The Power of Patient Data

Christine Von Raesfeld (6s):
I do see the future of medicine, right? I see the value of our collective data and I think a lot of patients don't realize that. We like to look at ourselves, our own illness, and the journey that we're going through, but we're not looking how our information, and what we're going through, can actually help our entire community.

Terry Wilcox (6s):
Today, we hear from patient advocates on the power of precision medicine. Plus, the latest policy stories, including why you shouldn't expect your Medicare premium to go down anytime soon. Why? That's up next. Welcome to The Patients Rising Podcast. I'm Terry Wilcox, Executive Director of Patients Rising. We're an organization that advocates on behalf of the one-hundred and thirty-three million Americans with chronic illness. Each Friday, we highlight the latest health policy news that will shape healthcare access, and affordability, for the chronic disease community. I'm joined by my cohost, who was Tom Cruise's stunt double in Top Gun Maverick, which I hear is a great movie. He's Dr. Bob Goldberg, Co-Founder of The Center for Medicine in the Public Interest. Tom Cruise's stunt double. You were definitely wearing the black t-shirt!

Dr. Bob Goldberg (6s):
I was, but I was the stunt double for the eating scenes. I doubled as his food taster as well. If you see Tom chewing from the back...

Terry Wilcox (6s):
That's you?

Dr. Bob Goldberg (6s):
That's me. Absolutely, because as I've said many times, despite my other extracurricular activities, I'm in demand for the Olympics, and in demand for movies, but nothing comes between me and the podcast. There's no shortage of news to dive into this week, Terry, especially for us at Patients Rising, when we're gearing up for our, We The Patients Fly-In, in less than two weeks. That's on June 14th.

Terry Wilcox (6s):
That's right. We are so excited and today we'll be doing a round-up of the health policy news that patients and caregivers should know about. That includes the latest from the House and Senate, on user fee packages, which helps the FDA quickly review new prescription drugs, medical devices, generics, and biosimilars. In short, it helps keep the agency running smoothly, which patients depend on.

Dr. Bob Goldberg (6s):
If you saw your Medicare premiums go up this year, don't expect them to go back down anytime soon. We'll get into that "Why", behind the premium spike, shortly.

Terry Wilcox (6s):
Plus, patient advocate, Christine Von Raesfeld, shares how precision medicine helped her. She explains why access to genetic information can help tailor treatments and improve patient outcomes. All that and more is up shortly, but first, this week's Healthcare News headlines.

Robert Johnson (6s):
In your Health News, the White House is updating its playbook from the COVID-19 pandemic, to inform its response to a growing number of monkeypox cases in the US. Gearing-up the nation's laboratory capability is cited as an early step in the fight. As of last week, the CDC had identified ten cases of the rare disease in eight states. House Republicans are hard at work on a series of white papers, outlining their healthcare agenda, if they take control of Congress during elections this Fall. Their latest report would allow seniors to have permanent access to telehealth services from their homes. Others detail their ideas for drug pricing and public health. Telemedicine is getting good reviews in a study out of Children's Hospital in Philadelphia. Researchers there examined more than 5,800 cases involving kids with genetic diseases, and found they could just as accurately diagnose conditions remotely, as they could in-person. The only exceptions, it takes longer to collect genetic samples, and families using telemedicine, generally were white, had insurance and came from higher income neighborhoods. Finally, the American Medical Association wants doctors to learn how to treat people with opioid use disorder, but the organization doesn't want a federal law mandating the training. The group says it's not against the training, it just doesn't want an order from Uncle Sam. That's your Health News update for this week, I'm Robert Johnson.

Terry Wilcox (6s):
Catching up on some news from last week, HHS announced that it would not lower the Medicare Part B premiums for beneficiaries this year. If you're on Medicare Part B, in 2022 this year, you saw your premiums go up by $22 a month. It was the biggest spike in history with the goal of offsetting the price of a new Alzheimer's drug. However, the price-tag of that drug was cut-in-half and coverage was restricted heavily to only those in clinical trials. Despite all that, Medicare patients are still on the hook for that price-increase, all through the rest of 2022. Patients could see an adjustment in premiums for 2023. However, I have never seen the government lower something after they've raised it. It just doesn't happen. So, what's your take Pop?

Dr. Bob Goldberg (6s):
This whole thing is really just a lot of nonsense. First of all, there hasn't been a time in Medicare's history, I don't think, and I will double check just to make sure. If I'm wrong, I will talk about it the next show. This is the first time they said, we're going to raise the price, because of one drug, which by the way, we don't know what the price is going to be. We just saw what's in the paper and we're going to list....So to my mind, this was a political price hike that was not based on economics. Why? When you see the memo that CMS Actuary sent to the White House, they said, well, the premiums may have gone up by $160, and this is the best, Terry. We never had anyone raise premiums for a specific drug before, so we don't know if it's going to work out or not. Therefore, the actuaries are basically saying, we know this was made-up to begin with, but now that we've raised the prices, we can't do it midstream, because our computers won't handle it. This is basically what they're saying. Then they're saying, we're going to evaluate what new drugs might be coming down the pike next year. Now, they've found a new criteria for how they're going to raise your premiums. Last year, they were controlling for Aduhelm, and they said that the Part B premium would be $160. It would have gone up anyways. Now they're saying, that under the new scenario where the price-cuts, and the fact that you have to be in a clinical trial, had to take a trip around the world, and be left-handed, I think those are the limitations, that the premium.....

