Applications and authorisation procedures

A medicine is authorised for marketing through an application that runs through a regulatory procedure, and its authorisation is changed through further procedures once granted. The parts divide by the application or procedure the work serves: preparing a European marketing-authorisation application and the paediatric investigation plan it has to come with, the European procedures it can run through, the routes that speed access to medicines for unmet medical needs or authorise them in a public health emergency, the variations and reviews that change an authorisation once granted, and a US new drug application. What goes into the dossier is left to the dossier and its documents, the sponsor's exchanges with the agency, from scientific advice to answers to its questions in review, to dialogue with the agency, the legal bases and designations particular to one class of product, such as generics and orphan medicines, to product classes with their own requirements, the European network that makes and applies the rules to the regulatory framework, and the periodic safety reports and post-authorisation safety studies an authorised medicine owes to safety and pharmacovigilance.

• Marketing-authorisation applications — what an application is, deciding the legal basis early and checking patents and data exclusivity, the application form in Module 1, presenting and sending the dossier so a reviewer finds what the decision rests on, the guidelines and standards it meets, and a final checklist
• Paediatric investigation plans and waivers — what the plan is and why a European application has to come with one, the Paediatric Committee that decides on it, what it covers and when it is submitted, waivers, class waivers and deferrals, the guidance, forms, structure, team and timeline for preparing it, the background, strategy and justifications the application gives, frequent mistakes and completing the package, the procedure with the committee and the check of compliance, the US paediatric study plan, and the regulation's costs and results
• European authorisation procedures — the centralised, mutual recognition, decentralised and national procedures and what decides among them, how half a century of harmonisation produced them, the electronic application forms, the agency's opinions on medicines for use outside the EU, and assessments of generic applications shared with regulators outside the EU
• Early access routes to a European marketing authorisation — accelerated assessment, conditional marketing authorisation, authorisation under exceptional circumstances and adaptive pathways, with medicines assessed on an accelerated timetable and authorised conditionally, a recommendation to revoke a conditional authorisation after its confirmatory study failed, and the agency's pilot of adaptive pathways
• Authorising medicines in a public health emergency — the European agency's rapid procedures for COVID-19 medicines and its opinions before marketing authorisation, US emergency use authorisations, the regulators' decisions compared, adapting vaccines to variants, and the winding down of the emergency flexibilities and the lessons learned
• Variations and post-approval procedures — why an authorisation is varied, the variation types and the new variations framework, notification of type I variations, the start dates of type II assessments, who at the agency handles a procedure, reviews that narrow an indication and extensions of one, and the particulars and sites a holder needs in the European Economic Area after the United Kingdom's withdrawal
• US new drug applications — the application and its classification code, the FDA's review of it and the drugs it approves, the expedited pathways to review and approval, and the FDA's review times under the user fee act against those of other agencies