Published: 21 September 2023
In 2019 the NIHR collaborated with the commercial sponsors of research, clinicians and patients to pilot a new patient engagement service designed specifically for the life sciences industry. Since then, the NIHR has supported a number of life science companies and CROs (Contract Research Organisations) across a range of patient engagement projects. One recent project focused on a programme of early phase breast cancer research. A company representative, summarised the drivers behind the recent activities:
“Patient involvement is recognised as best practice, we are actively encouraged to have those interactions with patients when designing trials. For example, in previous projects we have gained incredible feedback on study materials around issues such as health literacy and informed consent.
“But we realised that we could improve our studies further, and make them more accessible, if we collaborate with patients at an earlier stage of study development. That’s when a colleague signposted us to the NIHR.”
The company approached the NIHR explaining that they have a growing pipeline in second line treatments for HER2 positive and HER2 negative metastatic breast cancer. Consequently, the patient engagement request this time around was broader than looking at one specific trial. The company representative explains:
Seeking the patients' perspective
“There are three elements to this engagement. The first is for us to better understand the lived experience of metastatic breast cancer. For instance, we were keen to discuss the burden of symptoms: Does the disease have an impact on their work situation, or their leisure and social activities? This information will help us to avoid creating further burden when we're designing new trials.
“The second area was asking for feedback on a proposed study design. We want to know how participants feel about different dosing schedules, the volume and frequency of hospital visits, and preferred ways of collecting quality of life data.
“Thirdly, we wanted to gauge the participants’ interest, awareness and understanding of pathways and processes for early access to medicines. We explained that the efficacy and safety data for medicines made available through these routes tends to be from smaller, earlier phase trials, compared to the conventional route via larger, later phase drug registration trials. The key difference being the size of the body of evidence. So the question here was: How do they feel about medicines going through early access pathways?”
The NIHR team set to work and engaged four breast cancer patients and one carer to take part in a virtual patient insight session. Mary, one of the patients, heard about the opportunity via a support group linked to Make 2nds Count - a UK-wide charity focusing on secondary breast cancer.
Mary was diagnosed with breast cancer in February 2010. In 2020 she learned she had secondary breast cancer in her stomach, which progressed into her bladder the following year. Since her initial diagnosis, Mary has undergone surgery and multiple treatment regimes including; three kinds of oral chemotherapy, two types of intravenous chemotherapy, two sets of hormone injections and 25 sessions of radiotherapy. Mary’s cancer stabilised after she was able to access a newly approved drug in 2022.
Previously Mary had participated in document reviews for clinical trials, but she was keen to share her experiences further:
“I was motivated to take part because of my own personal situation,” explains Mary. “I was working my way through treatment after treatment and I wanted to know what else might be available. I also believed that clinical trials were going to possibly be my answer ahead.
“I'm also very passionate about patients having a say in research and evolving treatments. I have a daughter and I want to make sure that she, and the 31 women who die of secondary breast cancer every day in the UK, have greater access to new treatments and don't have the worry, stress and anxiety of limited treatments.”
But having never before engaged directly with a pharmaceutical company, Mary wasn't sure what to expect:
“I was quite apprehensive about it from the social [perspective] and also from the point of view that I thought it was maybe going to be quite scientific and perhaps my knowledge was not going to be sufficiently adequate to participate effectively. I felt that perhaps I was going to be listening a lot more - hearing about scientific innovations and new treatments.
“But I needn't have worried at all because it was so welcoming and it was so interactive. The session was genuinely about hearing each participants’ voice, and their concerns. I think that's really important for the researchers to hear what it is like to be actually living, day-to-day, on treatment. And I think it's really important that the researchers understand that people with secondary cancer, in particular, just want to get on with living, as opposed to just existing.”
Key ingredient: The independent intermediary
NIHR patient insight sessions are planned and delivered in a way that enables a frank and open discussion. Two NIHR representatives and two colleagues from the pharmaceutical company joined this particular insight session, with the five patients/carers. All participants are fully briefed on their roles and responsibilities in advance. This includes the company representatives who often have information to present. Crucially, it is the NIHR representatives who facilitate the conversation.
