The power of the Parkinson's patient according to Tom Isaacs: a call to action

For me, one of the biggest challenges and yet also the biggest opportunity is galvanizing people who live with Parkinson's from day-to-day to engage with their condition. If everyone with Parkinson's were to communicate their experiences of living with PD; if everyone participated in clinical trials; if everyone took the time to become more knowledgeable and was more committed to partnering the scientific community in the search for new treatments, then there is no doubt in my mind that we could significantly accelerate progress in Parkinson's research. Tom Isaacs The global Parkinson's community suffered a great loss with the passing of Tom Isaacs, a powerful advocate, spokesperson and visionary, one whose legacy will continue to have a profound impact on our goal to relegate Parkinson's disease to the history books. Fundamental to Tom's beliefs was the power of the patient voice; that central to the expert team of researchers and clinicians dedicated to solving this enigma of a disease, were patients themselves. That every aspect of research from its conception to development including clinical trials and the drug approval process by regulatory bodies, could be expedited and had a greater chance of success if patients were involved. His vision was not of a passive community of patients, the grateful recipients of the efforts of research, but instead active participants in the search for better treatments and a cure. He believed that in order to ensure progress, patients must be involved at every level, as a treatment journeyed from lab counter to pharmacy shelf and ultimately into the hands of patients. Although impossible to completely capture the breadth of his vision, there are a number of core principles central to his work, that we in the Parkinson's community can consider as calls to action. There is a … source which is as yet untapped and probably represents the most important opportunity for progress in Parkinson's and that is to capture information and experiences from us, the people living with Parkinson's from day-to-day. Who better than patients to provide information about this disease and its impact on their life experience with this illness? According to Tom, the equation was very simple. Experience plus expertise equals a fast-track to new treatments. But the experience part of the formula cannot be easily defined. As those of us with Parkinson's know, although we may share some symptoms, most of us experience our own unique version of this disease. We differ in terms of age of onset, symptoms, progression, response to treatment and prognosis. This variability in experience can inform researchers, and perhaps raise questions which may direct a whole new avenue of research in the future. That data must be captured and recorded, analyzed and studied. Tom recognized that the current standard Parkinson's measurement scales and tools were limited in that they only provide a snapshot of a patient's condition at any particular moment in time and don't take into consideration the inherent variability that exists throughout the day and from one day to the next. They also do not take into consideration each patient's unique experience with this disease or their quality of life goals. This discordance between which aspects of Parkinson's disease are measured by current evaluation tools and the symptoms that are particularly impactful, is an issue. If “to measure is to know,” then an accurate assessment tool that measures how this disease truly impacts patients is the first step in improving the relevance of treatment from a patient's perspective which in turn may improve quality of life for patients. The impact of this lack of comprehensive and objective measurement tools affects not only how research outcomes are defined and measured but also management decisions that direct patient care as well. The medical model has evolved from paternalism to individualism. Information exchange is now the dominant communication model, and the health consumer movement has led to the current model of shared decision making and patient-centered communication. The move from a more paternalistic model of patient care to patient-centered medical management is evolving. No longer are patients satisfied with being told what is best for them without their own input. Shared decision making is now the expectation of patients. This expectation extends to patients’ involvement in research as well. How can we expect patients to partner with the research community if they are not informed or made to understand the current state and future needs of clinical studies? Central to this process is communication. Any gap in communication between the research and patient communities needs to be bridged. 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. Central to successful communication is understanding that not only what is conveyed is important, but also how it is conveyed is equally critical. This involves using language that is easily understood by those without a scientific background. Currently for patients the first source of information pertaining to research results is the media rather than researchers themselves. This source of information is often skewed and can be sensationalized. The patient community must push for the reporting of clinical trial results in an appropriate context from the very source of those findings, resulting in a realistic view of the research and appropriate expectations. Too often have we as a community been duped by the hype surrounding a research study that in reality does not yield tangible, clinically relevant results for patients. An educated and informed patient is an empowered patient. An empowered patient is more likely to take action and become involved. We must recognize that although money is crucial to enable progress, teamwork is the only thing that can realize it. Funding is vital for research to proceed and patients according to Tom, lend credibility and relevance to either a public appeal for fundraising or in the application process for other sources of financial support. It becomes much more personal and provides real context when patients’ stories and experiences are included. Collaboration between researchers and patients to leverage funds from third parties including government agencies, industry or philanthropic sources is necessary to ensure success. In addition, according to Tom, the neurodegenerative patient community itself is a powerful and untapped resource for raising funds either through their influence on policy makers, their commercial investment in industry or philanthropy. Engaging with regulators such as a country's national authority that evaluates and monitors the safety and efficacy of therapeutic products is important. By making them aware of the patient experience of Parkinson's and the community's unmet needs, they are better able to make informed decisions regarding funding and approval. Advocacy in this regard, can make a difference for example, in the case of fast tracking potentially impactful therapies. In order to make an informed decision, payers too need to understand the impact of a therapy on patient quality of life, and how that is more beneficial or advantageous than existing treatment options. This can have a profound effect on getting new therapies into the hands of patients. The only way we can develop new treatments is to ensure rigorous testing in clinical trials. Tom also believed that advocacy was more than procuring funding for clinical trials and should include involvement in all aspects of treatment development. It is in our best interests to ensure that our voices as a community are heard, that we have input into the clinical trial process. Researchers first and foremost must know our concerns so that the outcome measures of any research that is undertaken, are relevant. Although patient involvement is recommended in trial design, we must advocate for more robust participation and input. That trials and their inherent risks are directed towards the most pressing needs of our community. Once established, patients need to be part of the protocol development, to ensure the protocol being used meets the needs of participants and that possible adverse outcomes are defined with patient experience in mind. And that the information that is directed towards patients, is comprehensive and unambiguous, allowing participants to make informed decisions about their involvement. Patients also need to become advocates in their own communities, advancing patient involvement in clinical trials. For a new neurologic treatment to get from the lab counter to the pharmacy shelf it can take decades and well over a billion dollars. Approximately half of that time is spent in clinical trials. Yet close to 85% of all clinical trials are delayed due to recruitment difficulties and a shocking 30% fail to recruit a single subject. Any delay a study faces due to difficulty in finding participants, leads to a huge waste of resources, money and most importantly time. There are a multitude of reasons for this lack of participation, some logistical such as accessibility or not meeting the study criteria. But more significantly is that there is often a lack of knowledge and a number of myths about clinical trials that may influence a potential participant's decision. Education and dispelling myths and misconceptions about clinical trials is key to increasing informed and continued research participation. There are a number of roles that patients can play to improve research participation, recognizing first and foremost that without our involvement, research cannot progress. Tom Isaacs was an inspiring force, a kind and humble individual whose loss will always be felt. But even without his leadership we must continue the fight. We as a community must continue to collaborate with all other stakeholders to realize a future without this debilitating disease. In the words of Tom, “We, as patients, represent an enormous and untapped opportunity to bring urgency (experience, focus, passion and feedback) to the delivery of the science and to become a significant resource in the quest for a cure. Without such participation, there is little hope that new products will truly meet the actual needs of the people they are made for, nor will they be delivered with the urgency that is required.” None.

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