EMA claims development of ATMPs is booming
But there are challenges impeding their production
The Chair of the European Medicine Agency’s (EMA) committee of advanced therapies (CAT) has claimed a sharp increase in the development of advanced therapy medicinal products (ATMPs).
Paula Salmikangas, speaking at a European Biopharmaceutical Enterprises (EBE1), EMA and Italian government-run workshop, held in London, said there had been 'major growth' in the development of such medicines. EMA ATMP classifications now number 117; in addition, 200 cases of requested scientific advice and 40 sets of advice on ATMP paediatric investigation plans were communicated.'There are currently an estimated 250 ongoing clinical trials with ATMPs in Europe, most of which are still in early phases of development and with more than half originating from non-commercial organisations (hospitals and academia) and micro, small and medium-sized enterprises (SMEs),' she said.
The workshop entitled ‘Development Pathways for Advanced Therapy Medicinal Products’ discussed scientific advances in cell and gene therapies and their potential impact on public health. It heard how ATMPs offer groundbreaking treatment, including the use of stem cells, gene therapy and tissue engineering.
One size does not fit all approach needs to be borne in mind
Salmikangas acknowledged that while development was booming, only four ATMPs have received central EU authorisation by EMA since the European Union’s (EU) ATMP Regulation came into force in 2009. Five evaluations are currently ongoing and four applications for marketing authorisations have been withdrawn.
Challenges impeding ATMP production include manufacturing (e.g. good manufacturing practice (GMP) compliance, characterisation, development and availability of validated potency assays); pre-clinical testing (e.g. a dearth of relevant animal models, and proof of concept difficulties); and clinical development (e.g. problems related to trial methodology, blinding, comparator, feasibility of dose finding studies and biodistribution studies).
Other challenges include patient access, affordability, and healthcare system diversity, notably regarding payments.
Workshop participants discussed business development models for ATMP and current regulations and Salmikangas stressed that a ‘one size does not fit all’ approach needs to be borne in mind, while inviting participants to work together to support the delivery and early patient access to ATMPs.
