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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. [ABSTRACT FROM AUTHOR]

Background: The maintenance treatment of ESRD puts a great burden on individual patients, society, and the country of China as a whole. Hemoperfusion plus hemodialysis (HP + HD) in ESRD patients can yield modified outcomes such as prolonged life expectancy and improved dialysis quality and quality of life. This study aimed to systematically analyze the effect of HP + HD on the overall survival (OS) rates of ESRD patients and to provide support for clinical decision-making. Methods: A computerized search was performed in the PubMed, Embase, Cochrane Library, CNKI, WanFang Data and SinoMed databases for relevant original research articles. Studies were included or excluded based on their compliance with predefined selection criteria. Results: Twelve studies were included in the qualitative synthesis and quantitative synthesis (meta-analysis). The meta-analysis showed that the 1-year OS rate (odds ratio [OR]: 3.35, 95% CI: 1.89, 5.91, p < 0.05), 2-year OS rate (OR: 2.88, 95% CI: 1.84, 4.53, p < 0.05), and 5-year OS rate (χ2 = 4.3092, p < 0.05) of patients with ESRD treated with HP + HD were better than those treated with HD, but there was no significant difference in 3-year OS rate (OR: 1.97, 95% CI: 0.76, 5.06, p > 0.05). Subgroup analysis showed the same outcomes in different study designs (1-year OS rate: randomized controlled trials [RCTs]: OR: 4.38, 95% CI: 1.61, 11.88, p < 0.05; cohort studies: OR: 2.90, 95% CI: 1.44, 5.85, p < 0.05; 2-year OS rate: RCTs: OR: 2.99, 95% CI: 1.84, 4.84, p < 0.05) and different age-groups (1-year OS rate: 45–50 years: OR: 3.19, 95% CI: 1.55, 6.57, p < 0.05; 55–60 years: OR: 3.37, 95% CI: 1.07, 10.61, p < 0.05; 2-year OS rate: 50–55 years: OR: 2.86, 95% CI: 1.59, 5.16, p < 0.05: 60–65 years: OR: 4.41, 95% CI: 1.19, 16.30, p < 0.05). Conclusions: This meta-analysis suggests that the OS rates of ESRD patients treated with HP + HD were better than those of patients treated with HD. A speculative hypothesis for why this is the case may be that HP + HD can achieve the complementary elimination of metabolites, effectively preventing and treating complications caused by long-term dialysis and prolonging life expectancy. Therefore, HP + HD should be widely used in ESRD patients. [ABSTRACT FROM AUTHOR]

Background: SMAD6 variants have been reported in patients with radioulnar synostosis (RUS). This study aimed to investigate the genotypes and phenotypes for a large cohort of patients with RUS having mutant SMAD6. Methods: Genomic DNA samples were isolated from 251 RUS sporadic patients (with their parents) and 27 RUS pedigrees. Sanger sequencing was performed for the SMAD6 coding regions. For positive probands, co‐segregation and parental‐origin analysis of SMAD6 variants and phenotypic re‐evaluation were performed for their family members. Results: We identified 50 RUS probands with SMAD6 variants (13 co‐segregated with RUS in pedigrees and 37 in RUS‐sporadic patients). Based on the new and previous data, we identified SMAD6 mutated in 16/38 RUS pedigrees and 61/393 RUS sporadic patients, respectively. Overall, 93 SMAD6 mutant patients with RUS were identified, among which 29 patients had unilateral RUS, where the left side was more involved than the right side (left:right = 20:9). Female protective effects and non‐full penetrance were observed, in which only 6.90% mothers (vs. ~50% fathers) of SMAD6 mutant RUS probands had RUS. Pleiotropy was observed as a re‐evaluation of SMAD6 mutant families identified: (a) three families had axial skeletal malformations; (b) two families had polydactyly; and (c) eight families had other known malformations. Conclusion: SMAD6 was mutated in 42.11% RUS pedigrees and 15.52% RUS sporadic patients. The RUS patients with SMAD6 variants exhibit both non‐full‐penetrance, variable expressivity, pleiotropy, female protective effects, and the left side is more susceptible than the right side. [ABSTRACT FROM AUTHOR]

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., Competing Interests: The authors have no conflicts of interest to disclose., (2024, International Research and Cooperation Association for Bio & Socio - Sciences Advancement.)