Evaluating vonoprazan and tegoprazan for gastroesophageal reflux disease treatment in Chinese Healthcare: an EVIDEM framework analysis.

BACKGROUND: In Chinese healthcare settings, drug selection decisions are predominantly influenced by the Pharmacy & Therapeutics Committee (PTC). This study evaluates two recently introduced potassium-competitive acid blockers, vonoprazan (VPZ) and tegoprazan (TPZ), utilizing the Evidence and Value: Impact on DEcisionMaking (EVIDEM) framework. METHODS: The study employed the 10th edition of EVIDEM, which includes a core model with five domains and 13 criteria. Two independent expert panels were involved: the PTC expert panel, tasked with assigning weights using a 5-point scale, defining scoring indicators, examining the evidence matrix, scoring, and decision-making; and the evidence matrix expert panel, responsible for conducting a systematic literature review, creating the evidence matrix, and evaluating the value contributions of VPZ and TPZ. RESULTS: The analysis estimated the value contributions of VPZ and TPZ to be 0.59 and 0.54, respectively. The domain of 'economic consequences of intervention' showed the most significant variation in value contribution between the two drugs, followed by 'comparative outcomes of intervention' and 'type of benefit of intervention'. CONCLUSION: Employing the EVIDEM framework, VPZ's value contribution was found to be marginally superior to that of TPZ. The EVIDEM framework demonstrates potential for broader application in Chinese medical institutions.

[Clinical comprehensive evaluation of Chinese patent medicine based on EVIDEM].

The clinical comprehensive evaluation of Chinese patent medicine is an important direction in the evaluation of traditional Chinese medicine(TCM), which positively promotes the development of TCM industry. The evaluation system of Chinese patent medicine is helpful to comprehensively evaluate the clinical value of different Chinese patent medicine in the same category, different dosage forms, and specifications, from different manufacturers on the basis of evidence and value. The establishment of a scientific and reasonable comprehensive evaluation index system for Chinese patent medicine is an important prerequisite to ensure clinical value. However, there has been neither a recognized systematic review on the clinical comprehensive evaluation of Chinese patent medicine nor a methodological system used for reference. The evidence and value: impact on decision-making(EVIDEM), developed by the international research team, is used to evaluate the comprehensive value of medical interventions. EVIDEM provides a methodological tool for scientific decision-making to evaluate evidence and value for health technologies on the basis of the multi-criteria decision analysis(MCDA) model and health technology assessment(HTA). Based on the ongoing EVIDEM research, the present study put forward that EVIDEM-based clinical comprehensive evaluation of Chinese patent medicine consisted of four aspects, seven modules, and ten steps, which is expected to references and practical experience for the follow-up comprehensive evaluation of Chinese patent medicine in the TCM field.

Impact on decision making framework for medicine purchasing in Chinese public hospital decision-making: determining the value of five dipeptidyl peptidase 4 (DPP-4) inhibitors.

BACKGROUND: Medicine purchasing in Chinese public hospitals is decided by the hospital Pharmacy Management Committee (PMC), that is complex, subjective and requires efficient approaches to ensure transparency and consistency for the factors being considered. This study aimed to use the Evidence and Value: Impact on Decision Making (EVIDEM) framework to assess medicine in these hospitals. In this study anti-diabetic drugs DPP-4 inhibitors, which work by inhibiting the activation of the Dipeptidyl Peptidase 4 (DPP-4) inhibitors, were appraised. METHODS: Following EVIDEM methodology (EVIDEM-10th), we convened an appraisal group and asked each individual to express their perspectives by assigning weights to each criterion. A systematic literature search for information of each criterion of five DPP-4 inhibitors was completed. Then the appraisal group scored for each criterion of the five DPP-4 inhibitors. The estimated value of the five DPP-4 inhibitors was obtained by Multi-Criteria Decision Analysis (MCDA) which combined individual weighting of each criterion with individual scoring for each intervention in each criterion. RESULTS: By assigning weights, the most important criterion was the quality of evidence (4.01±0.52), and that the comparative cost consequences-non-medical cost was the least important criterion (2.87±1.03). Criteria included disease severity, size of the affected population, comparative effectiveness, type of therapeutic/preventive benefit and cost of intervention, all of which were assigned the same weight of 3.58. After MCDA, the overall value orders for each DPP-4 inhibitor included Sitagliptin (0.45), Linagliptin (0.44), Vildagliptin (0.43), Alogliptin (0.42) and Saxagliptin (0.40). CONCLUSIONS: Based on EVIDEM framework and MCDA, we found that overall value of five DPP-4 inhibitors was similar. It is feasible to use the EVIDEM framework and MCDA in purchasing medicine for Chinese public hospitals.

