatments was more effective in terms of symptom relief, avoidance of further surgery and costs. Four hundred and five women with endometriosis, who were not intending to get pregnant, participated in a clinical trial. Half of the participants took long-acting reversible contraceptives, and the other half took the pill for 3 years following endometriosis surgery. The choice of treatment was made at random by a computer to ensure a fair comparison, although those allocated to the long-acting contraceptive could choose between injections or the coil. Participants completed questionnaires about their symptoms and life quality at intervals up to 3 years. Both treatments were equally good at reducing pain but more women using the pill had repeat operations. The pill was a little more costly overall but associated with a slightly higher quality of life. Both treatments are equally effective in reducing pain up to 3 years after surgery for endometriosis. The differences in costs are small and the choice of treatment should be based on personal preference.

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OBJECTIVES: To estimate the cost-effectiveness of sacrum multilayer silicone foam dressings as an adjuvant prophylactic therapy compared to standard pressure ulcer prevention in a hospital population at high risk for pressure ulcer development. METHODS: An economic evaluation is performed from a healthcare payer's perspective. This evaluation is based on a Belgian publicly funded pragmatic randomized controlled trial (RCT), linked with real-world data from administrative claims database and a Belgian cost analysis. A cost-consequences analysis with a one-year time horizon is performed. RESULTS: The RCT has shown that the risk of developing a new pressure ulcer on the sacrum was statistically significantly reduced by 41 % in the treatment group (RR = 0.59, 95 % CI 0.35-0.98, p = 0.04). The absolute risk reduction of 2.0 % (95 % CI -0.1-4.1 %) coincides with a number needed to treat of 50.0 to prevent one new pressure ulcer of category II or worse. The evolution of quality of life is on average negative for patients who developed a pressure ulcer before day 3, while it is positive for patients without pressure ulcers. In a scenario with conservative assumptions, i.e. without inclusion of price discounts for the multilayer silicone foam dressings and only including costs during the hospitalization, pressure ulcer prevention with dressings on the sacrum was already cost-neutral. CONCLUSIONS: The preventive use of silicone adhesive multilayer foam dressings on the sacrum for a population similar to the pragmatic trial population can be supported both from a clinical and economic point of view.

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PURPOSE OF REVIEW: This health technology assessment aimed to systematically assess the efficacy and safety of yoga as therapy for burnout. Economic, ethical, legal, social and organizational aspects were considered as well. RECENT FINDINGS: Yoga as a therapy has been shown to have positive effects on a range of symptoms, including stress, anxiety and depression. Regarding work-related stress and burnout, the effects of yoga have mainly been examined in a preventative context. Meta-analyses revealed no effects on burnout severity comparing yoga with passive controls in general. Compared with passive controls, yoga had a positive effect on subjective stress. Compared to active control, yoga had an effect on the burnout subscale depersonalization on individual study level. Yoga may have positive effects on burnout, but the results are mixed. Common definitions and standardized diagnostic tools are necessary to improve research and further assess yoga as therapy for burnout. TRIAL REGISTRATION: The HTA is registered with PROSPERO, CRD42022299405, on 6th February 2022.

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BACKGROUND: Efficiency, equity and financial risk protection are key health systems objectives. Equitable distribution of health care is among the priority strategic initiative of the government of Ethiopia. However, data on the distribution of interventions benefits or on disease burden disaggregated by subpopulations to guide health care priority setting is not available in Ethiopia. METHODS: Aligned with policy documents, we identified the following groups to be the worse off in the Ethiopian context: under-five children, women of reproductive age, the poor, and rural residents. We used the Delphi technique by a panel of 28 experts to assign a score for 253 diseases/conditions over a period of two days, in phases. The expert panel represented different institutes and professional mix. Experts assigned a score 1 to 4; where 4 indicates disease/condition predominantly affecting the poor and rural residents and 1 indicates a condition more prevalent among the wealthy and urban residents. Subsequently, the average equity score was computed for each disease/condition. RESULTS: The average scores ranged from 1.11 (for vitiligo) to 3.79 (for obstetric fistula). We standardized the scores to be bounded between 1 and 2; 1 the lowest equity score and 2 the highest equity score. The scores for each disease/condition were then assigned to their corresponding interventions. We used these equity scores to adjust the CEA values for each of the interventions. To adjust the CEA values for equity, we multiplied the health benefits (the denominator of the cost-effectiveness value) of each intervention by the corresponding equity scores, resulting in equity adjusted CEA values. The equity adjusted CEA was then used to rank the interventions using a league table. CONCLUSIONS: The Delphi method can be useful in generating equity scores for prioritizing health interventions where disaggregated data on the distribution of diseases or access to interventions by subpopulation groups are not available.

