Cost-utility analysis of romiplostim for the treatment of chronic primary immune thrombocytopenia in China.

This study aimed to assess the cost-utility of romiplostim (ROMI) compared to eltrombopag (EPAG) as a second-line treatment for chronic primary immune thrombocytopenia (cITP) in Chinese adults. A decision tree-embedded Markov model with a lifetime horizon was used to estimate the quality-adjusted life years (QALYs) and costs for ROMI versus EPAG from the perspective of the Chinese health care system. The model was driven by platelet response with a 4-week cycle. Both QALYs and costs were discounted 5% per year. Clinical data comparing ROMI and EPAG were obtained by matching-adjusted indirect comparison (MAIC), utilizing individual patient data on ROMI and published Chinese Phase III trial data on EPAG. Costs were reported in 2022 US dollars and included drug acquisition costs, monitoring costs, bleeding-related costs, and costs associated with adverse events. Deterministic and probabilistic sensitivity analyses were performed. The CEA model indicated that treatment with ROMI resulted in an average of $4,344.4 higher costs for 0.004 QALYs. One-way sensitivity analysis (OSA) indicated that the model was most sensitive to the high bleeding rate in response (Markov stage) for EPAG and ROMI. Probabilistic sensitivity analysis (PSA) indicated that ROMI was likely to be cost effective in 0.16% cases at a willingness-to-pay threshold of $12039.1 (China per capita GDP in 2022) per QALY. If the price of ROMI is either lower than or equal to that of EPAG, ROMI could likely be considered cost-effective as a second-line treatment for Chinese adults with cITP.

Preferences for COVID-19 Vaccines: Systematic Literature Review of Discrete Choice Experiments.

BACKGROUND: Vaccination can be viewed as comprising the most important defensive barriers to protect susceptible groups from infection. However, vaccine hesitancy for COVID-19 is widespread worldwide. OBJECTIVE: We aimed to systematically review studies eliciting the COVID-19 vaccine preference using discrete choice experiments. METHODS: A literature search was conducted in PubMed, Embase, Web of Science, Scopus, and CINAHL Plus platforms in April 2023. Search terms included discrete choice experiments, COVID-19, and vaccines and related synonyms. Descriptive statistics were used to summarize the study characteristics. Subgroup analyses were performed by factors such as high-income countries and low- and middle-income countries and study period (before, during, and after the pandemic wave). Quality appraisal was performed using the 5-item Purpose, Respondents, Explanation, Findings, and Significance checklist. RESULTS: The search yield a total of 623 records, and 47 studies with 53 data points were finally included. Attributes were grouped into 4 categories: outcome, process, cost, and others. The vaccine effectiveness (21/53, 40%) and safety (7/53, 13%) were the most frequently reported and important attributes. Subgroup analyses showed that vaccine effectiveness was the most important attribute, although the preference varied by subgroups. Compared to high-income countries (3/29, 10%), a higher proportion of low- and middle-income countries (4/24, 17%) prioritized safety. As the pandemic progressed, the duration of protection (2/24, 8%) during the pandemic wave and COVID-19 mortality risk (5/25, 20%) after the pandemic wave emerged as 2 of the most important attributes. CONCLUSIONS: Our review revealed the critical role of vaccine effectiveness and safety in COVID-19 vaccine preference. However, it should be noticed that preference heterogeneity was observed across subpopulations and may change over time. TRIAL REGISTRATION: PROSPERO CRD42023422720; https://tinyurl.com/2etf7ny7.

A comprehensive value-based method for new nuclear medical service pricing: with case study of radium [223Ra] bone metastases treatment.

