Draft toolbox guidance published for users of EMA’s PRIME scheme

The European Medicines Agency (EMA) has released a draft version for consultation of its toolbox guidance for its Priority Medicines (PRIME) scheme.

The PRIME scheme was setup to accelerate the authorisation of medicines that target an unmet medical need. The draft toolbox guidance aims to support European Union (EU) marketing authorisation applications (MAAs) for developers of PRIME designated medicines by summarising the scientific and regulatory tools that they should use. Specifically, the guidance focuses on how these resources can be used to quickly and effectively complete Module 3 quality data packages.

Scientific tools

Initially, the guidance describes the availability of general scientific tools. Prior knowledge is the first such tool and relates to the use of pre-existing knowledge. This could include company knowledge from developing and manufacturing approved products with similar compounds for instance. Another general tool relates to risk assessment and the importance of it in managing the higher levels of risk needed to approve products for unmet clinical needs.

Process validation protocols are another category of scientific tools described in the guidance. It outlines when these protocols can be accepted as substitutes for final validation reports as another way to accelerate the assessment process. It is noted that validation related activities that accelerate the normal application process will often only happen following a case specific analysis.

Scientific tools related to control strategy is another area outlined in the guidance. For applicants with a detailed knowledge of their product and process, they can be more flexible with their approved controlled strategy. However, when this isn’t the case, the EMA notes that some process development and evaluation studies may be deferred to the post-approval phase (following a risk/benefit analysis). In this way control strategy can be implemented in a way to facilitate accelerated patient access to medicines.

Advice on good manufacturing practice (GMP) compliance in the context of accelerated assessment procedures stresses the importance of maintaining quality whilst being flexible. For instance, there may be a scenario where one can launch from an investigational medicinal product site. However, this can only be done following an adequate level of GMP compliance is maintained (as determined by an assessment).

Further scientific tools detailed in the document relate to stability testing and comparability studies.

Regulatory tools

This section of the guidance describes the EMA’s aim to establish an early dialogue between regulators and manufacturers in order to support prospective planning.

The first regulatory tool relates to accelerated assessment. This is a procedure intended to shorten the active review time of a marketing authorisation application from 210 to 150 days. A conditional marketing authorisation (CMA) is another regulatory that can be utilised. It essentially allows a marketing authorisation to be granted before comprehensive clinical data has been submitted on the condition that a risk/benefit analysis has been conducted and is considered positive. Other regulatory tools include post-approval change management protocols (PACMPs) and post-authorisation measures (PAMs).

This draft guidance is now open for public consultation until 31 July 2021.

To read the draft toolbox guidance in full, click here.