A. General rules on medicines

A medicinal product (medicine) is a substance or combination of substances that is used for the treatment or prevention casino terpercaya 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 medicines has been centralised through the European Medicines Agency (EMA) since its creation in 1993 and a centralised authorisation procedure was put in place in 1995 to guarantee the highest level of public health and to secure the availability of medicinal products. The main pieces of legislation 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.

This set of rules has been repeatedly updated, most recently in 2019 in accordance with the principles of Better Law-Making (amending Regulation (EU) 2019/1243). 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 (Regulation (EC) No 141/2000), medicines for children (Regulation (EC) No 1901/2006) and advanced therapies (Regulation (EC) No 1394/2007). More information on this topic is set out in section D.

B. Clinical trials

Clinical trials are systematic investigations of medicines in humans that are intended to study the efficacy 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. Standards have been developing progressively – both in the EU and internationally – since 1990 and are codified in EU legislation, a process that is mandatory for the pharmaceutical industry. 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. Following the development of a fully functional EU clinical trials portal and database, the regulation took effect at the end of January 2022.

C. Advanced-therapy medicinal products

Advanced-therapy medicinal products 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) 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. Directive 2004/23/EC on setting standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells is therefore of great relevance to these products. A committee for advanced therapies was created at the EMA with responsibility for assessing the quality, safety and efficacy of advanced-therapy medicinal products and 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. 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 which 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. To date, the EU has authorised few orphan medicines, and owing to the low number of people who are affected by rare diseases, research in this field has been neglected. With this in mind, different measures, such as the Innovative Medicines Initiative, have been established 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. A legislative proposal is expected to be presented within this legislative period. 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.

Orphan and paediatric medicines will also be addressed by the new pharmaceutical package of April 2023.

E. Medical devices

New pieces of legislation concerning medical devices and in vitro diagnostic devices were adopted in 2017. Medical devices cover a broad range of products, from simple bandages and glasses to special medical equipment used by doctors and hospitals. In vitro diagnostic devices are used for the external examination of samples taken from the human body, e.g. in a test tube (‘in vitro’ literally means ‘in the glass’).

Regulation (EU) 2017/745 and Regulation (EU) 2017/746 set the rules on placing medical and in vitro diagnostic devices (IVD) 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. The new regulations are a significant step towards strengthening patient safety as they 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 of the European Parliament and of the Council of 15 March 2023 amends the abovementioned regulations as regards the transitional provisions for certain medical devices and in vitro diagnostic medical devices. This new regulation introduces a staggered extension of the transition period provided for in Regulation (EU) 2017/745 on medical devices (Medical Devices Regulation – MDR), subject to certain conditions. It also deletes in both the MDR and IVD Regulation 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.

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