Journal of market access & health policy最新文献

Background: Clinician's choice of hypoallergenic formulas in the first-line management of cow's milk protein allergy (CMPA) should be informed by evidence on clinical efficacy and cost-effectiveness.

Objective: We compare the cost-effectiveness of amino acid-based formula (AAF), extensively hydrolyzed casein formula with Lactobacillus rhamnosus Gorbach Goldin (EHCF+LGG), extensively hydrolyzed whey formula (EHWF), and rice hydrolyzed formula (RHF) in non-breastfed children in France.

Methods: Immunotolerance and atopic manifestations' prevalence were based on a prospective non-randomized study with a 36-month follow-up. Resource utilization was sourced from a survey of French clinicians, and unit costs were based on national data. Costs and health consequences were discounted at 2.5% annually. Results were reported using the Collective and French National Health Insurance perspectives.

Results: Children receiving EHCF+LGG were predicted to require less healthcare resources, given their reduced prevalence of CMPA symptoms at 3 years. In the base case, EHCF+LGG led to savings of at least €674 per child compared to AAF, EHWF, and RHF at 3 years, from both perspectives. Nutrition had the highest economic burden in CMPA, driven by hypoallergenic formulas and dietetic replacements costs. Results were robust to one-way and probabilistic sensitivity analyses.

Conclusions: EHCF+LGG was associated with more symptom-free time, higher immune tolerance, and lower costs.

Involvement of all relevant stakeholders will be of utmost importance for the success of the developing EU HTA harmonization process. A multi-step procedure was applied to develop a survey across stakeholders/collaborators within the EU HTA framework to assess their current level of involvement, determine their suggested future role, identify challenges to contribution, and highlight efficient ways to fulfilling their role. The 'key' stakeholder groups identified and covered by this research included: patients', clinicians', regulatory, and Health Technology Developer representatives. The survey was circulated to a wide expert audience including all relevant stakeholder groups in order to determine self-perception by the 'key' stakeholders regarding involvement in the HTA process (self-rating), and in a second, slightly modified version of the questionnaire, to determine the perception of 'key' stakeholder involvement by HTA bodies, payers, and policymakers (external rating). Predefined analyses were conducted on the submitted responses. Fifty-four responses were received (patients 9; clinicians: 8; regulators: 4; HTDs 14; HTA bodies: 7; Payers: 5; policymakers 3; others 4). The mean self-perceived involvement score was consistently lower for each of the 'key' stakeholder groups than the respective external ratings. Based on the qualitative insights generated in the survey, a RACI Chart (Responsible/Accountable/Consulted/Informed) was developed for each of the stakeholder groups to determine their roles and involvement in the current EU HTA process. Our findings suggest extensive effort and a distinct research agenda are required to ensure adequate involvement of the key stakeholder groups in the evolving EU HTA process.

Objectives: In this paper, we outline and compare pharmaceutical pricing and reimbursement policies for in-patent prescription medicines in three Maghreb countries, Algeria, Morocco, and Tunisia, and explore possible improvements in their pricing and reimbursement systems.

Methods: The evidence informing this study comes from both an extensive literature review and a primary data collection from experts in the three studied countries.

Key findings: Twenty-six local experts where interviewed Intervieweesincluded ministry officials, representatives of national regulatory authorities, health insurance organizations, pharmaceutical procurement departments and agencies, academics, private pharmaceutical-sector actors, and associations. Results show that External Reference Pricing (ERP) is the dominant pricing method for in-patent medicines in the studied countries. Value-based pricing through Health Technology Assessment (HTA) is a new concept, recently used in Tunisia to help the reimbursement decision of some in-patent medicines but not yet used in the pricing of innovative medicines in the studied countries. Reimbursement decision is mainly based on negotiations set on Internal Reference Pricing (IRP).

