Sara Riggare

Not patient but im-patient

Category: In English

  • Four reasons for involving patients in improving research (plus the one that matters most)

    Four reasons for involving patients in improving research (plus the one that matters most)

    There has been a lot of research on why patients should be involved in improving research. The arguments usually sound convincing — but they often miss the real point. Here are the four most common reasons, and the reason that I think should always be mentioned first. I have also added some scientific references at the end in case you want to dive deeper.

    Reason 1: It’s the morally and ethically right thing to do

    The literature repeatedly frames patient involvement as an ethical obligation. It respects autonomy, promotes justice, values lived experience, and strengthens accountability. Instead of treating patients as passive subjects, involvement recognises them as partners with a legitimate claim to influence research that affects their lives. It also fits democratic ideals: publicly funded research should be transparent, fair, and responsive to those it serves.

    All true.
    But ethics alone rarely changes research practice.

    Reason 2: It improves research design

    When patients help shape questions and protocols, studies tend to become more relevant and feasible. Patient input can sharpen outcome selection, clarify consent materials, refine inclusion criteria, and reveal practical burdens researchers miss. Involving patients early makes studies look less elegant on paper — but more workable in reality.

    Good design is a strong argument.
    Still, it doesn’t explain why patient input makes design better in the first place.

    Reason 3: It improves participant recruitment and retention

    Patient involvement can make studies clearer, more acceptable, and more trustworthy. When study materials reflect patient perspectives, people are more willing to enrol and stay engaged. Collaboration also signals partnership rather than extraction, which strengthens trust — and trust drives participation.

    Useful, yes.
    But recruitment is a downstream effect, not the core reason.

    Reason 4: It improves value for money

    Studies that ignore patient realities are more likely to struggle with recruitment, require redesign, or produce findings that never get implemented. Involving patients early can reduce waste, improve efficiency, and increase the chance that research investments actually lead to change.

    This argument resonates with funders.
    But it still treats involvement as a tool for optimisation, not as a core part of knowledge production.

    Reason 5: Patients have knowledge researchers don’t

    Here’s the real reason.

    Patients and researchers don’t just have different views — they have different epistemic positions. Researchers are trained to generate analytic, comparative, and generalisable knowledge from the outside. Patients develop situated, embodied, and longitudinal expertise from the inside. They know how illness unfolds over time, how treatments collide with everyday life, how systems behave in practice, and how decisions are made under real constraints.

    This isn’t anecdotal input. It’s operational knowledge of how health and care actually work.

    When that knowledge is missing, research often rests on assumptions that seem reasonable to professionals but fail in real life. When it’s present, studies are more realistic, interpretations make more sense, and implementation becomes possible.

    The reason patient involvement improves research is not primarily ethical, financial, or procedural.
    It’s epistemic.

    Conclusion

    Ethics, design quality, recruitment, and efficiency are all good reasons to involve patients. But they are secondary. The fundamental reason is that research improves when it integrates both outside analytic expertise and inside lived expertise. Without that combination, we risk producing studies that are methodologically sound — and practically wrong.

    (Secondary) patient involvement isn’t mainly about being nice, fair, or efficient — it’s about fixing a knowledge gap in research that only patients can fill.

    References

    • Beresford, P., & Russo, J. (2020). Patient and public involvement in research. In Achieving Person-Centred Health Systems.
    • Boivin, A., Richards, T., Forsythe, L., Grégoire, A., L’Espérance, A., Abelson, J., & Carman, K. (2018). Evaluating patient and public involvement in research. BMJ, 363. 
    • Domecq JP, Prutsky G, Elraiyah T, Wang Z, Nabhan M, Shippee N. Patient engagement in research: a systematic review. BMC Health ServRes. 2014;14.14
    • Dumez, V. and A. L’Espérance, “Beyond experiential knowledge: a classification of patient knowledge,” Soc Theory Health, Jun. 2024,
    • Frank L, Basch E, Selby JV, Patient-Centered Outcomes Research I. The PCORI perspective on patient-centered outcomes research. JAMA. 2014;312:1513-1514.13. 
    • Hoos A, Anderson J, Boutin M, et al. Partnering with patients in the development and lifecycle of medicines: a call for action. TherapeuticInnovation & Regulatory Science. 2015;49:929-939.
    • Levitan B, Getz K, Eisenstein EL, et al. Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project. Ther Innov Regul Sci. 2017.
    • Røssvoll, T. B., Rosenvinge, J., Liabo, K., Hanssen, T. A., & Pettersen, G. (2023). Patient and public involvement in health research from researchers’ perspective. Health Expectations, 26, 2525–2531.
    • Sacristán, J. A., Aguaron, A., Avendaño-Solá, C., Garrido, P., Carrión, J., Gutiérrez, A., Kroes, R., & Flores, A. (2016). Patient involvement in clinical research: why, when, and how. Patient Preference and Adherence, 10, 631–640.
    • Smith MY, Hammad TA, Metcalf M, et al. Patient engagement at a tipping point—the need for cultural change across patient, sponsor, and regulator stakeholders: insights from the DIA Conference, “Patient Engagement in Benefit Risk Assessment Throughout the Life Cycle ofMedical Products.” Therapeutic Innovation & Regulatory Science. 2016;50:546-553
    • Staley K, Minogue V. User involvement leads to more ethically sound research. Clin Ethics. 2006;1:95-100.12. 
  • Checklist and resources for meaningful engagement of patients