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When individuals face life pressure or significant family changes, individuals with better family adaptation can better survive the crisis. Although the influencing factors of family adaptation have been investigated, the application of potential profile analysis has yet to be found. This analytical approach can reveal different potential categories of family adaptation, providing new perspectives for theoretical development and interventions. This study used latent profile analysis to explore family adaptation levels in breast cancer patients and identify different latent categories, examining their characteristic differences. A cross-sectional study was conducted in Jinzhou, China, from July 2023 to March 2024. The questionnaire included Sociodemographic and clinical characteristics, Benefit Finding Scale (BFS), Dyadic Coping Scale (DCI), Chinese Perceived Stress Scales (PSS), and Family adaptability and cohesion evaluation scales (FACES). Mplus8.3 and SPSS26.0 software were used for data analysis. The latent profile analysis (LPA) method was used to fit the family adaptations of breast cancer patients. Three latent categories of family adaptation were identified: low-level family adaptation (21.5%), medium level family adaptation (47.8%), and high-level family adaptation (30.6%). All 14 items with high levels of family adaptation scored higher than the other two groups. In particular, out of all the categories, item 9, "The idea of educating children is sound," scored highest. Compared with the low-level group, the influential factors of family adaptation in the high-level group were BFS, DCI, PSS, relapse and personal monthly income; The factors influencing family adaptation at the middle level are DCI, BFS, breast cancer type, family history of breast cancer, and personal monthly income. Compared with the medium level group, PSS and DCI were the influential factors of family adaptation in the high-level group. Family adaptation in breast cancer patients can be divided into three categories: low-level, medium-level, and high-level. There were significant differences among different categories of family adaptation levels in "personal monthly income", "family history of breast cancer", "type of breast cancer", "recurrence", "dyadic coping", "benefit finding", and "perception stress".

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BACKGROUND: Tuberculosis (TB) infection screening of high-risk groups is an important strategy for achieving End TB targets. A TB infection screening program was implemented for quarry workers from a Portuguese high-incidence area in 2018-2022. We aimed to calculate the cost-benefit of the screening program from the societal perspective. METHODS: We calculated medical and non-medical direct and indirect screening costs and compared them with the cost savings from averted cases of TB disease. We estimated the number of potentially averted TB disease cases based on the risk of progression of TB infection to TB disease found in the literature. RESULTS: During the screening program, 997 workers were screened. TB infection was diagnosed in 215 workers, 150 of those initiated preventive treatment. Screening program total costs were €136,295. Twenty-nine TB cases were potentially prevented, what would have costed €152,386. Savings of €16,091 were obtained (€4516, €40898, and -€29322 from the workers, employers, and NHS, respectively). CONCLUSIONS: The monetary benefit of a TB infection screening program directed to quarry workers in a high-incidence area was greater than its cost. Companies and workers saved substantially more money. TB infection tests that are better predictors of progression to TB disease could reduce NHS costs.

