Part 8: The Role of Patients

The photo above was taken by Anders M. Leines and is part of a campaign called This is Parkinsons. Click here to learn more.


On the most basic level, the model governing who makes decisions in the development of new therapies can be summed up in pretty simple terms. Clinicians tells scientists what the needs of patients are, and then scientists go into their labs and use the tools available to try and address those needs. Patients get looped in typically well after most of the important decisions have been made.




It is the way of medicine, and in most branches, it is the only practical way for the system to work. Patients usually have neither the expertise nor the capacity to contribute to decision making.

But neurology and neuroscience are different. They deal with a system that is much more complex and difficult to access than any other in medical science. These constraints have made it incredibly difficult for this model to come up with effective therapeutic interventions.

However, there is an opportunity, particularly in Parkinson’s disease, to use a different model that just might produce more effective outcomes. Because the disease progresses relatively slowly, patients have the time needed to develop some expertise in their disease, and many take it upon themselves to do so. To date this expertise has been underutilized, however, times are changing. There is a growing recognition among clinicians and researchers of the added value brought by involving expert patients at various stages of therapeutic development.

Below is a collection of excerpts taken from interviews I conducted with experts in the field on the value of involving patients in therapeutic development. I hope more will follow in their lead and put these words into practice.



on the Importance of Engaging Patients in Therapeutic Development


Prof. Andrew Lees

Professor of Neurology at the National Hospital for Neurology and Neurosurgery at Queens Square, London and University College London


“In my view the greater problem is that the doctors dictating research agendas in medical science see fewer and fewer patients. Many chairmen of medicine in prestigious Ivy League universities have given up clinical duties entirely.”

“In the last twenty years clinical pharmacological studies to evaluate new compounds have almost disappeared from university departments because of legal restrictions. I think hope lies now with small companies depending on venture capital that are prepared to take risks and work closely with academics and patients.”




Prof. Hilal Lashuel

Associate professor of life sciences and director of the laboratory of molecular and chemical biology of neurodegenerationat at Ecole Polytechnique Fédérale de Lausanne (EPFL)


“Today, researchers rely on clinicians and pathologists to tell us how these diseases affect people. We (scientists) are then left to our own devices to connect what they tell us with what we see in the lab. Adding to the problem is that we are preoccupied with reductionist approaches that take us away from the true complexity of these diseases.

When I came back from the HDdenomore event in the Vatican (an event where scientists interacted with patients and families affected by Huntington’s disease), I began to question what we know about the human pathology and whether our current understanding truly reflects what happens in the human brain. Could the clinical heterogeneity be explained by a pathological diversity that we are not able to capture?

It quickly became clear to me that we know very little about what is actually happening in the brains of people with these diseases and that there is an urgent need to revisit and reconsider our simplistic and reductionist view of the pathology of neurodegenerative diseases, which does not take into account the large biochemical, structural and morphological diversity of the pathology in different patients and in different regions of the brain.

I personally believe that more effective interactions and collaborations between scientists and clinicians with patients and their families is crucial to understanding the complexity of neurodegenerative diseases and accelerating the translation of research from bench to clinic to novel therapies. My experience has been that such interactions inform and inspire new research directions and priorities that are aligned with patient’s needs.”


Dr. Kalpana Merchant

Former Pharmaceutical Executive now Chief Scientific Officer at Vincere Biosciences


“I think that when academic and industry scientists get to interact with patients and see how their work could ultimately be applied, the therapeutic goal becomes a beacon for them. We are all human beings at the end of the day, we want to do the right thing. At the start of any therapeutic program it is important to interact with patients to understand the disease better and to set the right goals in mind. As programs progress towards the clinic, getting patient input on the design of the trials is also critical. Over the last decade, there has been a clear recognition that patient engagement and input in all aspects of a therapeutic program – from formulation development to the design of clinical trials to patient recruitment strategies – are critical for the ultimate success of a therapy. But patient engagement remains sub-optimal; we need more interactions at scientific and strategic conferences, as well as all the way through the drug development process.”



