Reflections on Patients as Partners 2020
My key takeaways from this year’s conference, held in January.
I wrote this blog before lockdown, and since then the world has changed drastically in many ways. But one thing that hasn’t changed is the need to ensure that patients and the public are involved in research — from the outset. There’s been lots of great progress over the last few years, but the coronavirus pandemic has highlighted that we still have a way to go before patient involvement is truly embedded as a core part of the research process. Sharing best practice and highlighting areas for improvement remain important at this time in order to ensure patient involvement becomes a higher priority for researchers and companies.
This year’s Patients as Partners Europe conference brought together patients, patient organisations, industry and other key stakeholders to share best practices for how to involve patients throughout the medicines development life-cycle.
A stand-out feature of this conference — now in it’s 4th year — is the genuine and demonstrable involvement of people affected by health conditions throughout the event itself. The program is developed with patients, public sector voices and industry, and patients were included as panellists and speakers throughout the program. There also seemed to be more patients in attendance compared to last year’s event, giving the opportunity for more engaging, thought-provoking discussions and helping keep the focus of the conference at the forefront at all times.
Below is a summary of my highlights and key takeaways from the event.
Raising the profile of Parkinson’s
I was delighted to see this year’s keynote delivered by Lesley Gosden, who has been living with Parkinson’s for over 15 years. In 2014, Lesley enrolled on the groundbreaking GDNF trial, funded by Parkinson’s UK. Since then, she has been sharing her experiences with others affected by the condition, giving talks up and down the country. Lesley has also been working alongside Parkinson’s UK and research teams from academia and industry, using her insights to help improve the way that Parkinson’s research is done.
The results of the GDNF trial revealed promising signs of the drug’s potential. Brain scans suggested that damaged cells were rekindled and participants reported significant and sustained improvements in their symptoms. But sadly, the crucial scientific assessments fell short of conclusively proving that GDNF was better than a placebo, and so the trial did not meet its primary endpoint.
Lesley spoke about the impact this had on her and fellow trial participants, and discussed how vital it is that researchers and companies learn from the trial rather than disregarding it as a failure.
“Failure is an opportunity, not an ending.”
For pharma companies, failure isn’t new — the success rate for clinical trials is frustratingly low. But for patients, Lesley said, failure can mean the end of hope. To keep hope alive, we need to embrace failure and learn from it, so that we can improve things for the future.
Another thing Lesley highlighted was the vital need for researchers to identify meaningful and accurate outcome measures for Parkinson’s trials that reflect participants’ experiences. Patient reported outcome measures can play a key role in increasing scientific integrity. “We’re trying to use standardised measures to measure an unstandardised condition”. Lesley urged researchers to think outside the box, and have courage and confidence to challenge the status quo.
“If we ask the wrong questions, we’ll continue to get the wrong answers.”
Increasing the emphasis on patient reported outcome measures
The importance of including patient reported outcome (PRO) measures in trials was consistently raised across the two-day conference, as was the need to move away from focusing only on quantitative measures.
Patient advocates highlighted the need for greater focus on quality of life measures, and the importance of looking more holistically at people’s lives throughout a trial. An industry colleague pointed out that primary outcomes are always quantitative, and noted that researchers are missing out on a wealth of rich insights that qualitative data would provide.
Importantly, simply having a PRO measure included in a trial doesn’t solve the problem — it’s vital to ensure that the PRO is measuring what researchers are intending it to measure. Patient reported outcome measures must be co-created with patients, to ensure they are fit for purpose.
At Parkinson’s UK, we’re working on a project doing just that — partnering with Parkinson’s Foundation, UCB and six patient advisors to co-create better ways to measure the benefits of new treatments tested in clinical trials. We’ll be sharing the outcomes of this pioneering collaboration later this year.
Highlighting the vital role of carers
For me, one of the most moving sessions at the conference was hearing from Grace and Alfred Samuels, a married couple who shared how Alfred’s diagnosis of prostate cancer in 2012 had impacted both of their lives.
Since diagnosis, Grace has been a crucial support for Alfred, playing an active role in his care and also supporting him to take part in a clinical trial which ultimately saved his life, they said.
Grace described the impact Alfred’s condition had on her and her family — both mentally and physically. Yet too often, the importance of her role as a carer and support for Alfred was overlooked and unrecognised. Rarely was Grace asked “how are you getting on?” by healthcare professionals. She described one experience where she attended an appointment with Alfred and was completely ignored by the doctor for the entire visit.
“Healthcare teams need to include caregivers in meetings — they give a vital perspective.”
Grace spoke about her unique perspective as a carer, describing how she would often notice things about Alfred that he would not recognise himself. These insights can be vital information for understanding a patient’s progression and experience. It’s crucial that researchers and healthcare professionals recognise this, and put in place the necessary steps to support carers in their active role as partners in research and care.
Increasing involvement in HTA processes
One area that was identified as an important focus for the coming years is increasing patient involvement in Health Technology Assessment processes.
Health Technology Assessment (HTA) is the systematic evaluation of new health technologies — including medicines — to assess their impact. The aim of HTA is to inform policy decisions about whether or not a health technology should be adopted, and which patients are most likely to benefit from it.
The panellists of the session noted that at present, there is not enough involvement of patients in HTA processes — and where there is, influence and impact feels limited. There is a strong push to increase patient involvement in the early stages of drug development. While this is crucial, it is also important that we have an eye on the other end of the continuum too.
Patients have a key role to play in this area. Understanding the unmet need in patient groups is hugely important for assessing the benefits and risks of new healthcare technologies. It’s therefore vital that HTA organisations integrate patient involvement into their processes.
At present, the Scottish Medicines Consortium are the only HTA organisation who hold their decision-making meetings in public, enabling patients and the public to be part of the full discussions. The panel discussed how other organisations can follow suit, and what processes need to be in place to enable meaningful patient involvement in HTA processes.
Developing processes and tools for meaningful patient engagement is the focus of the PARADIGM project — an IMI initiative co-led by the European Patients’ Forum and EFPIA. ‘Early dialogue with regulators and HTA bodies’ is one of the key decision-making points that the project is focusing on, and so it will be great to see what tools are available to support this when the project finishes later in 2020.
Summary
There were some really interesting discussions at this year’s conference, and I came away feeling challenged and invigorated, thinking about what I can do in my role at Parkinson’s UK to act on some of the points raised across the two days.
Towards the end of the second day, we were all asked to write down 2–3 action points on a postcard, which the conference team would be posting out to us in 6 months’ time. Here are mine:
- Explore how Parkinson’s UK can work more closely with HTA bodies and facilitate the involvement of people affected by Parkinson’s in HTA processes.
- Work to ensure carers and family members are not forgotten when it comes to research, and think about how we can do more to support carers and family members to contribute their insights to the research process.
I’ll be reporting back later this year! In the meantime, what one thing could you do over the course of this year to increase patient involvement in research? You could start by taking the PFMD pledge to patients and joining the growing patient engagement movement.
At Parkinson’s UK, we’ve been supporting researchers and people affected by Parkinson’s to connect remotely for a number of years now. So during this pandemic, we’re still keen to hear from researchers and companies who want to explore how they can work together with people affected by Parkinson’s to plan research. Visit our website or email us: researchinvolvement@parkinsons.org.uk.
