onsistently, patients participate in clinical trials for altruistic reasons, and this is never more true than when the clinical trial targets a disease with no cure, or a life-limiting diagnosis. Many are placing their hope in the trial that is running: that it will prolong survival and at the same time afford, critically, an increased quality of life and a delay of symptoms. As the industry tries to find cures for these illnesses, death naturally occurs. As many have said, life is a terminal condition; there is nothing new there, but how do we, as clinical researchers, design trials that are compassionate, can help patients have a good death, and can help caregivers both support their loved ones and themselves feel supported?
Death is the hardest of all the objective, measurable endpoints. There are numerous articles about overall survival endpoints, but in the context of patient-centric research, should we be redressing the balance between objective and subjective data? Gaining a greater understanding of what matters to the patient—could that be the path to more holistic treatment? Moving our focus increasingly towards quality of life and what is important to the patients and their families as they come to terms with their diagnosis and prognosis?
I know from experience that freedom from disease—both the symptoms and the treadmill that treatment can become—is critical to overall mental health and well-being. I believe that, as clinical researchers, we have both the power and the gift to design research that meets the desires of patients. But how do we do that?
Give Patients a Choice
True choice, the opportunity to make decisions, can feel out of reach when living with life-limiting illness. When my husband was first diagnosed, I read books, I changed our diets, I changed our exercise regimen. I needed to take control of what was uncontrollable, I needed to show active participation in the management of the disease. I did not want to be a bystander and let nature take her course. I did not want our lives to be governed by tests, results, treatment.
With the advancement of digital technologies, we now have the ability to design studies that allow data to be collected without the patient having to attend a physical site. We no longer have to ask them to spend hours in clinics, staring at hospital walls and wondering what news they are going to hear.
Where patients want or need flexibility, visits can be conducted over the internet and tests can be performed remotely. Patients, and their caregivers, can talk about what path is right for them with their clinical team. Some people might want to visit the site, but some days they might not want to. Sometimes that trip in the car, catching a bus, taking the time off work, is just one bridge too far. We can design studies where lack of physical presence at the site does not mean lost data and a threat to endpoints. So, rather than lose the patient or appear inflexible, we can let them make the call. But importantly, digital health is location agnostic: an ePRO can be completed in the clinic or at home, on a bus, or at the gym. And because of this, the same process can be used whenever the requirement is there, relieving the burden of sites having to master multiple processes in order to execute a study.
Open Communication Channels
Having a seat at the table is important and never more so than when dealing with a life-limiting illness. Listening to news once is hard, but having to repeat it can be more challenging, especially as people then say “Did you ask if…?” “Did you check that…?” But open communication channels between all the stakeholders within the person’s care environment really encourages engagement and investment.
During my late husband’s illness, I made all the “big” decisions; his brain trauma, disease progression, two craniotomies, and extensive radiotherapy robbed him of this ability. Over the course of the 13 years of his illness and decline, my faith and trust in our clinical teams was unwavering. I was supported throughout his final weeks, helping me make decisions and giving him the “good death” he wanted. I was living proof that engagement needs to go beyond the patient themselves. In today’s society, many families live apart, and engaging with the family, who are often decision makers, gives them the opportunity to nurture possibly the most important end-of-life relationships. By designing studies that incorporate caregivers, we are providing holistic treatment options. And technology makes it possible, using telehealth and patient portals to meet and share documentation.
Allow Patients to Identify as Themselves
Before identifying themselves as patients, people say they are a friend, parent, child, comedian, artist, sportsperson, animal lover, … the list goes on. We are all patients, but I doubt most of us would put “patient” in our top-20 list of self-identifiers. If we can design research studies that allow symptoms to be alleviated, to allow patients to be themselves again in the most critical time of their lives, to live their lives and invest their best hours in doing what they love, not doing what they have to do to keep themselves alive, then we are succeeding. We have the ability to give them freedom, to allow the clinical teams to monitor them safely from a distance.
In Conclusion
My final word, and what I live by when I am looking at study strategy, is to truly place yourself in the patient’s position and that of their loved ones. If the study strategy I help to design shows compassion and empathy, will allow patients to live and caregivers to breathe, then I have helped create something truly wonderful. I will have assisted patients to fulfil the main reasons that they take part in clinical research: to help advance science and the treatment of disease, and to help others who suffer.
Clare’s debut book, Choosing to Float, shares her family’s story of learning to cope with a brain tumor diagnosis. The book is available via her website: https://www.clarecampbellcooper.com/.