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Long-term excessive use of fertilizers and intensive cultivation not only decreases soil organic carbon (SOC) and productivity, but also increases greenhouse gas emissions, which is detrimental to sustainable agricultural development. The purpose of this paper is to identify organic amendments suitable for winter wheat growth in the North China Plain by studying the effects of organic amendments on the economic benefits, carbon emissions, and carbon sequestration for winter wheat fields and to provide a theoretical basis for the wide application of organic amendments in agricultural fields. The two nitrogen rates were N0 (0 kg ha-1) and N240 (240 kg ha-1), and the four organic amendments were straw, manure, mushroom residue (M R), and biochar. The results showed that, compared to N0, N240 significantly increased the yield by 244.1-318.4% and the organic carbon storage by 16.7-30.5%, respectively, but increased the carbon emissions by 29.3-45.5%. In addition, soil carbon stocks increased with all three types of organic amendments compared to the straw amendment, with the biochar treatment being the largest, increasing carbon storage by 13.3-33.6%. In terms of yield and economic benefits, compared to the straw amendment, the manure and biochar amendments increased winter wheat yields by 0.0-1.5% and 4.0-13.3%, respectively, and M R slightly decreased wheat yield; only the economic benefit of the M R amendment was greater than that of the straw amendment, with an increase in economic benefit of 1.3% and 8.2% in the 2021-2022 and 2022-2023 seasons, respectively. Furthermore, according to the net ecosystem productivity (NEP), N0 was the source of CO2, while N240 was a sink of CO2. The TOPSIS results showed that N240 with a mushroom residue amendment could be recommended for increasing soil carbon stocks and economic benefits for winter wheat in the NCP and similar regions. Low-cost M R can increase farmer motivation and improve soil organic carbon, making a big step forward in the spread of organic materials on farmland.

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Background: There is interest in using treatment breaks in oncology, to reduce toxicity without compromising efficacy. Trial design: A Phase II/III multicentre, open-label, parallel-group, randomised controlled non-inferiority trial assessing treatment breaks in patients with renal cell carcinoma. Methods: Patients with locally advanced or metastatic renal cell carcinoma, starting tyrosine kinase inhibitor as first-line treatment at United Kingdom National Health Service hospitals. Interventions: At trial entry, patients were randomised (1 : 1) to a drug-free interval strategy or a conventional continuation strategy. After 24 weeks of treatment with sunitinib/pazopanib, drug-free interval strategy patients took up a treatment break until disease progression with additional breaks dependent on disease response and patient choice. Conventional continuation strategy patients continued on treatment. Both trial strategies continued until treatment intolerance, disease progression on treatment, withdrawal or death. Objective: To determine if a drug-free interval strategy is non-inferior to a conventional continuation strategy in terms of the co-primary outcomes of overall survival and quality-adjusted life-years. Co-primary outcomes: For non-inferiority to be concluded, a margin of ≤ 7.5% in overall survival and ≤ 10% in quality-adjusted life-years was required in both intention-to-treat and per-protocol analyses. This equated to the 95% confidence interval of the estimates being above 0.812 and -0.156, respectively. Quality-adjusted life-years were calculated using the utility index of the EuroQol-5 Dimensions questionnaire. Results: Nine hundred and twenty patients were randomised (461 conventional continuation strategy vs. 459 drug-free interval strategy) from 13 January 2012 to 12 September 2017. Trial treatment and follow-up stopped on 31 December 2020. Four hundred and eighty-eight (53.0%) patients [240 (52.1%) vs. 248 (54.0%)] continued on trial post week 24. The median treatment-break length was 87 days. Nine hundred and nineteen patients were included in the intention-to-treat analysis (461 vs. 458) and 871 patients in the per-protocol analysis (453 vs. 418). For overall survival, non-inferiority was concluded in the intention-to-treat analysis but not in the per-protocol analysis [hazard ratio (95% confidence interval) intention to treat 0.97 (0.83 to 1.12); per-protocol 0.94 (0.80 to 1.09) non-inferiority margin: 95% confidence interval ≥ 0.812, intention to treat: 0.83 > 0.812 non-inferior, per-protocol: 0.80 < 0.812 not non-inferior]. Therefore, a drug-free interval strategy was not concluded to be non-inferior to a conventional continuation strategy in terms of overall survival. For quality-adjusted life-years, non-inferiority was concluded in both the intention-to-treat and per-protocol analyses [marginal effect (95% confidence interval) intention to treat -0.05 (-0.15 to 0.05); per-protocol 0.04 (-0.14 to 0.21) non-inferiority margin: 95% confidence interval ≥ -0.156]. Therefore, a drug-free interval strategy was concluded to be non-inferior to a conventional continuation strategy in terms of quality-adjusted life-years. Limitations: The main limitation of the study is the fewer than expected overall survival events, resulting in lower power for the non-inferiority comparison. Future work: Future studies should investigate treatment breaks with more contemporary treatments for renal cell carcinoma. Conclusions: Non-inferiority was shown for the quality-adjusted life-year end point but not for overall survival as pre-defined. Nevertheless, despite not meeting the primary end point of non-inferiority as per protocol, the study suggested that a treatment-break strategy may not meaningfully reduce life expectancy, does not reduce quality of life and has economic benefits. Although the treating clinicians' perspectives were not formally collected, the fact that clinicians recruited a large number of patients over a long period suggests support for the study and provides clear evidence that a treatment-break strategy for patients with renal cell carcinoma receiving tyrosine kinase inhibitor therapy is feasible. Trial registration: This trial is registered as ISRCTN06473203. Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment Programme (NIHR award ref: 09/91/21) and is published in full in Health Technology Assessment; Vol. 28, No. 45. See the NIHR Funding and Awards website for further award information.

