In recent years, there has been a growing recognition of the importance of a patient’s voice in the trial design. As the term “patient-centric” becomes the new buzzword, patient groups and charities continue to advocate and highlight the need to include those living with health conditions in the cycle of research and development (R&D).

In an exclusive interview with Clinical Trials Arena, Nick Hartshorne-Evans, CEO and founder of Pumping Marvellous Foundation, shares his thoughts about the state of cardiovascular clinical trials and what needs improvement.

In 2009, Hartshorne-Evans suffered from chronic heart failure at the age of 39. After digging through the internet without any positive information, his mental health was challenged. But being an entrepreneur, he tended to see the positive side of things and started the charity in 2010, almost as a way to self-medicate, he explains.

The charity was set up to do two things: help people live with heart failure and advocate for patients at a high level such as policy, management, care plans and pathways, and have patient insights within that decision-making process. More than a decade later, Pumping Marvellous Foundation is a global leader in patient insights, working at local, regional and global levels across the UK and Europe.

Urtė Fultinavičiūtė: Quality of life is an important aspect for heart failure patients, but it is often missed in the regulatory decision-making and clinical trial design. Why is this the case?

Nick Hartshorne-Evans: I am not saying that everything is easy here, but getting hard endpoint data around mortality and cost is what the system does well. What the system does not do well is understand what quality of life is. We need to measure the quality of life better from a clinical trial research perspective. At the end of the day, after all the R&D, where the drug predominantly sits is within people's lives when they go home. How does that medication or treatment affect people?

What is really important is that we need to learn to measure the quality of life differently. It is not just in a sterile clinical trial research environment where you are using a number of certified health questionnaires that researchers use every time. That only impacts the patient cohort you are studying. I think there is a better way of learning about the quality of life and what it is like to live a life with a condition rather than a snapshot in time over a 12- or 36-month period.

It is quite a challenging conversation and I do not think the system fully understands how to measure quality of life because it is different for everybody. But surely, with technology and the realisation that quality of life is when people live with the condition at home 99% of the time and maybe 1% of the time is spent with doctors and nurses, that 1% is minuscule when compared to the 99%.

We recently did a whitepaper across multiple countries in Europe and what we saw is that quality of life was on a parallel of importance to mortality. While the system likes talking about the quality of life, it is working in opposition to what the patient wants. I always say that if we can solve the Covid-19 issue, we can work out how to measure the quality of life in people with long-term conditions.

The key driver here is the government. It needs to indicate that they want better measurements of quality of life. Therefore, life science industries and research facilities could design better ways of measuring it and implement that in research.

UF: What is the importance of including patients’ voices in clinical trial design? How might it benefit the patient and the sponsor?

NHE: It benefits everybody. You could be cynical about why you are developing treatments, but actually, you are developing treatments for patients. If it does not work, why develop it? The patients can give you helpful insights through the life cycle of R&D. It is also important for relevance because it is not just the clinical impact of medical treatment but also the psychological impact and patients can help package it up.

UF: Would you say that the changes need to happen at the government level first for everyone to change that mindset?

NHE: Unfortunately, it is health economics, and it is driven by money. The instigator needs to be the government as life sciences companies will question why they have to do this because it is an extra process that they need to incorporate in the R&D. It needs to be instigated from a payer’s perspective. We do not think about it, but the quality of life has a huge economic benefit. There is a big conversation going on in the UK about NHS asking GPs to prescribe health and wellness coaching.

Researchers do not bring the emotional reason why we do things whereas patients can. It is very important when you are trying to influence and change how we do things. Data by itself does not change things; it complements the emotions.

UF: What advice would you give to clinical trial sponsors so they could improve their cardiovascular clinical trials?

NHE: You should always design your research around who is going to use it. If the end user is going to be the patient, then always think about why a patient would want this. There is a lot of compliance and challenges about involving patients in early stage R&D, but it is really important to bring the user into the process right at the start as it is as important to keep them on board and in larger quantities as you go through the R&D cycle.

As you start Phase II or Phase III trials, you need to engage with the users of that medication. In the trials that we get involved in, we have patient and public involvement teams that are made up of patients who are potentially end users of this treatment and they will give you a real-life answer to the questions asked.

By involving patients, they can give their insights to life science companies to develop the right treatments for the right people at the right time.

What is also important is giving patients the sight of up-and-coming treatments because patient groups are the eyes and ears of the system. That does not mean that there are any conflicts or confidentiality issues because you are building awareness of what is going to be published in medical journals. As a patient advocate and leader, I know that what I see in medical journals, the chances are it is going to come through National Institute for Health and Care Excellence (Nice), and I am NICE’s patient expert. The more time I have to learn about this new treatment and a clinical trial, the better. Communication is always key.

It is not just the responsibility and domain of people in research to do that research. There are lots of people who have a stake in it. Apart from the government that reimburses research and development of that treatment, the person that should have the biggest stake in it is the patient. By involving patients, they can give their insights to life science companies to develop the right treatments for the right people at the right time.

UF: Is there anything else you would like to add that is worth mentioning?

NHE: It is also the responsibility of life science companies to democratise research. Oncology research does not have the same problem as cardiovascular. We need to make it more viewable, understandable, and relevant. The majority of the patients never talk to their doctor, physician or nurse about research. Whereas many patients are approached for research when they are diagnosed with cancer.

We need to take the research out of the labs, put it into the hands of the patients and the public and tell them that research is important. If we don't research, we don't get better at what we did. But you need to get people interested and enthusiastic and get them to believe that it is important.