There have been many developments on rare diseases since the EU Orphan Medicinal Products Regulation came into force in 2000. As the European Commission is evaluating its effectiveness, EHA discussed with Prof. Giampaolo Merlini, Director of the Amyloidosis Research and Treatment Center and researcher at the Department of Molecular Medicine of the University of Pavia, how far we have come and what still needs to be done to improve access to treatment for rare diseases.

The European Cooperation in Science and Technology (COST) is an EU-funded, intergovernmental framework that aims to create pan-European research networks in all science fields and promote excellence, foster interdisciplinary research and empower young researchers and innovators.

Research and academic groundwork funded with public means should be free and open to everyone. That is the principle behind Open Access that was formulated in 2003 in the Berlin Declaration. More than a decade has passed since and for years no significant action was taken. Scientific research, paid for by the taxpayer, is still mostly locked behind paywalls.

EHA has recently brought key stakeholders around the table to discuss bureaucratic obstacles in clinical research. Regulators, patient organizations, the European Commission and industry representatives were invited for a day-long discussion at EHA offices, building on an informal dialogue with the European Medicines Agency (EMA) that had been initiated by a group of prominent clinical researchers.

In recent weeks, two Calls for proposals were published that offer relevant funding opportunities for hematologists: IMI2 – 18^th Call for proposals, a jointly funded partnership between the European Union, and Horizon 2020 – Call for cancer research proposals, call for research proposals in the category ‘Societal Challenge 1 (SC1): Health, demographic change and wellbeing’. . 

In 2014 the European Parliament approved the Clinical Trials Regulation (CTR) that is supposed to replace the Clinical Trials Directive (CTD) from 2001. Five years later, the regulation has not yet become applicable. What is the aim of the ‘new’ regulation, what issues does it address, and why was its application delayed?

2019 will see significant changes within the European institutions. The first already took place in late May, as 50% of EU citizens – the highest turnout since 1994 – went to the ballot box to elect their Members of the European Parliament (MEPs). The result: Confirmation that nationalism and Euroscepticism are on the rise, and a set-back of center political parties.