Prof. Ray Dorsey

Professor of Neurology and Director of the CHET at the University of Rochester Medical Center


“It starts with the needs of the patient. If you think about the way we deliver care for people with Parkinson’s disease, it couldn’t be done much worse. We ask what are usually older individuals with impaired mobility, living in suburban or rural environments, to be driven by over-burdened care givers, often long distances, to receive care once every 3-6 months. Instead, clinicians should be delivering care to patients on their terms. My colleagues and I have been using video conferencing to connect to patients in their homes and we have had great success. Other colleagues are doing us one better by actually visiting patients in their home.

Our current setup is designed to optimize efficiency from the standpoint of clinicians. We ask patients to go to holding cells and wait there until the doctor is ready, and then they get processed, much like cattle, some places even assign a number, to then be seen for a very fixed period of time before being told when to come back. We now have technology in the 21st century to allow us to deliver care wherever people live and should be taking advantage of that.”


Prof. Roger Barker

Professor of Clinical Neuroscience and Honorary Consultant in Neurology at the University of Cambridge


“Patients can also offer fantastic insight into what risks they are willing to accept. Generally, regulatory bodies are very conservative with what trials they allow to go forward. I think having patients say they are prepared to take the risk, after having heard all the evidence, would help.

Another area patients should have a voice in is how we design the clinical trials around these therapies. This is particularly true in cell therapy as the current double-blind, placebo controlled trial model forces us to do sham surgeries. This is pretty controversial, especially with cell replacement therapy because it generally takes 2-3 years to receive benefit, meaning some patients will spend three years in such a trial and receive nothing. Patients should have a say in whether or not they are willing to go through that.”



Dr. Todd Sherer

Chief Executive Officer of The Michael J. Fox Foundation for Parkinson’s Research


“One of the things that we are learning from the PPMI study is how varied Parkinson’s is from individual to individual. The only way we can get that information is through the participation of people with Parkinson’s. They are the true experts in this disease, and only through partnering with them can we increase our understanding and ultimately develop effective treatments.”



Dr. Jon Stamford

Neuroscientist Turned Patient Advocate


“I have absolute, unwavering faith (in basic science). I don’t see any other route. But I don’t think we should channel all funding into primary research, that is a significant part of it and that it where the major leaps are going to come from, but those decisions have to be informed by patient input. Patients have to lead the agenda and be able to say to scientists, ‘these are my priorities and these are the things that need addressing.’ The idea of scientists alone making the decisions about what they perceive to be Parkinson’s is insane. If I think back to who I was before (my) diagnosis, I wouldn’t trust myself to make those decisions.

Scientists need to be aware of what patients are experiencing. A clinician might know what Parkinson’s looks like, but patients know what it feels like, and I think there is quite a distinction between those two things. It’s easy to describe dystonia for instance, but until you have experienced it you really only have a dim picture of it. I believe that more research needs to be led by patients.”




  1. Thank you for presenting another interesting piece. This trend is proving to be a better method for the patient living with Parkinson’s Disease. Providing opportunities for others to have a more meaningful and purposeful life is key. Discovering new pathways for improved treatments, and ultimately a cure, by working together is the goal.

  2. Beni,

    Just a thought:

    There seems to be consensus that Patients involvement would be beneficial in looking for therapies for PD. However, the big question is how: How to capture and systematize patients’ experience. How about you start leading this effort. Maybe taking to the big data people can give you some directions. Start thinking about your own experience and try to incision how this would aid researchers and physicians.


    Sent from my iPhone


  3. Another great post, Ben. The quotes from the experts you cite really help support the Advocate Project Dashboard concept, esp. w.r.t. to research prioritization and study design projects. More followup on this soon.

    Wishing you a very Happy New Year!


  4. I see you don’t monetize, don’t waste your traffic, you can earn extra bucks every month with new monetization method.
    This is the best adsense alternative for any type of website (they approve all sites),
    for more details simply search in gooogle: murgrabia’s tools

Leave a Reply