Patient and Stakeholder Engagement

Why the time’s right for a Rare Disease Film Festival

Premier Research is proud to be a presenting sponsor of “Disorder: A Rare Disease Film Festival,” being held in Boston in October. Festival co-founder Daniel DeFabio shares with us why having such an event is important for raising awareness, sparking conversations and hopefully, maybe even a cure or two.

A couple years ago, I made a documentary to bring awareness to my son’s rare disorder Menkes Disease. That short film enjoyed some successes: Oscar-nominated actress Mary McDonnell agreed to replace my voice as the narrator, the film won best documentary at a festival, it got thousands of views online, and it screened to audiences of 500 orphan-drug professionals at a conference to standing ovations. Each of those successes felt great, perhaps even like progress. But if your aim is advocacy, then your goal is to get better treatment or a cure. By that standard, the film fell short. I felt I needed to find more audiences for it.

At a Global Genes rare disease conference, I met Bo Bigelow, whose advocacy for his daughter’s condition impressed me. Her disorder related to the USP7 gene was so rare it didn’t have a name yet. It was only through Bo’s efforts that the few USP7 families in the world each other and found a researcher looking into the mutation. Bo had also made films. We got to talking. Months later he joined me at a film festival for a screening of my documentary. While it was a very nice event, the crowd was small and more interested in the films with a local connection than a disease they’d never heard of.

My experience submitting to various film festivals made me realize that while a few festivals might have a focus on science or medicine, there weren’t any festivals dedicated to rare disease. And while medical conferences were in some ways the ideal place to screen a rare disease film, film screenings tend to be an exception there, a rarity.

Bo and I began to think: What if there was a film festival that combined the general interest from the public of a typical festival with the more motivated specific interest from advocates, industry, and researchers found at a conference. Could we create that event?

I had experience running a general interest film festival, the Ballston Spa Film Festival. I had created the event and run it for four years. It is now in its tenth year. So creating a rare disease film festival seemed feasible. Bo liked the idea and agreed to co-direct the event, in part because any rare disease advocacy film, including mine or his, can struggle to reach audiences beyond the core group already concerned. Gathering multiple films on multiple disorders seemed a logical extension of the advocacy slogan “Alone we are rare, together we are powerful.”

And so, “Disorder: The Rare Disease Film Festival” was born.

We decided to host the event in a city where neither of us lived but each had to travel to for advanced care for our children: Boston. The concentration of medical, research, biotech, and genetic professionals in that city meant we’d be going where our best audience already was.

The response to our idea has been overwhelmingly enthusiastic. Early on advocacy groups like NORD, The Mighty, RDUF, MassBio, and Global Genes agreed to become our promotional partners. And soon great sponsors came on board like Premier Research, The Menkes Foundation, The Patient Experience Project, Horizon Pharma, Sanofi-Genzyme, Shire, Alnylam, Cambridge BioMarketing, 32 Mile Media, and FDNA. Filmmakers have responded as well, with 30 selected films covering two dozen different diseases already added to the agenda.

Will Disorder meet our goals of better treatments or cures? We’ll find out after October 2-3. Grab your tickets, and we hope to see you there.