Ask anybody working in the rare disease space and they will all agree: raising awareness is one of the key ways to driving research and effecting meaningful change in patient lives. But for those who are not directly affected by the disease, it can be challenging to convince them to care.
“When you hear a term like ‘rare disease’ it feels so abstract, so far away,” said Kristopher Sarajian, vice president of marketing at Signant Health. “Film has a unique power to close that gap and connect audiences with the real people and the real families behind the statistics in order to share their stories, convey empathy, raise awareness, and ultimately drive action.”
Signant Health, the patient technology company formerly known as CRF Bracket, develops technology-driven solutions to enhance patient engagement and clinical trial efficiency across many therapeutic areas, including rare disease. Yesterday, they announced that they’re one of the latest sponsors of DISORDER: The Rare Disease Film Festival (DISORDER: RDFF), a two-day event to be held at Mission Bay Conference Center in San Francisco, CA.
The film festival will feature more than 50 films on rare disease topics from the creative minds of grassroots organizations and major motion picture filmmakers. Bo Bigelow and Daniel DeFabio, co-founders of the festival and parents of children with a rare disease, hope that the collection of documentaries, animations and fictional stories will highlight the struggles and experiences of people living with rare disease.
“These films put the often abstract and dire clinical information into the context of real people living their lives,” said DeFabio. “We believe increased awareness can connect patient families, inspire conversations about translation applications of current science, lead to new paths for research and save lives.”
DeFabio’s son lives with Menkes disease, an X-linked disease that affects physical and cognitive development and is characterized by progressive deterioration of the nervous system. DeFabio teamed up with Bigelow after meeting at a patient advocacy conference and learning that each had created films about their child’s rare disease. Bigelow’s daughter lives with USP7, a genetic disorder characterized by autism, intellectual disability and seizures.
These are two of more than 7,000 known rare diseases, many of which are incurable. Despite the prevalence of patient advocacy groups and research labs dedicated to finding a cure for one rare disease, there still exists a gap between how this research actually reaches patients.
“All credit to the people who dedicate their lives to researching a drug, but they are often the first to admit that sometimes they get lost in the lab and forget the human being they are working for,” said DeFabio.
It’s not just the scientists that may forget about the patient, but the clinical trial sponsors, too. Patients with rare disease may face geographical or financial barriers to participating in clinical trials, which deny them the chance to access lifesaving medicine. To address this, companies like Signant Health are focused on increasing patient engagement through removing these barriers and keeping patients on track with study requirements. One of their digital solutions is TrialGuide, a mobile application which increases study compliance and completion.
By uniting industry professionals, researchers, advocates and rare disease patients through film, Bigelow and DeFabio hope more people will start talking about rare disease.
“We believe increased awareness can connect patient families, inspire conversations about translation applications of current science, lead to new paths for research and save lives,” said DeFabio. “It all comes back to that well-known slogan: ‘alone we are rare, together we are powerful.’”
Signant Health joins Vertex, Ovid Therapeutics, Openly Rare, Biomarin, BridgeBio, Bausch Health and the Menkes Foundation as a producer sponsor for DISORDER: RDFF.