Exploring physicians and patients' perspectives for current interventions on thyroid nodules using a MCDA method.

BACKGROUND: The detection of thyroid cancer has rapidly increased over last few decades without an increase in disease specific mortality. Several studies claim that the diagnose of thyroid nodules through routine ultrasound imaging is often the trigger for cascade effects leading to unnecessary follow-up over many years or to invasive treatment. The objective of this study was to explore physicians' and patients' insights and preferences regarding the current interventions on thyroid nodules. METHODS: An online survey was developed using a comprehensive multi-criteria decision analysis (MCDA) framework, the EVIdence based Decision-Making (EVIDEM). The EVIDEM core model used in this study encompassed 13 quantitative criteria and four qualitative criteria. Participants were asked to provide weights referring to what matters most important in general for each criterion, performance scores for appraising the interventions on thyroid nodules and their consideration of impact of contextual criteria. Normalized weights and standardized scores were combined to calculate a value contribution across all participants, additionally differences across physicians and patients' group were explored. RESULTS: 48 patients and 31 physicians were included in the analysis. The value estimate of the interventions on thyroid nodules reached 0.549 for patients' group and 0.5 was reported by the physicians' group, compared to 0.543 for all participants. The highest value contributor was 'Comparative effectiveness' (0.073 ± 0.020). For the physicians' group, 'Comparative safety' (0.050 ± 0.023) was given with higher value. And for the patients' group, 'Type of preventive benefits' (0.059 ± 0.022) contributed more positively to the value estimation. 51% participants considered 'Population priorities and access' having a negative impact on the interventions of nodules.66% participants thought that the 'system capacity' had a negative impact. CONCLUSION: Our study shows participants' preferences on each criterion, i.e., physician indicated keeping the interventions safe and effective more important, patients indicated quality of life after receiving interventions more important. Through comparison among participants, differences have been highlighted, which can make better communication between physicians and patients. This study provides a supportive decision-making for healthcare providers when they explored the interventions on thyroid nodules.

[Study on establishing a methodology of comprehensive clinical evaluation for Chinese patent medicine based on health technology assessment].

At present, most of Chinese patent medicines have problems, such as weak scientific basis, unclear clinical value evaluation, etc., which lead to the lack of sufficient evidence for the entry into the essential medicine list, medical insurance catalog and other important health resource allocation tools. This has a negative impact on the development of traditional Chinese medicine. It is urgent to establish a comprehensive evaluation methodology system of Chinese patent medicine in China. There are at least three reasons including producing evidence for clinical rational use scientifically, developing an objective and transparent selecting strategy, eventuating and highlighting its clinical value. The process and method of health technology assessment(HTA) can provide reference for the establishment of comprehensive evaluation method of Chinese patent medicine. However, HTA is rarely carried out in the field of traditional Chinese medicine. The latest domestic and international research showed that HTA combined with multi-criteria decision analysis(MCDA) can increase the transparency of decision-making and improve the quality of decision-making. The combined two formed the EVIDEM framework, which is conducive to drug selection and its clinical use. In this paper, the EVIDEM framework was introduced to establish the methodology framework of the comprehensive clinical evaluation system of Chinese patent medicine. A preliminary research idea has been put forward. In the future, we can establish a set of comprehensive clinical evaluation methods for Chinese patent medicine in cooperation with relevant national drug decision-making departments and management departments. It is believed that such cooperation could promote the full implementation of the re-evaluation for Chinese patent medicine.

Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs-a Systematic Literature Review. Next Steps in Methodology Development?

Background: Multi-criteria decision analysis (MCDA) is a decision-making tool that can take into account multidimensional factors and enables comparison of (medical) technologies by combining individual criteria into one overall appraisal. The MCDA approach has slowly gained traction within Health Technology Assessment (HTA) and its elements are gradually being incorporated into HTA across Europe. Several groups of scientists have proposed MCDA approaches targeted toward orphan drugs and rare diseases by including criteria specific to rare diseases. The goal of this article is to provide an overview of the current state of knowledge and latest developments in the field of MCDA in HTA for orphan drugs, to review existing models, their design characteristics, as well as to identify opportunities for further model improvement. Methods: A systematic literature search was conducted in January 2018 using four databases: MEDLINE (Pubmed), EBSCO HOST, EMBASE, and Web of science to find publications related to use of MCDA in the rare disease field (keywords: MCDA/orphan drug/rare disease and synonyms). Identified MCDA models were analyzed, e.g., structure, criteria, scoring, and weighting methodology. Results: Two hundred and eleven publications were identified, of which 29 were included after removal of duplicates. 9 authors developed own MCDA models, 7 of which based on literature reviews intended to identify the most important and relevant decision criteria in the model. In 13 publications (8 models) weights were assigned to criteria based on stakeholder input. The most commonly chosen criteria for creation of the MCDA models were: comparative effectiveness/efficacy, the need for intervention, and disease severity. Some models have overlapping criteria, especially in the treatment cost and effectiveness areas. Conclusions: A range of MCDA models for HTA have been developed, each with a slightly different approach, focus, and complexity, including several that specifically target rare diseases and orphan drug appraisal. Models have slowly progressed over the years based on pilots, stakeholder input, sharing experiences and scientific publications. However, full consensus on model structure, criteria selection and weighting is still lacking. A simplification of the MCDA model approach may increase its acceptance. A multi-stakeholder discussion on fundamental design and implementation strategies for MCDA models would be beneficial to this end.

