In the summer of 2014, the ALS Ice Bucket Challenge took the world by storm, becoming a global social media phenomenon and raising more than $220 million in the fight against ALS. Pat Quinn, who had been diagnosed with the disease in March 2013 and launched the challenge with fellow patient and advocate Pete Frates, never imagined it would take on such a life of its own.
“It’s really amazing, the power of social media. What resulted from it is the most amazing thing. It created hope among patients who really didn’t have any,” said Quinn, closing out MassBio’s Patient Advocacy Summit on Oct. 27. He was joined at the event by Andrew Frates, Pete’s brother.
“August 2014 was a crazy, crazy month for my family and I like to think of it as the first building block in the end to ALS,” said Andrew, who has become his brother’s caretaker.
Both Quinn and Frates were diagnosed with ALS before their 30th birthdays, and the pair—hailing from Yonkers, N.Y. and Beverly, Mass. respectively— came together in the hope of spreading greater awareness of the disease. Their campaign elevated ALS to a topic that was discussed in the homes of millions of people. The funds raised are now being used to support ALS care services, public policy initiatives and research, including four collaborative research initiatives to build understanding of thedisease, target new therapies, expedite clinical trials, and make DNA and RNA sequencing data available to the entire ALS research community.
“I think a big lesson is that advocacy starts with the patient,” said Quinn. “If you’re not willing to fight, who’s going to? I knew early on I would be proactive. You have to put yourself out there to make things happen.”
Quinn’s lesson and the theme of building a patient-centered biotech was consistently repeated throughout the second annual summit, which brought industry leaders together with patient advocates and other stakeholders to examine ways in which life sciences companies can more fully incorporate the patient voice into the work they do—not just approaching regulatory applications or at commercialization, but throughout the drug development cycle.
Conversation circled around the importance of the patient dialogue, particularly as medicine is becoming more personalized.
“With rare diseases, the patients and their families are really the ones who know the disease better than anyone. Patient advocacy is part of the fabric of what we do. I would cut everything else before I would cut that. Having someone remind us of our purpose is critical,” said Richard Peters, Head of Rare Disease at Sanofi Genzyme. He joined bluebird bio Chief Financial Officer Jim DeTore to discuss their companies’ efforts to build, maintain and measure patient advocacy programs.
“As rare patients, we realize we can’t sit back and wait for an individual doctor or company to come up with a cure,” said Kathleen O’Sullivan-Fortin, board member of ALD Connect—an international, non-profit group of ALD patients, patient advocates and researchers, who collaboratively educate, advocate, and conduct clinical research among those affected by X-linked adrenoleukodystrophy (ALD).
O’Sullivan-Fortin, who has a 10-year-old son with ALD, joined Michele Rhee of bluebird bio to talk about collaborating to achieve impressive outcomes including ALD being added to the federal Recommended Uniform Screening Panel. The pair was presented with MassBio’s Caring
“It’s amazing what you can accomplish when you don’t care who gets credit,” said O’Sullivan-Fortin. “We’re trying to build a big, strong net to catch the cure. As a mom, it doesn’t matter who finds a solution first. I hope everyone is wildly successful.”
Another parent, Ron Suskind, a Pulitzer Prize-winning journalist and best-selling author, shared his experiences as caregiver to his son, Owen, who was diagnosed with regressive autism at the age of three.
Recognizing Owen’s affinity for Disney movies, Suskind began channeling characters
to open up communication with his son.
“Look through the eyes of the patient, it’s amazing what you’ll learn,” said Suskind in an emotionally-charged keynote address.
Capping off the day’s case studies, Victoria Dibiaso of Sanofi and Dr. Steven Edelman of Taking Control of Your Diabetes (TCOYD) talked about their work together to design a clinical trial that fits the individual needs of each patient.
“We wanted a two-way conversation to incorporate patient insights, reinvent research models and make smarter, faster decisions,” said Dibiaso. “Based on feedback, we’ve been able to simplify our prototype and reduce development timelines. If we have the ability to make someone’s life better, we have an obligation to do it fast and efficiently.”
The day-long event, held at Google’s Cambridge campus, also included panel discussions on how technology and patient data are changing drug discovery and development and how companies can set clear expectations for the patient in the case of expanded access.