    Checklist and resources for meaningful engagement of patients

    This post is a followup of my recent post Why are patients the only ones around the table expected to work for free? In this new post, I have made a checklist and compiled some resources that may be useful for for patients when invited to contribute to for example research, care improvement, conferences, products, etc.

    The term “meaningful engagement” that I have used in the title comes from a WHO report that was published in 2023, and can be found under Resources further down.

    I see meaningful engagement as engagement that leads to meaningful change.

    Checklist

    First the checklist, let me set the scene:

    You have received an invitation of some sort to contribute in some way… In my experience, often the person inviting you is not entirely sure what they want your help with. They have heard, maybe from their boss, their colleagues or someone else, that it is very important to involve patients. Maybe they don’t fully understand why, and that’s OK. For me, many great collaborations have started with a very unclear beginning. The key factor is if the person contacting you have the right mindset and the only way to establish that is by interacting with them. It can take some time but my experience is that it is worth investing that time early on instead of encountering difficult issues later in the process.

    I hope the checklist below can help you. Use it to quickly assess whether the invitation treats you as a knowledge contributor or as free labor.

    1) Clarify the role

    2) Clarify the expectations

    • How many meetings? How long?
    • What preparation is expected?
    • Will I be asked to read materials in advance?
    • Are follow-ups or written feedback expected?

    If preparation is required → this is work.

    3) Ask directly about compensation

    • Is there an honorarium or hourly compensation?
    • Are preparation time and meeting time both compensated?
    • Are travel time and expenses covered?

    If the answers are vague, that’s a signal.

    4) Watch for “replacement compensations”

    Be alert if you hear:

    • “You’ll get visibility / contacts”
    • “You can use our system/app for free”
    • “Others do this voluntarily”
    • “There is no budget”
    • “It’s complicated administratively”

    These are red flags, not benefits. For common arguments and counterarguments, see this post.

    5) Check how professionals are treated

    • Are the researchers/organizers/project team members doing this during paid work hours?
    • Are speakers, consultants, or moderators paid?

    If yes → the principle should apply to you.

    6) Ask how your input will be used

    • How will my contribution be documented?
    • Will I receive feedback on how it influenced the project?
    • Is this advisory or actually consequential?

    Vague answers often mean symbolic involvement.

    7) Consider accessibility and fairness

    • Would someone without financial flexibility be able to say yes to this?
    • If not, the setup is structurally excluding many patients.

    8) Ask for it in writing

    • Role description
    • Time expectations
    • Compensation terms
    • Reimbursement of expenses

    If it feels uncomfortable for them to write it down, that’s informative.

    9) Decide your boundary

    You can say:

    “I’m happy to contribute, but I don’t do this type of work without compensation.”

    This is not rude. It is professional.

    10) Remember

    You were invited because of what you know.
    That knowledge has value.

    Resources

    Below, I have listed some resources that support meaningful engagement.

    WHO framework for meaningful engagement of people living with noncommunicable diseases, and mental health and neurological conditions

    Link to document

    Highlights:

    • Target audience: Member States and other stakeholders
    • “The overall objective of the framework is to support WHO and Members States in meaningful engagement of people living with NCDs, and mental health and neurological conditions to co-create and enhance related policies, programmes and services.”
    • Practical advice and recommendations
      • 4. Principles of meaningful engagement
      • 5.1 Sustainable financing. Meaningful engagement should be supported by sustainable financing for all engagements with individuals with lived experience remunerated at a rate equivalent to that for technical experts.
    • Conclusions:
      • A lot of work still remains
      • The way forward lies in collaboration

    WHO Guidance for best practices for clinical trials

    Link to document

    Highlights:

    • Target audience: Everybody working on clinical trials
    • “strengthening clinical trials to provide high-quality evidence on health interventions and to improve research quality and coordination to identify and propose best practices and other measures to strengthen the global clinical trial ecosystem and to review existing guidance and develop new guidance as needed on best practices for clinical trials.”
    • Main focus is RCTs
    • Emphasizes meaningful engagement:
      • 2.2 Good clinical trials respect the rights and well-being of participants
      • 2.3.1 Working in partnership with people and communities
      • 3.2.1 Patient and community engagement

    Update to the Declaration of Helsinki

    I have also written a blog post on the update to the Declaration of Helsinki, the post is in Swedish, use your favorite translator to read it: Link to post

  • Why are patients the only ones around the table expected to work for free?

