Asking patients to tell us what we don’t know

By Lisa Willemse
Note: Names of patient advocates and other identifying information have been omitted for privacy considerations.
Twenty-six people had gathered in the room. One, a patient with a spinal cord injury, arrived in a wheel chair. Another had Huntington’s disease. Two of the women had multiple sclerosis and another had vision loss. In all, eight of those assembled had first-hand experience living with a severe, debilitating illness. 
All were patient advocates, some of whom worked for their representative health charities. They joined the remaining 18, representing health charities and organizations focused on research and clinical translation.
They came to listen and to share their expertise and perspectives on clinical trials and regenerative medicine, something that strangely doesn’t happen very often. It’s not hard to imagine why not:  although everyone in the room wants the same thing – treatments or cures for injury and disease – each group has its own focus. Different branches of the same tree, reaching in the same direction, but not necessarily touching. We amass a great deal of knowledge and experience that doesn’t simply jump from one side of the tree to the other. We sometimes forget that it doesn’t.
That separation can create barriers to communication: jargon or assumptions about the level of knowledge – dangerous things if it makes patients more susceptible to the risks of unproven therapies. 
Event organizers, the Ontario Institute for Regenerative Medicine (OIRM) and Clinical Trials Ontario (CTO), brought everyone together in Toronto to understand the gaps in knowledge in order to understand how they could align communications efforts to enable patients, medical professionals and health charities obtain better information and make better decisions about their health. The day was bookended by presentations from OIRM and CTO to provide background information and set the stage for the focused discussions.
Even within this small group, there were a range of experiences. One MS patient spoke openly about her travels to the United States and her hope that paying for unconventional therapies that are unapproved in Canada would stall or perhaps even reverse the progression of her disease. Another woman, who had taken part in a stem cell clinical trial in Ottawa, is now considered cured of a rare and aggressive neurological disorder. 
But, despite many differences, some clear commonalities emerged. The first part of the discussion asked what information was needed about clinical trials to help patients. Patient advocates and health charity staff provided these insights:
  • Ensure specialists and other health care practitioners are kept up-to-date with clinical trial information, since these are where they turn first for advice.
  • More support for patients from health care providers. They should not be expected to have all the answers but should at least be able to advise on informed consent and provide credible resources.
  • Need improved ways to search for relevant clinical trials.
  • Need for basic explainers on clinical trials for patients to help them determine eligibility and understand protocol.
  • Need clear Information about clinical trial stages and why many are for “end-of-life”.
  • Patients should be a part of the process from research to clinical trial to help develop protocols and ethical discussions so that it better represents what is happening in the real world with those with the disease.
  • Need clear language on travel or other costs of clinical trial and what follow up it will entail.

The second part of the discussion focused on stem cells specifically, as asked what information is needed about stem cell therapies available through clinical trials and those available outside clinical trials. Feedback included these main points:

  • Scientists need to be better trained to provide direct, jargon-free answers about stem cell clinical trials and future therapy timelines for patients. As one participant commented, “We’re kind of in the wild west where we have a lot of scientists who are new to the clinical space and they need to have the tools to communicate more effectively as experts.”
  • Alignment of patient engagement protocols across the various organizations. 
  • Need for better resources on one site, including information on risks, where Canada is in relation to other countries that are offering treatments. 
  • Need a list of questions to ask when considering a stem cell therapy. List should include questions for the prospective clinic but also for the patient’s Canadian health care provider. (Author note: Lists of this sort do exist, notably one produced by the International Society for Stem Cell Research, and another produced by the Huntington Society of Canada.)
As one patient advocate exclaimed: “I didn’t know what I didn’t know! I had filled out an application online was accepted to a stem cell clinic in Panama. The cost per treatment was $21,000 and I didn’t know what questions to ask!”
The many questions about stem cell treatments outside of Canada indicated that this was an area of both interest and concern on behalf of patient advocates. They included questions regarding success rates, availability of funds for post-treatment in Canada, where treatments can be obtained and what preparation should be done in advance of treatment. In essence, they were asking “How do I assess a therapy being offered by a clinic outside of Canada?”
One reason it is challenging to answer this question is that there is no systematic way of assessing the quality or accuracy of the information, since most unregulated clinics do not publish data, so there is no way to establish an effective or safe protocol. However, it was obvious that the importance of published data and treatment protocols is not yet fully understood by the patient audience, and thus presents another item for consideration that was not explicitly raised. 
Many of the 26 people who were in that Toronto room had never had the opportunity to share or listen to the questions and experiences that framed understanding and perceptions of clinical trials and stem cell treatments. The organizers walked away with a long list of ideas, some feasible, others less so, that they can use to improve patient engagement and to create better communications products. The patient advocates and health charities will know that their insights were valuable and that further engagement will follow.