Medicines III [EU]
Medicinal products for paediatric use
This law aims to ensure that medicines for children are fully adapted to their particular needs. It lays down specific obligations on the pharmaceutical industry, accompanying these with rewards and incentives.
Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on products for paediatric use and amending Regulation (EEC) No 1768/92, Directives 2001/20/EC and 2001/83/EC and Regulation (EC) No 726/2004 (see amending acts)
Purpose
This law aims to:
encourage high-quality research into the development of medicines for children;
guarantee – over time – that the majority of these are specifically authorised for such use ;
ensure the availability of accurate and helpful information on paediatric drugs.
Companies must draw up paediatric investigation plans containing data on the use of a medicine in children when applying for authorisation to market their products. In exchange, and as an incentive, a company receives a 6-month extension of its supplementary protection certificate – a form of intellectual property right. For orphan medicines, a manufacturer has an extra 2 years’ market exclusivity.
An independent paediatric committee inside the European Medicines Agency advises on questions raised by children’s medicines and is responsible for scientific assessment and approval of the paediatric investigation plans.
The law established:
an EU inventory of the therapeutic needs of children to focus the research, development and authorisation of medicines;
an EU network of investigators and trial centres to carry out research;
a system of free scientific advice for the industry;
a public database of paediatric studies;
EU funding to promote research into off-patent medicines for children.
In 2013, the Commission published a report which noted that, given the development cycle of medicines, it would require at least 10 years to gain full understanding of the law’s impact. It pointed out that the requirement for companies to screen every new adult product for its potential paediatric use means that this factor is now an integral feature of a product’s overall development.
References
Act
Entry into force – Date of expiry
Deadline for transposition in the Member States
Official Journal
Regulation (EC) No 1901/2006
OJ L 378 of 27.12.2006
Amending act(s)
Entry into force
Deadline for transposition in the Member States
Official Journal
Regulation (EC) No 1902/2006
26.1.2007
–
OJ L 378 of 27.12.2006
RELATED ACTS
Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency (Official Journal L136 of 30.4.2004)
Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use (Official Journal L 311 of 28.11.2001)
Directive 2001/20/EC of the European Parliament and of the Council of 4 April 2001 on the approximation of the laws, regulations and administrative provisions of the Member States relating to the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use (Official Journal L 121 of 1.5.2001)
Report from the Commission to the European Parliament and the Council Better medicines for children – From concept to reality: General report on experience acquired as a result of the application of Regulation (EC) No 1901/2006 on medicinal products for paediatric use (COM(2013) 443 final of 24.6.2013 – not published in the Official Journal)
EU rules for new medical products based on genes and cells
Scientific advances bring new medicinal products based on gene or somatic-cell* therapy and tissue engineering* to treat human diseases. Since December 2008, legislation aims to ensure these are both safe for patients and can be available throughout the EU.
It lays down rules for authorising, supervising and monitoring advanced therapy medicinal products, known in the jargon as ATMPs. These can be used, for instance, in correcting knee cartilage defects in adults. The legislation protects patients from scientifically unsound treatments.
Key Points
— The legislation creates a Committee for Advanced Therapies (CAT) within the European Medicines Agency. Its role is to give scientific opinions on the quality, safety and efficacy of advanced therapy medicinal products.
— The Committee for Medicinal Products for Human Use bases its authorisation decisions on the CAT’s opinions.
— Once authorisation is granted, the item is considered safe for human use throughout Europe.
— Manufacturers must comply with detailed labelling and packaging rules.
—  Manufacturers must ensure that each product, and its raw materials, can be traced from initial sourcing through packaging, storage and delivery to its final destination.
— Hospitals and other medical locations must maintain traceability systems for both patients and the products used.
— Manufacturers must specify the remedial measures they will take if an authorised product is found to cause adverse reactions.
— Where there is particular cause for concern, the European Commission may request a manufacturer to establish a risk management system to identify, prevent or minimise any possible risks.
— The Commission draws up detailed guidelines on good clinical and manufacturing practice specific to advanced therapy medicinal products.
Background & Context
A Commission report of March 2014 examined the status of advanced therapies in the EU and how the regulation had worked in practice. It noted that the legislation had established a recognised framework for assessing new advanced therapies.
Significant research takes place in the EU into advanced therapies. The EU’s clinical trials database, EudraCT, recorded up to 250 between 2004 and 2010. Almost 70 % of the entities involved were SMEs or not-for-profit organisations. Multinational pharmaceutical companies accounted for under 2 %.
KEY TERMS
* Somatic-cell therapy: experimental method of cloning genes and reintroducing them into cells to correct an inherited disease.
* Tissue engineering: manufacture of organs to be implanted and used in the human body.
ACT
Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004
REFERENCES
Act
Entry into force
Deadline for transposition in the Member States
Official Journal
Regulation (EC) No 1394/2007
30.12.2007
–
OJ L 324, 10.12.2007, pp. 121–137
Amending act(s)
Entry into force
Deadline for transposition in the Member States
Official Journal
Regulation (EU) No 1235/2010
1.1.2011
–
OJ L 348, 31.12.2010, pp. 1–16
RELATED ACTS
Report from the Commission to the European Parliament and the Council in accordance with Article 25 of Regulation (EC) No 1394/2007 of the European Parliament and of the Council on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 (COM(2014) 188 final of 28.3.2014)