Applying Reflective Multicriteria Decision Analysis (MCDA) to Patient-Clinician Shared Decision-Making on the Management of Gastroenteropancreatic Neuroendocrine Tumors (GEP-NET) in the Spanish Context.

INTRODUCTION: Unresectable, well-differentiated nonfunctioning gastroenteropancreatic neuroendocrine tumors (GEP-NETs) can be monitored (watchful waiting, WW) or treated with systemic therapy such as somatostatin analogues (SSAs) to delay progression. We applied a reflective multicriteria decision analysis (MCDA) shared-decision framework (previously developed for the USA) to explore what matters to Spanish patients and clinicians considering GEP-NET treatment options. METHODS: The EVIDEM-derived framework was updated and adapted to the Spanish context. During a Chatham House session, five patients and six physicians assigned criteria weights using hierarchical point allocation and direct rating scale (alternative analysis). Informed by synthesized evidence embedded in the framework, participants scored how each criterion favored SSA treatment (reference case lanreotide) or WW and shared insights and knowledge. Weights and scores were combined into value contributions (norm. weight × score/5), which were added across criteria to derive the relative benefit-risk balance (RBRB, scale - 1 to + 1). Exploratory comparisons to US study findings were performed. RESULTS: Focusing on intervention outcomes (effectiveness, patient-reported, and safety), the mean RBRB favored treatment over WW (+ 0.32 ± 0.24), with the largest contributions from progression-free survival (+ 0.11 ± SD 0.07), fatal adverse events (+ 0.06 ± SD 0.08), and impact on HRQoL (+ 0.04 ± SD 0.04). Consideration of modulating criteria (type of benefit, need, costs, evidence, and feasibility) increased the RBRB to + 0.50 ± 0.14, with type of therapeutic benefit (+ 0.10 ± SD 0.08) and quality of evidence (+ 0.08 ± SD 0.06) contributing most towards treatment. Alternative weighting yielded similar results. Results were broadly comparable to those derived from the US study. CONCLUSION: The multicriteria framework helped Spanish patients and clinicians identify and express what matters to them. The approach is transferable across decision-making contexts. FUNDING: IPSEN Pharma.

DRUG EVALUATION AND DECISION MAKING IN CATALONIA: DEVELOPMENT AND VALIDATION OF A METHODOLOGICAL FRAMEWORK BASED ON MULTI-CRITERIA DECISION ANALYSIS (MCDA) FOR ORPHAN DRUGS.

OBJECTIVES: The aim of this study was to adapt and assess the value of a Multi-Criteria Decision Analysis (MCDA) framework (EVIDEM) for the evaluation of Orphan drugs in Catalonia (Catalan Health Service). METHODS: The standard evaluation and decision-making procedures of CatSalut were compared with the EVIDEM methodology and contents. The EVIDEM framework was adapted to the Catalan context, focusing on the evaluation of Orphan drugs (PASFTAC program), during a Workshop with sixteen PASFTAC members. The criteria weighting was done using two different techniques (nonhierarchical and hierarchical). Reliability was assessed by re-test. RESULTS: The EVIDEM framework and methodology was found useful and feasible for Orphan drugs evaluation and decision making in Catalonia. All the criteria considered for the development of the CatSalut Technical Reports and decision making were considered in the framework. Nevertheless, the framework could improve the reporting of some of these criteria (i.e., "unmet needs" or "nonmedical costs"). Some Contextual criteria were removed (i.e., "Mandate and scope of healthcare system", "Environmental impact") or adapted ("population priorities and access") for CatSalut purposes. Independently of the weighting technique considered, the most important evaluation criteria identified for orphan drugs were: "disease severity", "unmet needs" and "comparative effectiveness", while the "size of the population" had the lowest relevance for decision making. Test-retest analysis showed weight consistency among techniques, supporting reliability overtime. CONCLUSIONS: MCDA (EVIDEM framework) could be a useful tool to complement the current evaluation methods of CatSalut, contributing to standardization and pragmatism, providing a method to tackle ethical dilemmas and facilitating discussions related to decision making.