HM1: Update on EU new regulatory legislation − EMA, industry and others

Regulatory Rapporteur

December 2023  |  Volume 20  |  No.11

 Key points

• The session gave insight on the proposal adopted by the Commission in April 2023 to revise and replace the existing general pharmaceutical legislation (Regulation 726/2004 and Directive 2001/83/EC) and the legislation on medicines for children and for rare diseases (Regulation 1901/2006 and Regulation 141/2000/EC, respectively).
• Based on the two draft reports recently issued by the two MEP Rapporteurs, the EU Parliament may finally move away from the Commission’s initial proposals on some key aspects, notably on the ‘launch conditionality’, and on the question of transferable exclusivity vouchers, and on the reduction of incentives for orphans, and finally on the removal of accelerated timelines for MA review.
• Measures to improve access to medicines will, however, remain difficult to overcome because the majority of access barriers must, in the majority of cases be resolved at national level.

Olga Solomon highlighted the Commission proposals in relation to the reform of the EU pharmaceutical legislation, and notably the package adopted by the Commission in April 2023 which includes a new Regulation and a new Directive as well as an initiative aimed at preventing antimicrobial resistance (AMR). She reminded the session audience that the political objectives of this ambitious reform were to improve access to medicines in the EU, strengthen measures to prevent shortages of medicinal products and secure the supply of critical products, while reinforcing affordability for patients and ensuring environmental sustainability. These measures were prepared keeping in mind that they should not negatively impact the capacity of innovation of the pharmaceutical industry and maintain competitiveness.

Proposed solutions to improve access to medicines are based on the introduction of a modulation of incentives for innovative medicines which translates into a potential one-year extension of the data protection (and up to three years market exclusivity for orphan medicines).

To address the increasing number of shortages and secure thew supply of critical products, the solutions proposed by the EU Commission include a better coordination, monitoring and earlier notification of shortages which would translate into the development of prevention plans and a community list of critical medicines that deserve a better and harmonised follow-up. The EMA will have a pivotal role in this regard on top of the National Competent Authorities and the EU Commission. These proposed measures will complement other initiatives which are not in the pharmaceutical package but ultimately share the same objectives.

The Commission also made proposals aimed at supporting the affordability of medicines, including measures to ensure an earlier market entry of generics and biosimilars as well as additional measures to increase transparency on public contribution to R&D, comparison of clinical trials to support national decisions on pricing, and further support for information exchange between Member States regarding pricing and reimbursement considerations.

To reinforce the competitive and innovation-friendly regulatory framework, the Commission have proposed procedural changes to reduce the Marketing Authorisation timelines (from 277 to 226 days) and improve regulatory efficiency (eg., by allowing authorities to reject immature applications, authorising rolling or phased review, as successfully experienced for COVID products, abolishing MA renewals, etc.). These measures should lead to changes in the EMA organisation, a better use of data and digitalisation (e.g., via more systematic use of RWE), as well as an increase of pre-submission support and lower regulatory burden for SMEs and academic organisations.

Needless to say, the package includes measures to ensure environmental sustainability, by providing a better implementation of the environmental risk assessment (ERA), and notably by extending the ERA to medicines already on the market before 2005 – notably, this generated a few questions from the audience during the subsequent panel discussion.

As part of the proposed solutions to combat AMR, the ‘AMR voucher’ is of particular interest. Indeed, this type of incentive/mechanism, ie., a transferable voucher allowing an additional year of data protection, was deemed to be first of a kind for Europe.

Paula Loekemeijer, Exec. Director of the MEB, then provided the National Competent Authorities’ perspective on the above-detailed pharmaceutical package and the probable impact of these changes for the Netherlands MEB. She first reminded session participants of the valuable contribution of the MEB in the EMA and EU procedures on top of national activities, which represent a significant investment in time and resources for a national agency.

Overall, Loekemeijer acknowledged the benefits that this reform should have on the MEB activities, and she recommended to consider all the lessons learned from the Veterinary legislation implementation in 2022. Among them, it is important to allow sufficient time for the teams to prepare and understand impact, training the staff, and make sure the agency will have sufficient resources to handle, and to implement all these changes in time. As with the EMA and other Agencies, the MEB also faces challenges to recruit resources and this important aspect should not be underestimated as it may slow down the transition to the new framework.

Valentin Plouchard, representing EuropaBio, the European Association for Bioindustries, then presented the industry perspective. He acknowledged that there were a lot of interesting measures proposed by the Commission in April 2023, and also informed the audience that in early October 2023 the two EU Parliament Rapporteurs published their draft reports (presenting different visions for the future EU pharmaceutical legislation), and that the deadline for amendments was mid-November.

The draft reports clearly imply substantial changes to the two texts as a result of the legislative debate. EuropaBio considers the following points as positive for the European life science industry^[1]:

• The extension of data protection for innovative medicines.
• The removal of the ‘launch conditionality’ replaced by an obligation to submit an application for Pricing and Reimbursement.
• The acknowledgement that the EU SME definition should be revisited.
• The increased harmonisation of the transparency for hospital exemption.

If there are positive aspects, EuropaBio also considers the following points as less favourable to the industry:

• The reduction of incentives for orphans and stricter criteria to get orphan drug designation (ODD).
• Validity of the centralised MA remains limited to 5 years (renewal of MAs will not disappear).
• EMA MA procedure timelines will remain unchanged (no acceleration as initially envisaged).
• Removal of the transferable exclusivity vouchers.

Steffen Thirstrup, CMO of EMA, then concluded with his view on the Commission’s proposals. He also welcomed the changes in legislation and acknowledged that the EMA need to evolve and that some processes and ways of working deserve to be revisited. To some extent, he echoed Laura Loekemeijer, by emphasising that the activities conducted at EMA and national competent authority levels were increasingly substantial, and of course time and resources are limited which can become a concern, or at least may slow down the transition process. He also welcomed all initiatives aimed at improving the innovation and competitiveness of Europe and reminded that it was key to share data and knowledge and therefore the collaboration with other agencies and health technology agency (HTA) bodies was paramount to reach this goal.

Reference: 

^[1]  LinkedIn: Valentin Plouchard