High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study.

Background: in the field of rare diseases (RDs) most of the European studies on budget impact analysis of drugs that have been conducted often lay on theoretical assumptions and focus only on Orphan drugs (ODs). Objectives: we aimed to estimate the budget impact of specific drugs for non-oncological RDs, both ODs and non-ODs, using real-world data about patients residing in Veneto Region (Italy) and to describe its expenditure structure and dynamics. Methods: a population-based multi-source observational study was conducted using data from Regional administrative databases; an ad-hoc drugs' list specific for RDs including both ODs and non-ODs and classifying them by ATC codes has been created. Results: In 2019, the total expenditure for drugs specific for RDs was EUR 97.2 million (6.6% of the total Regional budget). The RD drug list included 58 ATC codes, of which 15 ATC had an annual budget impact over EUR 1 million ("blockbuster drugs"). The most expensive treatment was a non-OD drug (Coagulation factor VIII). The two most represented therapeutical areas were the metabolic and the hematological ones. Conclusions: Cost analyses on RD high-cost drugs expenditure should consider any specific RD drug, not only ODs. Expenditure dynamics for RD drugs are peculiar showing "blockbuster drugs". Some therapeutical areas seem to be lacking in the drug research field.

Virtual hospitals: The future of the healthcare system? An expert consensus.

Today, social and healthcare systems at a global level are facing constant challenges dictated by an increasing mismatch between the demand for care services and the supply of human and economic resources. Such a situation has been exacerbated in the past two years by the Covid-19 pandemic. This has led to an increase in the leverage of digitalisation, which has proved to be a crucial tool for the development and application of new organisational models at both hospital and territorial levels, thus addressing the various criticalities already present in the system. In this sense, the Virtual Hospital has emerged as a potential model for increasing effectiveness and efficiency in delivering sociomedical services. Starting from these premises, an EFTE (estimate, feedback, talk, estimate) approach was used to acquire an expert consensus within a multidisciplinary panel of academics and healthcare managers of the Veneto Region in Italy. This article reports the expert opinion on the possible application of the Virtual Hospital model in the national context, starting from the existing international evidence and good practices, highlighting the potential advantages and barriers to its implementation. Furthermore, the article analyses the most relevant areas of investment for the development of intangible assets and the acquisition of tangible assets necessary for its implementation.

Preparing healthcare, academic institutions, and notified bodies for their involvement in the innovation of medical devices under the new European regulation.

INTRODUCTION: Favoring innovation by making timely medical technology available to people and by securing patients' safety is a challenge. AREAS COVERED: The new European Medical Device Regulation (MDR) will have a central implication in the development of new devices and could affect their innovation and availability, as well as discourage investment in research within Europe. EXPERT OPINION: Start-ups and small companies might not be able to cope with the increasing complexity and the required changes of perspective. Health-care institutions are facing an increasing availability of complex technologies, while data on their clinical efficacy and cost-effectiveness are rarely provided. A partnership/collaboration between health-care institutions, academia, and private industries will enhance their own specific interests with the common goal of improving overall health and quality of life. The complexity of the subject combined with the variety of specialists and stakeholders involved requires the implementation, in hospital centers of clinical excellence, of units dedicated to the whole path of the medical device innovation. Stakeholders should quickly provide adequate measures to facilitate the complex medical device innovation path under the more stringent MDR aimed to increase safety and quality of care.