Bringing patients and the life science industry together: Patient Advocacy +

By Robert Schultz

“Progress is personal,” according to Bob Coughlin, President and CEO of the Massachusetts Biotechnology Council (MassBio) and the father of a graduating high school senior with cystic fibrosis (CF). Coughlin’s keynote opened a unique virtual event called Patient Advocacy +, organized by swissnex Boston in partnership with Inspire.

Patient Advocacy + was a perfect example of why Switzerland opened swissnex Boston in 2000 in Cambridge, Massachusetts. swissnex Boston is Switzerland’s first hub of science and innovation and, as such, a pioneer in intellectual diplomacy. The office has become critical in the continued growth and development of the Swiss pharmaceutical industry. I am humbled to have had the opportunity to represent Inspire in this significant collaboration with swissnex Boston and share some of the insights I have learned working with these patient advocacy experts.

Mr. Coughlin and patient advocates from the pharmaceutical industry and advocacy organizations spent the morning of Tuesday, June 2 sharing their ideas and experiences of collaboration. Andrew Frates, of the Peter Frates Family Foundation, delivered an inspirational final keynote and there were several workshop conversations focused on hot issues led by Dave Bjork, Director of Development at the FRAXA Research Foundation (and recent Inspire partner), Brian Loew, CEO and Co-Founder of Inspire, Mike Walsh of Patient Advocacy Strategies, Lindsey Smith of Corbus Pharmaceuticals, and Seth Rotberg of Inspire and Our Odyssey.

The message coming loud and clear from all participants was the importance of money, collaboration, patient community and persistence in therapeutic drug development to address unmet patient needs.


March 13, 2012 is etched in Andrew Frates’ memory. It was the night when Pete Frates, his 27-year old brother, sat down with his family at their kitchen table in Beverly, Massachusetts. A professional baseball player who had never been sick in his life, Pete had just been diagnosed with amyotrophic lateral sclerosis or ALS. ALS is a rare but progressively devastating disease in which motor neurons controlling voluntary muscles die.

Seventy-five years ago, Lou Gehrig was diagnosed and died of this same disease. The experience of no treatment and no cure for ALS patients has remained the same. Sadly, the life expectancy after diagnosis averages two to five years. “At his appointment, Pete asked his doctor, ‘What can I do to help you?’ His doctor told him to raise one billion dollars for research.” That night, Pete told his family, “‘get on your work boots’ because “I’m going to get this disease in front of mega-philanthropists like Bill Gates,’” Andrew remembers.

By the time Pete married in 2013, ALS was affecting his ability to walk and talk. But Pete and Team FrateTrain were not giving up. From “walks to 5ks to Pub Nights” they kept fundraising, said Andrew. Then in 2014, Pete was introduced to the fundraising idea of the Ice Bucket Challenge where people are asked to video themselves dumping a bucket of ice water over their heads and to post the experience on social media to raise awareness and funds for a charity of their choice. Pete began asking others to take the Ice Bucket Challenge (IBC) for ALS. The FrateTrain engaged athletes from all over Boston, until athletes from The New England Patriots, the Boston Bruins, and others joined in. Soon, athletes from around the country, entire sports teams and celebrities took the challenge. On August 14, 2014, Pete took the challenge himself in the outfield of Fenway Park.

According to Andrew, from June 1 to September 1, Facebook reported more than 17 million Ice Bucket Challenge videos, viewed by more than 440 million people over 10 billion times. In just those three months, the #IceBucketChallenge was used on Twitter so often that it is rated # 6 in a recent listing of all hashtags since Twitter’s inception. Prior to the challenge Wikipedia was searched for ALS only 163,399 times in 2014, while, after the challenge in August, searches increased to almost three million. The ALS Association of the US received $115 million in just eight weeks. Worldwide donations to ALS and international affiliates equaled over a quarter of a billion dollars. “The ALS Association received donations from 2.4 million new donors,” Andrew stated. Bill Gates was indeed one of the donors.

Although Pete Frates passed in December 2019, his work and the work of FrateTrain to raise awareness and funds has led to the discovery of five new genes associated with ALS. Two new antisense drugs targeting the SOD1 and C9orf72 genes are going into clinical trials with patients.



“If you are not persistent, you don’t belong here. I truly believe that,” said David Bjork. He brings collaboration between FRAXA Research Foundation, a patient advocacy organization that supports people with Fragile X founded in 1994, and the pharmaceutical industry. “I think patient advocacy is business development.” Fragile X is a genetic condition causing intellectual disability.

Bjork, in his leadership at FRAXA, sees collaboration between patient advocacy and biotech as crucial to finding treatments. “It takes risk, it takes investment, it takes persistence, and it takes patient groups working together in collaboration with industry.” Bjork’s favorite story of working with industry involves a biotech called Tetra Therapeutics.

“They had a compound that they were exploring with Alzheimer’s and the CEO had a hunch that this might be something that would work for Fragile X, so they contacted us. We have a validation facility – a Fragile X mouse facility – so we tested this compound. The company ended up getting Orphan drug status for Fragile X. We helped fund a phase II clinical trial with them with 30 patients, we found patients for the clinical trial and worked in collaboration with Tetra, determining outcome measures that are important to move this forward.”

FRAXA advocacy is also moving toward Precision Medicine. Patients with Fragile X have a single gene, which has been identified causing Fragile X symptoms and a protein that has been shut off. “We know what causes Fragile X, so if we can find treatments that have an impact on the Fragile X population, we believe that this would have impact on patients across the Autism spectrum.”

