EPHA published a position paper on medicine shortages in Europe. EHA welcomes its  recommendations to address the increasing shortages crisis, which threatens patient outcomes and patient safety.

Advanced therapy medicinal products (ATMPs) are a game changer for improving the lives of patients with severe to life-threatening diseases. Yet, despite fast-paced innovation in the field, few ATMPs have reached the EU market and benefited patients. The challenges facing their adoption by health systems are complex. A test case is the uptake of CAR T-cell therapy; what are the hurdles and what action is needed from stakeholders?

While health policy remains the primary responsibility of Member States, in many areas the added value of EU-level collaboration and harmonization has increased. EU initiatives in health have shown a mixed record in terms of impact, but hopes are high for the latest one, the ambitious Europe’s Beating Cancer Plan.

Clinical trials and drug development have become more complex over the years. One complicating factor is the increased administrative burden, as was pointed out in the previous edition of HemAffairs. Doctors and clinical researchers are more and more bound to rules they deem neither necessary nor effective. At first sight, the requirements for clinical trials seem clear and thorough. Nonetheless, the interpretation and implementation of these rules leave room for improvement.

Blood, tissues and cells (BTC) are used in medicine and in hematology on a daily basis. EU legislation has been in place for over 15 years to regulate those practices, and to ensure safety and quality standards for substances of human origin (SoHO). For instance, through technical and traceability requirements for BTC, the reporting of serious adverse reactions (SARs), and verification procedures for imported BTC.

To solicit input from a wide range of non-ICH members about the revision of ICH E8(R1), ICH held a Public Meeting on ICH E8(R1) “General Considerations for Clinical Studies” (Silver Spring, USA, October 31, 2019).

On September 24, Prof Ulrich Jäger, former EHA President and current member of the EHA European Affairs Committee, was elected as co-chair of the Health Care Professionals Working Party (HCPWP) of the European Medicines Agency (EMA). He will lead the working party during its 2019-2022 mandate alongside Juan Garcia-Burgos, EMA’s head of Public Engagement.

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.

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.

EHA has given its formal endorsement to the “Manifesto for a new approach for better medicine in Europe: Establishing Treatment Optimization as part of personalized medicine development”.