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In April 2023, the European Commission (EC) published two proposals to revise the existing (and outdated) pharmaceutical legislation.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, the legislative framework will:

Be simplified to reduce administrative burdens and speed up procedures
Become future-proof
Include environmental safeguards

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 the 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.