Patient and Stakeholder Engagement

Stories of Courage and Hope: Key Takeaways from Disorder: The Rare Disease Film Festival

Every so often, you stumble onto a marketing partnership that just seems to fit. When we were approached earlier this year about a rare disease festival happening in October, we knew immediately that we had to be a part of it. Who could pass up the opportunity to be part of a first-of-its-kind event that would bring patients, advocates, researchers, and drug developers together outside the standard conference (and conference room) setting?

We knew from co-founder Daniel DeFabio that hopes were high for such an event, and Disorder: The Rare Disease Film Festival did not disappoint – what started as a one-day festival had grown into two days across two venues, featuring some 30 films addressing two dozen different diseases.

The films, ranging anywhere from two minutes to an hour or more in length, shared incredible patient and family journeys. While each film addressed a different disease, there were similarities among the messages: children missing milestones, parents facing financial challenges, sacrifices made, and overwhelming pain, and some joy, as well.

And hope. There’s always hope.

The festival itself was a huge success. More than half of the selected filmmakers attended and spoke about their films. Additional speakers included patients and their families sharing their journeys, patient advocates helping increase awareness, sponsors and innovators highlighting their work and technology, and our own Juliet Moritz, head of patient engagement here at Premier, discussing the importance of patients sharing their voices in the clinical research process.

Favorite moments?  When Magic Wheelchair presented a My Little Pony cloud chariot to Dalia, a young lady with MERRF(myoclonic epilepsy with ragged-red fibers) syndrome. And we won’t forget Rare Disease United Foundation unveiling a new portrait of young Purnell, a Niemann-Pick disease type A patient, as part of their “Beyond the Diagnosis” campaign.

Engaging Patients in Clinical Trials w/ Alan Thomas

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Here are some other memorable takeaways from our two days in Cambridge:

“People are more compelled by stories than causes.”
Consider this quote confirmed, with stories ranging from two teen girls trying to find a magical plant in the Peruvian forest to watching Jimmy Cahill train for hours a day, while monitoring his lung function and sitting through multiple medications and nebulizers for several hours each day before his lung transplant.

“Not hitting milestones.”More often than not, each family’s struggle began with parents and doctors noticing children not hitting developmental milestones. Whether it was speech or motor skills, the kids just weren’t developing as expected, red-flagging parents and setting them off on their journeys.

“There’s no one way to look sick.”
A good thing to keep in mind. Patients don’t always look sick, their symptoms aren’t always external, their symptoms don’t always present.

“We don’t have to accept things the way they are.”
Too often, parents of children with rare diseases – and older patients themselves – are faced with low awareness, and even less research happening around their disease. But no one says things have to stay the way they are.

“Just be a little bit more…understanding. A little more kind.”
When asked what one thing people can do to help, one patient offered up this statement. While there may be nothing we can do to actually help the situation, just being understanding and showing a little kindness can go a long way.

“You can choose to go down the path of despair, or you can choose hope.”
It’s perhaps one of the most important decisions patients and their families (and honestly, researchers and developers) can make: choosing to move through life with hope, or giving up.

“Our job was to get the ball rolling and hopefully the next generation can keep it going.”
Parents who started a patient advocacy group from scratch knew their effort might not help their child, but might benefit patients to come.

“Tell me what it’s like to live with a disease so I can help find a therapy that provides meaningful impact. Your voice is critical. Share your voice.”
From the drug developer standpoint, the more information we the researchers know, the more we can use that information to design our trials, to work with our patients.

“I have to come to terms with the fact that I may never be diagnosed.” and “Patients would rather have a bad diagnosis than no diagnosis at all.”
In the U.S., it takes an average of 7.6 years to uncover a rare disease diagnosis. That’s countless doctor visits, a barrage of tests, and a lot of waiting.

“Go have a life, learn as much as you can, and KNOW yourself.”
Patients aren’t defined by their rare diseases, so while it can consume their lives and those around them, patients still must learn to live with it, learning who they are and what they can accomplish.

“Every day we wait, is a critical day in my daughter’s development.”
If ever there was a plea for more research, and faster, it’s this. All too often patients’ lives hang in the balance, waiting for a therapy to come to market.

“I’ve been involved in a lot of drug trials that have prolonged my life. Drug studies are really important.”Cystic fibrosis patient Jerry Cahill, the subject of the last film, closed the festival by mentioning that before he underwent his double lung transplant, he participated in many drug studies that he knows prolonged his life.

“Our children wrote the lyrics years ago but we are here to sing their songs.”
Said by a mother who lost her child to a rare disorder but continued her advocacy work, making it her life’s mission to create awareness for the disease.

“You’re not alone, and we are all in some way connected to each other.”
These two days in Cambridge bore this out, as attendees found ways to connect with each other, sharing stories, information, strength, and hope.

And one day, hopefully, a cure.