"Politically Correct..."
“What I am about to say is likely not politically correct…”. I turned my attention back to the computer screen. The conversation and polite scientific debate between researchers, that was part of a virtual Parkinson’s scientific advisory board meeting I was attending, had unexpectedly taken on a more curious tone. As I braced myself for what was going to follow, I was entirely unprepared for the words that were spoken. “…Why do we need patient representatives? What do they add to this group?”. After a moment of awkwardness, a few spoke to their support for patient involvement, but unfortunately to me, this comment was a clear indication that in some cases the patient voice remains undervalued in the research process.
I’ve been living with Parkinson’s disease for 24 years and have been deeply involved in patient advocacy for much of that time, donating my time and experience to help this diverse and remarkable patient community with whom I share the daily, progressive, and ultimately disabling challenges that this disease brings. As a physician, I am well aware of the dated paternalistic nature that has, and can still, characterize the doctor-patient relationship. I have seen and experienced many an interaction where the patient’s views and insight have been marginalized. Situations where decisions are made, not based on the patient’s quality of life, but on the disease or symptoms being treated. Thankfully this is evolving, and I have witnessed the shift towards more patient-centric care, where the concerns, limitations and impact on life experience are foundational. This type of shared decision making has allowed patients to become empowered in their own disease management.
The research community in some cases has done the same, recognizing that patient input in clinical research is not only admirable, but necessary. The saying “Nothing about us without us” rings true for them and patients become a valuable partner in their work. But unfortunately, in many cases still, research is performed on patients, not with patients. And in these cases, patients are viewed mainly as a source of data and further involvement can be merely tokenistic.
So, to answer the question that was raised, the first answer is more ethical than anything else. How can the research community do their work independent of the community that their life work is committed to serve? How are the most impacted stakeholders not part of the equation? Who else knows the depth of this disease, the insidious, progressive impact on patients’ quality of life? Who else lives with the disabilities that this disease relentlessly creates? Who else can not only help with the direction of research based on the needs of the patient community, but add the sense of urgency to the search for better treatments and ultimately a cure?
Direction
And if those reasons are less compelling, one only has to look at the evidence. First, studies have shown that the patient perspective directs research that is grounded in relevant clinical need or research that is necessary to achieve that goal. One cannot assume researchers know all the subtle nuances of this pervasive disease that may in fact be significant contributors to poor quality of life for patients. The risk is that research questions may be scientifically relevant but may not be relevant to patients.
Design
Poor recruitment and retention of patients are major sources of research inefficiency – delaying studies, inflating cost, and potentially resulting in biased findings. Involvement of people with lived experience of the condition has been shown to be significantly associated with improved enrolment. Including patients from the very early stages of study design ensures patient centricity of study protocols in terms of practicality, recruitment materials and outcome measure selection, resulting in increased patient interest and improved recruitment and retention.
Take outcome measures. Currently we have no widely accepted, reliable, objective biomarker for Parkinson’s disease that may be used for diagnosis, monitoring progression or responses to treatment. The research outcomes we often use are clinical and rely on evaluation of the patient at a moment in time. They do not take into consideration the heterogeneity, variability, and unpredictability in symptoms that patients experience which differ day to day, sometimes hour to hour. Traditional end points may not be ideal and the choice of outcome measure for a study and may be improved by input from those that stand to benefit from the intervention. Even in disease states that have established target end points, there may be a mismatch in the goals of an intervention. For example, in a study that surveyed a group of patients with diabetes, the majority of patients surveyed would prefer that clinical trials include variables that measure the impact of interventions on quality of life, such as time to dialysis and blindness, rather than HgbA1C (a test that measures your average blood sugar levels over the past 3 months) alone. And in the field of oncology, end points that have to do with patient experience and quality of life while on treatment are important to those with cancer, not just traditional survival-based end points.
Beyond the design, the patient voice can also be influential in personalizing the need for funding to government, industry or philanthropic sources and can also be integral to the approval process by regulatory agencies.
Dissemination
Once completed, patients want to know if a research study has resulted in tangible, clinically relevant outcomes that will help to optimize their quality of life in a timely manner or has contributed to our evolving knowledge of this disease which is integral to further research. Currently the first source of information pertaining to research results is the media, and this source of information is often skewed and sensationalized. In contrast patients are able to help with the dissemination of clinical trial results or research in an appropriate context to the patient community, resulting in a realistic view and informed expectations. Well informed and educated patients are more likely to stay involved and support the research process.
This is not a divisive issue. As a patient I am indebted to the dedicated clinical and research communities that devote their efforts to understand this disease, find better treatments and ultimately a cure. Both the medical research and the patient communities need each other. Neither can succeed in isolation. Our goal is the same – to end the suffering and disability that Parkinson’s inevitably represents, to improve the lives that are currently, or in the future may be devastated by the challenges this incurable disease brings. But instead of asking why patients deserve a seat at the table, perhaps the more important question is how can we best collaborate to end this disease once and for all?