On 26 April 2023, the European Commission (EC) proposed reforming the European Union (EU) pharmaceutical legislation. This revision constitutes the first major overhaul of the pharmaceutical legislation since 2004. It will adapt the legislation to the needs of the 21st century.
The reform includes two legislative proposals: a new Directive and a new Regulation. The new Directive will amend and replace the current Directive on medicinal products for human use (Directive 2001/83/EC) and will incorporate and amend relevant parts of the Paediatric Regulation (Regulation (EC) 1901/2006). The new Regulation will amend and replace the Regulation on the European Medicines Agency (Regulation (EC) 726/2004), the EU Regulation on Orphan Medicinal Products (Regulation (EC) 141/2000) and the Paediatric Regulation (Regulation (EC) 1901/2006).
The reform also includes a Council Recommendation on antimicrobial resistance (AMR).
The European Parliament adopted its position on the proposal at first reading on 10 April 2024, but the EU Council has not yet found a common position due to some controversial topics. Depending on the progress of the negotiations, an agreement between the Member States could be reached in the first half of 2025, however, it is still difficult to predict the timing of legislation adoption at this stage.
The EU pharmaceutical reform aims to achieve the following 6 main objectives:
1. Create a single market for medicines ensuring that all patients across the EU have timely and equitable access to safe, effective, and affordable medicines
Currently access to medicines across EU Member States is not timely, and not equally accessible – there is up to 90% variance in accessibility between Northern and Western European countries and Southern and Eastern European countries. Average waiting time for access across the EU is 4-29 months.
New incentives would encourage companies to make their medicines available to patients in all EU countries and to develop products that address unmet medical needs. Currently, there is “a one-size-fits-all” system that grants all innovative medicines 8 years of data protection and 2 years of market protection. To create a single market for medicines, the reform would move the current system towards a more targeted approach aimed at achieving public health objectives, i.e., 6 years of standard data protection and 2 years of market protection, plus conditional extensions, such as + 6 months if a medicine addresses an “unmet medical need”; + 6 months if the applicant has carried out comparative clinical trials; + 2 years if a medicine is available in all Member States where the marketing authorisation is valid within 2 years after authorisation (3 years for small and medium-sized enterprises [SMEs] and not-for-profit entities); + 1 year for any additional therapeutic indications.
The current maximum regulatory protection (data and market protection together) is 11 years. In the future, it will be 12 years.
Moreover, for orphan medicines, the reform will bring a standard market exclusivity of 9 years, plus conditional extensions, such as +1 year if a medicine is available within 2 years after authorisation (3 years for SMEs and non-profit entities) in all Member States where the marketing authorisation is valid; +1 year if they address a high unmet medical need within rare diseases (all orphan medicines are considered unmet medical need); +1 year for any additional therapeutic indications for an already authorised orphan medicine (up to 2 years).
The current maximum regulatory protection for orphan medicines is 10 years. In the future, it will be 13 years.
2. Expansion of an attractive and innovation-friendly framework for research, development, and production of medicines in Europe
The reform would create an innovation-friendly regulatory environment for the development of new medicines and the repurposing of existing ones:
- Early regulatory and scientific advice for developers of promising medicines to facilitate the expedited approval and supporting SMEs and non-profit developers.
- Developers of innovative products will receive advice on their product years before applying for marketing authorisation which will facilitate their decisions.
- Regulatory “sandboxes” that allow testing new regulatory approaches for novel therapies under real world conditions.
- Easier use of adaptive clinical trials, real world scientific evidence and health data to reduce costs of developing new medicines and improve patient outcomes.
3. Reduce administrative burden drastically by speeding up procedures, thereby significantly reducing authorisation times for medicines to reach patients faster
The proposed reform introduces faster authorisations of new medicines. For its assessment, the European Medicines Agency (EMA) will have 180 instead of 210 days. For the authorisation, the EC will have 46 instead of 67 days. This will help to reduce the current average of around 400 days between submission and market authorisation. For the assessment of medicines that are of major public health interest, EMA will only take 150 days.
The regulatory burden and costs will be reduced through simplified procedures (e.g., by abolishing in most cases marketing authorisation renewal and introducing simpler procedures for generic medicines) and digitalisation (e.g., electronic submissions of applications and electronic product information).
