Cancer clinical trials – how patient centric are they?
As part of Customer Faithful’s ongoing research work in oncology patient experience, we caught up with our former intern Georgina Powell - just graduated in biomedical science from Imperial College London. Having spent time working with the National Institute for Health Research’s oncology clinical trials team, we were keen to learn from her direct experience, and commissioned her to write a series of articles.
In this first blog, she explores how well clinical trials can claim to being truly patient centric.
In order to examine cancer clinical trials from a patient-centric perspective, my starting point was a simple question: “What are clinical trials for?” Their primary purpose is to gather data to contribute to scientific knowledge, and it’s indisputable that clinical research produces knowledge valuable for understanding human disease, preventing and treating illness, and promoting health to benefit society and future patients.
The need for cancer clinical trials to support saving lives is also clear. Cancer is the second leading cause of death worldwide after heart disease, responsible for 9.6 million deaths in 2018. Globally the number of new cases per year is rising and expected to reach 27.5 million by 2040. This can be attributed in part to better diagnosis, and to a growth and ageing in global population.
However, finding cancer drugs that work isn’t easy – with some studies of approved cancer drugs showing they were ineffective for 57% of patients, and chemotherapy rendering an overall survival of less than 5% in adjuvant treatment of breast, colon, head and neck cancers. These somewhat shocking statistics are due to the heterogeneity in tumours, each with distinct molecular dysfunctions. The treatment landscape of oncology is shifting from a “one drug fits all”, treating per tumour type, towards a more personalised approach.
Precision medicine
73% of cancer drugs in development today are ‘precision medicine’ which has large and mostly positive media coverage. But I’ve been fortunate to get a close up, practical experience of where these cancer trials start from a patient involvement perspective. From my time last year working with the National Institute for Health Research (NIHR) oncology clinical trials team at London’s Hammersmith Hospital, I’ve had a unique opportunity to facilitate patient recruitment directly from the ward on to trials and also to gain insight and reflect on what benefits clinical trials really have to patients more generally.
Dissemination of cancer trial research is often presented with ambitious and exaggerated claims of benefits. From the patients’ viewpoint, the new treatments may provide an illusion of certainty as they are based on their specific tumour. This hides the uncertainty in the behaviour of cancer and how the tumour will respond to treatment.
Precision medicine considers molecular and cellular features of the tumour and its microenvironment with additional factors such as genetics and lifestyle of the individual to create a tailor-made treatment. This molecule-specific therapy is particularly attractive to patients, since they are largely self-administered oral medications without requiring intravenous chemotherapeutic infusions.
Trials can be seen as an opportunity for a patient to take an active role in their own cancer treatment. However, since most trials are randomised, the patient is not given a choice of which treatment arm they receive. Not only does this affect the patient but also can bias the results of the study. A “matched” patient to a treatment arm may underestimate the risks. Conversely a “non-matched” patient may believe the therapy will not work, which could result in the over-reporting of adverse events.
A change in perception of the concept of precision medicine is needed among cancer patients, through publication of accurate lay summaries and the use and explanation of more generalised terms such as “therapy-based genomics”. Clinicians need to be trained to disseminate this information and be aware of misguided or unrealistic expectations.
I was shocked to discover that most oncology clinical trials have the primary goal of minimising disease progression to extend patient survival in terms of months. The sad truth is that only a minority of patients experience an objective response to the treatments. What’s a year of life worth? Patients are also asked to complete highly personal surveys on their physical and emotional well-being to assess the QALY (Quality Adjusted Life Years) that the new drug will bring.
Access benefits and risks
All patients have to be screened before entering a trial which itself can be a stressful process with a risk of complications in providing a tumour biopsy. However, all patients screened do obtain valuable genetic information about their tumour and learn more about their disease. Although this genetic information will aid researchers, it remains to be determined whether establishing a comprehensive tumour molecular profile directly improves patient outcome.
The opportunity to participate in a trial extends hope for patients and proves difficult to acknowledge their terminal status rendering them less receptive to end-of-life discussions and advanced care planning. The practice of informed consent needs to be updated with more information provided to the patients about benefits from the screening process such as the opportunity to undergo another already approved treatment.
It is important to remember that clinical trials are not a way of patients being able to bypass rigorous clinical testing for expedited access to unproven treatment options. Due to the increasing complexity of decision making, there is a need for further training of oncologists to ensure knowledge is accurately translated from clinical trials and implemented in practice.
Of course, trials have tremendous possibility to benefit patients. Participation in trials provides extra consultation, more frequent monitoring, and access to the latest medications, therefore study participation can be a significant advantage over standard medicine. Although frequent visits to the hospital are time consuming, patients have reported higher levels of care and attention. Jonathan Sheffield, CEO of NIHR, shares this optimism, saying “there’s a growing body of evidence which suggests that research-active hospitals have lower mortality rates, and better patient outcomes.” One certain rewarding aspect of participating in a trial is that patients are contributing to research that may save lives in the future.
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Georgina Powell facilitated the NIHR oncology clinical trials team during 2019/20 in screening, data entry and patient recruitment from wards and clinics. Her critically analysis of current systems in the NHS used for recruiting patients to clinical trials was submitted as part of her thesis for her biomedical science degree from Imperial College London.