Treatment breaks in cancer are of significant interest to patients and health professionals. Renal cell carcinoma is the most common type of kidney cancer. Sunitinib and pazopanib are both targeted treatments. They were commonly used to treat advanced kidney cancer but often cause side effects, sometimes requiring use of a reduced dose or even stopping treatment. The STAR trial was designed to see whether planned treatment breaks made patients with advanced kidney cancer being treated with sunitinib and pazopanib feel better, without substantially affecting how well the treatment worked. After 24 weeks of treatment, patients took sunitinib and pazopanib either as they normally would or in the alternative way with planned treatment breaks. Treating patients in this way was continued until drug-related side effects stopped treatment, patients' disease worsened while taking treatment or the patient died. The trial compared how well the different treatment strategies worked in terms of how long patients lived and their quality of life over that time. This trial is the largest United Kingdom trial in advanced renal cell carcinoma. Patients took part from 60 United Kingdom centres between 2012 and 2017. It was funded by the National Institute for Health and Care Research Health Technology Assessment Programme and run by the Leeds Clinical Trials Research Unit. In total, 920 patients took part. Four hundred and sixty-one patients were allocated to continue treatment and 459 were allocated to start at least one treatment break. Treatment breaks lasted on average 87 days. The length of time patients lived in both arms of the trial appeared similar, but this cannot be concluded due to insufficient information. Being allocated to have treatment breaks rather than continuing treatment did not negatively impact a patient's quality of life. Additionally, allocating patients to have treatment breaks was shown to have significant cost savings compared to just continuing treatment. Importantly planned treatment breaks were shown to be feasible.

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The utilization and application of genomic information generated from precision medicine continues to increase with the goal of improving health outcomes. Increasingly researchers, health care professionals, and public health teams include an examination of the ethical, legal, and social issues (ELSI) in their consideration of the use of precision medicine for newborn and pediatric health. In addition to ELSI considerations, stakeholders could benefit from an understanding of economics, the other "E" in ELSI. The use of an economic evaluation could aid decision-making on whether to screen newborns who may be at risk for disease, to diagnose newborns and children who present with symptoms, to inform the treatment and management of diagnosed individuals. In this manuscript we review the core concepts of economic evaluation, the framework of decision-analysis, and key parameters for consideration in assessing the economics of NBS program(s). We describe the common language used in the economic evaluation and provide a practical overview of health economic evaluations including 1) their purpose, 2) different types and components, 3) evaluation of the different types and components of economic evaluations (i.e., cost-effectiveness vs. cost-benefit analysis), 4) impact of societal or healthcare perspectives on the analysis, 5) health outcomes, 6) time horizon for the analysis, 7) identification of appropriate comparators, and 8) resources for economic data. We conclude with a use case to demonstrate the application and understanding of economic considerations for in the advancement and expansion of NBS.

