The role of patient advocates in trial design, participation

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Published: 24 Jun 2017
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Sarah Liptrott - Eurpean Institute of Oncology, Milan, Italy

Sarah Liptrott spoke to ecancer at EHA 2017 about patient engagement and advocacy, following her role as co-chair of a conference session on bringing patient perspectives into trial design,

Paola Kruger talked to ecancer about involving patients in their own therapy options and how the patients input can be valuable within a clinical trial at IFCPE 2017.

Patient engagement is also discussed by Prof Gabriella Pravettoni, here, and Dr Ethan Basch spoke with ecancer at ASCO 2017 about the clinical benefits of patient reported outcomes, here.

ecancer's filming has been kindly supported by Amgen through the ECMS Foundation. ecancer is editorially independent and there is no influence over content.

The session today, I was invited by EHA to co-Chair the session run by the patient advocates and this was about clinical trials, innovative clinical trial designs, patient involvement in clinical trials and also adaptive pathways, so new ways of actually getting drugs out there and available to clinicians and to the patients.

What were some of the highlights from this session?

The session, as I say, was co-Chaired by Sophie Wintrich. Sophie is a patient advocate for MDS in the UK so as well as her experience in the advocacy field we had views from patients. We also had views from regulatory authorities and also from clinicians and then finally one of the doctors actually spoke about the new EU clinical trial regulation that’s going to be coming in in 2018 and how that is actually going to be, hopefully, moving forward clinical trials for patients.

The key thing, especially in haematology is the difficulty that we have in getting evidence of safety and efficacy in trials when we’ve got quite small populations. I know that’s true in other disease groups as well but especially in haematology. It’s making sure that we’ve got trial designs that can actually give us some concrete evidence. So it was really talking about adaptive pathways, so things that the EMA have brought forward, about how we can actually use those, use different innovative trial designs and get those drugs into practice a little bit quicker but making sure that we’ve got the correct evidence to actually support their use.

Patient powered research?

Yes. We also listened about involving patients in trial designs and things like that. It was interesting to see, we got a show of hands at the end of the session asking how many of those who had attended the session were pharma, which was about a third, we had patient advocates and we also had clinicians but there were a lot of patient advocates that were there, probably over a third. We asked how many were actually involved in clinical trials in the design, so asking for their opinion about clinical trials, how they were going to be set up, patient information, those kinds of things and hands dropped to about three. So it was really quite small numbers of patient advocates that are actually getting involved in the design of trials which is really quite important.

Maybe it will take the larger organisation, like you say from the EU, of reshaping trials to involve them from the start rather than trying to introduce yourself to the trials later.

Yes. We were talking today with some of the clinicians who had actually experience of involving patients in their design in how to get access to that particular patient group, getting patients involved, having a look at the ethical aspects of designing trials so that patients will participate and will want to stay in that trial so we’re not getting high drop-out rates even. So even thinking about those types of things and the future is getting them to share their experience with other clinicians so that people realise that you don’t have to be afraid of getting patients involved, it’s not going to slow things down and it can be really useful. But until we get a chance to show some concrete evidence of how useful that can actually be there is always going to be a little bit of reluctance or some uncertainty to take that first step.

There was of course the research that we mentioned from ASCO of Ethan Basch where patients feeding back their symptoms ongoing throughout the clinical trial was improving trial participation, chemotherapy endurance and outcomes even.

Definitely. This is something really important. There’s one thing, safety and efficacy, and when we’re talking about evaluating side effects of treatment in an objective way when you’ve got common terminology criteria that you can evaluate exactly what’s happening to patients. But what does that mean for a patient? So patient reported outcomes, what’s the burden of that symptom for a patient? It may only be a grade 1 diarrhoea but a grade 1 diarrhoea to a patient may mean can I actually go out of the house? Do I need to think about where there’s going to be the nearest bathroom? These can be really important things and those are the types of things that we need to capture as well when we’re doing clinical trials, yes.

Then there was a session yesterday that you mentioned.

The session yesterday, yes. This was another session about quality of life and also patient reported outcomes. This was really quite an interesting session; there’s some research going on, I think it’s Professor Salek and his colleagues and I think it’s Hertfordshire University. Now they’re actually doing research into developing a patient reported outcome and this will actually be within haematology. So they actually have been doing I think he said over 120 qualitative interviews with patients looking at the vast range of haematological malignancies that there are, haematological conditions. Also different stages of the disease, so newly diagnosed patients, relapsed patients. So having to do so many interviews to make sure that they’ve got saturation of data, if you like, to be able to make sure that they are identifying what the key problems are for all different types of patients at different stages within their journey. If I remember correctly, 2017/2018 they’re looking to conclude those items and then do some psychometric testing. So hopefully 2018, maybe 2019, that tool may be available actually for testing and that will be something really exciting.