Article

According to information published on the official websites of the competent authorities of the European Union (hereinafter also referred to as the “EU”), the European Commission (hereinafter also referred to as the “EC”) proposed an ambitious revision of EU pharmaceutical legislation, which has been the subject of extensive discussion among experts. According to the EC, this revision is intended to be the first major update of pharmaceutical legislation since 2004; thus, after more than 20 years, these regulations are to be adapted to the current needs of the 21st century in light of developments that have occurred during this time (e.g., the COVID-19 pandemic, new vaccines, or shortages and disruptions in the supply of medicines—including the substances used to produce them—also due to environmental factors). The proposals were debated in the European Parliament in April 2024, and Members of the European Parliament approved them on April 10, 2024. However, further steps regarding this legislation will be taken by the new European Parliament following the European elections, which will take place from June 6 to 9, 2024.

The purpose of this article is to briefly describe the content and scope of the new legislation, as well as its stated goals and purpose, based on official information published by the competent authorities.

According to the EC, the main objectives of this revision are:

1. to create a single market for medicines and ensure that all patients across the EU have timely and equitable access to safe, effective, and affordable medicines,
2. to continue to offer an attractive framework for research, development, and production of medicines in Europe that supports innovation,
3. significantly reduce the administrative burden by substantially speeding up procedures and shortening the time required for drug authorization, so that medicines reach patients faster,
4. improve the availability of medicines and ensure that medicines can always be supplied to patients regardless of where they live in the EU,
5. combat antimicrobial resistance and the presence of medicines in the environment through a “One Health” approach,
6. increase the environmental sustainability of medicines.

This revision consists of the adoption of two legislative proposals intended to establish a new regulatory framework for all medicines (including medicines for rare diseases and medicines for children):

1. a new Directive
2. a new Regulation

It also includes a Council Recommendation on antimicrobial resistance.

These proposals replace the existing general pharmaceutical legislation (Regulation (EC) No. 726/2004 of the European Parliament and of the Council (EC) No. 726/2004 of March 31, 2004, laying down Community procedures for the authorization and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency, and Directive 2001/ 83/EC of the European Parliament and of the Council of 6 November 2001 laying down the Community code relating to medicinal products for human use) and legislation on medicinal products for children and for rare diseases (Regulation (EC) No. 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for pediatric use, amending Regulation (EEC) No. 1768/92, Directive 2001/20/EC, Directive 2001/83/EC, and Regulation (EC) No. 726/2004, and Regulation (EC) No. 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products).

New legislation under the EC proposal

The revision builds on strict standards of quality, safety, and efficacy in the authorization of medicines and proposes new tools:

a shift from a universal incentive system for pharmaceutical companies to a modulated incentive system, under which companies that meet important public health objectives—such as providing access to medicines in all Member States—are rewarded, the development of medicines that address unmet medical needs, the conduct of controlled clinical trials, and the development of medicines that can also be used to treat other diseases. For medicines for rare diseases, a similar modulation is proposed in the form of market exclusivity,

faster availability of generic and biosimilar medicines and transparency in public funding,

addressing medicine shortages and ensuring their supply,

a modern and simplified regulatory framework enabling faster authorization of new medicines (e.g., the EMA will have 180 days instead of 210 days for evaluation. The Commission will have 46 days instead of 67 days to grant a marketing authorization. This will help reduce the average time between submission of an application and the issuance of a marketing authorization, which currently stands at 400 days. The EMA will have only 150 days to assess medicines of major public health importance under experimental regulatory frameworks designed to support the development of innovative medicines, electronic submissions, and electronic package inserts),

support for innovation and competitiveness,

better environmental protection,

the fight against antimicrobial resistance.

