EMA’s PRIority MEdicines (PRIME) scheme and FDA’s breakthrough therapy (BT) designation program are designed to help speed development of innovative products which address unmet medical needs.
For products included in these expedited development programs, the marketing application is still expected to include all the clinical, non-clinical, and chemistry, manufacturing, and control (CMC) information to meet approval standards. Because generating CMC information on more compressed
timelines can present challenges for companies, EMA and FDA have been engaging in open dialogue with industry stakeholders in order to explore approaches to expedite the development and approval of these products without lowering the standards that patients have come to expect in a medicine.
To this end, on 26 November 2018, EMA and FDA organized a stakeholder workshop on quality development in early access approaches, such as PRIME and Breakthrough Therapies. This workshop focused on potential scientific and regulatory approaches to address challenges associated with expedited product development, so that robust quality and manufacturing data packages will be submitted to enable timely access to medicines for patients whilst assuring that product safety, efficacy, and quality will not be compromised.
During the workshop, challenges and solutions were explored by a combination of real case studies from industry [covering chemical molecules, biologicals, and advanced therapy medicinal products (ATMPs)] and regulators’ perspectives and panel discussions.
Based on the experience with PRIME and BT programs, regulators and industry selected the following areas for discussion: process validation, control strategy, compliance with Current Good Manufacturing Practice (GMP) requirements, comparability, stability and regulatory tools.
The discussions and main conclusions from the workshop, including scientific elements and regulatory tools which already exist or which would benefit from exploration, to help address development challenges, were captured in a meeting report. During the workshop, FDA and EMA also reflected on areas that would benefit from further discussion between both regions and identified the following topics: control strategy, innovative process validation approaches, stability data, and launching from the clinical manufacturing site or with investigational medicinal product batches.
EMA–FDA joint Q&As on Quality and GMP aspects of PRIME/Breakthrough therapy applications