Through the Eyes of the Patient: MassBio’s 2nd Annual Patient Advocacy Summit

Nov 02, 2015

PA Summit Logo draft final

MassBio’s 2nd Annual Patient Advocacy Summit educated & inspired the 200+ attendees that gathered from the life sciences community.

Keynote: Ron Suskind

The day kicked off with an unforgettable keynote by Pulitzer-prize winning journalist and best-selling author Ron Suskind. Ron is the father of Owen, who was diagnosed with regressive autism at the age of three. Ron lamented over the years during which he and his wife struggled to connect with Owen. Finally, Ron broke through. How? He listened to the patient.

Owen is obsessed with Disney movies. Ron took a leap of faith one day and picked up an Iago puppet from Aladdin off Owen’s bedroom floor. Ron channeled the character and opened up a dialogue with Owen. It was a pivotal moment that would change the rest of their lives.

ron and owen

Finally, Ron had broken through and found a way to communicate with his son.

Ron’s heartfelt keynote drove home a patient-centric message. His closing words were powerful ones:

“Look through the eyes of the patient, it’s amazing what you’ll learn.” – Ron Suskind


Panel: The Business Case & Metrics for Patient Advocacy

After Ron’s moving keynote, we got right down to business for “The Business Case & Metrics for Patient Advocacy” panel. The panel featured Richard Peters, Head of Rare Disease at Genzyme and Jim DeTore, Chief Financial Officer at bluebird bio. Bob Tedeschi, a reporter from Stat News, moderated. The discussion was focused on truly walking the walk in patient advocacy and working to incorporate patients across the entire drug development process.

Key Takeaways

  • Richard Peters earned applause from the audience when he said, “I would cut everything else before I cut patient advocacy,” and advised, “Don’t use patients as props!” His perspectives illustrated that a genuine, authentic patient advocacy program is critical for a life sciences organization.
  • Keep open lines of honest communication. Even if patient groups disagree, that’s OK. Peters said that he worries if patients stop communicating.
  • At bluebird bio, Patient Affairs reports to the COO because it is integrated into every piece of the organization. DeTore and Peters stressed the importance of having the patient voice “at the table” for decision-making.

Case Study #1: International FOP Association & Clementia Pharmaceuticals

Next on the agenda was a case study featuring the patient-industry partnership between the International Fibrodysplasia Ossificans Progressiva Association (IFOPA) and Clementia Pharmaceuticals. The strong collaboration allowed the two organizations to make a real impact. IFOPA bridged the gap between patients and industry and helped Clementia recruit over 50 patients for their Natural History Study. Both organizations garnered a wealth of information from this study to inform education and research initiatives.

Betsy Bogard of IFOPA and Donna Grogan of Clementia credit their success to open, honest, and ongoing communication and an understanding of each others’ goals.

Check out their lessons learned:

lessons learned case study 1

Fireside Chat: The Value of Medicine

Following the first case study was a Fireside Chat on the value of medicine. This panel looked at Alzheimer’s from the patient perspective and the researcher perspective. It examined the important role that detection and medicine can play, and the economic and policy challenges the life sciences industry faces as it works to bring innovations and therapies to patients.

value of med 2

The panel featured Mike Simmons, Director of Advocacy and Professional Relations at Eli Lilly, Dr. Paul Solomon, Clinical Director at the Boston Center for Memory, James Wessler, President & CEO of the Alzheimer’s Association, Massachusetts and New Hampshire Chapter, and was moderated by Susan Tracy, President of The Strategy Group.

Caring Collaborations Award

And the award goes to….

bluebird bio & ALD Connect! Michele Rhee of bluebird bio and Kathleen O’Sullivan-Fortin of ALD Connect were shocked and honored to be presented with MassBio’s first ever Caring Collaborations Award recognizing an outstanding patient-industry partnership.

Michele & Kathleen went on to present a case study on their partnership. Michele was clear that bluebird bio is committed to understanding the patient perspective and incorporating it into all decisions.

caring collab 2

Kathleen made a critical point when she stated:

“It’s amazing what you can accomplish if you don’t care who gets credit. We just want to save people suffering.” – Kathleen O’Sullivan-Fortin

The two organizations worked together to achieve impressive outcomes including ALD being added to the federal Recommended Uniform Screening Panel.

