The Cell and Gene Therapy Catapult hosted a daylong workshop on “Optimising the design of clinical trials for advanced therapies” on the 4th of September. The event was co-organised by the Advanced Therapy Treatment Centre network, the Association of the British Pharmaceutical Industry, UK Bioindustry Association and Cell and Gene Therapy Catapult In partnership with the Advanced Therapies Network and London Advanced Therapies. Approximately eighty guests attended drawn from the National Health Service, academia, industry and government regulatory agencies
The event consisted of talks, panel discussions and a breakout session to present multiple aspects of the design and execution clinical trials in this complex landscape. Major messages from the day were:
- To engage early with competent authorities early, especially regarding innovative trial designs early. The authorities are willing to guide companies before a trial design is formally locked down
- Establish interdisciplinary teams within facilities to improve the execution of clinical studies
- Include health related quality of life measures in early phase studies to build data for reimbursement.
The importance of collaboration was also underscored as many trials with advanced therapies will have smaller patient cohorts. This is partially due to many candidate therapies addressing rare conditions. Researchers will need to focus on the similarities of administration and patient care between these studies to inform risk benefit profiles of advanced therapies. More information will be available on the ATTC website in the near future.