PCORI Workshop post 3: Danny van Leeuwen (@HealthHats)

Danny, aka "Health Hats"

Danny, aka "Health Hats"

Next in our series of posts by SPM members on their experience at last weekend’s PCORI workshop. See Monday’s introductory post.

I recently met (face to face) Danny van Leeuwen, one of the newer members of our society. He’s a sensitive, caring patient and RN, and one of the patient SPM members to attend last weekend’s PCORI workshop on patients engaging in research. This appeared Monday on his blog, here.

Patient Centered Outcomes Research

I attended the PCORI workshop in DC last weekend. Congress authorized PCORI The Patient-Centered Outcomes Research Institute (PCORI) to conduct research to provide information about the best available evidence to help patients and their health care providers make more informed decisions. PCORI’s research intends to give patients a better understanding of the prevention, treatment and care options available, and the science that supports those options. About 130 attended: patients, patient advocates, representatives of disease specific organizations, researchers, clinicians, home schoolers. PCORI aligns well with my personal mission: Increase the sense of balance patients, caregivers, and clinicians feel as they work together towards best health.

My main takeaways from the weekend are:

  1. Current research focuses disproportionately on the medical end of the health spectrum. Which therapy (mostly drugs) works better? Many participants would like to shift focus of research to the social and behavioral factors on the spectrum. Social factors are the conditions in which people are born, grow, live, work and age, including the health system. Behavioral factors include risky behavior, developing and following health plans, management of stress, pain and illness
  2. The gold standard of double-blind studies does little to help those confronting illness make decisions. As my neurologist tells me, it’s very challenging to apply research about populations to people. I am not a population. There are other models of sharing information and building on experience.
  3. Dissemination of research results is last funded and least rigorous. Many results are not published. Most patients don’t read peer-reviewed journals.
  4. The power gradient in research favors those managing the money. Rather than researchers seeking consumer involvement, communities could control the funding and seek researchers
  5. Health disparities affect communities (the unfair and avoidable differences in health status shaped by the distribution of money, power and resources at global, national and local levels). Research into health disparities can inform action by self-defined communities.
  6. The reservoir of experience, expertise, passion, and commitment of the PCORI participants is awesome, deep and diverse. I  am humbled to be included in this movement.



Posted in: policy issues | pts as teachers | research issues




3 Responses to “PCORI Workshop post 3: Danny van Leeuwen (@HealthHats)”

  1. Stephanie says:

    It’s very exciting to see the medical paradigm being shifted to better encompass the patient, their doctor, healthcare providers, and families – it is only by such a holistic approach that we can hope to see a greater number of informed decisions being made and more positive outcomes achieved. It sounds like the PCORI workshop was a phenomenal forum for the clarification and dissemination of important information about the medical decision-making process. Furthermore, by empowering patients to advocate for themselves and to harness new technologies for better medical understanding and information sharing, we will be better able to personalize medicine towards better outcomes. As stated by Dr. Eric Topol in his book The Creative Destruction of Medicine, we now have the tools to “leverage the science of individuality, getting the relevant digital readout from a person to fashion a therapy, instead of a mass-population-directed strategy.”
    By working together as a patient and medical community, it will be easier to understand the complex interplay of biological and behavioral factors that promote disease. All patients are certainly different, and it is not just the biochemical side of the equation that determines the incidence of disease in a population. By understanding what certain groups of individuals are doing “right,” i.e. contributing to a better quality of and longer life, we can hopefully share this information with greater numbers of people. This responsibility does not just exist for the patient, but also for the medical-scientific establishment. As an undergraduate conducting research at Rice University, I am familiar with the academic side of research and feel that better patient education would go a long ways in improving care outcomes. However, it will take everyone, the patients, doctors, and researchers, working together to truly create sustainable positive changes in medical decision making.

  2. Alan Kravitz says:

    I have taken a close look at PCORI, it’s Board and Advisory Committee. It looks to me like another way to mine data about the social and behavioral aspects of health care. I don’t doubt that there were a number of well minded individuals at the Workshop but see little evidence that there will be any real effort at critical analysis coming out of this group. There are systemic reasons why pharma refuses of release all trial data, why “peer reviewed” articles are edited and not made available to patients (we are not peers), and why approved products are marketed without subsequent reviews of off label use, and why clinical trials are registered without the information that a patient would require to make an informed decision about participation, and why companies continue to design clinical trials that involve a placebo or comparator arm that is known to provide limited efficacy ( even in the face of legislatively mandated regulations providing for alternative approaches designed for biological drugs both targeted and immunologic), As a patient activist, I don’t see this as a productive

    • Edited substantially a few minutes after initial posting

      I understand your view, Alan; all I can say (having been there, which I think you haven’t, right?) is that my impression is different from yours. My view is that it’s a big messy complicated initiative with many many stakeholders who have irons in the fire (mixed metaphor – stakes and irons), and the people in the center (PCORI staff) are poked at with all those hot and pointy things.

      My impression is that they’re trying to do good work, headed in the right direction, but with an uphill climb. Time will tell, IMO – but I don’t think you yet have a specific factual basis for these concerns, just an impression, right?

      I’m not arguing or flaming – that’s not my style – just asking. My wording seems a bit ingracious – apologies.

      Just for the record, my personal impression is that there’s a subtle but decisive difference in different people’s views of what “patient centered outcomes research” means. Is it:

      1. Outcomes research that’s centered around which outcomes patients find important, from a scientist-defined menu


      2. Research into pursuing patient *defined* outcomes (not ones presented after the fact by the scientists)


      3. Research whose very purpose – whose scientific objective – is defined by the people whose bodies are on the line? For instance, are we talking about outcomes in the domain of cure, or outcomes in the domain of symptomatic relief, or….?

      Who gets to say?

      And then there’s the issue of how the scientific method is applied: who gets to call the shots on that? In analyzing the data, is it more important to avoid false positives, or more important to avoid false negatives? Is an apparent placebo effect “noise,” or a golden opportunity to be pursued and investigated?

      I’ve personally seen that many people in the research establishment – including many well-meaning ones – believe that it’s their job to know what should be researched, for the benefit (paternally) of sufferers (who couldn’t possibly understand the scientific issues); at some point in the process they ask patients which of the discovered outcomes is valuable to those patients.

      This points in the right direction; in the past the patient’s preferences have been pretty much immaterial, even among the well-meaning.

      Meanwhile, other researchers are not so well-meaning: they’re frankly arrogant, dominating, “Who do you think YOU are to tell me what I should research?” (I heard one PCORI board member – a minority – say “Yes, we asked the experts, so the patient perspective has been covered.” Doh!)

      And a very small number of researchers ask, “Hello, patients. What objectives would YOU like us to pursue? A cure? Better data on drugs for maintenance for your status? Research on relieving symptoms?”

      In any other industry, the person with the need (aka the customer) would TELL the contractors (researchers) what outcome they want. The traditional view in medicine is that the “customer” couldn’t possibly understand, and in my personal experience the people running PCORI are working to change that. But it’s a long haul, because unlike other industries, here the whole establishment is necessarily being dragged along.

      There are at least two ways to assess these questions: science (the pursuit of knowledge) and financial. As America looks to reduce the sheer volume of medical spending, I think it makes sense to gather everyone, like a family at the kitchen table, and say, “Okay, folks – we have to make some cuts; we can’t keep doing everything we’ve been doing. What do we think is most important?”

      In the medical context it seems clear to me that this conversation MUST include, as a priority, the voice of the patients. And that’s what I want PCORI to focus on, the most. Not to the exclusion of the others, but as the “true north.” Because to me the whole reason we have medicine is to help patients and families have better lives.

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