Documents considered by the Committee on 25 April 2018 Contents

12State of Paediatrics Medicines in the EU

Committee’s assessment

Politically important

Committee’s decision

Cleared from scrutiny; drawn to the attention of the Health Committee

Document details

Report from the Commission: State of Paediatric Medicines in the EU—10 years of the EU Paediatric Regulation

Legal base


Health and Social Care

Document Number

(39173), 13779/17, COM(17) 626

Summary and Committee’s conclusions

12.1In its assessment of the impact of the EU’s Paediatric Medicines Regulation,155 the European Commission concluded that the Regulation had boosted the development of paediatric medicines, but that these positive developments were not spread evenly across all therapeutic areas. They were often linked to research priorities in adults rather than children and there had also been issues about the availability of licensed medicines and long deferrals of clinical trials.

12.2The Parliamentary Under-Secretary of State for Health (Lord O’Shaughnessy) has responded to queries raised by the Committee in our Report of 10 January 2018. In particular, the Minister makes a commitment to undertake a discrete piece of analysis, considering: the impact of the Regulation in the UK specifically; the possible implications of EU exit on paediatric medicines development; and the future of paediatric medicines development for the UK. He says that this work should be concluded by the end of 2018, but warns that the Government would not be able to publish anything that would prejudice the UK’s negotiating position.

12.3Writing before the Prime Minister’s Mansion House speech—in which she expressed an intention to seek associate membership of the European Medicines Agency—the Minister explains current arrangements for cooperation between the EU and third countries in this area and emphasises the UK’s desire for continued close cooperation.

12.4We welcome the Minister’s commitment to a discrete piece of analysis on paediatric medicines development in the UK in the specific context of the UK’s withdrawal from the EU. We recognise the potential sensitivity of this material as the UK negotiates its exit from the EU, but we would caution against an assumption that none of the assessment could be published.

12.5We emphasise the desirability of including clinical trials within the Government’s analysis. Continued cooperation on clinical trials, including the ability to share and access data through mechanisms such as the European clinical trial database (EudraCT), is crucial to the ability of UK paediatricians to engage in research on rare congenital diseases in particular.

12.6Since the Minister wrote, the Prime Minister has expressed the Government’s intention to seek associate membership of the European Medicines Agency (EMA). We note that EU legislation on the EMA currently makes no such provision and would therefore need to be amended. Equally, however, the European Council guidelines on the negotiation of the future relationship between the EU and the UK appear not to preclude some form of UK non-voting participation in EU agencies in the future. The Health Committee is taking these issues forward with the Department.

12.7We will monitor with interest the Department’s work on paediatric medicines development and evolving discussions on future engagement between the EU and the UK. We clear this document from scrutiny and draw this chapter to the attention of the Health Committee.

Full details of the documents

Report from the Commission: State of Paediatric Medicines in the EU—10 years of the EU Paediatric Regulation: (39173), 13779/17, COM(17) 626.


12.8The Paediatrics Regulation sets up a system of obligations, rewards and incentives, and puts in place measures to ensure that medicines are regularly researched, developed and authorised to meet children’s therapeutic needs. The Regulation obliges companies to agree at an early stage of development a paediatric investigation plan (PIP) with the European Medicines Agency (EMA). In its report, the Commission suggested that further scrutiny was required of why major therapeutic advances had failed to materialise in diseases that are rare and/or unique to children and which, in many cases, were equally supported by the orphan legislation.156 Full details on the background to, and content of, the Commission’s report were set out in our Report of 10 January 2018.

12.9In his original EM, the Minister was unable to confirm the extent to which the Government agreed—or disagreed—with the report. This was largely because the report’s conclusions were based on an EU-wide sample and further work would be needed to confirm whether the conclusions applied to the UK. The Minister nevertheless confirmed that, while the UK was still represented at the EMA Paediatric Committee, the UK would work with other regulators and stakeholders to undertake the activities proposed in the report. The degree to which the UK would collaborate with the EU on paediatric medicines post-Brexit was still to be confirmed.

