Well conducted patient and public engagement can have a positive impact on recruitment and retention. To really increase your chances of success you need to be engaging patients as early as possible in the clinical development process, says Dr William Van’t Hoff, a Clinical Director in the National Institute for Health Research (NIHR) Clinical Research Network.

In the UK, the NIHR has made huge efforts to improve recruitment into clinical trials over the last decade, with justifiable success. Last year the National Institute for Health Research (NIHR) Clinical Research Network (CRN), published record levels of research participation. Over 870,000 people took part in clinical research (including both commercial and non-commercial) during 2018/19, a 54 per cent increase from 2010/11. Drilling down to commercial contract research alone reveals an impressive 229 per cent increase in recruitment to 46,064 participants in 2018/19 from 13,987 over the same period.

Whist we celebrate these results, it is also important to focus on the need to further improve our performance in the set-up and delivery of research – offering research to participants quicker and completing studies to time and target is important to patients and industry alike. Working with patients can help enhance our performance. We use the term ‘patient engagement’ to talk about this important relationship between patients and the life sciences industry. Other terms may be used in other contexts but the key thing is the principle underpinning the approach, which is about collaborating with patients and the public throughout research design and delivery, rather than doing research ‘to them’ or ‘for them.’

In the UK meaningful patient engagement has been taking place across the NIHR for over twenty years; NIHR Involve1 was created in 1996. Activities such as research priority setting, advising on recruitment strategies and reviewing the wording of patient information leaflets have become commonplace. Yet it’s fair to say that patient engagement has, to date, been more visible in non-commercial research (funded by government, universities, research charities) than in commercial research (funded by the life sciences industry). This needs to change if the UK is to maintain its position as a global leader in clinical research delivery.

As a paediatrician and a researcher, I really see, and experience, the challenge of conducting clinical trials with children. There are many factors which influence the family’s decision whether or not to take part, but I would say that first and foremost we need to ensure that the ‘ask’ of the research is balanced with the burden of the disease.

For example, for a life-limiting disease like cancer you might expect a trial to be quite intensive in terms of treatment sessions, hospital visits and tests. But that same regime would be less acceptable for a long term condition like asthma, which many people manage with medicine, at home, on a daily basis. If we have a mismatch, in other words if the ask of the research is heavy where the burden of the disease is light, patients are less likely to participate and it will have a significant impact on a study, from approvals right through set-up and recruitment.

It’s really important to prevent a mismatch from happening and I believe the key is to ensure that there is an opportunity for patient engagement and input to protocol review. Spending time consulting with patients, representative of potential participants, should reap dividends in a study set-up and delivery. Patient’s experience of their disease and its impact on their daily lives can really help us achieve the right balance between the ‘ask’ of the research and the burden of the disease.

However, bringing patients and life-sciences’ researchers together is not easy and needs to be done appropriately. Some members of the public are unclear or suspicious about what motivates and drives the life science industry and how they operate. Codes of practice and regulations governing the life science industry, which are entirely appropriate, may seem like an impediment to this form of engagement at times. When the NIHR Clinical Research Network team held discussions with industry representatives about patient engagement, a number of practical issues were raised. These included:

  • Identification of patients/public who are willing and able to work with industry
  • Managing the legal requirements, professional standards and commercial sensitivities, often across different nations
  • Appropriate formats companies should use to communicate and work with patients/public
  • Recompense and thanks for patients/public for their time, commitment and expertise
  • Public perception of patient engagement with the pharmaceutical industry
  • Cost-effectiveness in the absence of clear evidence of positive impact

Last October the NIHR Clinical Research Network announced a pilot project which aimed to address some of these challenges while focusing on facilitating patient and public engagement activities earlier in the clinical development process – specifically at the protocol review stage. The initiative’s long term aim is to create a level playing field, on a national scale, in which patient engagement activities can take place unhindered.

Throughout 2018 we collaborated with patients and a global pharmaceutical company, Pfizer Ltd, to design and pilot this new service. The pilot involved two Pfizer studies looking at a new treatment for eczema which were both paediatric and adult studies. The NIHR Clinical Research Network team facilitated two meetings at Alder Hey Children’s Hospital NHS Foundation Trust, Liverpool, firstly with a group of young people2 and then with a group of parents/carers.

Sophie Evett, Feasibility Lead for Pfizer UK, who led Pfizer’s participation in the pilot, said: “At the study level, the clinician who wrote the protocol met with both patient groups in person, as did my colleague who supports recruitment for Pfizer globally. They were surprised at some of the questions they were asked by the young people in particular. Some of the questions were very scientifically technical, for example around how the drug might alter your immune system and what effects that might have on the body. The young people were not afraid to say what they felt and were very clear about what they wanted to see included in the informed consent and assent document. For the adult study, one element of the draft protocol relating to prohibiting other medications was amended as a direct result of engagement with the parents/carers, and the study has since been approved to move to site selection stage.

“Looking at the pilot more broadly, it took a little longer than expected to satisfy our legal and compliance teams initially, mainly because it was a completely new way of working. However, it was a worthwhile exercise because it has enabled us to work through the many legal and compliance challenges and develop documentation that we can use time and time again for patient engagement activities, regardless of the study, patient group or therapeutic area.”

A review of the pilot suggests that one of the factors contributing to successful engagement between early-adopter companies and patients is the role of an independent ‘go-between’ or facilitator – a role which the NIHR Clinical Research Network is undertaking. Keith Wilson, Patient Research Ambassador for Liverpool Heart and Chest Hospital NHS Foundation Trust, worked with NIHR Clinical Research Network on developing the pilot. Keith explains:

“The main challenge in the past has been around trust. Patients and the public have historically been wary of the life sciences industry, and some may still harbour some misconceptions.

“I thought the pilot was a great idea and long overdue but, at the same time, I was initially sceptical that the project would achieve the desired outcomes. However, having the NIHR Clinical Research Network – which has worked in partnership with our NHS for over a decade – facilitate the service has made an enormous difference. Patients love their NHS, and if the approach comes from the NIHR Clinical Research Network, patients and members of the public are more open to having those direct discussions with life sciences companies. The NIHR Clinical Research Network is absolutely instrumental and fundamental to brokering that deal.”

The pilot continues and is expected to publish case study material by the end of the year describing how this patient engagement process has an impact on clinical trial design. The ambition is then to establish a national framework for earlier patient engagement by 2020, with the CRN providing a clear route for life sciences companies to connect with relevant patients to help ensure patient-friendly clinical trial designs.


1. https://www.invo.org.uk

2. YPAG – Young Person’s Advisory Group –