The power of patients engaging in research and clinical care

)Prof Thierry Trooster Thierry Troosters: the allied health professional's perspective

22 March, 2017

I was going to write this blog on the important meeting that the World Health Organization (WHO) hosted on the significance of rehabilitation in their headquarters on 6-7th February, 2017. The meeting was vital as it highlighted the importance of an intervention that is also of benefit to many patients with chronic respiratory conditions including COPD, cystic fibrosis, interstitial lung disease, pulmonary hypertension, oncological problems, and asthma just to name a few.

WHO has now clearly stated that there is, and will continue to be, an increasing need for rehabilitation. Policy and advocacy actions are required and health care systems need to be prepared to adopt rehabilitation in general. This call for action can be easily translated to pulmonary rehabilitation. Tools are available within the documents of leading societies(1, 2), and hence I decided to leave this important topic aside this time, as more important news crossed my path very recently.

This news was the sad message from the COPD Foundation, announcing that their founder, Mr. John Walsh, had passed away. John was an extraordinary person and an inspiration to many. I had the privilege of being introduced to this entrepreneur-patient by Dr. Richard Casaburi a few years ago when we were developing a patient-reported outcome tool to capture physical activity with the PROactive consortium. It is truly a meeting I’ll remember, and it was followed by subsequent and equally enjoyable meetings. As a clinical researcher I was not aware of the impact the COPD Foundation had. I quickly learned that it’s impact should not be underestimated. The Foundation has, through the leadership of Mr Walsh, found a unique place as a bridge between researchers and patients. The Foundation has a scientific journal, the COPD-Journal of the COPD Foundation, with whom they partner in scientific projects like COPDGene, and they also facilitate interactions with regulators. The foundation hosts the “COPD Biomarker Qualification Consortium”, a consortium of academics, the pharmaceutical industry and the Foundation, largely supported by pharmaceutical industry funding. This consortium helps to prepare submission dossiers to the regulatory authorities in order to seek qualification for end-points of clinical trials. Having such end points or biomarkers qualified obviously helps to get novel treatments for patients approved. To that end, the Foundation provides ‘neutral ground’ to host large databases with the trial results on key biomarkers or end points from stakeholders in the industry and academia. As an example, Fibrinogen levels ≥ 350 mg/dL were found to identify individuals with COPD with an increased risk of exacerbations and death. Fibrinogen has now been accepted by the FDA as a biomarker for enriching clinical trials in the COPD population(3). These are just two examples of projects where the patient organisation plays a pivotal role.

In addition, the Foundation is there for patients. In their COPD360° social community they offer a platform for patients to share experiences and ideas. For researchers, this can be a resource to identify real-world problems in patients with COPD. The website of the Foundation offers a very rich resource for patients, families and researchers. Much credit for these developments and particularly for the open minded, inclusive vision of this patient organisation goes to its founder, the late John Walsh. I have met many patients but very few had such great passion to ensure the success of a patient association.

Truth be told, I have been reluctant to see the benefit of teaming up with patients to move ahead in solving research questions. I was one of the people that believed that ‘patient representatives’ were rather professional, well-educated people that were by no means representative of my daily patient. John has singlehandedly changed my opinion in showing me that a patient organisation is much more than that. The reason why he succeeded in changing my (typically stubborn) line of thought was the fact that John exploited the best of researchers and focused on true patient engagement. In as far as I can see it, the COPD Foundation is not trying to set the research agenda, but rather wants to be informed and engages in research where it can see the benefit for the patient, taking a medium or long-term perspective. In addition the Foundation acts as a ‘connector’ between important stakeholders so that patients benefit ultimately through more efficient and rapid progress. I see it as the perfect example of the win-win in collaborating with such visionary patient associations. Through patient organisations like the COPD Foundation, patients can be informed by peers and experts and can become an active and empowered partner in their health care.

On the side of the researcher or clinician, there is surely value in reaching out to patient organisations in making scientific facts understandable and explain why we can’t (yet) solve questions such as ‘can you repair my lungs’. For example, the COPD Foundation has prepared a nice and carefully drafted statement on stem cell therapy, which many patients would potentially find appealing as a possible solution to their problems. The COPD Foundation, in clear lay language, explains that this is not a valid treatment option yet.

A patient organisation can play an active role in advocating for therapies that lack powerful lobbyists. It brings me back to my starting point. On the website of the COPD Foundation, there is convincing publicity including patient testimonies on pulmonary rehabilitation. Patients can even search for a Pulmonary Rehabilitation programme in their neighbourhood. That, however, is rather disappointing as there are still large areas in the US where no pulmonary rehabilitation seems available. Alternatively (and perhaps more plausibly) these are regions where clinicians or researchers have not yet connected to the patient organisation to make their programme known.

Dr. Casaburi, who has worked with the COPD Foundation for a number of years, helped me see the human side of the passing of John Walsh. “John Walsh”, Rich told me, “was someone who exuded competence and confidence. You always believed he knew how to get things done. He magnetically drew people in to help meet his goals; you never wanted to say “no” to John. I believe he was the most important force for progress in COPD I’ve met in my lifetime and will be sorely missed.”

In Europe, the patient association landscape is more scattered. Language is obviously an important issue and complicates rolling out a large European patient organisation that truly engages large groups of patients. The European Lung Foundation can nevertheless be the ‘glue’ between these regional societies and can assist them to lobby and speak with one voice to enhance their impact on local or regional policy. Clinicians and scientists should find their way to these societies and show an interest to be involved. You can check the societies in your region on the website of the European Lung Foundation. This tool will allow you to reach out and check what you can do for your patient association and what they can do for you. Promoting pulmonary rehabilitation is just one example, but there are many others that can connect your expertise to the patient. When properly led and carefully embedded, patient organisations are powerful engines to move multicentre and multi-stakeholder research ahead. You will not find such inspiring leaders as John Walsh everywhere, but I’m sure you’ll be able to find a reliable partner to push regionally-important aspects of respiratory care, bring them to the agenda of regional policymakers and make progress together with the people we care for.

Reference List

  1. Spruit, M. A., S. J. Singh, C. Garvey, R. ZuWallack, L. Nici, C. Rochester, K. Hill, A. E. Holland, S. C. Lareau, W. D. Man, et al. 2013. An official American Thoracic Society/European Respiratory Society statement: key concepts and advances in pulmonary rehabilitation. Am.J.Respir.Crit Care Med. 188:e13-e64.

  2. Rochester, C. L., I. Vogiatzis, A. E. Holland, S. C. Lareau, D. D. Marciniuk, M. A. Puhan, M. A. Spruit, S. Masefield, R. Casaburi, E. M. Clini, et al. 2015. An Official American Thoracic Society/European Respiratory Society Policy Statement: Enhancing Implementation, Use, and Delivery of Pulmonary Rehabilitation. Am.J.Respir.Crit Care Med. 192:1373-1386.

  3. Mannino, D. M., R. Tal-Singer, D. A. Lomas, J. Vestbo, B. R. Graham, K. Tetzlaff, M. Lowings, S. I. Rennard, J. Snyder, M. Goldman, et al. 2015. Plasma Fibrinogen as a Biomarker for Mortality and Hospitalized Exacerbations in People with COPD. Chronic.Obstr.Pulm.Dis. 2:23-34.

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