12.10When the Committee first considered the document, at our meeting of 10 January 2018, we asked the Minister to:

The Minister’s letter of 6 February 2018157

12.11The Minister makes a commitment that the Medicines and Healthcare products Regulatory Agency (MHRA) will undertake a discrete piece of analysis on paediatric medicine development in the UK with support from the DHSC. The analysis will consider:

12.12The Minister explains that the analysis will be completed by the end of 2018 so that outcomes can be fed into wider EU exit work. He expresses confidence that the Committee will understand that such analysis will be sensitive during this time of negotiations with the EU, and will appreciate that the Government has a duty not to publish anything that could risk exposing its negotiating position. However, he assures the Committee that undertaking this work will ensure future decisions on paediatric medicines regulation in the UK are backed by a solid foundation of evidence.

12.13The Minister re-iterates the UK’s position on medicines regulation post-Brexit in the following terms:

“we wish to retain a close working partnership with the EU. Our overall aim is to ensure that patients in the UK and the EU continue to access the best and most innovative medicines, and are assured that their safety is protected through ongoing cooperation and the strongest regulatory framework for all medicines, including paediatric medicines in every respect.”

12.14Regarding current third country engagement with the EU on the development of paediatric medicines, the Minister explains that the European Medicines Agency (EMA) and medicines regulators in third countries cooperate in a number of ways on regulatory approaches to development of paediatric medicines. He notes that, while there are different legal requirements in relation to medicines between the EU and third countries, there is a great deal of voluntary collaboration between regulators around the world. This collaboration includes the MHRA and EMA, and aims to promote effective and streamlined approaches to paediatric drug development internationally.

12.15The EMA, he says, holds regular meetings with other non-EU regulators to discuss global development plans for paediatric medical products (such as internationally coordinated paediatric trials) and to exchange information on specific products and other topics related to development of drugs for children. The intention is to avoid the unnecessary duplication of paediatric clinical trials and to maximise the exchange of information on safety and efficacy of paediatric medicines. The EMA has also arranged joint workshops with international partners on the development of specific paediatric regulatory guidelines to foster common approaches.

12.16The Minister goes on to reference the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA), which is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children. Enpr-EMA’s main objective is to facilitate studies in order to increase availability of medicinal products authorised for use in children. Participation is not limited to networks from EU countries and Enpr-EMA works with international partners specialising in the regulation of medicines for children including the World Health Organisation (WHO) through the EMA’s membership of the Paediatric Medicines Regulators’ Network (PmRN) and with the US Food and Drug Administration (FDA) through the EMA’s existing interaction on paediatric therapeutics.

12.17In terms of prospects for future collaboration between the EU and the UK, the Minister concludes:

“This range of international collaboration activities illustrate well that the regulation of medicines and the sharing of best practice already happens globally, not just within the EU. The UK already collaborates with countries both within and outwith the EU on a range of regulatory issues, and intends to do so once we leave the EU, including on paediatric medicines. It shall continue to be in the best interest of both the UK and EU to collaborate and support the development of better medicines for young patients whether they are in the UK or in the EU as illustrated in the Commission’s 10-year report. The Paediatric Regulation has been achieving its aims of increasing the number of well-evidenced medicines for children with age-appropriate formulations in the EU, but more can be done to reach its full potential.”

12.18The Minister emphasises that the UK is fully committed to continuing and evolving the close working relationship with its European partners. Its aim is to ensure that patients in the UK and across the EU continue to be able to access the best and most innovative medicines and be assured that their safety is protected through the strongest regulatory framework and sharing of data. The Minister believes that the health needs of children will be best served by a continued close relationship between the UK and EU and its associated paediatric networks.

12.19Finally, the Minister emphasises that there is no immediate prospect of any change to the UK’s regulatory relationship with the EU in the light of the planned post-Brexit implementation period and the conversion of EU law into UK law. Longer term, he says, the UK will be pragmatic in establishing UK regulatory requirements, bearing in mind the needs of patients and industry, and giving sufficient time to consult on, and implement, any new requirements.

Previous Committee Reports

Ninth Report HC 301–ix (2017–19), chapter 5 (10 January 2018).

155 Regulation (EC) No 1901/2006 of 12 December 2006 on medicinal products for paediatric use.

156 Regulation (EC) No 141/2000 of 16 December 1999 on orphan medicinal products. Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union. Due to the small usage, the pharmaceutical industry has little financial interest in their development.

157 Letter dated 6 February 2018 from the Lord O’Shaughnessy to Sir William Cash MP.

Published: 1 May 2018