First results of a study into factors associated with successful development of Advanced Therapy Medicinal Products (ATMPs) were reported today by Utrecht University at European Biopharmaceutical Enterprises’ (EBE) 6th Annual Regulatory Conference in London. The study, carried out via the Escher platform in the Netherlands and supported by an unrestricted grant by EFPIA and EBE has highlighted the challenges faced by developers of ground breaking new treatments such as gene and cell therapies and tissue engineering. 

Although this new group of innovative treatments offers patients across a range of conditions new hope in tackling their disease, innovators face significant challenges in realising their potential. 

EBE Executive Director Barbara Freischem said: “Developing ATMPs is incredibly challenging. This new study explores and, for the first time quantifies these challenges. It provides an important evidence base to develop policy in Europe that can deliver these potentially life-saving therapies to patients faster and more safely.” 

The study highlights a number of challenges from regulatory hurdles to clinical trial design and uncertainties related to market access.  It underlines the disparity between European-wide and national regulation, complexity involved in manufacturing and the need to develop new health technology assessment and reimbursement models. 

The study calls for greater collaboration between innovators, regulators, HTA bodies and payers to reduce the uncertainty and complexity in delivering ATMPs to patients.  

Nathalie Moll, Director General of EFPIA, concluded ”Europe was a first mover in the field of ATMP legislation. The European Commission and the European Medicines Agency engage in improving the implementation of the European legislation. The study shows that further action is now necessary to ensure that healthcare systems and patients ultimately can have access to these incredibly promising new therapies. We look forward to working collaboratively with stakeholders to that end.”

About EBE
The European Biopharmaceutical Enterprises (EBE) represents the voice of biopharmaceutical companies of all sizes in Europe and is a specialised group within the European Federation of Pharmaceutical Industries and Associations (EFPIA). Established in 2000, EBE is recognised as the leading biopharmaceutical association in Europe. To learn more about EBE, visit www.ebe-biopharma.eu

About EFPIA
The European Federation of Pharmaceutical Industries and Associations (EFPIA) represents the pharmaceutical industry operating in Europe. EFPIA is the voice on the EU scene of 1,900 companies committed to researching, developing and bringing to patients new medicines that will improve health and the quality of life around the world. To learn more about EFPIA, visit: www.efpia.eu

Media Contact
Barbara Freischem, Executive Director
European Biopharmaceutical Enterprises – EBE
Tel: +32 2 626 25 64
email: barbara@ebe-biopharma.org