    Why are patients the only ones around the table expected to work for free?

    (Detta inlägg finns även på svenska: LÄNK)

    (This post is complemented with a checklist: LINK)

    I have been part of literally hundreds of conversations and meetings with researchers, healthcare professionals, companies, agencies, conference organizers and others wanting to involve patients. Here, I have listed 12 arguments that I have heard when asking if the offer comes with some form of payment (plus one bonus argument, which I think is the true reason that patients are often expected to work for free)

    First, let’s make sure that we’re talking about the same thing:

    Primary or secondary patient involvement?

    When I teach or present on patient involvement, I always spend some time at the start to ensure that we all agree on what we’re talking about. If the topic is how to engage patients in participating in research studies or clinical trials, I refer to it as primary patient engagement. For conversations on getting patient input on the structure and/or process of studies or trials to improve them for upcoming participants, I use the term secondary patient engagement. I have written about this before => link to post.

    Many regulatory bodies, research funders, governmental authorities and others all around the world are increasingly promoting, encouraging, and even demanding secondary patient engagement for applications and projects. And that is what this post is about: secondary patient engagement.

    I want to point out that the same principles apply to engaging patients to help improving healthcare processes or facilities, medtech products, evaluating research applications, speaking at conferences etc.

    Here we go, 12 arguments I’ve heard on why not to pay patients:

    (edit: now with five additional arguments, based on comments and feedback, between reasons 12 and 13)

    Reason 1: “This is voluntary engagement, not work”

    Imagine a project meeting where a researcher, a clinician, a project manager—and a patient—are all invited to help improve a study design. The meeting has an agenda, background material is sent out in advance, and everyone is expected to read it, think it through, and contribute informed input.

    The researcher, clinician, and project manager are all attending as part of their paid workday. The patient is described as “volunteering.”

    Yet the expectations are the same: show up on time, be prepared, share expertise, and help shape decisions the project depends on. The only real difference is how the contribution is labeled—and whether it is compensated.

    How can this argument be met?

    Counterarguments

    • The tasks are defined, scheduled, and requested by the organization → this is work.
    • Volunteering is initiated by the individual. Here, the system initiates.
    • This is organized knowledge work, not volunteering.

    Reason 2: “Compensation threatens independence”

    Imagine the same meeting where everyone is paid for their time—except the patient, who is told that payment might make their input less independent. The implication is that being unpaid makes their perspective more trustworthy. Strangely enough though, the paid professionals’ independence is never questioned.

    Counterarguments

    • Everyone else in the room is paid without their independence being questioned.
    • Transparency about compensation increases credibility.
    • Independence is secured by transparency, not by lack of payment.

    Reason 3: “But researchers (doctors, nurses, etc) do this (review funding applications, sit on advisory boards, present at conferences, etc) without payment”

    No, they don’t. This argument overlooks that these activities usually take place within paid employment, during salaried work hours, and contribute to professional merit, networks, and career progression. What appears as “unpaid” is in fact embedded in a system where time, status, and long-term benefit are already compensated. Patients, in contrast, are expected to contribute outside any such structure.

    Counterarguments

    • For professionals, this work happens within paid roles and paid time; for patients, it does not.
    • These activities build careers and merit for professionals—patients receive no equivalent benefit.
    • What looks unpaid for staff is still supported by salary, status, and infrastructure; patients stand outside all three.

    Reason 4: “There’s no budget for paying patients”

    Another type of argument relates to financial aspects. It is often said that compensation simply wasn’t planned for, as if budgets were fixed facts rather than reflections of priorities. Yet the same project easily finds funds for venues, catering, consultants, and staff time—which simply means that the real issue is not lack of money. It’s about what the organization sees as important.

    Counterarguments

    • Budget reflects priorities, not constraints of nature.
    • The same budgets accommodate consultants and communication activities.
    • If it is not in the budget, it is because it was deprioritized.

