NextGen conference proceedings highlight a new model for patient-researcher collaboration

The proceedings of the NextGen Therapies for SJIA & MAS 2012 have been published in the July 2020 edition of the Pediatric Rheumatology journal online. The proceedings summarize the scientific sessions, the patient stories and the discussions about potential treatments.

The annual NextGen conference is unique in that it brings together researchers, patients, clinicians and regulators to discuss upcoming therapies for SJIA & MAS. It is a small, intimate event to facilitate collaboration between diverse groups. We often hear from participants how the conference helped them better understand the disease and treatment strategies, and decided to publish conference proceedings so these same lessons could reach a larger group.

The publication is indeed a landmark for the SJIA Foundation. We started this journey, seven years ago, after our son’s diagnosis, first with SJIA and subsequently with lung disease – a rare complication of a rare disease. We realized soon after that there were no experts to turn to and we needed to help create that expertise ourselves.

It has taken five years of work to reach this landmark – conference proceedings co-authored by researchers and patients. Not just paying lip-service to the concept of patient participation in research, but a true collaborative effort.

We started with the assumption that in rare diseases, patients are the true experts on the disease. You can mine this expertise to accelerate the search for a treatment and a dedicated rare disease foundation can help drive this effort.

Let me explain how this can work.

Typically, researchers meet patients within the walls of the clinic and learn of individual cases. It can take years to start noticing rare patterns and start research projects to find more patients who fit those patterns.

In the alternate patient-driven model, patients (even rare ones) find each other in online patient groups, sometimes within days of being diagnosed. In the daily discussions, they start finding other similar patients. A rare disease foundation can bring together this group of patients with researchers interested in that disease.

Soon after its founding, the SJIA Foundation started organizing events to bring together researchers and these groups of similar patients (we have organized 7 events so far).

Each event was carefully designed to break down the walls between physician and patient, to enable interaction and learning on both sides. Every event starts with patients on the stage telling their stories. This sets the agenda for the meeting. It also both informs and inspires researchers – they know what the acute problems in the community are and are motivated to solve it.

When we decided to write up the proceedings, we took the same approach – to make sure the writeup represents the scientific discussions without diluting the patient voice. There are many patient case reports in scientific literature, I cannot recall any written by the patients themselves. In these proceedings, the patient stories are written by parents themselves.

Another objective was that patients should be able to access the full proceedings. Much of scientific research is hidden behind paid walls. We chose to submit to a journal which is open access.

Ultimately, the goal of this effort was to surface the story of emergent lung disease and accelerate finding a treatment ultimately a cure.

Next blog post, we will talk about our search for a drug for MAS and lung disease in SJIA, and share progress with that effort.