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
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&Dby 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 havenever been in the same room with someone who has the
condition. It is enormously inspiring.'
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.
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.'