Conclusion: Whereas each country has its specific regulations, there are many similarities in the pricing and reimbursement policies of in-patent medicines in Algeria, Morocco, and Tunisia. The ERP was found to be the dominant method to inform pricing and reimbursement decisions of in-patent medicines. Countries in the region can focus on the development of explicit value assessment systems and minimize their dependence on ERP over the longer-term. In this context, HTA will rely on local assessment of the evidence.

Purpose: The success of reverse total shoulder arthroplasty (RTSA) has expanded its use for a broader range of shoulder indications worldwide. Evidence regarding the relative efficacy and long-term safety of medical technologies used in RTSA is subjected to rigorous assessment. Nonetheless, substantial challenges impede market access for innovative shoulder implant technologies for RTSA in Australia, resulting in delayed patient access.

Approach: This paper addresses the key challenges associated with generating evidence for the health technology assessments of innovative medical technologies for RTSA that are required for access to the Australian market. The transition to value-based care requires establishing a benchmarking reference that incorporates patient-reported outcome measures (PROMs) and combines revision outcomes with additional clinical outcomes to increase patient cohort sizes. Establishing the benchmark would require agreement on the outcome measures to be collected for each indication, and investment in reporting patient-reported outcomes for RTSA to the national orthopaedic registry.

Implications for practice: The need for increased flexibility in developing evidence for health technology assessment of RTSA medical technologies is required. Optimised approaches for benchmarking RTSA require extensive stakeholder discussions, including the agreement on evidence requirements and follow-up periods, selection of clinical outcomes, as well as pre-operative and post-operative PROMs as a value assessment.

Background: Copay cards are intended to mitigate patient out-of-pocket (OOP) expenses. This qualitative, exploratory focus group study aimed to capture patient perceptions of copay cards and copay adjustment programs (CAPs; insurers' accumulator and maximizer policies), which redirect the copay card utilization benefits intended for patients' OOP expenses.

Methods: Patients with chronic conditions were recruited through Janssen's Patient Engagement Research Council program. They completed a survey and attended a live virtual session to provide feedback on copay cards.

Results: Among 33 participants (median age, 49 years [range, 24-78]), the most frequent conditions were cardiovascular-metabolic disease and inflammatory bowel disease. Patients associated copay cards with lessening financial burden, improving general and mental health, and enabling medication adherence. An impact on medication adherence was identified by 10 (63%) White and nine (100%) Black respondents. Some patients were unaware of CAPs despite having encountered them; they recommended greater copay card education and transparency about CAPs.

Conclusion: Patients relied on copay cards to help afford their prescribed medication OOP expenses and maintain medication adherence. Use of CAPs may increase patient OOP expenses. Patients would benefit from awareness programs and industry - healthcare provider partnerships that facilitate and ensure access to copay cards.

Introduction: Prolonged Hospital Length of Stay (PLOS) is an indicator of deteriorated efficiency in Quality of Care. One goal of public health management is to reduce PLOS by identifying its most relevant predictors. The objective of this study is to explore Machine Learning (ML) models that best predict PLOS.

Methods: Our dataset was collected from the French Medico-Administrative database (PMSI) as a retrospective cohort study of all discharges in the year 2015 from a large university hospital in France (APHM). The study outcomes were LOS transformed into a binary variable (long vs. short LOS) according to the 90^th percentile (14 days). Logistic regression (LR), classification and regression trees (CART), random forest (RF), gradient boosting (GB) and neural networks (NN) were applied to the collected data. The predictive performance of the models was evaluated using the area under the ROC curve (AUC).

Results: Our analysis included 73,182 hospitalizations, of which 7,341 (10.0%) led to PLOS. The GB classifier was the most performant model with the highest AUC (0.810), superior to all the other models (all p-values <0.0001). The performance of the RF, GB and NN models (AUC ranged from 0.808 to 0.810) was superior to that of the LR model (AUC = 0.795); all p-values <0.0001. In contrast, LR was superior to CART (AUC = 0.786), p < 0.0001. The variable most predictive of the PLOS was the destination of the patient after hospitalization to other institutions. The typical clinical profile of these patients (17.5% of the sample) was the elderly patient, admitted in emergency, for a trauma, a neurological or a cardiovascular pathology, more often institutionalized, with more comorbidities notably mental health problems, dementia and hemiplegia.

