EVERY year on May 20, Clinical Trials Day commemorates the clinical study conducted by James Lind in 1747 to investigate treatments for scurvy. It is an occasion to recognise scientific progress and the contributions of researchers, healthcare professionals, regulators, sponsors, patient organisations and, most importantly, the people who participate in clinical research.
However, Clinical Trials Day should be more than a celebration of scientific milestones. It should prompt us to ask a fundamental question: Who is research really for?
Clinical trials are not solely about generating data, securing approvals or developing new treatments. Their central purpose is to improve lives. Behind every protocol is a patient, a caregiver and often a family navigating hope and uncertainty. Each participant brings more than a diagnosis. They bring prior healthcare experiences, present concerns and expectations for the future.
Patients must be recognised as equal partners
One of the greatest misunderstandings within the healthcare research ecosystem is the assumption that patients are simply recipients of expert knowledge. Patients may not possess formal qualifications in medicine or clinical research, but they possess another form of expertise: lived experience.
During CRM Trial Connect, Dato’ Dr Saunthari Somasundaram, President of the National Cancer Society of Malaysia, highlighted this imbalance. She observed that conversations about patients are still frequently conducted by healthcare professionals, organisational leaders and policymakers when a person with direct lived experience should also be at the table.
Her own family’s experience illustrates the gap. Her mother survived seven different cancers, yet clinical trial participation was never discussed with her –not even once. If a family with strong medical connections was not informed about possible research opportunities, we must consider how many other patients may never hear about clinical trials at all.
Malaysia has policies, ethical frameworks and research structures. However, the gap between having a framework and implementing it meaningfully remains significant. Patient participation cannot be reduced to obtaining a signature on a consent form or inviting a representative to attend a meeting after all major decisions have already been made.
Patients should be engaged early enough to influence the questions being asked, the design of the study, the outcomes being measured and the way information is communicated. Their contribution must have a visible impact on decision-making.
The principle of “nothing about us without us” should not be treated as a slogan. It should be reflected throughout the research process.
There is sometimes a belief that patients understand only their individual experiences and therefore cannot contribute to wider research decisions. This overlooks the fact that expertise is developed through learning and opportunity.
Researchers and clinicians were not born with an understanding of research methods, clinical medicine or healthcare systems. They were educated and given opportunities to develop that knowledge. Patients and caregivers should similarly be provided with the training, information and platforms required to translate their lived experience into meaningful research contributions.
A person living with a condition every day may recognise burdens and changes that are not captured by conventional clinical measurements. Caregivers understand the practical consequences of treatment schedules, side effects, repeated hospital visits and complex medication regimens. They know which outcomes genuinely improve daily life and which outcomes may appear statistically significant without making a meaningful difference to the patient.
This is particularly clear in rare diseases, where patients and families may spend years searching for a diagnosis and become highly knowledgeable about conditions that many healthcare professionals rarely encounter. However, the same principle applies across cancer, chronic illnesses, infectious diseases, disabilities and other health conditions.
Patients need more than promising research
Clinical trials depend heavily on trust. Yet the relationship between patients and the research system often contains an inbuilt imbalance.
For clinical research to serve patients, we must ask more than whether a trial is scientifically promising.
First, is the trial available? A suitable clinical trial may not be offered in the patient’s country, state or hospital, leaving them with no opportunity to participate.
Second, is it accessible? Even when a trial is available, patients may not know about it, receive a referral or understand the enrolment process. Information must therefore reach patients in a clear and timely way.
Third, is participation affordable? Although the treatment may be provided free of charge, families may still face transport, accommodation, meals, childcare and loss of income. These additional costs may make participation unrealistic.
Fourth, is the trial acceptable and practical? Its design should respect the patient’s culture, faith, diet, disability, age and family responsibilities. A medicine may be difficult to swallow, contain unsuitable ingredients or require frequent hospital visits that a patient or caregiver cannot manage.
Finally, is it sustainable? Patients deserve to know what happens after the trial ends. Will an effective treatment become available in Malaysia? Will participants continue to have access to it?
Patients should not be asked to carry the risks and burdens of research only to discover that the resulting treatment will remain inaccessible to them. A trial that creates hope without a credible post-trial access pathway risks damaging rather than strengthening public trust.
When lived experience shapes science
Scientific progress is often celebrated through groundbreaking discoveries, cutting-edge technologies and medical innovations. Yet the true measure of progress is not found solely in laboratories or research papers, but in the lives of the people those discoveries are meant to serve.
For Dr Pooja Sharma, founder and CEO of APAR Health Clinical, years spent across surgery, clinical research, patient advocacy, philanthropy and research support have reinforced a simple but profound truth: the most enduring lessons in healthcare are often taught by patients themselves.
Clinical practice has a way of revealing humanity in its purest form. A terminally ill patient, despite facing the end of life, found the strength to ask whether a young doctor had taken the time to eat during a demanding residency shift. A pharmacist, entrusted with administering the first dose of a newly developed vaccine to his healthy infant, demonstrated unwavering confidence in the scientific process by placing his own child at its forefront. A child diagnosed with retinoblastoma, abandoned by parents overwhelmed by the burden of illness, not only survived but went on to establish a survivor support group, inspiring countless others through poetry and drama. These are not simply moving stories. They are powerful reminders of why research exists in the first place.
Scientific advancement can sometimes become preoccupied with metrics and technological breakthroughs. While these remain essential, they should never overshadow the human purpose that underpins every clinical trial. Behind every protocol lies an individual waiting for hope, better treatment or a chance at a fuller life.
The experiences encountered throughout a medical and research career continue to shape every discussion around science and policy, serving as a constant reminder that innovation without empathy risks losing sight of the very people it is intended to benefit.
Perhaps the most important question for every researcher, clinician and policymaker is not how advanced a new discovery may be, but how meaningfully it will improve a patient’s everyday life. When lived experience is allowed to shape science, innovation becomes more than progress – it becomes purpose.
Dr Akhmal Yusof is CEO of Clinical Research Malaysia while Nadiah Hanim Abdul Latif is President of Malaysian Rare Disorders Society.
