Europe – Quality data packages in early access approaches : workshop report published

Supporting medicine developers in generating quality data packages in early access approaches (PRIME and breakthrough therapies)

EMA and the US Food and Drug Administration (FDA) have published today a  
 on their joint workshop with stakeholdersheld on 26 November 2018. The aim of this workshop was to discuss scientific and regulatory approaches to address quality and manufacturing challenges encountered during the development of medicines under early access programmes, such as the PRIority MEdicines scheme (PRIME) in the European Union and the Breakthrough Therapy designation in the United States. The report contains recommendations from participants on next steps and areas to be further explored by EMA and the FDA.

“With PRIME, we have established a platform that supports the development of promising medicines to help patients with unmet medical needs to benefit from these as early as possible,” said EMA’s Head of Human Medicines Research and Development Support Division, Enrica Alteri. “Some challenges, for instance to complete quality and manufacturing development and data requirements are universal to early access programs. We have therefore teamed up with our partners at the FDA to organise this workshop and discuss together with pharmaceutical companies possible scientific and regulatory approaches that facilitate the preparation of robust quality data packages. This is the basis which enables timely access to medicines for patients and gives the necessary assurance that patient safety, efficacy and product quality are not compromised. We will continue to cooperate with our FDA colleagues to further facilitate the successful development and authorisation of priority medicines.”…