IMPORTANCE: Innovative nuclear medicine services offer substantial clinical value to patients. However, these advancements often come with high costs. Traditional payment strategies do not incentivize medical institutes to provide new services nor determine the fair price for payers. A shift towards a value-based pricing strategy is imperative to address these challenges. Such a strategy would reconcile the cost of innovation with incentives, foster transparent allocation of healthcare resources, and expedite the accessibility of essential medical services. OBJECTIVE: This study aims to develop and present a comprehensive, value-based pricing model for new nuclear medicine services, illustrated explicitly through a case study of the radium [223Ra] treatment for bone metastases. In constructing the pricing model, we have considered three primary value determinants: the cost of the new service, associated service risk, and the difficulty of the service provision. Our research can help healthcare leaders design an evidence-based Fee-For-Service (FFS) payment reference pricing with nuclear medicine services and price adjustments. DESIGN, SETTING AND PARTICIPANTS: This multi-center study was conducted from March 2021 to February 2022 (including consultation meetings) and employed both qualitative and quantitative methodologies. We organized focus group consultations with physicians from nuclear medicine departments in Beijing, Chongqing, Guangzhou, and Shanghai to standardize the treatment process for radium [223Ra] bone metastases. We used a specially designed 'Radium Nuclide [223Ra] Bone Metastasis Data Collection Form' to gather nationwide resource consumption data to extract information from local databases. Four interviews with groups of experts were conducted to determine the add-up ratio, based on service risk and difficulty. The study organized consultation meeting with key stakeholders, including policymakers, service providers, clinical researchers, and health economists, to finalize the pricing equation and the pricing result of radium [223Ra] bone metastases service. MAIN OUTCOMES AND MEASURES: We developed and detailed a pricing equation tailored for innovative services in the nuclear medicine department, illustrating its application through a step-by-step guide. A standardized service process was established to ensure consistency and accuracy. Adhering to best practice guidelines for health cost data analysis, we emphasized the importance of cross-validation of data, where validated data demonstrated less variation. However, it required a more advanced health information system to manage and analyze the data inputs effectively. RESULTS: The standardized service of radium [223Ra] bone metastases includes: pre-injection assessment, treatment plan, administration, post-administration monitoring, waste disposal and monitoring. The average duration for each stage is 104 min, 39 min, 25 min, 72 min and 56 min. A standardized monetary value for medical consumables is 54.94 yuan ($7.6), and the standardised monetary value (medical consumables cost plus human input) is 763.68 yuan ($109.9). Applying an agreed value add-up ratio of 1.065, the standardized value is 810.19 yuan ($116.9). Feedback from a consultation meeting with policymakers and health economics researchers indicates a consensus that the pricing equation developed was reasonable and well-grounded. CONCLUSION: This research is the first study in the field of nuclear medicine department pricing methodology. We introduce a comprehensive value-based nuclear medical service pricing method and use radium[223Ra] bone metastases treatment pricing in China as a case study. This study establishes a novel pricing framework and provides practical instructions on its implementation in a real-world healthcare setting.

Comparison of Rare and Common Diseases in the Setting of Healthcare Priorities: Evidence of Social Preferences Based on a Systematic Review.

Background: In light of the limited availability of healthcare resources, providing universal access to healthcare is a challenging task. As a result, prioritizing healthcare services has emerged as a crucial issue. This study aims to explore the preferences of the public regarding healthcare prioritization for rare and common diseases. By examining public attitudes, this study seeks to inform government decisions concerning resource allocation and distribution within healthcare. Methods: "Social preference" and "rare disease" were searched as MeSH terms in the electronic databases of Ovid Medline, Web of Science, Embase, and Econlit for articles published since their establishment, and the information on the characteristics of the articles and the results of social preferences for rare diseases were analyzed and summarized. Results: The public held predominantly neutral views on the setting of healthcare priorities for rare and common diseases. The results of the included studies showed that with all else being equal, no social preference for rarity was found, but when the public considered the proportional advantage of rare diseases or when the respondents were young, a social preference for rarity existed. In addition, the public weighed attributes such as the health benefits of treatments, the effectiveness of treatment options, the safety of treatment, equity, unmet needs, and disease severity in the process of setting of treatment priorities for rare diseases. Furthermore, in consideration of equity, the public showed a willingness to pay for rare diseases in spite of the high medical costs. Conclusion: International studies on social preferences provide some evidence for the setting of healthcare priorities for rare diseases, and health policymakers should consider social preferences in an integrated manner in order to set healthcare priorities appropriately.

Discrete Choice Experiments in Health State Valuation: A Systematic Review of Progress and New Trends.

BACKGROUND: Discrete choice experiments (DCEs) are increasingly used in health state valuation studies. OBJECTIVE: This systematic review updates the progress and new findings of DCE studies in the health state valuation, covering the period since the review of June 2018 to November 2022. The review reports the methods that are currently being used in DCE studies to value health and study design characteristics, and, for the first time, reviews DCE health state valuation studies published in the Chinese language. METHODS: English language databases PubMed and Cochrane, and Chinese language databases Wanfang and CNKI were searched using the self-developed search terms. Health state valuation or methodology study papers were included if the study used DCE data to generate a value set for a preference-based measure. Key information extracted included DCE study design strategies applied, methods for anchoring the latent coefficient on to a 0-1 QALY scale and data analysis methods. RESULTS: Sixty-five studies were included; one Chinese language publication and 64 English language publications. The number of health state valuation studies using DCE has rapidly increased in recent years and these have been conducted in more countries than prior to 2018. Wide usage of DCE with duration attributes, D-efficient design and models accounting for heterogeneity has continued in recent years. Although more methodological consensus has been found than in studies conducted prior to 2018, this consensus may be driven by valuation studies for common measures with an international protocol (the 'model' valuation research). Valuing long measures with well-being attributes attracted attention and more realistic design strategies (e.g., inconstant time preference, efficient design and implausible states design) were identified. However, more qualitative and quantitative methodology study is still necessary to evaluate the effect of those new methods. CONCLUSIONS: The use of DCEs in health state valuation continues to grow dramatically and the methodology progress makes the method more reliable and pragmatic. However, study design is driven by international protocols and method selection is not always justified. There is no gold standard for DCE design, presentation format or anchoring method. More qualitative and quantitative methodology study is recommended to evaluate the effect of new methods before researchers make methodology decisions.

