Medicines and Medical Devices
Medicines and medical devices are subject to the rules of the single market and have a direct impact on people’s health. A robust legal framework is in place to protect public health and guarantee the safety of these products. Access to affordable medicines, the fight against antimicrobial resistance, the ethical conduct of clinical trials, the use of artificial intelligence in medical devices and incentives for research and development are just some of the key issues the EU deals with in this field.
Legal basis
Articles 168 (public health) and 114 (single market) of the Treaty on the Functioning of the European Union.
Context
Member States are responsible for devising health policies and organising and delivering health services and medical care. Therefore, in this domain, the EU has a complementary competence that enables it to support and coordinate actions and adopt binding legislation concerning medicines and medical devices.
Achievements and current developments
A. General rules on medicines
A medicinal product (medicine) is a substance or combination of substances that is used for the treatment or prevention of diseases in human beings. With the aim of safeguarding public health, the market authorisation, classification and labelling of medicines has been regulated in the EU since 1965. The evaluation of most medicines has been centralised through the European Medicines Agency (EMA) since 1995 to streamline approvals and maintain a stable supply of medicinal products. The primary legislative frameworks in this area are Directive 2001/83/EC and Regulation (EC) No 726/2004, which lay down the rules for establishing centralised and decentralised procedures for marketing authorisations. These frameworks were amended by Regulation (EU) 2019/1243, which introduces specific measures to ensure the availability of medicines and to manage shortages within the EU.
In April 2023, the Commission published a proposal for a new directive and a new regulation, which, together, revise and replace existing general pharmaceutical legislation. On 10 April 2024, Parliament approved the two respective files, paving the way for negotiations with the Council in the future.
Once medicines are placed on the market, they are monitored throughout their entire lifespan by the EMA under the pharmacovigilance system, which records any adverse drug effects in daily clinical practice.
In addition to the general rules on medicines, specific regulations are also in place for orphan medicinal products for the treatment of rare diseases, medicines for children and advanced therapies. More information on these topics is available in sections C and D.
B. Clinical trials
Clinical trials are systematic investigations of medicines in humans that are intended to study the effectiveness and safety of a given medicine. In order for a medicine to be placed on the market, it must be accompanied by documents indicating the results of the tests that it has undergone – a step that is mandatory for the pharmaceutical industry. Standards have been developing progressively – both in the EU and internationally – since 1990 and are codified in EU legislation. The latest revision of the EU legislation from 2014 established harmonised rules for the authorisation and conduct of clinical trials (Regulation (EU) No 536/2014). Clinical trials must undergo a scientific and ethical review and must receive prior authorisation. Furthermore, they may only take place if the rights, safety, dignity and well-being of participants are protected and prevail over all other interests, and only if the trial is designed to generate reliable and robust data. Regulation (EU) No 536/2014 took effect at the end of January 2022 and established the EU Clinical Trials Information System to centralise submitted applications for clinical trials and provide the results of these trials, enhancing transparency and accessibility in clinical research.
C. Advanced therapy medicinal products (ATMPs)
ATMPs are a relatively new kind of product or pharmaceutical based on advances in cellular and molecular biotechnology and novel treatments, including gene therapy, cell therapy and tissue engineering. These complex products, which involve pharmacological, immunological or metabolic actions, cannot be treated in the same way as conventional drugs, and they require specific legislation as laid down in Regulation (EC) No 1394/2007 and Directive 2009/120/EC. Because of the risk of disease transmission that they pose, tissues and cells must be subject to strict safety and quality requirements. On 24 April 2024, Parliament approved a new regulation on standards of quality and safety for substances of human origin intended for human application (SoHO Regulation). At the EMA, a committee for advanced therapies was created to assess the quality, safety and efficacy of ATMPs, as well as following scientific developments in this emerging field of biomedicine, which has enormous potential for patients and industry.
D. Orphan medicinal products and medicines for children
Paediatric medicinal products are also specifically regulated (Regulation (EC) No 1901/2006) to ensure that they have been tested specially for children in an ethical way, that they meet the needs of children and that they have age-appropriate doses and formulations. Regulation (EU) 2019/5 amends Regulation (EC) No 1901/2006 to account for the fact that the specific obligations subject to financial penalties are now laid down in Regulation (EC) No 726/2004. Pharmaceutical companies carry out studies on children to obtain evidence about the safety and efficacy of new medicines before requesting marketing authorisation. The EMA’s Paediatric Committee assesses those studies and the data generated by them.
In the EU, rare diseases are those that affect no more than five in every 10 000 people. Orphan medicinal drugs are specifically designed to treat these illnesses. Regulation (EC) No 141/2000 lays down the centralised procedure for the designation of orphan drugs. Owing to the low number of people who are affected by rare diseases, research in this field is not very economically attractive. The EU has therefore launched the Innovative Medicines Initiative to encourage the pharmaceutical industry to develop orphan drugs. In 2017, the Commission began its evaluation of the legislation on medicines for children and rare diseases. Between May and July 2021, the Commission conducted a public consultation on this issue. In November 2022, the Commission published the Notice Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another. Given that rare diseases are a worldwide concern, the EMA collaborates extensively with its global counterparts to designate and evaluate orphan medicines.
In 2023, the Commission presented a proposal for a new regulation as part of the ‘Pharmaceutical Package’, which will repeal existing regulations on paediatric and orphan medicinal products. The proposal was approved by Parliament on 10 April 2024 and is awaiting the Council’s decision.
E. Medical devices
A medical device is any product used for medical purposes, including diagnosis, prevention, treatment, investigation or changes to anatomy, as well as contraception devices and sterilising medical equipment.