Support for innovation in the proposed legislation

• The proposed measures primarily concern the simplification of procedures and the new structure of the EMA (hereinafter also referred to as “EMA”)
• The EMA will provide scientific advice from the outset of the processes, thereby improving the quality of applications
• “interim assessments” (i.e., the gradual review of data as soon as it becomes available) and temporary emergency marketing authorizations in the event of health emergencies (based on experience with the COVID-19 pandemic) will be introduced
• companies developing innovative medicines will be able to obtain advice regarding their medicine years before applying for a marketing authorization
• experimental regulatory environments will enable the testing of new regulatory approaches to new therapeutic procedures under real-world conditions
• Special provisions and incentives for changing drug indications will make it easier for researchers and non-profit organizations to conduct research on authorized medicines
• Special incentives will be introduced for medicines that address high unmet medical needs in rare diseases
• companies that bring innovative medicines to market will be subject to regulatory protection lasting at least eight years, which includes six years of data protection and two years of market protection
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• Companies may benefit from additional periods of protection, extending the total period to a maximum of 12 years, whereas the current maximum is 11 years
•  
• These additional periods of protection may be granted to companies if
• a) they market the medicine in all Member States (+2 years),
• b) the medicine addresses an unmet medical need (+6 months), or
• c) controlled clinical trials are conducted (+6 months)
• For medicines for rare diseases, the standard duration of market exclusivity will be 9 years. Companies may be eligible for additional periods of market exclusivity if:
• a) they address a high unmet medical need (+ 1 year),
• b) they market the drug in all Member States (+ 1 year), or
• c) they develop new therapeutic indications for an already authorized drug for an orphan disease (+ 2 years).

Regulatory protection periods may last a total of up to 13 years, whereas the current maximum is 10 years

References to the fight against antimicrobial resistance

• the situation in this area is complex—according to the EU, it is caused by market failure, as the more antimicrobial agents are used, the higher the risk of resistant bacteria emerging. On the other hand, however, there is a lack of motivation for drug development and innovation in this area due to restrictions on the sale of these products or the prospects for their sale. The result may be that in the future there will be no new drugs capable of combating multidrug-resistant pathogens
• In light of the above, a voucher system is proposed—companies developing new, revolutionary antimicrobial agents will be granted “transferable data exclusivity vouchers” that these companies can use themselves or sell. Thanks to the voucher, such a company will be granted data protection against competitors for the next year in relation to the drug to which the voucher applies

Patient access to innovative medicines

• Since not all patients across the EU have timely and equitable access to safe and effective medicines—particularly innovative medicines—the reform proposes that the two-year data protection period be conditional on the medicine’s launch in all Member States
• Shortening the standard period of regulatory protection by two years (compared to the current situation) and measures that support the timely entry of generic and biosimilar medicines into the market will, at the same time, provide patients with more affordable options and result in reduced spending on medicines by healthcare systems

Simplifying the decision-making system will shorten the timeframes for authorizing new medicines.

• In addition to existing paper-based package inserts, patients will be provided with accurate electronic information about medicines in their own language, in the form of electronic leaflets. This will also help address medicine shortages by facilitating the redirection of medicine stocks to countries that need them.

Changes to the EMA’s operations to accelerate drug authorization

• In the legislative proposal, the EMA’s structure is streamlined into two main scientific committees for medicines for human use: the Committee for Medicinal Products for Human Use and the Pharmacovigilance Risk Assessment Committee (PRAC). The Committee for Orphan Medicinal Products, the Paediatric Committee, and the Committee for Advanced Therapies will be abolished
• ; under this new, simplified structure, the two remaining committees will receive stronger support (through working groups and expert groups). This will shorten the time taken to assess medicines, free up scientific resources to strengthen support for companies developing medicines prior to authorization, and increase the effectiveness
• the mandate of the EMA and its Executive Steering Group on drug shortages and safety will be expanded to include the management of critical drug shortages and the security of supply of critical medicines

Drug shortages

• the reform goes beyond crisis preparedness and response and will help address systemic shortcomings under any circumstances. The new legislation will strengthenthe monitoring and mitigation of medicine shortages, particularly critical shortages, at the national level, as well as through changes to the EMA’s operations
• pharmaceutical companies will also have to address critical shortages and report on the results of the measures taken (such as increasing or reorganizing production capacity or adjusting distribution to improve supply)
• an EU list of critical medicines will be established, i.e., medicines that are considered most essential to healthcare systems under all circumstances. Recommendations will be provided to companies and other relevant stakeholders on the measures they should take to strengthen the supply chains for these medicines, thereby ensuring the continuity and security of the supply of medicines to EU citizens. Under this legislation, the EC will have the option to adopt measures in the form of an implementing act to strengthen the security of the supply of medicines, including requirements for the establishment of emergency stocks.

Criticism

The proposed legislative changes have not been met with only positive feedback. Criticism from expert circles has focused primarily on the unrealistic nature of the targets, particularly regarding generic medicines and their market entry, as well as intellectual property protection, which serves as a tool to ensure a return on investment in innovative medicines. According to experts, some of these measures actually reduce the EU’s competitiveness and lead to a slowdown in investment activities (one reason is that bringing a drug to market in all member states following its registration is reportedly unrealistic given the differences in healthcare systems and approaches to pricing and reimbursement across EU countries).