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.

Patient-centric treatments such as gene and cell therapies have been a game-changer in tackling hematological disorders. Nonetheless, their development remains slow and erratic, limiting benefit for patients.

Learning how to recognize and treat a disease that is relatively new to many parts of Europe, and spreading, is crucial to limit, if not avoid, irreversible impact and provide proper care to patients.

Gathering clinical, genetic and molecular information into a single database: this is the challenge taken on by the HARMONY Alliance. By bringing together data currently scattered across different clinical trial databases and registries, the HARMONY Alliance is harnessing the enormous potential of Big Data and Big Data analytics to deliver insights that will help improve the care of blood cancer patients.

The European Commission (EC) launched a new online knowledge-sharing platform – the European Platform on Rare Disease Registration (EU RD Platform) – on February 28, 2019.

Advanced therapies, such as gene therapy, hold promise for treating a wide range of chronic diseases and improving patients’ quality of life. As promising as these treatments are, they remain out of reach for many. 

Gene (editing) therapy continues to break ground in hematology, showing promising results in tackling severe blood cancers.

Every European citizen should have access to the best quality medical care at the best possible price. Pan-European cooperation on health technology assessments (HTA) is essential for such aspirations.

Joint Actions are projects designed and financed by Member State Authorities and the EU to address specific priorities under the EU Health Program.

EHA welcomes initiatives but calls for more focus on causes

Medicine shortages have a negative impact on the quality and cost of treatments and on patient access to the best possible care. EHA has helped flag the problem with policymakers and regulators, but lack of data and understanding of the causes make it notoriously difficult to tackle.

Precision medicine approaches the treatment of a disease via an individual’s genes, environment, and lifestyle.