Journal of market access & health policy最新文献

Background and Objective: China has managed to control the coronavirus disease (COVID-19) with confinement measurements and treatment strategies, while other countries are struggling to contain the spread. This study discusses the guidelines related to COVID-19 in China in order to provide important references for other countries in the fight against COVID-19. Methods: Chinese guidelines relevant to COVID-19 were systematically searched via the China National Knowledge Infrastructure database, YiMaiTong database, and World Health Organization (WHO) COVID-19 database on March 20^th, 2020. Guideline information was extracted, including date of publication, source, objectives and the target population. Guidelines specific to the pharmacological treatment of COVID-19 were further investigated to identify the types of antivirus drugs recommended and to report on how treatment recommendations for COVID-19 have evolved overtime. Results: A total of 100 guidelines were identified, of which 74 were national guidelines and 26 were regional guidelines. The scope of included guidelines consisted of: the diagnosis and treatment of COVID-19, the management of hospital departments and specific diseases during the outbreak of COVID-19. Fifty-one of the included guidelines targeted overall COVID-19 patients, while the remaining guidelines concentrated on special patient populations (i.e. geriatric population, pediatric population, and pregnant population) or patients with coexisting diseases. Fifteen guidelines focused on the pharmacological treatments for all COVID-19 patients. Interferon, Lopinavir/Ritonavir, Ribavirin, Chloroquine, and Umifenovir represented the most recommended antivirus drugs. Among them, 7 Chinese guidelines have recommended Chloroquine Phosphate or Hydroxychloroquine for the treatment of COVID-19. Conclusions: China has generated a plethora of guidelines covering almost all aspects of COVID-19. Chloroquine, as one widely affordable treatment, was recommended by Chinese national guidelines and provincial guidelines. Considering the continuous debates around Chloroquine, confirmatory studies with robust methodology are awaited to address the unanswered questions on its potential benefits and risks on COVID-19.

Background: Non-medical switching refers to a change in a stable patient's prescribed medication to a clinically distinct, non-generic, alternative for reasons other than poor clinical response, side-effects or non-adherence.

Objective: To assess the perceptions of high-volume Medicare and/or Medicaid physician providers regarding the impact non-medical switching has on their patients' medication-related outcomes and health-care utilization.

Methods: We performed an e-survey of high-volume Medicare and/or Medicaid physicians (spending >50% of their time caring for Medicare and/or Medicaid patients), practicing for >2 years but <30 years post-residency and/or fellowship; working in a general, internal, family medicine or specialist setting; spending ≥40% of their time providing direct care and having received ≥1 request for a non-medical switch in the past 12 months. Physicians were queried on 15-items to assess perceptions regarding the impact non-medical switching on medication-related outcomes and health-care utilization.

Results: Three-hundred and fifty physicians were included. Respondents reported they felt non-medical switching, to some degree, increased side-effects (54.0%), medication errors (56.0%) and medication abandonment (60.3%), and ~50% believed it increased patients' out-of-pocket costs. Few physicians (≤13.4% for each) felt non-medical switching had a positive impact on effectiveness, adherence or patients' or physicians' confidence in the quality-of-care provided. Non-office visit and prescriber-pharmacy contact were most frequently thought to increase due to non-medical switching. One-third of physicians felt office visits were very frequently/frequently increased, and ~ 1-in-5 respondents believed laboratory testing and additional medication use very frequently/frequently increased following a non-medical switch. About 1-in-10 physicians felt non-medical switching very frequently/frequently increased the utilization of emergency department or in-hospital care.

Conclusion: This study suggests high-volume Medicare and/or Medicaid physician providers perceive multiple negative influences of non-medical switching on medication-related outcomes and health-care utilization.

Purpose A international registry analysis led by Mehra et al. to investigate the use of hydroxychloroquine (HCQ) and chloroquine (CQ) with or without a macrolide in 96,032 hospitalised COVID-19 patients were published on Lancet, which has raised considerable discussions in the public health community. This study aimed to critically review the quality and limitations of the Mehra et al. publication and discuss the potential influences on the use of HCQ/CQ worldwide. Method A critical review of this publication was conducted to examine the potential study bias in the study objectives, methodology, confounding factors and outcomes and summarise the external reviews. Results The very high homogeneity of the patients' characteristics at baseline was inconsistent with region specific epidemiology and several critical confounding factors. The results indicated that angiotensin converting enzyme inhibitors were associated with a hazard ratio of 0.5, which suggested a technical problem in the estimation of the propensity scores. Several major risk factors for mortality identified in the analysis were treated as a minor risk or neutral or even protective factors. Antiviral treatments were recognised as an effective method to reduce mortality and were neither further studied nor integrated in the multivariate Cox model. Conclusion This research appeared to carry multiple biases. An extensive audit of the study, conditions of review and acceptance for publication in the Lancet of that study are requested to avoid damage to the publics' trust on the scientific community at this critical time of COVID-19 pandemic.

