News / Press Releases - April 11, 2024

European Parliament adopts reports on the Revision of the EU General Pharmaceutical Legislation

On 10 April 2024, the European Parliament adopted the two reports on the Revision of the EU General Pharmaceutical Legislation, namely the Directive on medicinal products for human use and the Regulation on authorisation and supervision of medicinal products for human use and governing rules for the European Medicines Agency.

Establishing the position of the European Parliament, the reports set out reforms of the EU pharmaceutical landscape, aiming at enhancing access, affordability, and availability. While the Parliament has clearly tried to strike a balance to foster innovation and competitiveness among pharmaceutical companies, there is still a need for improvement of the legislation.

The Parliament’s general acknowledgement of the need for a robust incentive framework to support innovative small and medium-sized pharmaceutical companies is encouraging. It remains critical to ensure amenable access to incentives for these companies to support predictable growth.

Fostering simplification and modernisation of the regulatory framework is a pivotal step in reducing administration for regulators and industry. The Parliament reports reinforce the EU’s position as a global regulatory leader. This should expedite the availability of new, safe therapies for patients.

On selected topics, the adopted reports demonstrate enhancements over the Commission’s initial proposal in nurturing the EU’s pharmaceutical competitiveness. Nonetheless, some concerns remain, especially in areas where more burdens and obligations are introduced, while incentives weakened when compared to the current system.

EUCOPE’s key points following the European Parliament’s Plenary vote

(High) unmet medical need (H-UMN)

The criteria for UMN and HUMN remain broadly consistent with the Commission’s proposal. These include concepts such as “life threatening or severely debilitating disease”, “meaningful reduction of morbidity or mortality” and “exceptional therapeutic advancement”, which are unclear and open to interpretation. These concepts run the risk of overlooking certain patient groups and disease areas depending on their interpretation.

These amendments signify a recognition of the imperative need to better incentivise the most innovative treatments. However, uncertainties surrounding the definition and understanding of UMN and HUMN may stifle innovation and decisions regarding market access by introducing additional unpredictability into the system. Moreover, (H)UMN designation comes too early in the assessment process to measure the full impact of a therapy, and too late in the process for investors to be able to make an appropriate investment decision.

When addressing UMN, the reward is an increase from six to 12 months Regulatory Data Protection (RDP), while for HUMN there is an increase from one year to two years orphan market exclusivity (OME), when compared to the Commission’s original proposal.

Orphan market exclusivity (OME)

The adopted reports reduce baseline OME to nine years and extend the OME for addressing high unmet medical needs (HUMN) to 11 years. This contrasts with the current 10 years OME applicable to all orphan medicinal products (OMPs). EUCOPE recognises the necessity and ambition behind a modulated OME framework. However, it is paramount to develop modulation that takes into account the unique characteristics of rare diseases and their treatments. Rather than basing OME modulation solely on HUMN, a more nuanced framework, such as the one developed by the OD Expert Group which allows for both upward and downward modulation. Such an approach incentivises the development of therapies in areas with no meaningful treatment options, without discouraging innovation for conditions where patients can continue to benefit from it.

More consideration is required regarding the ‘global orphan marketing authorisation’ (GOMA) which can promote additional research and incentivise companies to explore further uses of existing, patented compounds. It is unfortunate that this principle is not reinforced, for example by extending the period of duration of exclusivity and number of times an extension can be offered, to boost innovation to the benefit of patients and healthcare systems.

Regulatory Data Protection (RDP)

Regulatory data protection is a crucial incentive to ensure return on investment, in particular for small and mid-sized companies. By incentivising innovation, developers are able to reap the rewards of years of R&D efforts and high-risk financial investment. The adopted Parliament reports improve on the Commission’s initial proposal (six years of baseline RDP), by increasing the RDP baseline to seven and a half years. This could be extended up to eight and a half years, upon fulfilling certain conditions, such as addressing an UMN, conducting comparative clinical trials or carrying out (pre-clinical) R&D in the EU in cooperation with an EU public entity. We encourage Member States to amplify further the proposal of the European Parliament.


The new access proposal presents a more nuanced approach compared to the Commission’s initial proposal. While the Commission’s proposal requested the marketing authorisation holder to “release and continuously supply” the medicinal products in all 27 Member States in a strict timeframe, the Parliament reports propose to file for Pricing and Reimbursement (P&R) only in Member States that request it, with some derogations (for instance, for OMPs).

EUCOPE appreciates that the Parliament’s access proposal recognises the role of other stakeholders, besides marketing authorisation holders. Moreover, the Parliament reports take into account the specificities of certain products, such as OMPs and ATMPs and introduce a conciliation mechanism. Nonetheless, concerns remain regarding the feasibility for smaller developers, as well as the implications brought by the possibility of facing financial penalties for non-compliance with the requirements, and how these would be applied. Furthermore, uncertainties persist regarding the interpretation and implementation of these proposals.

Medicine shortages and security of supply

The provisions on medicine shortages and notification requirements in case of supply disruptions remain broadly unchanged in comparison to the Commission’s proposal, with some additional measures introduced. The reports distinguish between foreseeable and unforeseeable disruptions, and reinforce responsibilities and role of the European Medicines Agency (EMA), the Medicines Shortages Steering Group (MSSG) and Member States on monitoring and addressing shortages. These provisions continue to worry EUCOPE. Introducing excessive, unrealistic requirements will disproportionately burden small and mid-sized companies.

Environmental Risk Assessment (ERA)

The Parliament reports expand the scope of ERAs to incorporate the full lifecycle of medicines development, including onerous global requirements on companies. These provisions are concerning and present unrealistic requirements which should be avoided. Implementing excessive administrative requirements will significantly weigh on smaller companies and potentially impede timely authorisation and accessibility of treatments for patients. It is crucial to strike a balance, acknowledging the responsibilities of all parties involved.

Future-proofed Regulatory Network

The implementation of phased review processes for products offering exceptional therapeutic advancement and expanded expedited pathways for priority medicines under the PRIME scheme are key advancements. These measures are designed to accelerate the evaluation of promising treatments, ensuring quicker patient access for those most in need.

Openness to the use of Real-World Data (RWD) is encouraging. As the ability to collect good data continues to grow, our methods to assess and implement its applications in the EU should equally expand. Use of RWD can bolster the input of small-disease populations like the rare disease community on the true benefits of innovative treatments. Clear guidelines on how RWD can be used as part of the authorisation of medicines will be needed to complement the legislative provisions.

Other elements, such as Regulatory Sandboxes, aim to facilitate the assessment of technologies not currently covered by existing frameworks. It would provide a structured setting for experimentation, albeit with limited scope and usage, evaluated on a case-by-case basis for defined time periods. Such a structured approach to a ‘regulatory test environment’ is poised to be highly valuable for evaluating emerging technologies. Similarly, the legislation facilitates the assessment of Platform Technologies.

Restructuring the committee and working party structure of EMA can allow for a streamlining of advice and decision-making pathways. Inclusion of specific ad-hoc parties ensures necessary competence on key topics like OMPs and ATMPs is retained.

Ensuring a future-proofed regulatory network in the EU should support all innovative companies to work alongside regulators, utilising the best technology and creating seamless sharing of necessary information.