Discussion: The integration of ML, particularly the GB algorithm, may be useful for health-care professionals and bed managers to better identify patients at risk of PLOS. These findings underscore the need to strengthen hospitals through targeted allocation to meet the needs of an aging population.

Objectives: Since the initial release of biosimilars 18 years ago, regulations for their licensing have changed considerably; however, there is no global consensus on these regulations. Establishing harmonized regulatory guidelines for the approval of biosimilars with support from the ICH, an independent, non-profit association under Swiss law, will significantly enhance the affordability of biological drugs.

Methods: Regulatory guidelines from the Food and Drug Administration (FDA), European Medicines Agency (EMA), Medicines and Healthcare products Regulatory Agency (MHRA), and World Health Organization (WHO) were analyzed for historical changes and elements critical to the safety and efficacy of biosimilars.

Results: Analysis of all EMA and FDA filings show that none of the animal testing and clinical efficacy testing failed because animals do not have the required receptors to initiate pharmacologic responses, and efficacy studies cannot be statistically powered to conclude any results. New analytical technologies will enable good biosimilarity determination, avoiding both tests.

Conclusion: Scientifically based ICH guidelines that remove redundant studies will reduce development costs, improve safety, and allow global drug distribution based on single compliance. These guidelines are particularly necessary for emerging countries lacking the expertise and resources to evaluate biosimilar filings.

Background: Globally, healthcare has shouldered much of the socioeconomic brunt of the COVID-19 pandemic leading to numerous clinical trials suspended or discontinued.

Objective: To estimate the COVID-19 impact on the number of clinical trials worldwide.

Methods: Data deposited by 219 countries in the ClinicalTrials.gov database (2007-2020) were interrogated using targeted queries. A time series model was fitted to the data for studies ongoing, initiated, or ended between 2007 Quarter (Q) 1 and 2019 Q4 to predict the expected trials number in 2020 in the COVID-19 absence. The predicted values were compared with the actual 2020 data to quantify the pandemic impact.

Results: The ongoing registered trials number grew from 2007 Q1 (33,739) to 2019 Q4 (80,319). By contrast, there were markedly fewer ongoing trials in all four quarters of 2020 compared with forecasted values (1.6%-2.8% decrease). When excluding COVID-19-related studies, this disparity grew further (3.4%-5.8% decrease), to a peak of almost 5,000 fewer ongoing trials than estimated for 2020 Q2. The initiated non-COVID-19 trials number was higher than predicted in 2020 Q4 (9.9%).

Conclusions: This pandemic has impacted clinical trials. Provided that current trends persist, clinical trial activities may soon recover to at least pre-COVID-19 levels.

Objectives: Deliberative processes in Health Technologies Assessment (HTA) result in recommendations that determine the reimbursement of medicines, diagnostics or devices. These processes are governed by explicit criteria, but are also influenced by implicit factors. The objective of this work was to identify the implicit factors influencing HTA deliberative processes in five European countries (France, Germany, Italy, Spain and the UK).

Methods: A systematic review of literature published between 2009 and 2019 was conducted. The search was performed in Pubmed, The Cochrane Database of Systematic Reviews, Google Scholar and Center for Reviews and Dissemination. The ISPOR database was searched manually.

Results: Out of 100 eligible publications, eight articles were selected for data extraction and analysis. The implicit factors in the HTA deliberative process most frequently mentioned in the identified literature are value judgments, biases, preferences and subjectivity. Five out of the eight articles highlight the need to further improve the transparency of the process, and three provide recommendations on how to address the influence of implicit factors on the HTA deliberative process through a framework.

Conclusion: Even in countries with a long HTA history, evidence on implicit factors is scarce. Some methods have been recommended for addressing these factors. Further research is required to characterize the implicit factors in the HTA deliberative process at a country level and explore potential ways to mitigate the influence of these factors on the HTA deliberative process.