Royal College of Paediatrics and Child Health (RCPCH) – Written evidence (LSI0026)

Science and Innovation

1. Research with infants, children and young people is essential to improve our understanding of childhood disease, and provide healthcare based on the best possible evidence. Currently child health research is not given the same presence as adult research. It is important to recognise that children are not just small adults – they need biomedical and health services research that takes account of their changing physiology and addresses their problems directly, generating evidence to improve the quality of the treatments and healthcare they receive.

2. The growing burden of long-term conditions that have a substantial component of their origins in early life – such as obesity, cardiovascular disease, vascular dementia and diabetes – is placing a strain upon the NHS and adversely affecting the health and economic wellbeing of the nation.[1] The infants, children and young people of today are the productive future workers of the nation, and the parents of the next generation. As such, investment in child health research is also an investment in both adult health and future generations[2]. The improvements in child health which result from paediatric research are therefore a key factor in the long term sustainability of the NHS. In order to encourage investment in turning child health research into innovation in treatment, the long-term economic benefits of a healthy future workforce must be highlighted.

3. In the UK, both public sector and industry funding for child health research is limited. In 2012, the total UK public and charitable annual research expenditure was approximately £2.2 billion[3]. Although infants, children and young people represent nearly 20% of the UK population[4], only around 5% of this research expenditure was directed towards their needs[5]. The RCPCH’s 2012 Turning the Tide report[6] highlighted that in order to increase the evidence base for clinical care, develop appropriate treatments for children and reduce the costs of healthcare in adult life, the UK must fund and carry out more paediatric research.

4. Clinicians are crucial to successful research endeavour that involves children. Turning the Tide[7] evaluated the extent to which paediatricians are able to support clinical research. It found that major barriers to progress in this field were the limited opportunities for paediatricians to gain experience in research and acquire core research skills and knowledge. There is a critical shortage of paediatricians in the UK who can both carry out research and train future researchers in this field[8],[9]. In 2015, a RCPCH survey of all consultant and SAS (staff, associate specialist and specialty doctor) paediatricians identified the current level of paediatricians’ involvement in research as low, with 80.1% consultants having no programmed activities (PAs) in their job plans for research and 50.8% reporting to undertake no research work[10].

5. For paediatric clinical trainees, there is limited formal training in basic research methods, research regulation, organisation and governance. As such, in their final years of training, many are not confident in basic research tasks such as taking informed consent for participation. Whereas in previous years all consultants were expected to participate in research, today the majority of newly appointed consultant paediatricians have little or no research experience – with one in ten holding a higher research degree, compared to one in three of the paediatricians nearing retirement[11].

6. In order to overcome these barriers to progress within child health research, support is needed for paediatricians who wish to carry out research through advocating for sufficient supporting professional activity (SPA) time for non-direct clinical care activities. In particular, time for research activity needs to be made available for general and community paediatricians, who are considerably underrepresented in terms of research output in comparison to subspecialist paediatricians[12].

7. Communication to paediatricians and their employers regarding the possible opportunities for funding should be improved, and collaborative work between organisations encouraged. Key research successes by paediatricians, particularly where research has led to change and better outcomes for children, must be highlighted and promoted to encourage greater involvement in child health research across all career stages. Similarly, by studying the processes for paediatric research activity overseas, knowledge from existing models of best practice could be incorporated into paediatric research activity in the UK.

8. Training in research methods and associated skills must be effectively delivered in both undergraduate and postgraduate curricula, so that all paediatricians have the opportunity to get involved in research and the skills to understand and utilise evidence-based best practice. Capturing medical students’ enthusiasm in their early years, encouraging more trainees to get involved in academic research, and advocating for sufficient SPA time for non-direct clinical care activities is crucial to ensuring that students, trainees and consultant paediatricians have the necessary skills, opportunities and support to contribute to child health research. Encouraging them to gain experience in child health research will also help to identify and develop the future research leaders in this field[13]. This approach is supported the Academy of Medical Science’s recent report, Enhancing the use of scientific evidence to judge the potential benefits and harms of medicines[14].

Industrial Strategy

9. Neither the Life Sciences Industrial Strategy nor the Life Sciences Strategy for Scotland make any specific reference to or recommendation regarding child health. Given the current limitations in both funding and capacity for paediatric research, a strategy which specifically addresses these barriers to increasing the UK’s level of paediatric research activity is necessary in order for the UK to become world-leading in this area of the Life Sciences sector.

10. Charities make a key contribution to UK child health research – the government must support and facilitate continued charitable contributions. Conversely the RCPCH has reported evidence of a lack of significant industry support, particularly in the areas of children’s biotechnologies, devices and nutrition research[15]. Funding for the implementation of the Life Sciences Industrial Strategy should be maximised through collaborative working across industry, academic and charitable sectors.

11. The RCPCH State of Child Health report highlighted data for several indicators of child health are not consistently available across all four nations of the UK. Across all but one of the reported indicators, children from deprived areas of the UK have much worse health and wellbeing than those from wealthier areas[16]. Throughout the NHS, infants, children and young people should benefit from innovation equally. Since visibility in data is a vital part of driving improvement and innovation, it is therefore crucial that data collected are representative of all areas of the UK. In order to ensure that the UK is the best place in the world for child health and associated research which improves outcomes, we must move towards a robust indicator framework which is consistent across all four nations.

