Revision of the EU’s pharmaceutical legislation
Background on the reform
In April 2023, the European Commission (EC) published two proposals to revise the existing (and outdated) pharmaceutical legislation. This includes legislation on medicines for children and rare diseases.
The EC's proposed reform consists of a new:
- Directive on the European Union (EU) code relating to medicinal products for human use
- Regulation on the authorization and supervision of medicinal products for human use—and also on rules governing the European Medicines Agency (EMA)
You can read the original text of the proposals on the EC site.
What the proposed directive covers
The directive covers all requirements for authorization, monitoring, labelling and regulatory protection, placing on the market, and other regulatory procedures for every medicine authorized at EU and national level.
What the proposed regulation covers
The regulation defines specific rules for medicines authorized at EU level, particularly the most innovative ones, creates protocols for management of critical shortages and supply security of critical medicines, and stipulates rules governing the EMA.
Overarching aims of the EC's reforms
The main aims of this large reform are to:
- Ensure patients across the EU have timely and equitable access to safe, effective, and affordable medicines
- Improve security of supply and availability of medicines in the EU
- Offer an attractive and innovation-friendly environment for medicine research and development (R&D) and production in Europe
In addition, and in line with scientific and technological advances, the legislative framework will be simplified to:
- Reduce administrative burdens
- Speed up procedures
- Include safeguards to reduce the environmental impact
Why does it matter to EHA and hematology?
The EU's pharmaceutical reform directly touches on EHA’s core advocacy priorities, which are:
- Access to innovative therapies
- Support for research
- Better regulation
The legislation will have an impact, both directly and indirectly, on several of our concerns.
Potential impact on access
The legislation can address:
- Current inequities and delays in access across Europe
- Supply problems
- High prices
Potential impact on research
The legislation can:
- Stimulate patient-centric and needs-based development of medicines
- Create dedicated frameworks for personalized medicine and rare diseases
Summary of EHA's position
In line with our advocacy priorities, and in consultation with our European Affairs Committee, we have directed our focus to the following six topics:
- Hospital exemption
- Unmet medical need
- Transparency requirements
- Drug repurposing
- Incentives
- Shortages
In addition to responding to the EC consultation on the proposals, we have shared our stance on these topics will all the relevant policymakers. The following is a summary of those views.
Hospital exemption
EHA is in favor of protecting the hospital exemption provision, which should be seen as a complement to the commercial pathway in order to fill the gaps.
The hospital exemption is crucial to ensure patient access, particularly when:
- Their conditions are not commercially viable
- The patient requires personalized treatment
We explain our stance on hospital exemption in detail in our joint statement with the Association of European Cancer Leagues (ECL).
Unmet medical need
EHA calls for an inclusive definition of unmet medical need, encompassing:
- Severity
- Quality of life
- Burden of disease
- Availability of alternative treatments
Many non-lethal conditions in hematology gravely affect the lives of patients and their caregivers. They also create a financial burden for national health systems.
We believe that it should be mandatory to consult healthcare professionals and patients during the formulation of scientific guidelines on unmet medical need.
Transparency requirements
On transparency, EHA believes that more should be done.
Due to the extreme difficulty of making realistic estimates of costs incurred in R&D, this legislation provides a singular opportunity. Additional transparency requirements with regard to funding are vital to gain real insight into R&D costs, and ultimately improve affordability.
We support the disclosure of both direct and indirect public funding.
Incentives
EHA sees incentives as an important means to enhance access to therapies across the EU. We therefore support a lower baseline of regulatory protection, as proposed by the European Commission, in conjunction with (reasonable) conditional increases. Additional years of protection should result in a direct benefit for European patients.
The legislation should also incentivize R&D in the EU—not only to improve the EU’s strategic autonomy, but to account for the specificities of disease populations.
Drug repurposing
We highlight drug repurposing as a means to speed up the R&D process and save costs, thereby leading to more and lower priced innovative therapies.
The provision on drug repurposing should therefore not be confined to unmet medical need.
Shortages
EHA welcomes the measures introduced by the EC to address supply complications and shortages across the EU.
Next steps
The EC's two legislative drafts must be examined in detail by the:
- European Parliament (EP)
- Council of the European Union (Council)
During this process, the EP and Council will make changes to the EC's original proposals.
Whilst the EP has concluded its reading of the file, the Council's consideration is expected to take longer.
Once this process is concluded, the co-legislators and the EC will hold meetings (‘trilogues’) to negotiate what the final legislative text should look like.
In order to be final, the result of these negotiations must then be endorsed by the EP and Council.
If you have questions about our position
You can get in touch with us by emailing europeanaffairs@ehaweb.org.
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