Journal of Market Access & Health Policy

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...

Background : Delandistrogene moxeparvovec (SRP-9001) is an investigational gene therapy that may delay progression of Duchenne muscular dystrophy (DMD), a severe, rare neuromuscular disease caused by DMD gene mutations. Early cost-effectiveness analyses are important to help contextualize the value of gene therapies for reimbursement decision making. Objective : To determine the potential value of delandistrogene moxeparvovec using a cost-effectiveness analysis. Study design : A simulation calculated lifetime costs and equal value of life years gained (evLYG)...

Background: Like many developing countries, Colombia faces difficulties in financing health-care services as well as programs for health promotion and health education and there is evidence that its health-care system is underperforming. Objective: To provide evidence-based estimates of potential funding levels and assess the strengths, weaknesses, and viability of innovative funding mechanisms with a focus on treating rare diseases in Colombia. Methods: The strategy involved evidence-based projections of potential funding levels and a qualitative viability assessment using an expert panel...

BACKGROUND: Artificial intelligence (AI) enables remote patient monitoring (RPM) which reduces costs by triaging patients to optimize hospitalization and avoid complications. The FDA regulates AI in medical devices and aims to ensure patient safety, effectiveness, and transparent AI solutions. OBJECTIVES: Identify and summarize FDA approved RPM devices to provide information for the US medical device industry based on previous approvals and the markets' needs. METHODS: We searched publicly available databases on FDA-approved RPM devices...

INTRODUCTION: The Indian healthcare system is evolving towards better healthcare implementation and coverage. However, even today, the health-care system faces several challenges, a few of which are yet to be addressed. The present review is aimed to delineate the past and present healthcare scenarios in India, health-care policies, and other initiatives for achieving universal health coverage (UHC). METHODS: A literature search was done on various government databases, websites, and PubMed for obtaining data and statistics on healthcare funding, health insurance schemes, healthcare budget allocations, categories of medical expenses, government policies, and health technology assessment (HTA) in India...

INTRODUCTION: To ensure the sustainability of the AT access improvement, it is important that health system stakeholders have timely, analyzed information accessible for reference and decision-making support. In this study, we projected the direct costs required as well as the expected direct medical cost-offset and productivity benefits resulting from improving the disease control. METHODS: We implemented a deterministic, prevalence-based mathematical model to project the annual cost of rheumatoid arthritis (RA) management within the public healthcare system in Malaysia...

INTRODUCTION: Familial Mediterranean Fever (FMF) is a hereditary autoinflammatory disease that significantly reduces occupational productivity and quality-of-life in affected patients. Italy has an estimated FMF prevalence of 1 in 60,000 people. While colchicine is the primary treatment for FMF, biologics are administered to intolerant and non-responder patients. Anakinra and canakinumab are the only biologics approved and reimbursed for FMF in Italy. Both medicines have demonstrated efficacy in FMF patients yet differ in treatment costs...

BACKGROUND: Perianal fistula (PF), a complication of Crohn's disease (CD), affects health-related quality of life (QOL). OBJECTIVE: To elucidate QOL of health states corresponding to different stages of PF associated with CD in Japan. METHOD: This cross-sectional, observational, web-based questionnaire survey assessed eight different health states in patients with CD and PF and individuals without CD (non-patients) from the Medilead Healthcare Panel (MHP) and determined the utility values (QOL scores) in each health state by the time trade-off method...

BACKGROUND: The evidence base of tisagenlecleucel is uncertain. OBJECTIVE: To evaluate the cost-effectiveness of tisagenlecleucel. To conduct expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses. STUDY DESIGN: A three-state partitioned survival model. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 5 years; general population mortality with a standardised mortality ratio was then applied...

BACKGROUND: The increasing prevalence of obesity imposes a significant cost burden on individuals and societies worldwide. OBJECTIVE: In this nationally representative study, the association between body mass index (BMI) groups and the number of metabolic comorbidities (MetC) with total direct costs was investigated in the Finnish population. STUDY DESIGN, SETTING, AND PARTICIPANTS: The study cohort included 5,587 adults with BMI ≥18...

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...

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...

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)...

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...

Background: Precision oncology has a prominent role in nonsquamous non-small cell lung cancer (nsNSCLC) treatment progress; however, its access in a real-world scenario might be limited. Objective: To investigate the time spent in nsNSCLC molecular profile evaluation and its influence on clinical decisions. Methods: nsNSCLC patients who underwent molecular testing in a private referral Brazilian center between November 2015 and February 2020 were identified...

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...

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...

Background: Quantification of COVID-19 burden may be useful to support the future allocation of resources. Objective: To evaluate the public health impact of COVID-19 in French ambulatory patients with at least one risk factor for severe disease. Study design: A Markov model was used to estimate life years, costs, number of hospitalisations, number of deaths and long/prolonged COVID forms over a time horizon of 2 years. The hospitalisation probabilities were derived from an early access cohort, and the hospitalisation stay characteristics were derived from the French national hospital discharge database...

Background: In order to optimize positioning and associated drug price for both payer and investor, it is for a company essential to forecast the potential market access attractiveness for the new drug for different indications at the early onset of the clinical development program. This analysis must include the constraints from the perspective of the payer, but also the biotech companies, who require a minimum drug price to satisfy their investors. This paper aims to provide an Integrated Valuation Model for payer and investor, bridging concepts from health economics and economic valuation reflecting the perspectives of the payer and the investor for a drug in early clinical development phase...

Background: The economic impact of adverse events (AEs) for poly (ADP-ribose) polymerase inhibitors (PARPis) in ovarian or breast cancer has not been widely evaluated. Objective: Compare PARPi-related AE management costs from a US payer perspective. Methods: The frequency of treatment-related grade 3-4 AEs was obtained from published clinical trials of PARPis for the treatment of advanced ovarian cancer (AOC), platinum-sensitive recurrent ovarian cancer (PSROC), and metastatic breast cancer (MBC)...