Meg Didier was born with single ventricle physiology. Half of her heart does the work that two halves are meant to do, and she has had multiple open-heart surgeries so that it can. When she describes what a faster path to new rare disease treatments means to her, she doesn't reach for a statistic. She talks about seeing her daughter graduate from kindergarten, and about holding off a transplant a little longer.
That is where the new episode of the State of the Patient podcast begins, and it is a fair summary of why the Rise Awards exist. This Thursday, Patients Rising hosts the State of the Patient Summit in the Kennedy Caucus Room of the Russell Senate Office Building, and between panels on 340B and on Alzheimer's and caregiving, we will honor nine people and one film. Every one of them was told, at some point, that the thing they wanted to change was simply how healthcare works. Every one of them declined to believe it.
Three honorees changed the machinery rather than working one patient at a time. Dr. David Ridley, receiving the Policy Innovation Award, is the economist behind the priority review voucher: develop a treatment for a disease the market has no reason to pursue, and earn a faster review on another product, or sell that voucher to a company that wants one. The government spends nothing. As former FDA rare disease official Tim Coté puts it in the episode, the idea started as an academic paper, and then Senators Sherrod Brown and Sam Brownback — about as far apart as two senators could be — carried it into law together. Research!America's Ellie Dehoney calls the result a practical miracle.
Dr. Brian Blase, president of the Paragon Health Institute and recipient of the Policy Integrity Award, is honored for arithmetic that no one else was willing to do. Former White House Domestic Policy Council director Joe Grogan describes Brian's finding that, in some states, more people were receiving premium tax credits in a given income bracket than actually lived in that bracket — and asks the question at the heart of this show: what does coverage really mean if you can't get decent care?
Dr. Alex Oshmyansky, co-founder of the Mark Cuban Cost Plus Drug Company, receives the Access Disruptor Award. His colleague Jon Horbaly tells the origin story: a radiologist at Johns Hopkins watching families absorb a child's cancer diagnosis and then, in the same moment, realize they couldn't afford the medicine. His response became the title of this episode: it doesn't have to be this way. What he built is almost aggressively simple — what the drug costs, plus fifteen percent, plus pharmacy and shipping — and it has grown on word of mouth alone.
None of that happens unless someone builds the room. Deanna Darlington, receiving the Demeshia Montgomery Impact Award, has spent a career doing exactly that, bringing policymakers face to face with the people their decisions affect. Colleagues describe her work on a nearly two-year, large-scale survey of rare disease patients and caregivers in underrepresented communities — and the afternoon she left a focus group and got on the phone to help one participant enroll in her insurance. The Caregiving Impact Award goes this year to the documentary Caregiving, a film that makes the tens of millions of Americans doing unpaid care work visible to the people who make policy.
Four honorees won't be in the room on Thursday, and all four did their work in public, under their own names. Cynthia Fisher, founder of PatientRightsAdvocate.org and recipient of the Transparency Trailblazer Award, is the reason hospitals are required to publish their prices; former White House health adviser Katy Talento describes the night before a State of the Union when Cynthia's phone calls put two sentences on price transparency back into the speech, and Governor Mike Braun credits her with getting the idea into bill form with support from Senator Bernie Sanders. Baylen Dupree, receiving the Advocacy Out Loud Award, has built an enormous audience around living openly with Tourette syndrome, and a twelve-year-old boy tells us what that changed for him: the kids who used to tease him now understand. Dr. Elisabeth Potter, receiving the Courage in Care Award, is the Austin breast reconstruction surgeon who was called out of an operation — her patient already asleep, the procedure already approved — to justify her patient's care to an insurer, and then told the country about it. And Kris McCabe and Grams receive the Heart of Caregiving Award. Kris has cared for her grandmother through Alzheimer's for nine years, much of it live on TikTok. Asked by viewers whether her grandmother still knows who she is, Kris answers simply: "I don't let her forget me."
Near the end of the episode, Joe Grogan says the thing that explains the whole event: unless we celebrate people like this, there won't be nearly as many as we need. There is a person behind every patient, and nobody does this work alone. The system doesn't get better by drifting in the right direction. It gets better because someone decides it doesn't have to be this way.
[Full Transcript] [Terry's Substack Post]
The State of the Patient Podcast is produced by Patients Rising. New episodes every other Tuesday. Follow the show on Apple Podcasts, Spotify, YouTube, and wherever you listen.
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