Patient Community

“The mission of Inspire is to accelerate medical progress and we feel strongly that this can be done by actively involving patients. A lot of the best ideas for research come from patients themselves,” said Brian Loew, co-founder and CEO of Inspire. Started over 15 years ago, Inspire is the largest condition-specific healthcare community platform on the internet with over two million members.

Maintaining open, inclusive communities where patients and caregivers feel safe to share their experiences has been Inspire’s goal. Inspire members are able to discover the advice and support that they cannot find elsewhere from patients who have the same condition. Moreover, they obtain educational resources provided by the nonprofit advocacy organizations that partner with Inspire.

“When we started Inspire 15 years ago there was a lot of skepticism that it was even possible to create useful collaborations among non-profit patient advocacy organizations, life science companies and other partners like FDA and NIH. These were viewed as either competitive relationships or relationships without trust, and that has changed a lot as well,” Loew said.

Loew has been well ahead of his time in understanding the importance of patient data and patient involvement in pharmaceutical development. But in a world where some online platforms strip people’s data and, on the backend, work to “reveal you,” Lowe insists, “It shouldn’t be a feeling that, because you are sick, you should be willing to give up your privacy in order to find support from others online.” Recognizing the potential ethical threat to patient privacy, Inspire was carefully designed with data privacy in mind. Loew explained, “Ethically we have a model where we invite members to participate, we don’t force members to participate. If we ask people their permission and we ask them to affirmatively decide to participate in something or share their data, then they are willing to. We invite members to join in research, many of them do.”

Life science companies can conduct research with patients and caregivers to uncover insights that help to de-risk their product development and allow them to be more effective in their commercial efforts. Loew believes, “If you treat patients and caregivers respectfully, then what you will find is that people are thrilled to participate.”


In his keynote speech, Coughlin described how his personal story of work life dovetailed with his family life. In 2001, while running for the Massachusetts House of Representatives, his wife was pregnant with their third child. Having two healthy children, they were shocked to learn that they were both carriers of the cystic fibrosis (CF) gene and that their unborn child had CF. “It changed my world”, he said. “When Bobby was born, I thought doctors invented cures. I quickly found out that cures need to be bought–that the toughest thing in the world is to invent a therapy or a cure for an unmet medical need.”

Not long into his political career he realized that Massachusetts had become a state that was not friendly to industry. “I started gravitating to how could I make Massachusetts a better place for innovation.” The biotech industry held the key to cures. “I was spending more time working on the biotech industry because I was being consumed by the fact that I couldn’t sit idle. Not being a doctor, not being a scientist, what could I do so this beautiful baby wouldn’t die?”

Working with the Cystic Fibrosis Foundation (CFF), he learned about fundraising and investing in companies. “At the CF Foundation we set up Bobby’s Brigade, we got involved. Our goal was to raise $100 Million for the Cystic Fibrosis Foundation. We raised over $500 million and invested in companies like Vertex.”

Since 2007, Coughlin has been building collaboration through MassBio with academia, industry and government in Massachusetts. His efforts have brought top pharmaceutical companies to the state, saving the nearly 500 biotech companies that would have gone out of business during the Great Recession, and nurturing a climate favorable to the pharmaceutical industry.

patient advocacy

Image shared by Robert Coughlin at Patient Advocacy+ Credit:JONATHAN WIGGS/GLOBE STAFF

In the meantime, Coughlin’s son Bobby, continued living with cystic fibrosis. “Over the last 18 years, I have had a lot of ups and downs. The first few therapies for cystic fibrosis didn’t work for Bobby because of one of his tricky mutations. It’s devastating to be involved in the Cystic Fibrosis Foundation, to raise so much money and to see drugs invented that don’t work for his particular combination of mutations. But we never give up.”

On January 1st, 2019, Bobby Coughlin and his dad gave an interview to the Boston Globe. At that time, Bobby was taking between 50 and 100 pills a day, spending at least 45 minutes per day on a nebulizer and 20 minutes a day using a sonic defibrillator to loosen mucus. “We were on the front page of the Boston Globe,1 talking about the fact that we were running out of time.”

November 8, 2019, was a turning point for the Coughlin family. Trikafta, a new medication, was approved for children 12 and up.

“My little guy took his first dose of Trikafta. He’s graduating from high school on Saturday. We didn’t expect that. His lung function now is better today than it was three years ago. He’s gained 15 pounds in the last 6 months. He’s taller than me, he’s grown like 3 inches as well. So everything that all of you do as patient advocates and people that work in the life sciences industry, there isn’t anything more important. This is personal.”

What came out of this event is the understanding that science alone is not enough. Fundraising, collaboration and persistence are critical components to including the patient voice in pharmaceutical research. Amazing things can happen when people come together in support of those with unmet needs. Coughlin said,

“I’ll never give up. I’m not going to give up until every person with cystic fibrosis has a therapy, I’m not going to give up until there is something for ALS, Alzheimer’s, Duchenne muscular dystrophy.” He encouraged the life science industry professionals at Patient Advocacy + to keep working: “You go and go folks, because we have the most honorable profession in the world.”

More Is Yet to Come

While this event served as a basic overview of some of the hot issues in patient advocacy, we are just getting started. Let’s keep the conversation going via social media using the hashtag #patientadvocacyplus. What issues would you like to discuss and/or learn about at future Patient Advocacy + events?

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