Regulation (EU) 2017/745 (Medical Devices Regulation) and Regulation (EU) 2017/746 (In Vitro Diagnostic Medical Devices Regulation) set the rules on placing medical and in vitro diagnostic (IVD) devices on the market and on related clinical investigations. These regulations came into force in May 2021 and May 2022 respectively. Devices are grouped according to their risk category, each of which has a specific set of rules. These regulations introduce more stringent procedures for conformity assessment and post-marketing surveillance, require manufacturers to produce clinical safety data, establish a unique device identification system for the traceability of devices and provide for the setting up of a European database on medical devices. Regulation (EU) 2023/607 amends the abovementioned regulations as regards the transitional provisions for certain medical devices and IVD medical devices. This new regulation introduces a staggered extension of the transition period provided for in the Medical Devices Regulation, subject to certain conditions. It also deletes in both the Medical Devices and IVD Regulations the ‘sell-off’ deadline after which devices placed on the market before or during the transition periods that are still in the supply chain would have to be withdrawn.
F. Antimicrobial resistance (AMR)
Antimicrobial agents are substances that kill or inhibit microorganisms, including bacteria, viruses, fungi and parasites. The use (and misuse) of antimicrobial agents is linked to an increasing prevalence of microorganisms that have developed resistance to such agents, thereby posing a threat to public health and significantly increasing costs. EU-level action to tackle AMR dates back to the late 1990s. The European One Health Action Plan against AMR, adopted in 2017, ensures effective treatment of infections by reducing the emergence and spread of AMR and boosting the development and availability of new, effective antimicrobials. On 1 June 2023, Parliament passed a resolution addressing EU efforts to counter AMR. Additionally, on 13 June 2023, the Council approved a recommendation on stepping up EU actions to combat AMR through a One Health approach. In February 2023, the Commission published Delegated Regulation (EU) 2023/905 supplementing Regulation (EU) 2019/6 as regards the application of the prohibition of use of certain antimicrobial medicinal products in animals.
Additional challenges
The EU continuously strives to implement initiatives to foster research and innovation in the pharmaceutical sector. Research framework programmes have always supported health-related research. The current research and innovation funding programme, Horizon Europe, will promote health-related research and respond to the current challenges by addressing topics such as lifelong health, environmental and social health determinants, mental health, non-communicable and rare diseases, infectious diseases, tools, technologies and digital solutions for health and care, and healthcare systems.
Other EU funding programmes such as the 2021-2027 EU4Health programme, which was adopted to improve crisis preparedness, and the 2021-2027 European Social Fund Plus, which supports access to healthcare, also play an important role. Furthermore, the EU has given considerable support to developing innovative drugs and urgently needed treatments, and accelerating patient access to new treatments via the Innovative Medicines Initiative and its predecessors.
Access to essential medicines is part of the right to health, according to the World Health Organization. However, access to health treatment is becoming more and more heavily dependent on the availability of affordable medicines. Findings show differences in sales of innovative medicines between different Member States, which can be attributed to economic disparities and differences in healthcare infrastructure. The problem has been exacerbated by the economic crisis. Parliament, concerned with this serious situation, has published several own-initiative reports on access to medicines. In 2017, Parliament adopted a resolution on options for improving access to medicines. Access to affordable medicines remains a priority for the current Commission, as emphasised in its 2020 pharmaceutical strategy.
At the EMA, a Task Force on the Availability of Authorised Medicines for Human and Veterinary Use provides strategic and structural solutions to tackle disruptions in the supply of medicines and ensure their continued availability in the EU. The structures and processes that the EMA set up for dealing with medicine shortages in accordance with Regulation (EU) 2022/123 on the EMA’s reinforced role are primarily focused on activities related to crisis situations.
Given the increasing concerns about shortages of certain medicines, which have been aggravated by geopolitical events such as the war in Ukraine, the energy crises and high inflation, the supply aspect of medicines also requires attention. To this end, in 2019, the EMA issued guidance on the detection and notification of shortages of medicinal products.
In December 2021, Regulation (EU) 2021/2282 on health technology assessment (HTA) was published. The regulation entered into force in January 2022 and will gradually apply as of January 2025. The new regulation defines a support framework and procedures for cooperation on the clinical assessment of health technologies at EU level, and common methodologies for the same. Among other things, it will help accelerate access to new medicines.
On 7 March 2023, the Council adopted the Commission’s proposal which aims to extend the transition period to adapt to the new rules under the Medical Devices Regulation.
Role of the European Parliament
Parliament has consistently promoted the establishment of a coherent public health policy and a policy on pharmaceuticals that takes into account public health interest and industrial aspects. Recent pieces of adopted legislation include regulations on clinical trials, medical devices and in vitro devices, as well as HTA. Parliament has advocated for patient safety to be strengthened during the legislative process. Non-legislative resolutions and debates on current issues, such as the use of artificial intelligence in medical devices, access to medicines or AMR, highlight the attention that Parliament attaches to ongoing challenges and emerging threats. During the hearing of Commissioner Kyriakides, Parliament further emphasised the need to act on those challenges.
In the 2021 negotiations on the HTA Regulation, Parliament was keen to ensure that HTA would be used to promote innovations that achieve the best results for patients and society in general, and would enable medical staff, patients and medical institutions to determine whether a new health technology is an improvement on existing health technologies, in terms of its risks and benefits.
For more information on this topic, please see the website of the Committee on the Environment, Public Health and Food Safety (ENVI) and the Subcommittee on Public Health (SANT).
Filip Karan / Christian Kurrer