What is the Future of Rare Disease Research and Development in the EU?

Rare diseases are a major public health issue in the European Union (EU), affecting an estimated 30 million people, or between 6% and 8% of the population. (1)
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Valtermed: The Spanish National Health System’s information system that collects real world evidence on the therapeutic value of new therapeutics
In recent years, new pharmaceutical policies have been implemented in the Spanish National Health System (NHS) aiming to restrain pharmaceutical spending and improve transparency in healthcare decision making. Among them, there has been an increase in the use of performance-based agreements at national and regional level, as well as the development of the Valtermed program.
More price negotiations on new expensive drugs in the Netherlands
On the 24th of January 2023, the Dutch Minister of Health, Welfare and Sport Ernst Kuipers has decided to adapt the rules for admission of expensive (intramural) medicines to the basic health insurance package as from 1st July 2023. He is doing this by tightening the so-called lock criteria.
The AIFA’s innovativeness appraisal for medicines
A standardized procedure to evaluate the innovativeness of medicines has been implemented by the Italian Medicines Agency since 2017 (AIFA - Determina n. 1535/2017).
How to anticipate the 2025 EU HTA from now?
The European (EU) Regulation on Health Technology Assessment (HTA) came into force in January 2022 and will apply from January 2025 starting with oncology medicines and Advanced Therapy Medicinal Products (ATMPs). The three-year application period aims to set up the organizational framework and methodological guidelines of the common assessment and, to give health technology developers time to anticipate the requirements of the new framework.