Ambition and precision: How the EU’s pharma package can still be improved
June 2025On Tuesday 17 June, the European Parliament, the Council of the EU and the European Commission held their first trilogue meeting on the reform of Europe’s general pharmaceutical legislation – also known as the ‘pharmaceutical package’.
This meeting marks the start of intensive inter-institutional negotiations – a pivotal stage in the EU’s Ordinary Legislative Procedure. With both the Parliament and the Council having adopted their positions, trilogues now serve as the forum to resolve remaining differences and reach a potential final compromise. The outcome of these negotiations – on what is the most significant overhaul of EU medicines policy in two decades – will determine how medicines are developed, regulated and accessed across Europe.
Long before the European Commission published its initial proposals in April 2023, EURORDIS–Rare Diseases Europe and our community had been calling for legislative reforms that would foster innovation and deliver for the 30 million people living with rare diseases across Europe. Today, one in three people has not received a treatment directly linked to their condition – simply because one does not yet exist. In fact, dedicated therapies are still unavailable for 94% of all rare diseases. In addition, disparities in access to treatments for rare diseases persist across EU Member States[i].
Policymakers must now use this final phase to ensure the legislation responds to real-world needs – especially for those with rare diseases who still lack treatment options. Here is where the pharmaceutical package currently stands in the legislative process – and what still needs to be improved.
Where we are in the process
Since the Commission published its legislative proposals, both the European Parliament and the Council of the EU have adopted their respective positions.
The Parliament’s amendments were broadly welcomed by EURORDIS in 2024, as a “meaningful step” forward, as they introduced some important improvements. The Council’s position adopted earlier this month also advanced the text in some areas.
Yet, as the legislative process has advanced, proposals to remove or weaken certain key provisions have unfortunately emerged – and several challenges remain unaddressed.
Now that trilogue negotiations are under way, time is short to ensure that the final legislation truly delivers on its promise. The weeks and months ahead will decide whether the legislation is improved in such a way that optimises both its precision and its ambition.
What improvements do we still need to see?
1. Regulatory support
EURORDIS welcomed the original inclusion of the PRIME scheme in the legislative proposal – a scheme run by the European Medicines Agency (EMA) to enhance scientific and regulatory support for the development of ‘priority’ medicines.
However, the Council’s latest agreed position removes the explicit reference to orphan medicinal products (OMPs) in Article 60, which sets out the rules governing the PRIME scheme and the eligibility criteria for products able to benefit from it.
OUR PROPOSED UPDATES
EURORDIS is calling for the explicit mention of OMPs to be reinstated in the final text of Article 60. PRIME has played a vital role in advancing treatments for rare diseases – with over half of designations and approvals awarded to OMPs. Removing this reference risks creating uncertainty for developers and weakening one of the EU’s most effective tools for promoting innovation in areas of unmet need.
To maintain legal clarity and continuity, the final legislation must restore a specific reference to OMPs within the PRIME criteria – without limiting access solely to those that meet strict definitions of “high unmet medical need”.
2. Market exclusivity
The Commission’s proposed legislation introduced a tiered system of market exclusivity to better address patients’ unmet needs, combining a baseline duration for orphan medicinal products with an additional reward mechanism.
The Commission’s proposal and the negotiated positions of the Parliament and Council differ in both the baseline duration of market exclusivity for OMPs and the structure of the tiered reward system. Some proposals would reduce the current 10-year baseline – a measure that continues to serve as a critical incentive for innovation in rare diseases.
In addition, there is disagreement over the tiered reward for the most underserved conditions. It remains unclear whether an additional exclusivity period will be granted for treatments addressing a high unmet medical need – despite the complexity and risk involved in developing such therapies.
OUR PROPOSED UPDATES
EURORDIS supports a simple, two-tier exclusivity system that preserves the 10-year baseline and adds one year for therapies meeting the high unmet medical need (HUMN) definition. A baseline of at least 10 years is essential to ensure that innovations continue reaching the market and the people who need them.