In addition, EMA’s structure would be simplified to two main scientific committees for human medicines: the Committee for Human Medicinal Products (CHMP) and the Safety Committee. The orphan, paediatric and advanced therapies committees (COMP, PDCO and CAT) would be discontinued. The new, leaner structure is complemented by stronger support for the two remaining committees (through working parties and expert pools). This will reduce the assessment time and free up scientific resources to strengthen pre-authorisation support to developers and gain efficiency.
4. Enhance availability and ensure medicines can always be supplied to patients, regardless of where they live in the EU
Shortages of medicines have been a serious concern in the EU for several years. The EU’s pharmaceutical reform aims to mitigate and address shortages of medicines and enhance security of supply so that medicines are available for citizens across the EU at all times. The proposed measures are focused on two main areas of actions: shortage monitoring and security of supply enhancement, especially for critical medicines. This would be achieved by:
- Earlier warnings from companies on shortages and withdrawals of medicines, including the establishment of prevention plans.
- Establishment of a list of medicines critical for EU health systems, helping to identify supply chain vulnerabilities and improve security of supply.
- Better monitoring and mitigation of shortages, at both national and EU level, with a stronger guiding role for the EMA and the EC on security of supply.
5. Address antimicrobial resistance (AMR) and the presence of pharmceuticals in the environment through a One Health approac
AMR affects humans, animals, and plants, as well as the environment, impacting healthcare and food production. It is a cross-border issue and is one of the top 3 health threats faced by the EU. Overuse and misuse of antimicrobials, such as antibiotics, lead to an increase in AMR.
The reform offers some incentives:
- A transferable data exclusivity voucher giving developers of new antimicrobials an extra year of regulatory data protection (+12 months), making it more attractive for them to develop innovative antimicrobials without direct financial contributions from Member States.
- Procurement mechanisms, including adapted packaging and the introduction of a mandatory prescription requirement, to provide access to antimicrobials, including those under development.
6. Make medicines more environmentally sustainable
Medicines can harm the environment and human health. In addition, the presence of antimicrobials in the environment exacerbates AMR. One of the proposed solutions is to strengthen the environmental risk assessment (ERA) for all medicines, including those already authorized (before 2005), to limit the potential adverse effects of medicines on the environment and public health. The ERA has been mandatory since 2006 for all pharmaceutical companies placing their medicines on the EU market. However, the incompleteness of the ERA in the MA application has no real consequence under the current legislation. To strengthen the role of the ERA in the MA procedure, this new legislation would sanction the lack of seriousness of the ERA. If the applicant fails to submit a complete or sufficiently substantiated ERA or does not propose risk mitigation measures to sufficiently address the risks identified in the ERA, the MA would be refused. Additionally, the scope of ERA would be extended to cover the entire product lifecycle in order to reduce the risk of AMR in the environment due to the manufacturing, use and disposal of medicinal product with an antimicrobial mode of action.
In conclusion, the EU pharmaceutical reform aims to create a Single Market for medicines, making them accessible, safe, and affordable for all EU citizens. It introduces incentives for companies to develop and market medicines across the entire EU, addressing unmet medical needs. The reform also aims to foster innovation, reduce regulatory burdens, enhance the availability of medicines, tackle antimicrobial resistance, and make medicines more environmentally sustainable. Overall, it seeks to ensure that patients across the EU have timely and equitable access to high-quality medicines while supporting innovation and protecting public health and the environment.
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Rasa Vencpoliene, Senior Regulatory Affairs Manager, has more than 19 years of industry experience across Phase 1 through 4 studies, including more than 9 years of Regulatory Affairs Manager experience in the CRO environment. She has a strong educational knowledge in pharmacy and medicine and is experienced in all aspects of ICH/GCPs, quality assurance, PV, and regulatory requirements. Her main areas of regulatory experience include European Regulatory and Ethics submissions, oversight of regulatory requirements in countries of operations, and regulatory consulting. Ms. Vencpoliene is a CTIS expert, also involved in EMA procedures, such as Orphan Drug Designation, Scientific Advice and PIP applications.