EU Prepares To Release Major Pharma Legislative Overhaul
Executive Summary
The much heralded – and oft delayed – revision of the EU pharmaceutical legislative framework is about to be published. R&D-based pharma firms as well as generic and biosimilar companies will be keeping a close eye on proposals such as reductions in regulatory data protection and market exclusivity periods.
Wednesday 26 April will see the publication of the most wide-ranging overhaul of the EU medicines legislation since 2004 when Regulation No 726/2004 took effect, introducing a raft of changes including compulsory use of the centralized marketing authorization system for novel medicines such as new active substances, biologics and orphan drugs.
The regulation updated the EU framework for medicines to harmonize regulatory procedures across the bloc and allow the system to keep up with the emergence of new therapies such as gene and cell therapies.
Now, nearly 20 years later, the framework is set for a new revision that the European Commission says is intended to promote R&D, particularly products for unmet medical needs, and to create a balanced system that promotes accessibility and affordability of medicines while rewarding innovation.
To be launched tomorrow after a series of delays, the package of legislative revision proposals, which is part of the EU’s Pharmaceutical Strategy launched in December 2020, will affect a whole swathe of rules and regulations governing the pharmaceutical sector.
Among the planned changes are expected to be reductions in regulatory data protection and market exclusivity periods, the removal of the five-year marketing authorization renewal requirement, and ways of better addressing supply chain issues and product shortages.
Other plans will likely include accelerating regulatory processes at the European Medicines Agency, for example by reducing the number of scientific committees and giving the agency the responsibility for decisions on orphan drug designation.
New Regulation And Directive
One of the pieces of legislation in the package is a new draft Regulation amending and replacing Regulation 726/2004 and incorporating and amending the orphan Regulation 141/2000 as well as relevant parts of the pediatric Regulation 1901/2006. There is also a draft Directive that will update and replace the pharmaceuticals Directive (2001/83/EC).
Also due for release on 26 April is the commission’s Intellectual Property Action Plan (or “patent package”), which notably includes controversial plans for a harmonized EU approach to compulsory licensing of medicines to increase the availability of critical medical products and technologies in future health emergencies.
A further document – a recommendation from the Council of the EU on tackling antimicrobial resistance – is scheduled for publication at the same time.
Much of what will be in the package is already known after the draft proposals were leaked, although some of the detail will no doubt change in the final version.
This could be the case for the proposed data protection and market exclusivity changes, where there are suggestions that the latest delay to the release of the package was due at least in part to strong lobbying for a more industry-friendly version. (Also see "EU Health Chief Under Pressure To Explain Delay In Legislative Overhaul" - Pink Sheet, 31 Mar, 2023.)
The data protection and market exclusivity plans are a particular bone of contention between the commission and the R&D-based pharmaceutical industry federation EFPIA, which claimed that if they were introduced as currently drafted they would drive investment away from Europe and damage the industry’s ability to compete at global scale. (Also see "EU Data Protection Reductions Would ‘Irretrievably Sabotage’ Pharma Industry" - Pink Sheet, 15 Feb, 2023.)
Issues That Need Addressing
In drafting the proposals, the commission intends to address a whole range of issues that it sees as afflicting the current regulatory framework. They include problems with patient access to medicines, security of supply, the need for stronger obligations on companies to notify potential or actual shortages, a greater focus on producing medicines for unmet needs, and the need to simplify the marketing authorization system and make it flexible enough to keep up with developments in science and regulation at global level.
According to the leaked documents, the proposed revision is “comprehensive but targeted and focuses on provisions relevant to achieve its specific objectives.”
The package will also link in with other legislation and initiatives including the EU Clinical Trial Regulation, the enhanced mandate of the European Medicines Agency in tackling shortage, the new EMA fees regulation, legislation on blood, tissues and cells, the new medical devices legislation, the Health Technology Assessment Regulation, as well as the Horizon Europe research program.
Likely Key Proposals
Based on the leaked proposals, it is likely that the package will include the following:
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Reductions in data exclusivity periods and market exclusivity, with the possibility to recoup some of the protection periods when products are launched in all EU member states or are intended for major unmet medical needs.
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A new temporary emergency marketing authorization for products to be used in health crisis situations such as pandemics, to sit alongside other regulatory flexibilities.
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A transferable exclusivity voucher to encourage research into new antimicrobials. (Also see "Antimicrobial Exclusivity Vouchers ‘The Only Way To Go’" - Pink Sheet, 27 Feb, 2023.)
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A simplification of the EMA structure, including reducing the number of scientific committees from six to two. (Also see "EU Proposes To Chop EMA Committees, Drop Five-Year Renewals" - Pink Sheet, 2 Mar, 2023.)
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Transferring responsibility for orphan drug designation from the commission to the EMA, together with a register of designated products.
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An “evolutionary” pediatric investigation plan: companies developing certain products such as first in human substances can present a high-level clinical development plan, with more detailed information to be provided at precise development steps.
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Making the EU regulatory framework fit for emerging scientific advances through the use of regulatory sandboxes, adapted clinical trials, real-world evidence, secondary use of health data, and so on.
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Abolition of the requirement for a five-yearly renewal of marketing authorizations, except in specific circumstances on safety grounds.
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The sunset clause also to be abolished – this is the requirement to place a medicine on the market in at least one EU member state within three years of its centralized approval.
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Stricter requirements for assessing the environmental impacts of medicines.
On the pediatric and orphan medicine front, the commission hopes that incorporating and updating the existing regulations on drugs for children and rare diseases into the new legislation will ensure “clarity and consistency” of all measures that apply to these products.