    Reason 5: “It’s so administratively complicated”

    This argument points to practical hurdles—tax forms, payment systems, insurance, procurement rules—as if these were insurmountable barriers. But the very same administrative machinery routinely handles fees for consultants, speakers, and external experts. What it reveals is not true complexity, but that the system was never designed with patients in mind as formal contributors.

    Counterarguments

    • The system already manages consultant fees and honoraria.
    • “Complicated” means the organization has not adapted to patients as contributors.
    • Administrative systems should adapt to reality, not the other way around.

    Reason 6: “We don’t want to create ‘professional patients’”

    This argument reflects a concern that some patients might become too experienced, too familiar with research and healthcare processes, and therefore somehow less “authentic.” The strange thing is that in every other context, growing competence and repeated involvement are seen as assets that improve quality. What is framed as a risk here is, in practice, the very process by which expertise is developed.

    Counterarguments

    • What is being described is competence development.
    • We do not question “professional researchers”.
    • Competent patients are an asset, not a threat.

    Reason 7: “We are giving patients a chance to contribute to research / future patients / their own community”

    This frames participation as a gift to the patient rather than a contribution to the project. It suggests that the opportunity to be involved is itself a form of compensation, shifting attention away from the value the patient adds. In doing so, requested work is seen as a favor granted, rather than expertise sought.

    Counterarguments

    • Being invited is not compensation when real work and expertise are requested.
    • Calling it a “chance to contribute” reframes sought-after expertise as a favor instead of a contribution.
    • It is frankly unethical to try to shame patients, who often are struggling with illness, worry and financial hardship, into contributing for free.

    Reason 8: “Patients benefit from participating”

    This is an especially infuriating argument, that patients should not be paid because they get benefit from their contributions in the form of strengthening of health and motivation. This argument suggests that any personal health gains a patient might experience from being involved somehow replace the need for compensation. It implies that improved understanding, empowerment, or a sense of purpose is “payment enough.” By that logic, however, no one whose work is meaningful, educational, or professionally rewarding would ever need a salary.

    Counterarguments

    • Researchers also benefit personally from salary and career advancement. So, by the same logic, they should also not get paid.
    • Personal benefit does not negate that something is work.
    • Self-interest does not disqualify work—otherwise no one would be paid.

    Reason 9: “Compensation could attract the ‘wrong kind of patients’”

    This argument assumes that financial motivation makes participation less genuine, as if only those who can afford to contribute for free are the “right” voices. In practice, it means that unpaid involvement filters out anyone without spare time or financial flexibility. Rather than protecting quality, it quietly ensures that only the most privileged patients are able to take part.

    Counterarguments

    • This argument is never applied to professionals.
    • Compensation enables participation from those who otherwise cannot afford it.
    • Without compensation, you only recruit the economically privileged.

    Reason 10: “Patients don’t know enough about what we do for us to pay them”

    This argument suggests that patients lack the technical understanding required to warrant compensation, as if payment were reserved only for formal expertise. Yet patients are invited precisely because they bring a different kind of knowledge—about living with a condition, navigating care, and seeing gaps professionals may overlook. Dismissing this as insufficient knowledge overlooks the very reason they were asked to contribute in the first place.

    Counterarguments

    • Patients are invited for what they know that professionals don’t—not for what they lack.
    • You’re compensating contribution and time, not academic credentials.
    • If their knowledge isn’t valuable, they shouldn’t be in the room in the first place.

    Reason 11: “We’ve never paid patients before”

    This argument rests on tradition: it has always been done this way, so it feels natural to keep doing it. But history mostly tells us how things used to work—not whether they still make sense. Many of the improvements in patient involvement have come from challenging exactly these old routines.

    Counterarguments

    • Tradition is not an argument in knowledge development.
    • Patient involvement as a field exists precisely to change past practice.
    • Historical practice is the reason patient knowledge has been underused.

    Reason 12: “Not all patients want to get paid” or “Not all patients are able to accept payment”

    This argument points out that some patients prefer to contribute without compensation, or may be restricted by benefit rules or employment status. While this may certainly be true for some individuals, it turns a personal choice into a general rule for everyone. The fact that a few people are willing—or forced—to work for free does not mean that unpaid work should be the norm.

    Counterarguments

    • Individual preference should not define the norm for everyone else.
    • The option to decline payment is fair; the absence of payment is not.
    • Basing the system on who can afford to work for free excludes those who can’t.

    These additional reasons have been added as a result of comments and feedback:

    Additional reason 1: “We can’t pay you, but you’ll get great connections by speaking at our conference”

    This frames visibility and networking as a substitute for compensation. It assumes that exposure, contacts, or prestige are adequate payment for preparation time, travel, and sharing expertise. In practice, it mirrors a pattern where patients are expected to trade real work for intangible benefits that primarily serve the organizer.