The company representative believes that the NIHR’s role, as the independent intermediary, is key to ensuring everyone feels at ease:
“We were quite clear on our role in the session being to present the information and then [the NIHR facilitator] generated discussion around the questions we wanted to explore. It felt like the right approach, rather than us asking the questions directly.
“It takes a real skill to make everybody feel comfortable in that kind of situation,” she continues. “We are there from a pharmaceutical company, with five participants who are going through something really significant, and yet they were so open with us. Being able to create an environment where everyone felt they could share was outstanding.
“All the participants were incredibly engaged and what really surprised me was their openness to share their experience of living with metastatic breast cancer. Not just in terms of their symptoms and treatment, but also the social and financial impact on their lives.
“We went into the insight session with the preconception that we knew quite a bit about the disease. But when you speak to people who are actually living with the disease, you realise that you understand only a very small amount of what they are going through. It was a very humbling experience.”
Translating patient insight into action
The team at the pharmaceutical company is now exploring how they can implement the feedback. Some of the insight is already translating into tangible actions. which will improve the experiences of future clinical trial participants. The company representative shared some examples:
“The discussion around dosing regimes was extremely interesting. Understanding why one participant would prefer a muscular injection once a month in hospital, compared to taking a tablet daily at home, is really helpful for us when we start to think of who might be suitable, or not suitable, for a trial. That kind of information is tremendously helpful for the researchers designing and conducting the trials.
“The insight we gained about collecting Patient Reported Outcomes (PROs) was also exceptional. For example, we had assumed that reducing the frequency of requesting PRO feedback would reduce the burden of participation. Our draft protocol suggested using one slightly longer questionnaire, once a month. In contrast, the session participants told us that a shorter questionnaire, taking 5-10 minutes, once a week, was fine. They also agreed that digital reminders would be helpful as brain fog is a common symptom of chemotherapy.
“Another key point that one of the participants made was that PRO questions should be more subjective. Instead of asking; can you score your activity or your emotions today? They said we should be asking questions like: Did you feel well enough to go for a swim today? Did you feel well enough to phone a family member today?
“We shared our learnings with our global team. We can already see changes being made in one trial in particular where one of the questionnaires is being adapted to make it more accessible and easier for participants to use. That is a direct response to the feedback in this engagement.”
Following the insight session the team from the pharmaceutical company have written to the participants thanking them for their involvement and outlining which aspects of their feedback they expect to be able to implement. This follow up is valued by those who take part and is essential to inspire and motivate future participation, as Mary explains:
“From a personal point of view, I feel that I'm doing something to help the future generations who are going to experience this illness. I can now see that it's not that difficult and that the people who are involved in developing treatments do actually want to hear your voice. They're genuinely interested in your opinion.
“This experience has helped me become more determined to make my voice heard in future. If someone asked me - should I get involved in this process? I would say ‘absolutely’! You feel good that you are getting your opinions out there and that you are helping to shape future drug treatments.”
The company representative expressed their intention to provide further feedback when the study protocol is finalised:
“We continue to discuss the outputs of this engagement activity internally and we are exploring potential solutions to the different points raised. We are keen to share as much detail as possible with the participants when the trial opens and we can concretely see what changes have been implemented.
“Beyond that, we have a whole pipeline of molecules in metastatic breast cancer that are at an earlier stage in clinical development. I'm confident that we will adapt future trial designs at the conceptual stage to further implement what we have learned.”
Reflecting on the experience as a whole, the company representative concludes with advice to other companies who are yet to embark on their patient engagement journey:
“What has really stood out, for me, is how much the patients want to be involved and want to contribute to science. That's hugely motivating for us. We understand the value patients add to our work and if you've not done it yet, you should actually be asking - why not?
“My advice to other companies would be that if they have any reservations, or if they're unsure about working with patients, having NIHR as the mediator is a great way to do it.”
Learn more about our patient engagement service for life sciences industry.