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The schedule of administering a drug has profound impact on the toxicity and efficacy profiles of the drug through changing its pharmacokinetics (PK). PK is an innate and indispensable component of the dose-schedule optimization. Motivated by this, we propose a Bayesian PK integrated dose-schedule finding (PKIDS) design to identify the optimal dose-schedule regime by integrating PK, toxicity, and efficacy data. Based on the causal pathway that dose and schedule affect PK, which in turn affects efficacy and toxicity, we jointly model the three endpoints by first specifying a Bayesian hierarchical model for the marginal distribution of the longitudinal dose-concentration process. Conditional on the drug concentration in plasma, we jointly model toxicity and efficacy as a function of the concentration. We quantify the risk-benefit of regimes using utility-continuously updating the estimates of PK, toxicity, and efficacy based on interim data-and make adaptive decisions to assign new patients to appropriate dose-schedule regimes via adaptive randomization. The simulation study shows that the PKIDS design has desirable operating characteristics.

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Objective: To evaluate whether improved progression-free survival (PFS) from radiotherapy (RT) translates into an overall survival (OS) benefit for diffuse large B-cell lymphoma (DLBCL). Methods: A systematic literature search identified randomized controlled trials (RCTs) and retrospective studies that compared combined-modality therapy (CMT) with chemotherapy (CT) alone. Weighted regression analyses were used to estimate the correlation between OS and PFS benefits. Cohen's kappa statistic assessed the consistency between DLBCL risk-models and PFS patterns. Furthermore, the benefit trend of RT was analyzed by fitting a linear regression model to the pooled hazard ratio (HR) according to the PFS patterns. Results: For both 7 RCTs and 52 retrospective studies, correlations were found between PFS HR (HRPFS) and OS HR (HROS) at trial level (r = 0.639-0.876), and between PFS and OS rates at treatment-arm level, regardless of CT regimens (r = 0.882-0.964). Incorporating RT into CT increased about 18% of PFS, and revealed a different OS benefit profile. Patients were stratified into four CT-generated PFS patterns (>80%, >60-80%, >40-60%, and ≤40%), which was consistent with risk-stratified subgroups (kappa > 0.6). Absolute gain in OS from RT ranged from ≤5% at PFS >80% to about 21% at PFS ≤40%, with pooled HROS from 0.70 (95% CI, 0.51-0.97) to 0.48 (95% CI, 0.36-0.63) after rituximab-based CT. The OS benefit of RT was predominant in intermediate- and high-risk patients with PFS ≤ 80%. Conclusion: We demonstrated a varied OS benefit profile of RT to inform treatment decisions and clinical trial design.