    Counterarguments

    • Exposure and networking do not replace compensation for time and expertise.
    • Professionals invited to speak are rarely asked to accept “connections” instead of payment.
    • If the talk creates value for the conference, it deserves proper compensation

    Additional reason 2: “We can’t pay you, but you can use our app/AI bot/system for free”

    This argument reframes access to a product or service as a form of compensation. It treats the patient’s time and expertise as something that can be exchanged for exposure, testing access, or early use of a tool. In reality, this shifts the relationship from collaboration to unpaid product testing, where the patient’s contribution creates value for the company or project without any financial recognition.

    Counterarguments

    • Access to a product is not compensation for professional input and time.
    • This turns patient involvement into unpaid user testing.
    • If the tool has real value, it should be offered in addition to fair compensation—not instead of it.

    Additional reason 3: “We can’t pay you, but why don’t you fundraise to cover your own costs and then contribute for free?”

    This argument asks the patient to take on the financial responsibility for enabling their own participation, while everyone else is paid from the project’s budget. It turns involvement into a personal funding problem and shifts the burden of resourcing the project onto the very person whose knowledge is being requested.

    Counterarguments

    • Asking someone to fundraise to work for free reverses who is responsible for resourcing the project.
    • No other contributor is expected to secure their own funding to be allowed to participate.
    • If the contribution is important enough to ask for, it is important enough to budget for.

    Additional reason 4: “We’d love to pay you – but how?” (followed by silence)

    This starts as apparent goodwill: praise for your contribution and an expressed intention to compensate you. You provide the requested invoice or details—and then nothing happens. Months pass. A year later, you’re warmly invited back because your previous input was “so valuable,” while the earlier payment is still unresolved.

    Counterarguments

    • Good intentions without follow-through are functionally the same as not paying.
    • If an organization can manage contracts and payments for others, it can do the same for patients.
    • Unpaid past work should be resolved before new involvement is requested.

    Additional reason 5: “If you won’t do it, we will find someone else who will”

    This argument relies on the fact that some patients, out of goodwill, passion, or necessity, are still willing to contribute for free. It shifts the focus from fairness to replaceability, implying that the issue is not the value of the contribution but the willingness of someone to accept unpaid work.

    Counterarguments

    • The availability of unpaid contributors does not make unpaid work ethical.
    • Replaceability does not reduce the value of the contribution.
    • This approach selects for who can afford to work for free, not for who is best suited to contribute.

    Reason 13: The unspoken underlying argument

    The real, unspoken argument is probably:

    “If we start paying patients, we acknowledge that their knowledge is work. And that challenges the existing knowledge hierarchy”

    This is not about money. It is about epistemic status.

    Counterarguments

    • Yes — that is the point.
    • If patients contribute to knowledge production, they are knowledge actors.
    • Compensation is an organizational recognition of this.

    This is about how we view knowledge, not about money.

    Here is an argument that can be used to close:

    Either this is a real contribution to research and healthcare improvement — and should be compensated.
    Or it is symbolic participation — and we should be honest about that.

    Which ones have you heard? Have you heard any other arguments for not paying patients? Let me know in the comments.

  • A problem to be solved or a life to be lived?

    A problem to be solved or a life to be lived?

    A while back, I had two different meetings on the same day. They were different in so many ways but at the same time, they were also similar. In the first meeting, I was discussing ways that AI can be used for persons living with Parkinson’s disease (PD) with researchers in medicine and technology. The second meeting was with researchers in philosophy and history of science. We were also discussing the possibilities of AI for people living with different forms of disabilities and PD was one of the examples.

    (more…)

  • Spetspatients in the BMJ

    My colleague Charlotte Blease and myself have written about spetspatients in the British Medical Journal (BMJ):

    https://www.bmj.com/content/390/bmj.r1634

  • Managing medication in PD – both complex and simple?

    Managing medication in PD – both complex and simple?

    Medication management is probably the absolutely most important part of my Parkinson’s disease (PD) selfcare since without my pills, my body would not function. During the day I take meds approximately every three hours to be able to move around, take care of myself and be able to work. When I wake up in the morning, I have not taken any meds for at least 7 hours and my movements are REALLY slow and stiff.

    My approach to PD is that I try to take my meds at the same times every day. I know that other persons with Parkinson’s (PwP) have other approaches and no-one really knows which approach is best, since there has been no research conducted (or at least not published) comparing different approaches (which is actually one of my main reasons for writing this post).