NHS Procurement and Collaboration

12. The RCPCH strongly advocates for addressing the gaps in child health research through collaboration between stakeholder organisations and the NHS. Through this, the aim is to ensure that both child health research activity in the UK and the next generation of paediatric academics are supported. The combination of increased fiscal pressures within the NHS, limited funding for child health research and the decline in paediatric academic posts all highlight the need for organisations to collaborate in order to overcome barriers to progress, maximise research impact, and encourage innovation in this field. Key to this is ensuring close integration of research and core NHS services. Many processes are common to both clinical care and research. By eliminating the duplication of such processes, efficiency and quality can be improved whilst reducing NHS costs and the burden on families and healthcare staff[17]. Similarly, NHS employers must adopt a flexible approach to support paediatricians in maintaining up-to-date knowledge about clinical research as part of their continuing professional development, thereby increasing their ability to contribute to development and innovation through child health research.

13. The NHS England research plan is focused on driving the direction of research so that the necessary evidence is produced and applied in practice, encouraging and promoting research activity, and supporting a collaborative approach which enables the sharing and application of evidence in decision-making and practice[18]. However, the research plan makes no specific reference to paediatric research. Given the current obstacles to increasing child health research activity in the UK, specific strategies to address this issue must be integrated into future plans.

Responsibility and Accountability

14. Charities make a vital contribution to UK child health research. In 2012 the RCPCH found that paediatricians were twice as likely to receive a research grant from a local or national charity than from the National Institute for Health Research (NIHR), Medical Research Council (MRC) and Wellcome Trust combined. The limited research spend of the majority of national children’s research charities largely impedes their ability to support large clinical trials and research programmes on their own[19]. Supported by the Wellcome Trust and MRC, in 2015 the RCPCH launched the UK Child Health Research Collaboration (UKCHRC). This partnership aims to support strategic alignment of smaller charities and large funding bodies to optimise research funding and raise awareness of the need to increase and strengthen children’s research[20],[21]. The charity sector’s potential should be further maximised through formal collaboration with industry and academic stakeholder organisations. Responsibility for the implementation of the Life Sciences Industrial Strategy should be shared through this collaboration, facilitated by the UK Government. In order to measure progress, the group should report to a dedicated Life Sciences Minister who maintains overarching accountability to Parliament for the life sciences sector across the UK.

Brexit

15. Brexit will undoubtedly have a challenging and acute impact on child health services and research. In a 2017 report on the paediatric workforce[22], the RCPCH reported that UK’s reputation as a global leader in clinical practice, applied research, and innovation is being compromised. This is due to both the existing serious shortfall in the paediatric workforce, and the great uncertainty around immigration status and terms and conditions of employment for non-UK nationals, due to the UK’s upcoming withdrawal from the EU. More than 40% of consultants and SAS paediatricians obtained their primary medical qualification outside the UK[23]. As such, the RCPCH’s recommendation that Government action must ensure immigration rules allow entry to the UK for healthcare professionals whose skills will benefit the NHS is more pertinent now than ever. In order to foster a world-class life sciences sector, following Brexit the UK must still work to attract and retain high-quality international talent in research, as well as cultivating home-grown talent in this field. The UK must also remain open to examining the processes of paediatric research activity overseas in order to learn from models of best practice which could be replicated in the UK.

16. Loss of access to EU funding is highly likely to have a damaging effect on UK Life Science research. As discussed above, both public sector and industry funding for child health research is already limited in the UK. It is a concern that following Brexit, funding is likely to be allocated chiefly to high-profile areas. As a research area which has been comparatively low-profile with regard to funding, a priority must be to ensure that child health is not overlooked.

17. The single regulatory system at EU level has benefited children. Historically, variation in legislation and standards presented a significant obstacle to conducting transnational research on innovative treatments for children. In addition to this, infants, children and young people are not well-served by market forces in the development and supply of medicines. Due to EU legislation and policy initiatives, this variation has decreased over the past decade – contributing to an increase in research into the development of medicines for children in Europe[24]. The closely-coordinated European medicines regulatory network, which includes the European Medicines Agency (EMA) and national competent authorities of EEA Member States, helps to regulate medicines efficiently and effectively through pooled resources, accelerated exchange of information and reduction of the administrative burden. The regulation of medicines for children benefits from sharing human resources, expertise and data. To wholly remove the UK from this network would require a large and costly expansion in the capacity of UK regulatory agencies, and would harm UK children due to delayed availability of new medicines and delayed recognition of unsafe medicines. The infants, children and young people of the UK will be best served by the strongest possible involvement with and contribution to the EMA and European medicines regulatory network. The World Health Organisation (WHO) has described the reasons for regulating medicines and the functions of regulatory agencies[25]. Any changes following Brexit must address these factors, taking into account the needs of infants, children and young people.

18. Changes to the UK regulatory framework for research must maintain patient safety as paramount, whilst ensuring that the regulatory environment does not deter investment due to the increased costs which result from growing bureaucracy. The opportunities for children in the UK to take part in and benefit from research must not be reduced. In order for UK children to continue to benefit from sharing the results of research with European children, a priority must be to ensure that the UK remains open to collaboration with European research projects.

19. In Turning the Tide, RCPCH reported that particularly in relation to the needs of infants, children and young people, there was marked variability across the country with regard to research integration and the regulatory framework[26]. The changes in the relationship between the UK and Europe will inevitably impact upon the relationship of the four UK nations. In order to maximise research integration and impact, the regulatory framework must actively support child health research across England, Wales, Scotland and Northern Ireland consistently. There have also been concerns about the lack of necessary paediatric expertise in research ethics committees, and disproportionate risk assessments by insurers and NHS Trust Research & Development offices for research involving children[27]. In order to support growth and leadership in child health research in the UK, paediatric representation within governance processes must be facilitated to ensure that the necessary expertise are brought to the table in this area of the Life Sciences sector.

20. Infants, children and young people are a vulnerable group. It must be ensured that the UK’s withdrawal from the EU will not lead to unintended consequences such as delayed access to innovative treatments, or reduced collaborative research efforts to improve child health across the UK and Europe.

14 September 2017