The European Parliament adopted by 488 votes to 67, with 34 abstentions, a legislative resolution on the proposal for a regulation of the European Parliament and of the Council laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006.

The position adopted by the European Parliament at first reading under the ordinary legislative procedure amends the proposal as follows:

Subject matter and scope

The proposed regulation: (i) lays down Union procedures for the authorisation, supervision and pharmacovigilance of medicinal products for human use at Union level, (ii) establishes rules and procedures at Union and at Member State level relating to the monitoring and management of shortages and critical shortages and the security of supply of medicinal products and (iii) lays down the governance provisions of the European Medicines Agency.

Environmental risk assessment

The environmental risk assessment of medicinal products consisting of or containing genetically modified organisms should include the identification and characterisation of risks to the environment, animals and human health throughout the life-cycle of the medicinal product, including its production, and the risk reduction and mitigation strategies proposed to address the identified risks.

Combating antimicrobial resistance (AMR)

In order to support the development of antimicrobials and address existing market failures, Members wish to introduce market entry rewards and intermediate reward payment systems. Accordingly, they suggested developing a milestone payment reward scheme , complemented by a subscription model voluntary joint procurement scheme, should be developed to ensure that a market exists for developers that delink volumes sold from payment received.

Milestone payments are an early-stage financial reward granted upon achieving certain R&D objectives prior to market approval. While such mechanisms would serve primarily to provide access to existing antimicrobials, they could also support new antimicrobials in the development phase.

Granting the right to a transferable data exclusivity voucher

Following a request by the applicant for a marketing authorisation, made before the marketing authorisation is granted, the Commission may, by means of implementing acts, grant a transferable data exclusivity voucher to a ‘priority antimicrobial’, under certain conditions based on a scientific assessment by the Agency. The voucher should give the right to its holder to a maximum of additional 12 months of data protection for one authorised medicinal product.

The Commission should adopt delegated acts by setting up the eligibility of pathogens for the protection periods referred to in the regulation in accordance with the WHO priority pathogens list or an equivalent established at Union level, with 12 months of data protection for an authorised product ranked ‘critical’ , 9 months of data protection for those ranked ‘high’ and 6 months of data protection for those ranked ‘medium’.

A voucher should only be used once and in relation to a single centrally authorised medicinal product and only if that product is within its first four years of regulatory data protection. The voucher should not be used for a product which already benefited from the maximum regulatory data protection period.

By five years from the date of entry into force of this regulation, the Commission should submit an evaluation report to the European Parliament and to the Council containing a scientific assessment measuring the progress with regard to antimicrobial research and development and the effectiveness of the incentives and rewards in this regulation.

Agency’s scientific advice

The Agency should, to the greatest extent possible, ensure that there is a separation between those responsible for providing scientific advice to a given medicinal product developer and those subsequently responsible for the evaluation of the marketing authorisation application for the same medicinal product. The Agency should ensure that at least one of the two rapporteurs for a marketing authorisation application has not taken part in any pre-submission activities concerning the medicinal product.

Orphan drugs

Orphan drugs (medicines developed to treat rare diseases) would benefit from up to 11 years of market exclusivity if they address a high unmet medical need. By 24 months from the date of entry into force of this regulation, the Commission should, following a consultation with the Member States, patient organisations and other relevant stakeholders, propose a needs-driven and goals-based Union Framework for Rare Diseases with a view to better framing and coordinating Union policies and programmes.

Transparency

To increase transparency of scientific assessments and all other activities, a user-friendly European medicines web-portal should be created and maintained by the Agency. The portal should provide information for all centrally authorised medicinal products, inter alia on safety, efficacy, environmental risk, patient populations, and where relevant information on antimicrobial resistance, shortages, and pending obligations for marketing authorisation holders. Sufficient budgetary resources should be allocated to the Agency to ensure its transparency obligations and commitments are appropriately implemented.

Medicine shortages

The marketing authorisation holder should notify and explain its decision to temporarily suspend the marketing of a medicinal product in that Member State as soon as possible and no less than six months before the start of the temporary suspension of supply of that medicinal product into the market of a given Member State by the marketing authorisation holder.

The Agency should be empowered to monitor shortages of medicinal products that are authorised through the centralised procedure, also based on notifications of marketing authorisation holders. Information on such shortages should be made available on the European medicines web-portal provided for in this regulation.