    When I occasionally make changes to my medication regime, I note down dates of the change and what I changed (for example types of meds added/taken away, changes in timings for intakes, and/or changes in combinations of meds for each intake). For example, a few months ago I noticed that I tended to get slightly dyskinetic (a side effect of PD meds, see here) around lunch so I decided to push my second dose of meds 30 minutes and started taking it at 10:00 am instead of 9:30 am. This changed resolved my dyskinesia. These notes are for myself, none of my neurologists have ever asked about changes I have made between visits, they have only shown an interest in my current regimen at the time of the visit. I can completely understand this, they have enough work to do as it is and don’t need more tasks to complete. Of course there is plenty that could be learned from systematically recording individual medication regimes for a large number of persons with Parkinson’s (PwP) including following up the effects of that regimen on an individual level but I have long since accepted that that is probably not the best use of healthcare resources.

    In my role as an academic researcher, I have applied for funding many times over the past decade to address different aspects of how to optimise PD meds on an individual level. Unfortunately my funding applications have not really succeeded despite the fact that this area is extremely under-researched. I think there are several reasons for this apparent disinterest in the field of medication optimisation in PD and I will mention the three at the top of my mind:

    1. Complexity

    PD is SUPER COMPLEX! We have heard this so many times but what does it actually mean? The complexity of PD is multi-layered and there is so much we currently don’t know. We don’t know how and why the condition starts on an individual level, we are not sure about what processes makes the condition progress, and we don’t know why some medications work well for some but do not work as well for others.

    On the level of individual neurologists, I think that many of the clinicians who have made it their profession to help us actually think that medication intakes are already optimized based on available guidelines and their clinical experience. Those guidelines are however based on group-level data, and do not often translate well to individual patients. There is a huge gap between what is measured in research trials (which subsequently forms the basis of guidelines) and what PwP experience in their daily lives.

    One aspect that is often overlooked is that taking medication is not as simple as just swallowing a pill at the right time. It requires remembering (which, ironically, can be difficult when you have a neurodegenerative disease), carrying medication with you at all times, for some PwP also planning around meals, and adjusting for unpredictable factors such as stress, sleep quality, and physical activity. Even something as basic as getting water to take a pill can be a challenge in certain situations. The effort required to consistently take PD medication is far greater than many neurologists or researchers realize.

    Another factor adding to the complexity is that we living with PD are not really interested in taking the pills, we actually have zero interest in the actual pill taking itself. The reason we take them is to get the effect the pills (hopefully) have on reducing our symptoms. But what does healthcare use to evaluate this process? They use medication adherence, meaning if we take our pills in the way our neurologists have prescribed. As far as I know, there are no standardized adherence scales available that take into account the complexity of finding your individual best timings for intakes of meds to achieve the optimal effect.

    And since this complexity doesn’t directly influence the work of neurologists and researchers, it’s not strange that they don’t see the need for research into how to best optimize PD meds.

    2. Money

    The reality is that the use (and non-use) of pharmaceuticals is (almost) entirely driven by money. And currently there is no money to be gained on a corporate level from helping the approximately 10 million PwP in the world getting a better effect from the many pills we take.

    The pharmaceutical industry mostly hinges on developing new drugs, not on optimizing the use of existing ones. This means that the funding available for research favor the discovery of new molecules over the fine-tuning of treatment regimens for individual patients.

    3. Simplicity

    As a person who has spent more time thinking about this A LOT over the past 10 – 15 years, probably more than is healthy…, I have come to the conclusion that the main problem with the issue of medication optimization in PD actually is simplicity. More precisely, the illusion of simplicity. Medication adherence and effectiveness in PD are mostly treated as simple, binary issues: “Did you take your medication? Yes or no?” But in reality, it’s much more complex. Timing, food intake, stress levels, sleep quality, and countless other factors can influence how well our meds works.

    Yet, our healthcare system is not set up to systematically track and adjust for these variables. Instead, PwP are mostly left to figure it out themselves, with trial and error as our primary tool.This means that many PwP, myself included, spend years fine-tuning our medication regimens without structured support. Some of us get lucky and find a pattern that works well; others struggle for years with inconsistent results.

    In conclusion

    For this area to develop, we need a better understanding of how PwP actually take their medication in daily life and not just how they are prescribed to take it. We need more research that focuses on the real-world challenges of medication adherence, from remembering doses to managing side effects and adjusting for the unpredictability of daily life with PD. We need a more nuanced discussion of what is working, what isn’t, and why. Until then, PwP will continue doing what we have always done: adapting, experimenting, and trying to make the best of an imperfect system.

