“From discovery to access – how can we ensure Europe is at the forefront of innovation and access delivery of gene and cell therapies?”

By SME Europe

On March 5th SME Europe, in cooperation with SME Connect and the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), hosted a Working Breakfast on ground-breaking treatments with gene & cell therapies. The event took place in the Member’s Restaurant of the European Parliament.

Dr. Paul Rübig MEP, Honorary President SME Europe of the EPP

The Breakfast was hosted by Dr. Paul Rübig MEP (Vice-Chair of Science and Technology Options Assessment, President of SME Connect and Honorary President of SME Europe).

Other speakers were Alexander Natz (Secretary General of EUCOPE), Irene Norstedt (Acting Director for Directorate Health and Head of Unit Innovative & Personalised Medicine at DG RTD, European Commission), Maria Kelleher (Director of market Planning at BioMarin), Karolina Hanslik (Senior Project Manager at EURORDIS – Rare Diseases Europe) 

The speakers mentioned the need for new and innovative payment models for so called once-and-done clinical treatments such as gene & cell therapy. Other points on the agenda were barriers between research and healthcare, opportunities to harmonise clinical approval pathways and the challenges that make access to special treatment of rare diseases difficult or even impossible.

“The areas of application of gene and cell therapy are cancer, haemophilia, muscular atrophy, SCID”

There might be only ten centres for gene & cell therapy in Europe. The Cross-Border-Healthcare-Directive (2011) was a good starting point for patients to access urgently needed treatments across the member states but it is time for more action. 

“It is our responsibility as the Commission but ultimately of course it’s also the responsibility of the member states to make sure that academic research outcomes are translated into official medical practices”

– Irene Norstedt, DG RTD European Commission

From the point of view of SMEs, Europe has become a favourable regulatory environment to accomodate innovative medical treatments. They are not always complex to administer and  there is opportunity for joint HTA assessments as well as exelareted assessments. It is also possible to mitigate short term fiscal arrangements through real-world evidence data collection and also to seize savings with once-and-done treatments.

“Europe has become a favourable regulatory environment with such initiative as the “Prime Programme”

Maria Kelleher, Director of market Planning BioMarin

The Commission will continue its funding in 2020 so that the competetiveness of the European pharmaceutical industry can remain. But in the end the main focus is on the patient. As Irene Norstedt from the European Commission stated in her speech: „ We often forget that there are real people on the other side of discussions about money, investments and projects we are funding.“