A tiered exclusivity model would provide a proportionate reward for developers working on particularly complex and underserved conditions, where commercial viability is limited and the need is greatest.
Despite common misconceptions, market exclusivity does not block innovation – it applies only to the authorised indication. Its predictability remains one of the few tools capable of attracting sustained investment in rare diseases. A graduated system sends a clear signal that Europe values bold, patient-centred innovation in areas of highest need.
3. Incentive modulation
The tiered reward system includes providing higher incentives to developers addressing a high unmet medical need (HUMN). It is important, however, to avoid legal uncertainty and overly complex assessments of the criteria required to qualify.
EURORDIS considers the proposed criteria for defining HUMN to be reasonable: either the absence of an authorised treatment, or an exceptional therapeutic advancement brought by the orphan medicinal product.
However, a third criterion remains – that the medicine must deliver a “meaningful reduction in disease morbidity or mortality.” This language is vague and difficult to apply consistently, as it would require a lengthy period to gather the necessary evidence – to the detriment of patients. It may also inadvertently exclude paediatric patients and those with severe, underserved conditions.
In parallel, the Council’s rewording of the proposed Directive removes the long-standing recognition that all orphan medicinal products address an unmet medical need (Article 83 of the proposed Directive).
The differing positions of the EU institutions also offer limited assurances that patient representatives and other stakeholders will be involved in developing the implementation guidelines to define unmet and high unmet medical needs – a step that is crucial for fairness and legitimacy.
OUR PROPOSED UPDATES
EURORDIS supports the principle of modulated incentives – provided they are predictable, inclusive, and workable in practice.
We support keeping the concept of HUMN, as long as the criteria for qualification are clearly defined. We therefore call for the removal of the ambiguous “meaningful reduction” criterion, while maintaining the more practicable conditions: the absence of an authorised treatment, or the exceptional therapeutic advancement offered by the orphan medicinal product.
The legislation should also reinstate the recognition that all orphan medicinal products address an unmet need – a foundational principle of rare disease regulation. Finally, the implementation of HUMN-related provisions must be co-designed with meaningful stakeholder input. Modulation can drive innovation in underserved areas – but only if the system is grounded in legal clarity and shaped through genuine collaboration with the patient community.
4. Patient involvement
The Parliament had strongly backed the Commission’s original proposals to formalise voting rights for patient representatives on both the Committee for Medicinal Products for Human Use (CHMP) and the Pharmacovigilance Risk Assessment Committee (PRAC) at the EMA. The Parliament had also introduced new requirements for stakeholder input in guideline development and the classification of unmet medical need.
In contrast, the Council’s position removed patient voting rights on the CHMP, reduced patient seats on the PRAC, and made stakeholder consultation optional rather than mandatory.
OUR PROPOSED UPDATES
Patient involvement must be protected and strengthened – not rolled back.
EURORDIS, the European Patients’ Forum (EPF), and more than 70 other patient organisations across Europe have warned that removing voting rights and reducing patient representatives’ input would sideline the very people whose lives are most directly affected by EMA decisions.
We are urging the EU institutions, particularly the Parliament and the Commission, to stand firm in defending patient representation – by reinstating voting rights in the CHMP, maintaining seats on the PRAC, and guaranteeing meaningful consultation across all EMA processes. These are not symbolic gestures – they are essential to more informed and overall better regulatory decision-making.
We are also inviting the wider rare disease and patient advocacy community to support our #KeepPatientsVoting campaign to make clear that the patient voice must remain at the heart of EU medicines regulation.
Looking Ahead
The pharmaceutical package presents a crucial, generational opportunity to reshape how Europe drives innovation and ensures access to medicines.
For people living with rare diseases, success will depend on clear rules, adequate incentives and meaningful patient involvement. These are the foundations of a system capable of delivering urgently needed treatments and game-changing breakthroughs.
As trilogue negotiations continue, the EU must rise to the challenge. Getting this legislation right means offering real and justified hope to those still waiting for a treatment.
Julien Poulain, Communications & Policy Liaison Senior Manager