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Early patient engagement and partnership in pediatric trials - Dr. William van't Hoff, NIHR

'Engagement and partnership is really important for pediatric trials and I also think it absolutely delivers results, which is of course in everybody's interest.'

Pediatrician Dr. William van't Hoff is director of the National Institute of Health Research (NIHR) clinical research facility at Great Ormond Street Hospital in London and a clinical director for NHS engagement.

Dr. van't Hoff's describes the NIHR as 'a whole system health research network running through the UK providing opportunities for translational clinical research right through to Phase IV and supporting a large network of people to help Sponsors and investigators deliver their studies.' His own role within the network is in 'trying to help the NHS health system recognize and take on research as core business'.

As part of PCT TV at Partnerships in Clinical Trials Europe (Nov 2017), we sat down with Dr. van't Hoff - read or watch the full exclusive interview below.

If you could change one thing in clinical trials, what would it be?

'I think the opportunity for Sponsors is to have a new relationship with networks such as NIHR, but also networks that exist in other countries, and to use that partnership to change the way they work. Why should they do this? Because the older traditional model of clinical trials of Key Opinion Leaders and selected sites is not really delivering and it's at risk of not delivering in the future stratified medicine in vulnerable groups - in my own clinical field of pediatrics that's very relevant.

So what we're looking for is a different way of working. Partnerships offer the opportunity for early engagement with the whole national system, which gives much more accessibility to reach more patients and a key opportunity for patient engagement. I think part of the key change we need to see in a partnership is bringing the patient in early on; the concept of early engagement with a Sponsor in a network discussing what their development pathway is and where patients could contribute to get real life questions. This will help Sponsors deliver a protocol in collaboration with experts, Key Opinion Leaders and patients that should be more deliverable. That should reduce the time required to run the clinical trial, bringing the product closer to regulatory authorization with a better market approach. So, for me its all about early engagement in partnership.'


So it's all about the patients?

'It's very much about the patients, who are absolutely ready and willing to talk. All the pharma companies we interact with are also increasingly aware. The issue is how to join that up. One of the things we've been doing in NIHR is to create base of patients called patient research ambassadors. Anyone who has taken part in research can sign up and they can do as much or as little as they want at a local level at their own hospital, or at a national or international level.

Once we have that base of patients that we're developing, then the key is how do we interact them with industry who are asking for that engagement.'



As a pediatrician you will be aware of the European Directive requirement to conduct pediatric trials. Do you have a view about how the clinical trials industry can support those studies?

'Everything I've said about early engagement and partnership is especially important for rare groups including children. Before collaborative networks, and in the UK before NIHR, it was almost impossible to conduct meaningful children's pediatric research because no one site had enough children available to answer the study questions and the protocol.

With national or international collaborations, the power is there.For instance, we were able though NIHR to conduct the largest intensive care study in children with over 1500 children and we did the same the next year with a new group of children. So there are opportunities that pharma can realize through network engagement.

All I've said about patient engagement applies just as much to children. We've created groups of children aged 8 to 18 who meet every six to eight weeks at a weekend to discuss research, randomization, and placebo. They interact with researchers and tell them what's good and what's not good about their research and how it could be improve.

So I think that engagement and partnership is really important for pediatric trials and I also think it absolutely delivers results, which is of course in everybody's interest.'

Partnerships and patient engagement are key topics at T3: Trials, Tech and Transformation being held in Raleigh, North Carolina on May 30-31, 2018. Explore the agenda or book a pass from as low as $399.

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