The hearings at the ENVI committee on the revision of pharmaceutical legislation

by Giuliana Miglierini

As a part of the ongoing process of revision of the pharmaceutical legislation, the Committee on Environment, Public Health and Food Safety (ENVI) of the European Parliament hold a public hearing on 20 September 2023 with the EU Commission’s representative Mr Florian Schmidt (DG Santé). We summarise the main contents of the hearing.

The proposals of the Commission

The proposed package is comprehensive of a new Regulation introducing specific measures for innovative medicines, new rules on how to manage shortages and a new framework for the governance of the European Medicines Agency (EMA). The new Directive redefines pathways for regulatory authorisation and incentives for the development of various types of products. The third part of the package is the Council Recommendation on antimicrobial resistance (AMR).

As highlighted by Florian Schmidt, the proposed reform is characterised by six key political objectives, starting from the creation of a Single European market of medicines, claimed to represent a key factor to improve access to new medicines especially in the smaller Member States.

The Commission also aimed to improve the availability of critical medicines, acting on the long-lasting issue of shortages and security of supply, while addressing the sustainability of healthcare systems.

The global competitiveness of the European pharmaceutical sector would be supported by a more flexible regulatory framework, while the compulsory Environmental Risk Assessment (ERA) would improve the environmental sustainability of medical products. Last, but not least, is the fighting of antimicrobial resistance.

Main comments from ENVI rapporteurs

The hearing saw the intervention of the two rapporteurs of the EU Parliament (Pernille Weiss and Tiemo Wölken) and of several members of the ENVI Committee involved in the discussions on the proposals. The general objectives of the Commission are shared by Parliament, but some elements requiring optimisation emerged during the hearing.

As indicated by Rapporteur Pernille Weiss (Denmark), the ambitious target of improved access to a greater number of medicines for all EU citizens would require also a similar pricing structure and access across Member States. It would be important to ensure a fair playfield, as the pharma legislative package is expected to exert effects on EU competitiveness and access.

According to Ms. Weiss, the actual level of protection a new medicinal product might achieve would be dependent on how IP protection is regulated in different Member States. Pricing structure is also a matter falling under national competences. On this basis, the objective to guarantee access to medicines at lower prices to all Europeans should be considered vs the need to guarantee appropriate data protection. Without a national module on data protection, said Ms. Weiss, it would be very difficult to manage the system, and a realistic approach would be advisable.

Mr. Tiemo Wölken (Germany) supported the removal of the traditional one-fits-for-all approach, in favour of the modulation of regulatory protection. According to the Rapporteur, it would be a mistake to think that more R&D would be only generated with longer protection. Instead, a compulsory reinvestment of a certain percentage of turnover in R&D would be advisable in order to benefit from EU protection. This provision would help stopping the migration of companies towards China or India, despite they may had already experienced a very high level of protection in the EU.

Mr. Wölken was also not convinced the Transferable Exclusivity Voucher (TEV) would be the appropriate form of incentive to support research in new antimicrobials, as a great number of these activities are run by small and medium-sized companies (SMEs). The TEV would benefit big pharma players that would acquire the vouchers. According to the Rapporteur, this might lead to a sort of blockbuster scenario, delaying access of generics to market. TEVs would also not guarantee safety of supply: the Rapporteur mentioned the need of a new wording to be introduced in the text to guarantee the availability of antibiotics in the EU market, should the voucher be maintained in the final form of the proposal.

Finally, Mr. Wölken welcomed the provisions aimed to prevent and mitigate shortages and to ensure safety of supply of critical medicines. To this instance, he mentioned the transparency of marketing authorisation referred to production and the supply of medicines as key factors companies should be made accountable for.

Main comments from other ENVI members

MEP Tomislav Sokol (Croatia) highlighted the need to bring back innovation to the EU. Identified issues in the Commission’s proposals refer to a better balance between incentives for R&I, and the need to find the real value and access to new therapies. More in particular, incentives should focus in areas of major gaps, but for many companies their modulation may pose problems as they might have not enough capacity to serve all Member States.

Mr. Sokol proposed to better define these aspects so to improve the certainty and predictability for companies. He added some areas in rare diseases and paediatric medicines would need more incentives, while regulatory sandboxes can present opportunities for testing promising products.

According to MEP Monika Bénová (Slovakia), it would be very important to improve access to affordable medicines in small European countries by acting on rules on competitiveness, transparency and healthcare expenditure.

Ms. Bénová added it would be positive to have a shorter regulatory process, but not at the expense of safety and pharmacovigilance.

She also asked the Commission what the planned actions in the field of AMR are. Monika Bénová said the high profits pharma companies made in the last few years should be used for public health, and not only as a profit tool, especially during emergencies or pandemics. A goal that should be supported by new rules for better transparency in public procurement.