Preference to Family Doctor Contracted Service of Patients with Chronic Disease in Urban China: A Discrete Choice Experiment.

Objective: Shanghai is one of the pioneers proposing family doctor contract service (FDCS). However, there is no quantitative research focusing on the Shanghai experience from a demand-side perspective. This study investigated Shanghai chronic patients' relative preferences for FDCS using a discrete choice experiment method. Methods: A face-to-face discrete choice experiment (DCE) was performed to elicit the preference with 300 samples. Attributes and levels were extracted from the literature review and focus group consultation with patients. Seven attributes, follow-up frequency, medicine accessibility, family doctor competency, health management, referral convenience, appointment flexibility, and shared decision-making, were decided. Three levels were attached to each attribute. A mixed logit model was used to evaluate the multiple-choice data. Results: A total of 248 patients completed the survey. Patient valued FDCS medicine accessibility (ß=0.57, P < 0.05), and high family doctor competency (ß= 0.43, P < 0.05), regular health management activities (ß=0.36, P < 0.05), high follow-up frequency (ß=0.31, P < 0.05) the most. The good doctor-patient shared decision-making atmosphere (ß=0.12, P < 0.05), high referral convenience (ß=0.06, P < 0.05) and high appointment flexibility (ß=0.04, P < 0.05) are valued as less important. No significant preference heterogeneity was identified for patients with different sociodemographic characteristics. Respondents reported other FDCS needs, including online health consultation, specialist services in local institutes, higher reimbursement rates, free rehabilitation guidance for the disabled and personal health management. Conclusion: This research is the first discrete choice experiment FDCS preference research targeting on Chinese urban population. The results suggested that to increase the quality of FDCS, policy-makers should prioritize follow-up frequency, medicine accessibility, family doctor competency and health management. The service package should consider a higher reimbursement rate and rehabilitation guidance for the disabled if extra health-care resources available. Future FDCS policy should consider stated societal preference and be congruent with it.

Transcriptomic evidence for involvement of reactive oxygen species in Rhizoctonia solani AG1 IA sclerotia maturation.

Rhizoctonia solani AG1 IA is a soil-borne fungal phytopathogen that can significantly harm crops resulting in economic loss. This species overwinters in grass roots and diseased plants, and produces sclerotia that infect future crops. R. solani AG1 IA does not produce spores; therefore, understanding the molecular mechanism of sclerotia formation is important for crop disease control. To identify the genes involved in this process for the development of disease control targets, the transcriptomes of this species were determined at three important developmental stages (mycelium, sclerotial initiation, and sclerotial maturation) using an RNA-sequencing approach. A total of 5,016, 6,433, and 5,004 differentially expressed genes (DEGs) were identified in the sclerotial initiation vs. mycelial, sclerotial maturation vs. mycelial, and sclerotial maturation vs. sclerotial initiation stages, respectively. Moreover, gene ontology (GO) and kyoto encyclopedia of genes and genomes (KEGG) analyses showed that these DEGs were enriched in diverse categories, including oxidoreductase activity, carbohydrate metabolic process, and oxidation-reduction processes. A total of 12 DEGs were further verified using reverse transcription quantitative PCR. Among the genes examined, NADPH oxidase 1 (NOX1) and superoxide dismutase (SOD) were highly induced in the stages of sclerotial initiation and maturation. In addition, the highest reactive oxygen species (ROS) production levels were detected during sclerotial initiation, and enzyme activities of NOX1, SOD, and catalase (CAT) matched with the gene expression profiles. To further evaluate the role of ROS in sclerotial formation, R. solani AG1 IA was treated with the CAT inhibitor aminotriazole and H2O2, resulting in the early differentiation of sclerotia. Taken together, this study provides useful information toward understanding the molecular basis of R. solani AG1 IA sclerotial formation and maturation, and identified the important role of ROS in these processes.