What is Health Care Data & Why Should We Care?

Anything is possible, it seems, by harnessing healthcare data. But what is the data? Where does it live? Who has control over it? How can it be linked together and analyzed for good? This panel took a look at how technology and patient data is changing drug discovery & development, as well as the relationships between patients and industry.

healthcare data panel

The panel featured Andy Dorner, Vice President of Translational Medicine at Takeda, David Leventhal, Director of Clinical Innovation and Worldwide Research and Development at Pfizer, Ann Lucas, Genetic Counselor & DuchenneConnect Coordinator, and was moderated by Jennifer Joe, CEO, Medstro & Editor of MedTech Boston.

Key Takeaways:

  • Data sharing is critical. A study currently underway by Takeda Oncology and the MMRF is the first of its kind that provides open data. Data sharing must become the norm. Accessible data is what will bring us closer to cures.
  • Healthcare data is your data. A patient’s data belongs to the patient. It is important to ensure patients are aware of this and arm them with the knowledge that they can use it for the greater good.
  • Every patient has a story and it’s important that we listen. Patients and families have a huge voice to lend in data.

Case Study: Sanofi & Taking Control of Your Diabetes

Sanofi and Taking Control of Your Diabetes (TCOYD) took the stage next. Victoria Dibiaso of Sanofi talked about their mission to ensure the patient was involved from pre-clinical discovery all the way to commercialization. Together, Sanofi and Dr. Edelman of TCOYD worked together to design a clinical trial that fit the individual needs of each patient.

sanofi and tcoyd

Quotes from the collaborators capture the essence of their partnership:

If we have the ability to make someone’s life better, we have an obligation to do it fast & efficiently.” – Victoria Dibiaso

“I never realized that a pharma company could care about me.” – Patient in the diabetes clinical trial. “We do! Patients are what keep us motivated!” – Victoria Dibiaso.

“Bringing patients to the table is a game changer. Patients want to have a voice and make a difference.” – Dr. Edelman

Check out Sanofi & TCOYD’s lessons learned:

lessons learned case study 2

Navigating Expanded Access

Industry, regulatory and patient stakeholders discussed the various categories of expanded access (compassionate use, right to try, breakthrough therapies, etc.) and the complex decisions and discussions that take place in those scenarios.

expanded access

The panel featured Derek Gavin, Director of Development at NORD, Dan McIntyre, Senior Vice President of Corporate Affairs, Biogen, Rich Moscicki, Deputy Center Director for Science Operations, Food & Drug Administration, Dan McIntyre, SVP, Corporate Affairs, Biogen, and Kevin W. Mayo, Senior Director, Global Patient Access and Advocacy, Baxalta. The panel was moderated by Jamie Ring, Head of Patient Advocacy at Spark Therapeutics.

Key Takeaways

  • The FDA is very supportive of expanded access. It approves 99.4% of applications if they believe the potential reward outweighs risk factors.
  • It is important to have an expanded access policy and it must be a fair one that is created in tandem with patient advocates.
  • Doctors are responsible for setting clear expectations for the patient that the drug might not work.
  • Rich Moscicki, Deputy Center Director for Science Operations at the FDA reminded the audience to think about the patient’s interpretation of the press releases you publish:

“Think about what you say in your glowing press release aimed at investors but read by patients.”

Pat Quinn & Andrew Frates

The day ended with an empowering keynote from Ice Bucket Challenge Co-Founder Pat Quinn and Andrew Frates (Pete Frates’ brother).

Pat told the story of his devastating diagnosis, while Andrew shared the pain felt through the entire Frates family when Pete was diagnosed.

final keynote

Together the two talked about the astounding impact of the Ice Bucket Challenge. The social media campaign raised an unprecedented amount of funds and awareness but – equally important – it provided hope.

“We created hope among patients who didn’t have any.” – Pat Quinn

The advocates inspired the audience, communicating that the work of patient driven industry professionals is a critical part of finding cures.

“What you do is crucial. Engaging with patients is crucial.” – Pat Quinn

It was an incredible way to end a jam-packed day all about the patient. Thank you to all who attended! We hope you found it enriching and inspirational and we look forward to seeing you next year!

Be sure to check out the Storify recapping the #PatientDriven tweets from the Summit and follow this link to view the live-stream of the entire day!

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