New regulatory POC model

Question 1

Point of care POC – a new regulatory model

Answer 1

• In 2021 EU pharmaceutical law ended and UK law came into effect
• MHRA: Opportunity to develop new regulatory framework for POC manufacture

Question 2

Identified issues with current manufacture process

Answer 2

• Short shelf life
• Highly personalised products
• Large number of manufacturing sites
• Wide range of manufacturing locations
• Wide range of product types
• Main issue – maintaining quality drug control of drug manufactured at large number of sites

Question 3

Proposed manufacturing model

Answer 3

To have a central control site
• Holder of manufacturing license
• Responsible for qualified person certification and release
• Provide oversight of clinical trial sites

At the POC manufacturing sites
• Local manufacture of drug and release
• Authorised person
• Transmit manufacturing data back to central control siite
• So don’t need a qualified person and license at each site

Question 4

Standard marketing authorisation pathway

Answer 4

Approval based
 Treatment effectiveness
 Good strength and purity of drugs
 Safety + benefits

Pathway goes through each stage
• Safety, quality and efficacy
• Marketing authorisation phase 4

Question 5

Accelerated pathways for orphan drugs

Answer 5

• Marketing authorisation submitted earlier on
• Comprehensive data
• Approval earlier on, after phase I/II

Question 6

2 types of accelerated pathway

Answer 6

Conditional marketing authorisation
• Data shows positive benefit risk balance pending further confirmation
• Authorisation valid for 1 year – renewal basis
• Once data from pending studies provided – becomes standard marketing authorisation

Marketing authorisation under exceptional circumstances
• Comprehensive data can’t be provided
• Reviewed annually to assess risk benefit balance
• Will not normally lead to full application
• And will not gain standard marketing authorisation