    It’s ironic that while Parkinson’s medication is one of the most researched aspects of the disease, the actual experience of taking it—the daily struggles, the trial and error, the constant balancing act—is still largely unstudied. The truth is, many PwP are experts in managing their own meds out of necessity, yet our knowledge remains anecdotal and undervalued in clinical settings. If we truly want to improve Parkinson’s care, we need to start acknowledging the experiential expertise of us who live with it every single day.

    And no, there is nothing simple about managing PD meds – it’s complex all the way!

  • Understanding Parkinson’s: The curse of knowledge

    Understanding Parkinson’s: The curse of knowledge

    I just realised that I have probably been suffering from the “curse of knowledge” when trying to explain Parkinson’s (PD) to people. With this I mean that things that are obvious to me are probably not as obvious to others when it comes to understanding Parkinson’s. Let me demonstrate with a few examples:

    Biomarkers in Parkinson’s

    I often say that there are no biomarkers in PD but if you read the scientific literature there are many examples. What’s up with that? The problem is that none of them are fully deterministic. With that I mean that none of them are relevant for all people with PD. Many different attempts at biomarkers are used in projects on PD and one example REM-sleep behaviour disorder or RBD. This condition entails people not lying still while sleeping but rather can thrash about in bed and if someone is sleeping next to them the spouse or partner might even get hurt. This is considered one of the early markers of PD in the scientific community but I would say that its usefulness to patients is limited. This is because not all people with RBD will go on to be diagnosed with PD and conversely not all people with PD will ever experience RBD. There are also other suggested prodromal signs like depression, loss of sense of smell etc. They are all equally non-deterministic as RBD and without clear patterns in co-occurences between different individuals who go on to be diagnosed with PD (or not).

    Diagnosis of PD

    Also here there are important challenges. The condition is generally diagnosed based on observations of a few key symptoms:

    • Tremor. Not all PwP have this.
    • Slowness of movement (bradykinesia). I would say that all PwP have this symptom but there are MANY other conditions that also give this symptom
    • Stiffness (rigidity). Most PwP have this symptom in varying degrees but it can be difficult to distinguish from other conditions that result in stiffness.

    Comparison with type 1 diabetes

    Let me make a comparison with a different complex chronic condition: type 1 diabetes (T1d). There is a very distinctive and specific biomarker in blood glucose levels. All people with T1d need insulin to survive and they all use frequent blood sugar readings to regulate their treatment. 

    In short: both Parkinson’s and T1d are equal to being dealt a really shitty hand in the “poker game of life”. But while with T1d you get the two of clubs, seven of hearts, king of spades, ten of diamonds, and five of spades, with Parkinson’s your hand consists of an old bus ticket, a wrinkled up candy wrapper, your old business card from two re-organisations ago, a receipt from your latest visit to the pharmacy, and small piece of pocket lint…

  • Patient self-experimentation makes the headlines!

    Patient self-experimentation makes the headlines!

    What is self-experimentation?

    Sanctorio sitting in the balance that he made to calculate his net weight change over time after the intake and excretion of foodstuffs and fluids.

    Self-experimentation is a type of single-subject research where the researcher and the research subject are one and the same. This is a practice with ancient traditions and prominent examples exist throughout history. Some of the more well-known include: The Italian physician Sanctorius of Padua (1561-1636) who is considered the father of experimental physiology, largely as a result of him for thirty years using a ”weighing chair” to weigh himself, everything he ate and drank, as well as the urine and faeces he excreted. This led to him formulating new theories about our metabolism, some of which are still valid today. Another example is the German physician and researcher Werner Forssmann (1904-1979) who in 1929 performed the world’s first heart catheterisation, on himself. A more recent example is the Australian physician Barry Marshall who in 1984 infected himself with Helicobacter pylori, thereby proving that stomach ulcers are the result of a bacterial infection. Both Forssmann and Marshall were rewarded with Nobel prizes for their discoveries. 

    What happened the other day?

    And just the other day, self-experimentation made the headlines of Nature, one of the most prestigious scientific journals in the world. The article is titled “This scientist treated her own cancer with viruses she grew in the lab” and is definitely worth reading. It is about the Croatian virologist Beata Halassy who, after getting her second recurrence of breast cancer, combined her virology training with self-experimentation and administered oncolytic virotherapy to herself. Her oncologists agreed to monitor her and they are also coauthors to the scientific article in the journal Vaccines describing the study. The study was rejected by more than a dozen journals before being published by Vaccines and Halassy says that the reason for the rejections was ethical concerns. There are certainly plenty of interesting ethical challenges here and I would like to focus on a specific section in the Nature article, where law and medicine researcher Jacob Sherkow comments:

    “The problem is not that Halassy used self-experimentation as such, but that publishing her results could encourage others to reject conventional treatment and try something similar, says Sherkow. People with cancer can be particularly susceptible to trying unproven treatments. Yet, he notes, it’s also important to ensure that the knowledge that comes from self-experimentation isn’t lost. The paper emphasizes that self-medicating with cancer-fighting viruses “should not be the first approach” in the case of a cancer diagnosis.