Background: Organizational aspect is rarely considered in healthcare. However, it is gradually seen as one of the key aspects of the decision-making process as well as clinical and economic dimensions. Our primary objective was to identify criteria already used to assess the organizational impact of medical innovations. Our secondary objective was to structure them into an inventory to support decision-makers to select the relevant criteria for their complex decision-making issues.

Materials and methods: A search using the Medline database was conducted in June 2019. The records published between January, 1990 and December, 2018 were identified. The publications cited by the authors of the included articles and the websites of health technology assessment agencies, units or learned societies identified during the search were also consulted. The identified criteria were structured in an inventory.

Results: We selected 107 records of a wide range of evidence mostly published after the 2000s. We identified 636 criteria that we classified into five categories: people, task, structure, technology, and surroundings.

Conclusion: Criteria selection is a crucial step in any multi-criteria decision analysis (MCDA). This work is the first step in the development of a validated MCDA method to assess the organizational impact of medical innovations.

Background: Morbidity and mortality associated with non-valvular atrial fibrillation (NVAF) imposes a substantial economic burden on the UK healthcare system.

Objectives: An existing Markov model was adapted to assess the real-world cost-effectiveness of rivaroxaban and apixaban, each compared with a vitamin K antagonist (VKA), for stroke prevention in patients with NVAF from the National Health Service (NHS) and personal and social services (PSS) perspective.

Methods: The model considered a cycle length of 3 months over a lifetime horizon. All inputs were drawn from real-world evidence (RWE): baseline patient characteristics, clinical event and persistence rates, treatment effect (meta-analysis of RWE studies), utility values and resource use. Deterministic and probabilistic sensitivity analyses were performed.

Results: The incremental cost per quality-adjusted life year was £14,437 for rivaroxaban, and £20,101 for apixaban, compared with VKA. The probabilities to be cost-effective compared with VKA were 90% and 81%, respectively for rivaroxaban and apixaban, considering a £20,000 threshold. In both comparisons, the results were most sensitive to clinical event rates.

Conclusions: These results suggest that rivaroxaban and apixaban are cost-effective vs VKA, based on RWE, considering a £20,000 threshold, from the NHS and PSS perspective in the UK for stroke prevention in patients with NVAF. This economic evaluation may provide valuable information for decision-makers, in a context where RWE is more accessible and its value more acknowledged.

Background: Ischaemic stroke (IS) is a major cause of death and disability and affects the quality of life of patients. Previous studies focused on urban populations.

Objective: To evaluate the health-related quality of life (QoL) of patients with history of IS and living in a rural area in Poland.

Patients: Rural population of 172 patients discharged from a district hospital in Zakopane, Poland with a diagnosis of IS in the period from 01.01.2005 to 31.10.2006.

Intervention: QoL was evaluated using the European Quality of Life Scale-5 Dimensions EQ-5D-3 L (EQ-5D) and the Short Form Health Survey - 12 version 2 (SF-12).

Results: In the EQ-5D survey, 57.3% of patients had only some problems with mobility, 40.3% with usual activities, 63.2% with pain/discomfort, 59% with anxiety/depression, and 32.2% with self-care. In the SF-12 survey, both summary components (physical and psychological) were reduced compared to the population norm.

Conclusion: The quality of life in IS survivors is clearly reduced in the majority of domains assessed by the EQ-5D and SF-12 questionnaires. The most important factors affecting QoL were the functional state, depression and anxiety. A significant difference as compared to to urban and mixed populations was observed for a reduced SF-12 mental health component and for the EQ-5D visual analogue scale. We found no effect of gender, age or cognitive disorders on the outcomes of SF-12.

