EFPIA-EBE White Paper: Expedited CMC Development- Accelerated Access for Medicines of Unmet Medical Need
Since 2012 there has been a focus on faster translation of scientific breakthroughs to new, high quality medicines meeting unmet medical need through a programme initially referred to as Medicines Adaptive Pathways to Patients (MAPPs). In response to this in 2016 EMA introduced two new regulatory approaches; namely Adaptive Pathways (AP) and Priority Medicines (PRIME). At present, there is limited mention of CMC aspects for those accelerated access approaches in the public domain and this EFPIA-EBE White Paper seeks to outline options for the acceleration of CMC development that may be acceptable to Regulatory Authorities for medicines exhibiting the potential to meet an unmet medical need without compromising their quality or safety.
Underlying principles and illustrative examples of CMC approaches to development and manufacturing which a company may undertake to facilitate accelerated review or early access are described. A number of important regulatory considerations are also outlined and some initial considerations to support accelerated and novel approaches to CMC development are presented. Those suggestions must be viewed on a case by case basis and are in no way construed to represent a guarantee of accelerated review or early access. It is strongly suggested that sponsor companies wishing to adopt these approaches engage with the EMA through Scientific Advice as early as possible and that the Agencies reciprocate by enabling timely access to, and involvement of, the necessary technical experts to promote those discussions.
Consideration is given to the challenges posed by the accelerated development of both small molecules and large biopharmaceuticals. Detailed suggestions and a comparative assessment contrasting with conventional development activities are presented in two additional annexes with the aim of promoting further industry / regulatory collaborative development.
Read here the EFPIA-EBE White Paper
*It should be noted that the opinions shared in this document are the opinions of the EFPIA/EBE Trade Associations and should not be construed as defining any regulatory positions of the EMA. It is stressed that companies are urged to seek appropriate advice from competent authorities for specific application of any CMC expedited approaches.