When critical shortages are identified, both national competent authorities and the Agency should work in a coordinated manner to communicate the necessary information to patients, consumers and healthcare professionals, including on the estimated duration of the shortage and available alternatives, and manage those critical shortages.

Text adopted by Parliament, 1st reading/single reading

PURPOSE: to ensure the authorisation of high-quality medicinal products, including for paediatric patients and patients suffering from rare diseases throughout the Union.

PROPOSED ACT: Regulation of the European Parliament and of the Council.

ROLE OF THE EUROPEAN PARLIAMENT: the European Parliament decides in accordance with the ordinary legislative procedure and on an equal footing with the Council.

BACKGROUND: the Union pharmaceutical framework has enabled the authorisation of safe, efficacious and high-quality medicines in the Union, contributing to a high level of public health and a smooth functioning of the internal market of these products.

The Pharmaceutical Strategy for Europe marks a turning point with the addition of further key objectives and by creating a modern framework that makes innovative and established medicinal products available to patients and healthcare systems at affordable prices, while ensuring security of supply and addressing environmental concerns.

Addressing unequal patient access of medicinal products has become a key priority of the Pharmaceutical Strategy for Europe as has been highlighted by the Council and the European Parliament. Member States have called for revised mechanisms and incentives for development of medicinal products tailored to the level of unmet medical need, while ensuring patient access and availability of medicinal products in all Member States.

Previous amendments to the Union pharmaceutical legislation have addressed access to medicinal products by providing for accelerated assessment for marketing authorisation applications or by allowing conditional marketing authorisation for medicinal products for unmet medical need. While these measures accelerated the authorisation of innovative and promising therapies, these medicinal products do not always reach the patient and patients in the Union still have different levels of access to medicines.

The proposed revision of the pharmaceuticals legislation consists of this proposal for a new regulation and a proposal for a new directive, which will also cover orphan and paediatric medicinal products.

CONTENT: this proposal lays down Union procedures for the authorisation, supervision and pharmacovigilance of medicinal products for human use at Union level, establishes rules and procedures at Union and at Member State level relating to the security of supply of medicinal products and lays down the governance provisions of the European Medicines Agency (EMA).

This Regulation will not affect the powers of Member States' authorities as regards setting the prices of medicinal products or their inclusion in the scope of the national health system or social security schemes on the basis of health, economic and social conditions. Member States may choose from the particulars shown in the marketing authorisation those therapeutic indications and pack sizes which will be covered by their social security bodies.

Objectives

The objectives of the proposal are the following:

- guarantee a high level of public health by ensuring the quality, safety and efficacy of medicinal products for EU patients;

- harmonise the internal market for the supervision and control of medicinal products and the rights and duties incumbent upon the competent authorities of the Member States;

- make sure all patients across the EU have timely and equitable access to safe, effective, and affordable medicines;

- enhance security of supply and ensure medicines are always available to patients, regardless of where they live in the EU;

- offer an attractive innovation-and competitiveness friendly environment for research, development, and production of medicines in Europe;

- make medicines more environmentally sustainable .

The proposed regulation includes the following main areas of revision :

- promoting innovation and access to affordable medicines creating a balanced pharmaceutical ecosystem;

- modulation of the length of the market exclusivity for orphan medicinal products. For rare disease medicines, the standard duration of market exclusivity would be 9 years with the possibility of granting a one-year extension of market exclusivity, based on patient access in all Member States concerned;

- paediatric investigation plans for medicinal products for children, based on a medicinal product’s mechanism of action;

- measures related to antimicrobials and provisions on transferable data exclusivity vouchers. A voucher system will provide ‘transferable data exclusivity vouchers’ under strict conditions to developers of new antimicrobials. Such a voucher will grant an additional year of regulatory data protection to the developer of the priority antimicrobial, which the developer can either use for any product in their own product portfolio or sell it to another marketing authorisation holder;

- strengthening the scientific and regulatory support of the European Medicines Agency, in particular for developers of medicines that address unmet medical needs;

- enhanced pre-authorisation scientific and regulatory support;

- temporary emergency marketing authorisation;

- improving security of supply of medicines;

- a framework for activities to be undertaken by Member States and the Agency to improve the EU's ability to respond in an effective and coordinated manner to support the management of medicines shortages at all times;

- EMA capacity to inspect sites located in non-EU countries;

- reducing regulatory burden and providing a flexible regulatory framework to support innovation and competitiveness;

- improved structure and governance of EMA and the regulatory network.

