Cost-minimization analysis to support the HTA of Radiofrequency Echographic Multi Spectrometry (REMS) in the diagnosis of osteoporosis

This study aims at evaluating the costs of REMS vs. the conventional ionizing technology (dual-energy X-ray absorptiometry, DXA) for the diagnosis of osteoporosis from the perspective of the Italian National Health Service (NHS) using a cost-minimization analysis (CMA)

what type of clinical evidence is needed to assess medical devices

The objective of this mini-review is to discuss the role of real-world studies as a source of clinical evidence when experimental studies, such as randomised controlled trials (RCTs), are not available. Waiting for RCT evidence when the technology is diffusing could be anti-economical, inefficient from the policy perspective and methodologically questionable.We explain how real-world studies could provide relevant evidence to decision makers. Matching techniques are discussed as a viable solution for bias reduction.We describe a case study concerning a cost-effectiveness analysis based on real-world data of a technology already in use: Mitraclip combined with medical therapy versus medical therapy alone in patients with moderate-to-severe mitral regurgitation. The CEA has encountered the scepticism of most reviewers, due not to the statistical methodology but to the fact that the study was observational and not experimental. Editors and reviewers converged in considering real-world economic evaluations premature in the absence of a RCT, even if in the meantime the technology had been implanted >30 000 times. We believe there is a need to acknowledge the importance of real-world studies, and engage the scientific community in the promotion and use of clinical evidence produced through observational studies

The value of hearing aids for the Italian NHS: a cost-utility analysis

Objective: Hearing loss (HL) prevalence in Italy is expected to increase due to population aging. Hearing aids (HAs) are the main tool for HL rehabilitation; however, cost-utility analyses of HAs are limited. Our objective was to estimate the cost-utility of HAs use. Study Design: Cost-utility analysis. Setting: Italian National Healthcare Service, societal perspective. Patients, Intervention(s), and Main Outcome Measure(s): A multistate Markov model was developed to model a cohort of 55-year-old individuals starting from normal hearing and moving across HL states to compare cost-utility and net monetary benefit of HA use accompanied by post-purchase service, HA use alone, and no treatment. Parameters were estimated using secondary data. Incremental cost-utility ratio (ICUR) and incremental net monetary benefit (INMB) were computed against a €16,625/quality-adjusted life year (QALY) willingness-to-pay (WTP) threshold. Deterministic and probabilistic sensitivity analysis (DSA, PSA) was implemented to assess how uncertainty affected results. Scenario analysis was performed on different assumptions on costs, dropout and compliance rates. Results: The model suggests HAs use is a cost-effective strategy compared to no treatment (in the base case: incremental costs €429–€476, incremental QALY gain 0.18 and 0.19, ICUR €2‚404/QALY–€2‚450/QALY, INMB €2‚476–€2‚682 for male and female cohort, respectively). By assuming no dropout, INMBs increase up to €10,643–€10,728. DSA highlights that utility weights contribute the most to model uncertainty, PSA shows that the treatment has 97.8%–97.3% probability of being cost-effective at the WTP threshold considered. Conclusions: We proposed an original model to assess the cost-utility of HAs use; the application to the Italian setting suggests the treatment is cost-effective, reinforcing the importance of early uptake

Reflections on the importance of cost of illness analysis in rare diseases: a proposal

In the field of rare diseases (RDs), the evidence standard is often lower than that required by health technology assessment (HTA) and payer authorities. In this commentary, we propose that appropriate economic evaluation for rare disease treatments should be initially informed by cost-of-illness (COI) studies conducted using a societal perspective. Such an approach contributes to improving countries’ understanding of RDs in their entirety as societal and not merely clinical, or product-specific issues. In order to exemplify how the disease burden’s distribution has changed over the last fifteen years, key COI studies for Hemophilia, Fragile X Syndrome, Cystic Fibrosis, and Juvenile Idiopathic Arthritis are examined. Evidence shows that, besides methodological variability and cross-country differences, the disease burden’s share represented by direct costs generally grows over time as novel treatments become available. Hence, to support effective decision-making processes, it seems necessary to assess the re-allocation of the burden produced by new medicinal products, and this approach requires identifying cost drivers through COI studies with robust design and standardized methodology

The impact of HTA and procurement practices on the selection and prices of medical devices.

Technological innovation in healthcare yields better health outcomes but also drives healthcare expenditure, and governments are struggling to maintain an appropriate balance between patient access to modern care and the economic sustainability of healthcare systems. Health Technology Assessment (HTA) and centralized procurement are increasingly used to govern the introduction and diffusion of new technologies in an effort to make access to innovation financially sustainable. However, little empirical evidence is available to determine how they affect the selection of new technologies and unit prices. This paper focuses on medical devices (MDs) and investigates the combined effect of various HTA governance models and procurement practices on the two steps of the MD purchasing process (i.e., selecting the product and setting the unit price). Our analyses are based on primary data collected through a national survey of Italian public hospitals. The Italian National Health Service is an ideal case study because it is highly decentralized and because regions have adopted different HTA governance models (i.e., regional, hospital-based, double-level or no HTA), often in combination with centralized regional procurement programs. Hence, the Italian case allows us to test the impact of different combinations of HTA models and procurement programs in the various regions. The results show that regional HTA increases the probability of purchasing the costliest devices, whereas hospital-based HTA functions more like a cost-containment unit. Centralized regional procurement does not significantly affect MD selection and is associated with a reduction in the MD unit price: on average, hospitals located in regions with centralized procurement pay 10.1% less for the same product. Hospitals located in regions with active regional HTA programs pay higher prices for the same device (+23.2% for inexpensive products), whereas hospitals that have developed internal HTA programs pay 8.3% on average more for the same product