French MEP Catherine Amalric said a balanced framework would be needed to safeguard all interested parties. Support to research and innovation is essential in order to compete on the global scenario, but it would be important to avoid unrequested delays to investments and development.

According to Ms. Amalric, it would be also advisable to adapt to technological changes. Activities should be planned on all scales, from SMEs to big companies, while maintaining the autonomy and authority of the EU in the field of medicines. To this instance, more rapid procedures and a stronger role for EMA were suggested, among others.

It would be also advisable to have harmonised good practices in the field of pharmacovigilance. The digital revolution should be taken in mind, as well as the Green Deal: to this instance, Catherine Amalric supported the approach of the Critical Medicines Act.

According to MEP Frédérique Ries (Belgium), the proposals of the Commission may create a worrying hierarchy between rare diseases and medicines for very important needs. She said the Expert Group for incentives for orphan diseases proposed a much more pragmatic framework for prioritisation, asking the Commission if it was considered.

The participation of patients in the marketing authorisation procedures should be also approached from a pragmatic point of view, she added. As for access, Ms. Ries said active European support would be needed as for joint purchasing, something that would turn important for medicines entering the PRIME programme.

According to MEP Tilly Metz (Luxembourg), the balance between incentives and access is yet not optimal, as it is mainly focused on IP protection. New solutions are needed, including a greater emphasis on conditions for access and public subsidies independently from the form they assume.

Ms. Metz also indicated that the authorisation pathway for medicines for unmet medical needs appears too vague, thus potentially leading to some confusion. Available options should be better clarified, while in the field of antibiotics long-term solutions would be needed. Tilly Metz said TEVs would not represent the needed solution, as it would highly impact healthcare systems without solving the issue of access. Ms. Metz mentioned also the impact assessment of incentives targeted to R&D in the field of antibiotics, an area where would be advisable not to look only to solutions that please the industry. A European Fund for R&D in antimicrobials would be needed instead.

Furthermore, comparative studies should be always included, while their reference to the incentives scheme only would not represent the correct approach. Speaking on behalf of an absent MEP, Tilly Metz mentioned an issue with the Directive due to the non-inclusion of manufacturing in the Environmental Risk Assessment. It would be advisable to look at all the lifecycle of environmental risk, she suggested, as it was proposed in the first draft of the Commission.

MEP Joanna Kopcinśka (Poland) said Europe is still dealing with shortages of raw materials, something that also impacts access to medicines in different countries. It would be important to ensure a fair market in all the EU, by means of legal tools that would smooth out differences between countries.

Ms. Kopcinśka rejected the hypothesis of restrictions on marketing authorisations linked to environmental considerations, as this would be detrimental for patients. As for safety, the Directive would present some crucial aspects referring to active substances, especially in the case raw materials are manufactured outside the EU. It would thus be needed to include a clear definition of critical medicines, and the new methodology should be based among others on the therapeutic importance of critical medicines.

Speaking on behalf of an absent MEP, Ms. Kopcinśka said in the field of rare diseases is often difficult to reach a number of patients sufficient to test new substances. Innovation is key, and research should be of interest for investors in the EU.

MEP Peter Liese (Germany) strongly supported the TEV system, as according to him no other better proposal emerged in the past ten years. On the other hand, Mr. Liese judged not good enough the proposal on innovation, as six months’ incentives would not be sufficient.

The reply of the Commission

The hearing closed with the reply of Mr Florian Schmidt, who was happy to see sharing of the general objectives by MEPs even if not of all the proposed solutions. Mr. Schmidt mentioned the importance of SMEs and the specific measures planned to support them in the pre-authorisation phase and modelisation.

The Commission tested the different options available to model incentives, he added; the final choice was based on the ambition not to burden more on national systems, and the will to achieve a broader and earlier access for patients.

As for the voucher system, the Commission planned very closed conditions aimed at allowing for the issuing of no more than 10 TEVs in 15 years. According to Mr. Schmidt, this should improve the predictability of the system and the calculation of effects on healthcare systems.

He also mentioned the introduction of some new requirements in the Paediatric Investigational Plan in order to close some current loopholes in paediatric research. The Commission made no shortcuts on safety while reviewing the regulatory framework, he added, for example for the first time it was introduced the possibility to kick out the system for immature applications.

As for the proposed hierarchy of standard rare diseases and medicines for high unmet medical needs, according to Mr. Schmidt there was no intention in considering medicines for standard rare diseases less important. He also reassured MEPs on the involvement of patients in regulatory discussions and on ERA, that would not represent any risk for compliant companies.