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FDA and EMA to Hold Workshop on Breakthrough and PRIME Designations

Posted 31 July 2018 | By Zachary Brennan 

FDA and EMA to Hold Workshop on Breakthrough and PRIME Designations

The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) will hold a workshop on early access approaches, including PRIME and breakthrough designations, on 26 November at the EMA headquarters in London.
EMA said the aim of the workshop is for regulators and industry to discuss “technical quality challenges and scientific and regulatory approaches that could be used to facilitate development and preparation of robust CMC [chemistry, manufacturing and control] data packages,” as part of these expedited programs. 
Since the PRIME designation launched in March 2016, EMA says it has granted eligibility to 36 programs, 30 of which are for rare diseases and 19 of which are in oncology or hematology. Meanwhile, a review of all therapeutics receiving a breakthrough designation in the US and approved from 2012 to 2017 found a lack of randomization, double-blinding and control groups in pivotal trials supporting approval, a research letter published earlier this month in JAMA found.
“Experience to date has shown that applicants face challenges to complete quality and manufacturing development and data requirements during accelerated development,” FDA said. “In order to address/overcome these challenges regulators wish to support applicants with guidance and risk-based flexibility regarding their pharmaceutical development program.”
The workshop will include specific industry case studies covering chemical molecules, biologicals and advanced therapy medicinal products (ATMPs) and a discussion of experiences to date from early access approaches.
Those interested in participating are invited to register by sending an email to by 31 October. As the number of spaces is limited, EMA will allocate places per stakeholder group to allow attendance of a wide range of stakeholders. The conclusions from the workshop will be captured in a report that will be published.
“The development of further follow-up guidance may be considered,” FDA said.
Workshop with stakeholders on support to quality development in early access approaches (i.e. PRIME, Breakthrough Therapies)

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