    “I think it ultimately does fall within the line of being ethical, but it isn’t a slam-dunk case,” says Sherkow, adding that he would have liked to see a commentary fleshing out the ethics perspective, published alongside the case report.”

    These are relevant and important points and I would like to share my thoughts on this. 

    What are my experiences of ethical issues relating to self-experimentation?

    I have some personal experience from self-experimentation myself, although of a much less invasive nature. In one study I used an observational design to better understand how the effects from my medications vary over the day. I take a lot of pills to reduce my Parkinson’s symptoms. The pills have to be taken several times every day and the medication effect waxes and wanes as the substances in the pills are being taken up and metabolised in my body. This study is published in a scientific journal: “Precision Medicine in Parkinson’s Disease – Exploring Patient-Initiated Self-Tracking”.

    I have also used self-experimentation in an interventional study to investigate the effects of nicotine administered via an e-cigarette on levodopa-induced dyskinesia (a troublesome side effect of Parkinson’s medication). This study is also published in a scientific journal: “Patient-driven N-of-1 in Parkinson’s disease: Lessons learned from a placebo-controlled study of the effect of nicotine on dyskinesia”. 

    In 2018, my application to defend my PhD thesis at Karolinska Institutet was rejected, see a small excerpt from the decision of the Dissertation Committee here:

    ”[…] The primary focus of this process has been the issue of ethical approval and the conclusion is that The Ethical Review Act does not make exceptions for research on persons who themselves are scientifically involved in the study. […]”

    The full post is available here. 

    Subsequently, I went on to successfully defend my PhD thesis at Radboud University in the Netherlands, which you can read about here:

    As part of the process of transferring my PhD work from Karolinska Institutet to Radboud University, together with my main supervisor, professor Bas Bloem, my co-supervisors assistant professor Maria Hägglund and Martijn de Groot, and professor of biomedical ethics Annelien Bredenoord, I wrote a commentary on ethical issues in relation to my thesis work: “Ethical Aspects of Personal Science for Persons with Parkinson’s Disease: What Happens When Self-Tracking Goes from Selfcare to Publication?”.  

    What are my thoughts on what Halassy did?

    From a patient perspective, I completely understand why Halassy did what she did. She found herself faced with a personal health challenge with huge implications. For her it was literally a matter of life or death. At the same time, she had access to knowledge and resources that she could use to potentially address her problem, and hopefully survive. Of course she felt she had to do what she could with what she had at her disposal! In this case, my guess would be that there are few people in the world more suited than Halassy to make a genuinely informed decision about this specific treatment and the risks involved. And, in fairness, that was also what Sherkow said in the Nature article: the problem was not that Halassy had used self-experimentation. 

    Sherkow went on to say that the actual problem was that she had published her results, because “her results could encourage others to reject conventional treatment and try something similar”. Let me unpack that statement a bit: Is it reasonable to think that others with breast cancer would reject conventional treatment based on the publication of this study? I genuinely don’t think that is a likely scenario, for several reasons. First and foremost, very few patients have the knowledge needed and access to a virology lab. So why and how could the publication result in others rejecting conventional treatment? And if they did reject conventional treatment, for whatever reason, surely that is their prerogative? 

    To me, Sherkow’s statement seems to be an example of epistemic injustice in action, and in this case testimonial injustice, which according to the Wikipedia entry means “unfairness related to trusting someone’s word. An injustice of this kind can occur when someone is ignored, or not believed, because of their sex, sexuality, gender presentation, race, disability, or, broadly, because of their identity.” Before we ”become patients”, we have been able to live an independent life with responsibilities, obligations, and human rights. Why would we suddenly become unable to make our own decisions, just because we have been diagnosed with a life-altering medical condition, for example cancer?

    In contrast, I would like to ask: Would it have been ethical by Halassy and her coauthors NOT to have published what they learned from this?

  • “Precision Medicine in Parkinson’s Disease – Exploring Patient-Initiated Self-Tracking” as a podcast

    This is a podcast version of my published scientific article “Precision Medicine in Parkinson’s Disease – Exploring Patient-Initiated Self-Tracking” (https://content.iospress.com/articles/journal-of-parkinsons-disease/jpd181314) created by Google Notebook LM.