E-POSTERS - OUTCOME MEASURES: EP.51Expert advice to improve clinical trials: from TACT to ACT

Abstract

The TREAT-NMD Advisory Committee for Therapeutics (TACT), established in 2009, is a unique multi-disciplinary international group of academic and industry drug development experts. TACT meets twice a year to review and give advice on the translational and development pathway of therapeutic programs in rare neuromuscular diseases. Due to the success of TACT, we are now replicating the model for paediatric diseases, as part of the IMI2 project - Conect4Children (c4c). An ACT 'tool-kit' will be produced to support other disease networks in establishing their own advisory committees for therapeutics (ACTs). This will be disseminated through the European Joint Programme co-fund for rare diseases (EJP). We aim to replicate the TACT model in other, paediatric disease areas, to provide world-leading, expert advice and support to researchers and industry on translation from pre-clinical data to therapy approval, including clinical trial design. We will be producing a 10th anniversary paper evaluating the impact of previous TACT applications over the past ten years. We will support the set-up of large multi-stakeholder meetings, which include a wide range of drug development experts including clinicians, regulatory experts, academics, statisticians, toxicologists and patient representatives. We plan to create an ACT 'tool-kit' to allow other disease networks to set-up their own therapeutic advisory committees. Key findings: Feedback from previous applicants in the neuromuscular field has demonstrated that TACT has helped them to prepare for discussions with regulators, inform study design - including selection of endpoints and go/no-go decisions, enrolment and dosing decisions. TACT has reviewed over 50 applications in 9 years from academic groups and industry. Replicating the TACT model in other disease areas, particularly paediatric areas, will support better clinical trial design and guidance through the translational research pipeline. This will have a significant impact on patients.