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Background & Aims: International consensus has recently introduced a new definition of metabolic dysfunction-associated steatotic liver disease (MASLD). We sought to analyse epidemiological trends, prognostic features, and transplant survival benefits of patients with MASLD and without MASLD waiting for liver transplantation (LT) in Italy. Methods: Using the Italian Liver Transplant Registry database, we analysed data from adult patients listed for primary LT attributable to end-stage chronic liver disease between January 2012 and December 2022. Independent multivariable waiting lists and post-transplant survival models were developed for patients with and without hepatocellular carcinoma (HCC). A Monte Carlo simulation was used to create 5-year transplant benefit distributions based on the presence of MASLD, HCC, and model for end-stage liver disease (MELD)-sodium values. Results: A total sample of 1,941 patients with MASLD and 11,201 patients without MASLD was considered. A significant increase in the prevalence of MASLD as an indication for LT was observed from 2012 to 2022, for both cohorts with HCC (from 17.7 to 30%) and without HCC (from 9.5 to 11.8%) cohorts. Projections suggest that, as early as next year, MASLD will overcome HCV as the second most common indication for transplantation after alcoholic liver disease in Italy. According to univariate and multivariate analyses, MASLD was not an independent predictive factor for patient survival after transplantation. However, it increased the risk of death for patients on the waiting list without HCC (hazard ratio 1.62, p <0.001). At the same MELD-sodium, the 5-year transplant benefit was higher in patients with non-HCC MASLD, followed by patients with HCC, whereas it was lower in patients without HCC and without MASLD. Conclusions: Patients with non-HCC MASLD had an increased waitlist mortality and 5-year transplant survival benefit compared with other candidates. Impact and implications: The present research addresses the critical need to understand the evolving landscape of liver transplantation indications, mainly focusing on metabolic dysfunction-associated steatotic liver disease (MASLD) in Italy. Given the significant rise in MASLD cases, these findings highlight that patients with non-HCC MASLD face increased waitlist mortality and benefit more from liver transplantation within 5 years compared with other candidates. The significance of these results lies in their emphasis on the necessity of focusing on patients with MASLD on waiting lists to improve outcomes. By tailoring transplant eligibility criteria and resource allocation, the study provides actionable insights to improve patient survival and optimise liver transplantation practices.

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Hospice care is designed to support the medical and psychosocial needs of individuals with serious illness and their caregivers through the dying process. Some individuals, though, leave hospice prior to death, generally referred to as disenrollment or a "live discharge." Live discharge from hospice is a common and often distressing issue for hospice patients, their caregivers, and also for hospice professionals and agencies. This paper discusses common issues surrounding live discharge that clinicians and other healthcare professionals should consider when dealing with live discharge in their own clinical practices. Where applicable, we provide practical steps for hospice and palliative care clinicians to better support patients and families through this critical care transition. Further, we offer strategic directions interprofessional clinicians can take to affect systemic change to improve live discharge experiences.

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Complementary feeding is a critical milestone for parents, who must decide the extent to which they feed their infants with homemade foods (HMFs) or commercial infant foods (CIFs). The current study focused on how parents perceive and evaluate CIFs and what drives them to use these products. Understanding the wide variety of factors that influence parents' decisions to use CIFs as a food source is crucial to helping them make more balanced food choices for their infants. Data from an online survey with 858 German parents revealed their negative perceptions of CIFs, particularly in terms of nutritional quality and texture, and concerns that CIFs contain higher levels of pesticides and heavy metals compared to HMFs, despite acknowledging the expertise of the infant nutrition industry. These perceptions were likely to be driven by the 'natural-is-better' belief and parents' desire for control over the ingredients in their infants' meals, contributing to their skepticism towards CIFs. Additionally, our study found that while many parents know traditional nutrition guidelines, they are less familiar with recent updates. Parents' benefit beliefs about CIFs and parents' trust in the food industry strongly influenced the use of CIFs, while infant age and parent gender had a less strong, yet significant influence on CIFs consumption. Overall, the present study offers new quantitative insights into the factors that motivate parents to use CIFs. Furthermore, it underscores the need for health authorities to improve their communication strategies towards parents concerning new dietary guidelines.

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For public interest advocates engaged on issues of worker health and safety and environmental protections, regulatory cost-benefit analysis has long been seen as an obstacle to meaningful progress. In November 2023, the Biden administration overhauled Circular A-4, which provides guidance to agencies on how to perform cost-benefit analyses for their rules. The reforms seek to make cost-benefit analysis less biased against worker safety, public health, environmental, and other protective safeguards. As such, the new version of Circular A-4 offers important new levers to agencies to justify more stringent protections. By extension, those in the public interest community can use agency implementation of the new Circular A-4 as part of their advocacy efforts for specific rules they are tracking. This article seeks to support this tactic by providing a roadmap for advocates on how to incorporate into their comments critiques of agencies' cost-benefit analyses based on the Circular A-4 revisions.