Earlier this year, my local haemophilia treatment centre invited me to take part in a clinical trial for a new anti-haemophilia medication. This new medication would involve administering subcutaneous injections every week that should help prevent any bleeds from starting without affecting my inhibitor level (immune response) to the medication. This would mark the first time I switched from an “on-demand” to a prophylactic treatment for my haemophilia and also potentially halt the build-up of scar tissue in my limbs from a life-time of intravenous treatment.
After the initial phone call from the research nurse, I later received the participant information sheet in the post. I was dismayed that the information “sheet” was actually 45 pages long and written in language that was at times too detailed, yet also written in inappropriate scientific jargon. The methods of the study did not dispel my despair. The study required participants to visit the clinic every week for the first month to have a research nurse administer the treatment until they were confident and competent enough to self-administer. Each of these weekly visits could take up to five hours. If the participant still experienced a bleeding episode between doses of the new treatment (i.e. breakthrough bleeding), then they needed to visit their haemophilia centre within 48 hours.
I declined the invitation to participate in the trial—not because I doubted the efficacy or safety of the new treatment, indeed it appeared it could greatly improve my quality of life—but because of the methodological design of the trial described above which appeared to place the burden of participation on patients rather than clinical research staff. I understand that such oversight is necessary to monitor patients for adverse events, ensure that breakthrough bleeds are managed appropriately, and protect the haemophilia centre and hospital from litigation. I live in the city relatively close to my centre, however, I would have trouble getting there every week for an appointment. Do they expect participants with full-time employment to take annual leave to participate and attend follow-up appointments? What about people with mobility problems or who must travel for work or live in rural areas? How will they attend the mandatory clinic if they are away from home and their haemophilia centre? These questions raise a further question: how did the trial advisory group involve people with haemophilia or their carers in the design of the study? Answers to these questions may reveal how trials actively, even if unconsciously, imagine patients as participants in their studies. It may also highlight trialists’ narrow understanding and appreciation of patients’ obligations, values and activities outside of the clinic.
Participation should not be a burden for patients. Trialists and patients should work together to identify reasonable expectations of participation that values the time and knowledge of researchers, clinicians, and patients. Favouring one group’s input over the others creates inequalities in the labour involved (whether as clinician, research staff, or patient-participant) and hierarchies of knowledge where patients’ contributions are usually the most undervalued. Trialists, therefore, should co-design studies with the appropriate patient population to discuss potential methods to minimise risk and safety issues while also appreciating the lives of people with these conditions outside of the clinic.
All people, regardless of disability or state of health, live with other responsibilities and relationships that require their attention and obligate their time. Despite the prominence as the defining characteristic for the trial’s inclusion criteria, a person’s illness is only one aspect of their identity—one that may not be as important as other ones, such as their gender, ethnicity, sexuality, religious affiliation, or occupation. We should no longer accept tokenist or “tick-box” approaches to patient and public involvement (PPI). Let patients determine what meaningful involvement means to them commensurate with their skills and other responsibilities in an ongoing, living exchange. Major funding organisations (e.g. United Kingdom Research and Innovation, National Institute for Health Research, and Wellcome Trust), the Department of Health and Social Care and the Health Research Authority may have roles to play to incentivise, promote, or regulate better PPI arrangements in health, medical, and social care research.
I am fortunate that haemophilia has not greatly affected my career goals. I am an academic health and social care researcher in my own right. As a researcher, I know that we can do better. In fact, we must. As NHS England celebrated its 70th birthday this year, we need to not only think about person-centred care but also person-centred research. The argument for scientific validity is no longer an adequate pretence to disadvantage people from participating in studies. We must develop innovative techniques for robust and rigorous research that also considers the lived experiences of participants as people with full lives, not only patients in the clinic.
Matthew Lariviere is currently a UKRI Innovation Fellow at the Centre for Research on Care, Labour and Equalities at the University of Sheffield with research interests in cross-cultural understandings of ageing and care, technological interventions, and methodological innovation in health and care research. He lives with severe haemophilia A.
No competing interests: I have read and understood BMJ policy on declaration of interests and declare that we have no competing interests.