Legislative proposal

PURPOSE: to ensure the authorisation of high-quality medicinal products, including for paediatric patients and patients suffering from rare diseases throughout the Union.

PROPOSED ACT: Regulation of the European Parliament and of the Council.

ROLE OF THE EUROPEAN PARLIAMENT: the European Parliament decides in accordance with the ordinary legislative procedure and on an equal footing with the Council.

BACKGROUND: the Union pharmaceutical framework has enabled the authorisation of safe, efficacious and high-quality medicines in the Union, contributing to a high level of public health and a smooth functioning of the internal market of these products.

The Pharmaceutical Strategy for Europe marks a turning point with the addition of further key objectives and by creating a modern framework that makes innovative and established medicinal products available to patients and healthcare systems at affordable prices, while ensuring security of supply and addressing environmental concerns.

Addressing unequal patient access of medicinal products has become a key priority of the Pharmaceutical Strategy for Europe as has been highlighted by the Council and the European Parliament. Member States have called for revised mechanisms and incentives for development of medicinal products tailored to the level of unmet medical need, while ensuring patient access and availability of medicinal products in all Member States.

Previous amendments to the Union pharmaceutical legislation have addressed access to medicinal products by providing for accelerated assessment for marketing authorisation applications or by allowing conditional marketing authorisation for medicinal products for unmet medical need. While these measures accelerated the authorisation of innovative and promising therapies, these medicinal products do not always reach the patient and patients in the Union still have different levels of access to medicines.

The proposed revision of the pharmaceuticals legislation consists of this proposal for a new regulation and a proposal for a new directive, which will also cover orphan and paediatric medicinal products.

CONTENT: this proposal lays down Union procedures for the authorisation, supervision and pharmacovigilance of medicinal products for human use at Union level, establishes rules and procedures at Union and at Member State level relating to the security of supply of medicinal products and lays down the governance provisions of the European Medicines Agency (EMA).

This Regulation will not affect the powers of Member States' authorities as regards setting the prices of medicinal products or their inclusion in the scope of the national health system or social security schemes on the basis of health, economic and social conditions. Member States may choose from the particulars shown in the marketing authorisation those therapeutic indications and pack sizes which will be covered by their social security bodies.

Objectives

The objectives of the proposal are the following:

- guarantee a high level of public health by ensuring the quality, safety and efficacy of medicinal products for EU patients;

- harmonise the internal market for the supervision and control of medicinal products and the rights and duties incumbent upon the competent authorities of the Member States;

- make sure all patients across the EU have timely and equitable access to safe, effective, and affordable medicines;

- enhance security of supply and ensure medicines are always available to patients, regardless of where they live in the EU;

- offer an attractive innovation-and competitiveness friendly environment for research, development, and production of medicines in Europe;

- make medicines more environmentally sustainable .

The proposed regulation includes the following main areas of revision :

- promoting innovation and access to affordable medicines creating a balanced pharmaceutical ecosystem;

- modulation of the length of the market exclusivity for orphan medicinal products. For rare disease medicines, the standard duration of market exclusivity would be 9 years with the possibility of granting a one-year extension of market exclusivity, based on patient access in all Member States concerned;

- paediatric investigation plans for medicinal products for children, based on a medicinal product’s mechanism of action;

- measures related to antimicrobials and provisions on transferable data exclusivity vouchers. A voucher system will provide ‘transferable data exclusivity vouchers’ under strict conditions to developers of new antimicrobials. Such a voucher will grant an additional year of regulatory data protection to the developer of the priority antimicrobial, which the developer can either use for any product in their own product portfolio or sell it to another marketing authorisation holder;

- strengthening the scientific and regulatory support of the European Medicines Agency, in particular for developers of medicines that address unmet medical needs;

- enhanced pre-authorisation scientific and regulatory support;

- temporary emergency marketing authorisation;

- improving security of supply of medicines;

- a framework for activities to be undertaken by Member States and the Agency to improve the EU's ability to respond in an effective and coordinated manner to support the management of medicines shortages at all times;

- EMA capacity to inspect sites located in non-EU countries;

- reducing regulatory burden and providing a flexible regulatory framework to support innovation and competitiveness;

- improved structure and governance of EMA and the regulatory network.

Legislative proposal