Turning patients into partners

Bonnie Addario is a lung cancer survivor with an extensive family history of the disease. When she was diagnosed, she wanted to do something to help further the search for a cure. She thought she might hold some fundraisers and turn the money over to her oncologist. Instead, she formed two foundations that advocate, fundraise, and perform research.

That should be the modern model of a patient advocacy group, according to the Partnering for Cures panel discussion, “Powered by Patients.” The panel looked at the new breed of patient groups, weighed their importance in the process, and shared best practices of bringing patients into the R&D arena.

Patient advocacy groups are powered by the people who have the diseases or their family members, making them “wonderfully, positively motivating,” said National Institutes of Health Director Francis Collins. Families often get involved to expedite slow or nonexistent movement on disease research. Addario noted that when she was diagnosed, the survival rate for lung cancer hadn’t changed in 40 years. When Scott Johnson of the Myelin Repair Foundation was diagnosed with multiple sclerosis, he was told there would probably be a treatment within the next 25 years. But, 25 years later, there wasn’t.

Johnson looked into the medical research ecosystem and realized there was no cohesive manager of the research and translation process. For him, it made sense for a nonprofit to step into that role. “A nonprofit has more freedom to operate, fewer constraints,” he explained. Nonprofits can be the convener and manager of the process. His organization began by convening partners in academia, developing a research plan, and working closely with their own scientific team on research. The next step was to bring in pharmaceutical companies. Johnson said his organization does the work to validate and de-risk discoveries made in academia so they will be up to industry standards and will be able to be replicated. “We know we have to give them compelling enough data to get their interest,” he said of pharma companies.

Other stakeholders say the involvement of patient groups brings value to the research and translation process. Dennis Urbaniak of Sanofi reported that his company is changing the focus of how it runs its business to better serve communities in different disease areas. He said they want to listen to people who live with the diseases they treat and bring them into R&D by crowdsourcing questions on care to learn how patients actually want to be cared for.

“Most scientists welcome the opportunity to have that connection with what they are working on,” explained Collins. “They may have never been in the same room with someone who has the condition. It is enormously inspiring.”

N. Anthony Coles of Onyx Pharmaceuticals works with the Multiple Myeloma Research Foundation (MMRF) on a variety of projects, including leveraging MMRF’s clinical trials network to collaborate with patients and get information to them about trials, speeding up the enrollment process.

Onyx also invites patients to visit the company two or three times a year, “so we don’t ever lose sight of who we work for,” Coles said. “The most successful patient engagement groups are led by someone with personal experience,” he said, adding the hope that everyone who touches healthcare decisions has the kind of “transformational experience” through meeting patients he had in dealing with his son’s illness.

Patients want to contribute, stated Addario, but they don’t feel valued. “Until we invite them to our tables, to our board rooms, to the places where we are making decisions about their lives, they won’t feel valued,” she said.

She encouraged stakeholders to view patients in a new way. “We have to start to look at patients as someone who has the solution within their body. The biomarkers are there. All the keys to open the door are there in the patient,” she said.

The Patient-Centered Outcomes Research Institute was created as a provision of the Affordable Care Act to look further downstream in the translational pipeline, said Collins, and to figure out what works in the real world and in the minds of patients. “We want to collect information in a way that captures the patient experience and compares it across studies,” he said.

Lastly, Collins’ wish list included open access of information. “We can all go faster if we have the information in front of us, if we aren’t wasting our time doing experiments that already failed,” he said. He is encouraged by the strong movement to ensure that research publications are available to those who need them, and he is optimistic about the amount of clinical trial information available on clinicaltrials.gov.

Patient groups bring value to the ecosystem that can’t be ignored. To conclude the panel, moderator Susan Dentzer of Health Affairs summed up the panelists’ final sentiments: “If you are not bringing patients into your organization, you’re not just a dinosaur, you’re extinct.”