Implementation of value-based pricing for medicines

Value-based pricing (VBP) is well established in markets for common goods and services, but wide consensus on VBP for pharmaceuticals is lacking. In principle, VBP implies that prices are mainly driven by a drug's value (value for money) and that the impact on budget (sustainability) is a second-order driver of price regulation. Although the literature provides descriptive analyses on regulations governing medicine price negotiation, there are few insights on whether and how price negotiation regulations have been implemented. The goal of this article was to cover this information gap for 5 European countries and the United States. VBP has been applied according to two models: (1) direct models in which cost-effectiveness is a driver; and (2) indirect, multi-attribute models characterized by greater discretion on the integration between the different value domains and the evaluation of consistency between costs and value. In these models, cost-effectiveness is not a driver. In addition, it is hard to evaluate within these models the actual implementation of VBP. Identifying whether and how VBP is applied requires a clear predefined link between added value and the premium price, as well as transparency in the way added value is converted into a premium price. In general, for these countries, it remains difficult to determine whether pricing is mostly driven by value (value-for-money) or impact on budget (sustainability). In instances in which thresholds on the incremental cost-effectiveness ratio are used, it becomes easier to understand whether VBP has been implemented. If VBP relies on a multi criteria approach, greater transparency on which criteria have been used to assess a new drug and how they have been converted into a reasonable price may help in understanding whether a value-based approach has been used. (C) 2019 Elsevier Inc. All rights reserved

Cost per responder of Adalimumab biosimilars MSB11022 and ABP 501 versus the originator and methotrexate in chronic plaque psoriasis

Background: Pharmacoeconomic studies comparing the cost of adalimumab biosimilars versus the originator and conventional drugs in psoriasis are lacking. Research design and methods: To assess the cost per responder of adalimumab biosimilars versus the originator and methotrexate for psoriasis treatment. A cost per responder analysis comparing adalimumab biosimilars MSB11022 (Idacio®) and ABP 501 (Amgevita®), and methotrexate to the originator (Humira®) was performed. The incremental cost per responder was calculated by multiplying the cost of treatment based on the perspective of the National Healthcare System and number needed to treat for each therapy. Results: Considering the PASI75 response rate at 16 weeks, the cost per responder for MSB11022 and ABP 501 compared to the originator was € 500 versus 1,831 and € 968 versus 1,949, respectively. For the same endpoint, the cost per responder for subcutaneous or oral methotrexate was € 543 or 34 compared to 2,117 for adalimumab originator. At an indirect comparison among methotrexate, MSB11022 and ABP 501, the costs per PASI75 responder at week 16 were 2%, 26%, 27% and 50% of that of the originator, respectively. Conclusions: The use of biosimilars was confirmed as a valuable pharmacoeconomic strategy to lower healthcare cost in patients with psoriasis

Digitalizzazione nelle tecnologie per la salute: impatto sui livelli di governo del SSN

Il capitolo descrive il livello di digitalizzazione dei servizi sanitari analizzando in maniera critica il cambiamento avvenuto da pre a post pandemia da covid-19- Il capitolo riporta tutte le esperienze di digitalizzazione nelle regioni italiane con un focus specifico sull'aspetto della governance delle iniziative di digitalizzazione

Mobile health divide between clinicians and patients in cancer care: results from a cross-sectional international survey

Background: Mobile technologies are increasingly being used to manage chronic diseases, including cancer, with the promise of improving the efficiency and effectiveness of care. Among the myriad of mobile technologies in health care, we have seen an explosion of mobile apps. The rapid increase in digital health apps is not paralleled by a similar trend in usage statistics by clinicians and patients. Little is known about how much and in what ways mobile health (mHealth) apps are used by clinicians and patients for cancer care, what variables affect their use of mHealth, and what patients’ and clinicians’ expectations of mHealth apps are. Objective: This study aimed to describe the patient and clinician population that uses mHealth in cancer care and to provide recommendations to app developers and regulators to generally increase the use and efficacy of mHealth apps. Methods: Through a cross-sectional Web-based survey, we explored the current utilization rates of mHealth in cancer care and factors that explain the differences in utilization by patients and clinicians across the United States and 5 different countries in Europe. In addition, we conducted an international workshop with more than 100 stakeholders and a roundtable with key representatives of international organizations of clinicians and patients to solicit feedback on the survey results and develop insights into mHealth app development practices. Results: A total of 1033 patients and 1116 clinicians participated in the survey. The proportion of cancer patients using mHealth (294/1033, 28.46%) was far lower than that of clinicians (859/1116, 76.97%). Accounting for age and salary level, the marginal probabilities of use at means are still significantly different between the 2 groups and were 69.8% for clinicians and 38.7% for patients using the propensity score–based regression adjustment with weighting technique. Moreover, our analysis identified a gap between basic and advanced users, with a prevalent use for activities related to the automation of processes and the interaction with other individuals and a limited adoption for side-effect management and compliance monitoring in both groups. Conclusions: mHealth apps can provide access to clinical and economic data that are low cost, easy to access, and personalized. The benefits can go as far as increasing patients’ chances of overall survival. However, despite its potential, evidence on the actual use of mobile technologies in cancer care is not promising. If the promise of mHealth is to be fulfilled, clinician and patient usage rates will need to converge. Ideally, cancer apps should be designed in ways that strengthen the patient-physician relationship, ease physicians’ workload, be tested for validity and effectiveness, and fit the criteria for reimbursement

Social economic costs, health-related quality of life and disability in patients with Cri Du Chat syndrome

Cri du Chat syndrome (CdC) is a rare disease. The aim is to estimate economic costs related to CdC from a societal perspective, to assess the QoL and Disability in patients with CdC along with their caregivers in Italy