The results of a clinical trial comparing hydroxychloroquine with or without azithromycin to the standard of care for the treatment of COVID-19 were recently published by Philippe Gautret et al. This study provides outstanding results for the combination of hydroxychloroquine and azithromycin over the standard of care, but the evidence was deemed insufficiently robust to warrant a public health decision to widen the use of hydroxychloroquine for the treatment of COVID-19. We provide a scientific critical review of the Gautret et al. publication, put the results in the context of the current knowledge, provide an evaluation of the validity of the results (from a methodologic perspective), and discuss public health implications. The study has a number of limitations, including small sample size, lack of comparability between patients in active treatment and control arms, lack of blinding, use of interim analyses without controlling for the risk of type 1 error, use of analysis in the per-protocol population instead of the intention-to-treat population, and inconsistencies between the study protocol and article. However, none of these observations is of a nature to reverse the conclusions. The study brings useful knowledge consistent with available evidence and clinical practice from China and South Korea, which could have prompted quicker policy decision-making.

Background: Current detection methodologies are often unable to identify the location and extent of recurrent prostate cancer (PCa) leading potentially to 'futile' local therapies in the presence of metastatic disease. The use of ¹⁸ F-fluciclovine PET/CT may lead to better patient management. Objective: The aim of this study was to quantify the economic impact and cost-consequence of using ¹⁸ F-fluciclovine PET/CT in PCa recurrence. Study design: A decision analytic model based on recurrent PCa imaging guidelines. Setting: US hospital. Participants: PCa patients experiencing biochemical recurrence. Intervention: ¹⁸ F-fluciclovine PET/CT was compared to conventional imaging. Main outcome measure: Budget impact, correct diagnoses, futile treatments, and cost-consequence (cost per correct diagnosis) Results: For a hypothetical hospital serving 500,000 individuals, the model showed the use of ¹⁸ F-fluciclovine reduced 'futile' therapies by 19.2%. Re-imaging costs were reduced by 40.2% ($8.2 million); however, when assuming diagnostic and staging costs only, the total costs increased from $31.2 to $34.6 million (10.9%), driven by ¹⁸ F-fluciclovine imaging agent and procedure costs. The cost per 'correct' diagnosis declined $30,673 (46.8%). When including subsequent 5-year patient management, the cost per 'correct' diagnosis declined $410,206 (49.2%). Conclusion: ¹⁸ F-fluciclovine PET/CT imaging may improve the clinical management of men with recurrent PCa with minimal increase in healthcare spending.

Introduction and objective: Vernal keratoconjunctivitis (VKC) is a rare allergic eye condition that occurs in children and is characterised by a combination of debilitating symptoms. Repeated use of topical corticosteroid rescue therapy is often necessary in severe forms. This study aims to assess the validity of a new composite endpoint: the penalties-adjusted corneal staining score (PACS-S) proposed as primary endpoint in VEKTIS trial evaluating the efficacy of a new corticosteroid-sparing treatment, VERKAZIA® (ciclosporin 1 mg/ml eye drops), in severe VKC patients. Methodology: This research comprised a systematic literature review to identify efficacy endpoints being proposed in clinical trials for pediatric patients with severe VKC, followed by a remote expert advisory board assessing the validity of the PACS-S. Results: While no agreed or validated endpoint for assessing efficacy in VKC was identified when VEKTIS trial started, the experts' board acknowledged a high face validity of PACS-S as a subjective integrated measure matching the current clinical practice. A fair external validity was considered with regards to VEKTIS trial secondary endpoints. Conclusion: PACS-S appears to be a reliable, valid and clinically meaningful primary endpoint that allows significant improvement over existing endpoints in severe VKC trials. Additional research is needed to validate this endpoint.

Background: Limited previous research and guidelines on the design of economic evaluation for biosimilars have led to unresolved methodological questions on how to assess biosimilars. Objectives: We want to raise awareness of and explore methodological issues for the economic evaluation of biosimilars. Methods: We relied on a literature review, exploratory interviews, and our experiences. Results and Conclusions: In the majority of cases in which reimbursement for a biosimilar is sought, it will not be necessary to conduct an economic evaluation, given that the reference product is already reimbursed and standard of care. If the latter is not the case, a full economic evaluation of the biosimilar versus standard of care is needed. This might also be needed in the case of differences in administration form or adherence (for example, due to a nocebo effect) and to take into account value-added services. The entry of biosimilars and of next-generation biological products should trigger a re-assessment of the entire product class. HTA bodies and reimbursement agencies should provide clear guidance on how to assess the value of a biosimilar in each of these circumstances.