The revised pharmaceutical legislation
In April 2023, the European Union (EU) undertook a comprehensive evaluation of the pharmaceutical legislation. It adopted a proposal for a new pharmaceutical Directive (2023/0132) and a new Regulation (2023/0131), which revise and replace the existing general pharmaceutical legislation (Directive 2001/83/EC and Regulation (EC) No 726/2004). This evaluation also included a proposal to repeal the existing Orphan Medicinal Products Regulation (EC) No 141/2000 and the Paediatric Regulation (EC) No 1901/2006, and to identify strengths and areas for improvement in a new regulation.
This white paper discusses how the revised pharmaceutical legislation will affect:
- Scientific Advice procedures
- Orphan Drug Designation and maintenance
- Paediatric Investigation Plans (PIPs)
- Expedited EU pathways (PRIME)
- Marketing Authorisation Application preparation
Here is a snippet of some of the topics we cover in the whitepaper…
Orphan-designated medicines
The proposed pharmaceutical legislation introduces important changes for orphan-designated medicines. While the current prevalence threshold for orphan designation remains unchanged, prevalence can be adjusted for certain diseases. It also introduces breakthrough orphan medicinal products.
The duration of market exclusivity for orphan-designated medicines will also be updated.
Priority Medicines (PRIME) scheme
The PRIME scheme supports medicines targeting unmet medical needs through early regulatory interaction and accelerated assessment opportunities. The reforms propose enhanced support for innovative therapies, orphan medicines, and priority antimicrobials addressing antimicrobial resistance.
Paediatric medicine development
Paediatric medicine development will continue to require an agreed PIP or waiver before submitting an MAA. Procedural changes are proposed, including shifting responsibility for PIP review under the Committee for Medicinal Products for Human Use (CHMP) and providing free scientific advice on paediatric study design.
The Marketing Authorisation Application (MAA)
The pharmaceutical legislation also aims to streamline the MAA process through consolidated EMA committees, a simplified centralised procedure, and reduced evaluation timelines for new medicines.
Regulatory data and market protection
The regulatory data and market protection framework will become more conditional, with extension opportunities linked to unmet medical need, EU-wide launches, and additional indications.
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