FDA and EMA to hold workshop on breakthrough therapy and PRIME designations

RAPS

2 August 2018 - The US FDA and 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 haematology. Meanwhile, a review of all therapeutics receiving a breakthrough designation in the US and approved from 2012 to 2017 found a lack of randomisation, double-blinding and control groups in pivotal trials supporting approval, a research letter published earlier this month in JAMA found.

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Michael Wonder

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Michael Wonder