Tofacitinib immediate release (IR) formulation has been approved in both adult rheumatoid arthritis (RA) and pediatric juvenile idiopathic arthritis (JIA) patients. The modified release (MR) formulation has also been approved in RA patients.
In this presentation, a model-informed drug development strategy leveraging translational analysis to support the extrapolation of MR formulation approval from adult to pediatrics without conducting any pediatric trial is discussed. In this approach, prior knowledge across formulations/populations were integrated using a wide range of tools including PBPK, population PK modeling, exposure-response modeling as well extrapolation analysis. Upon establishment of translation between formulations in RA and between populations using IR formulation, an efficient development plan which doesn’t require any pediatric trial is proposed. The proposed plan was also adapted to the relevant regulatory guidance. In the end, the regulatory feedback from both FDA and EMA on this proposal as well as lessons learned will be shared.
Learning Objectives:
Upon completion, participant will be able to apply similar translational analysis to accelerate their own formulation development.
Upon completion, participant will appreciate the potential regulatory hurdles in applying study waivers through this case study.
Upon completion, participant will appreciate the ability to combine muliple modeling approaches in applying for study waivers.