Terry Wilcox (6s):
I mean it's so bad. It's so restrictive that Biogens, basically, just sort of let it go. I mean, it is what it is.

Dr. Bob Goldberg (6s):
The idea that they will provide a refund - I am sure that now that they're on the hook, they're on the hook, but like you pointed out, they now have an "out" saying, well, other things have gone up, so we're just going to keep the costs. Medicare is not an exemplar of efficiency. It's a functional program and a lot of people, such as myself, depend upon it, but the idea that you would single-out a drug and use that, by the way, which would be used, even at the list price, there's no way of explaining how you would raise the premiums $240 a year per 40 million patients. That's a lot more than what the drug would cost.

Terry Wilcox (6s):
Oh yeah, it literally has no mathematical sense. At all.

Dr. Bob Goldberg (6s):
None whatsoever, and by the way, I was horrible at math in high school, and I still came up with better calculations than they did. There ought to be some oversight hearings on this next Congress, because you can't go about making budgetary decisions, that have a programmatic impact like this, using back of the envelope calculations and political math to boot.

Terry Wilcox (6s):
Exactly. It's something we'll be watching for you guys, because it is interesting to see how HHS, and CMS, sort of get out of this one. There are also more updates to the FDA user fee packages, which I talked about at the top of the show, making their way through Congress. This is a big topic for our Fly-Ins, since user fees help keep the FDA running smoothly. Without them, reviews for new drugs, or devices, et cetera, would slow down exponentially. This is truly crucial for patients. We're also tracking any changes to the FDA's review process, that would impact patient access, like the accelerated approval pathway. Let's look at the Senate's user fee bill, which would give the FDA power to remove accelerated approval drugs from the market, if studies aren't completed, that show a clinical benefit. It might also require manufacturers have post-market studies already in process for it to be given accelerated approval. All of this is really technical, and I know that, but we're covering it, because it impacts patient access to new drugs and treatments. So, Bob, what was your takeaway from this proposed change to the accelerated approval? Is there any way you can kind of break-this-down, in patient-speak, for why this is important for them to understand?

Dr. Bob Goldberg (6s):
Sure. I think at the end of the day, very little has changed, except to say that when a product is going through the accelerated approval process, you begin to design, or come up with, ways to collect data to double-check whether there is a clear morbidity, mortality, or other clinical benefit of the drug.

Terry Wilcox (6s):
When we're talking about post-market studies, and things like that already sort of set-up in place, what does the post-market study look like exactly? Do they have a specific expectation, or can it be something like, we're measuring this, and we're measuring that, and it's real-world evidence? Are those designed between the FDA, and the manufacturer of whatever product, or what does the post-market study look like?

Dr. Bob Goldberg (6s):
The post-market study is really supposed to focus on specific clinical endpoints that the FDA, and the sponsor of the drug, agree upon. By the way, that process now has to take into account patient reported outcomes and patient considerations. In terms of the kind of information that needs to be done, there was initially an effort to eliminate the use of surrogate or intermediate endpoints. Those endpoints, surrogate or otherwise, there's still flexibility to do that. Then there's a discussion about, do you need randomized trials? The fact that they have not called specifically for randomized trials, is a very good sign, because one of the problems that accelerated approval has had in fulfilling post-market requirements is, if the drug is available, why would they want to be in a clinical trial?

Terry Wilcox (6s):
Especially when you're talking about a rare disease drug, you know what I mean? There has to be a conversation around these very easy to understand issues. This isn't complicated. It's like if the drug is already out there and available, why on Earth would I want to get the placebo?

Dr. Bob Goldberg (6s):
The other thing is, that to crack-down and require randomized clinical trials for those phase four studies, or for the follow-up studies, would basically contradict everything else that's going into the user fee bill where they're trying to come-up with more flexible end points for approving drugs for rare diseases, for instance. Where the call for novel end points, and the use of real-world evidence, and real-world data. Why would you impose a higher standard, and rely upon a brute randomization, when you're trying to get as much real-world data collected as possible? There is a devil in the details, as we like to say on the show, and the real fight is going to be in the development of the guidance, which will take some time, by the way. Since we've also been discussing pharmacy benefit managers, or PBMs, like when haven't we been discussing them?

Terry Wilcox (6s):
Exactly.

Dr. Bob Goldberg (6s):
We wanted to quickly highlight bipartisan legislation that's been introduced to curb the power of middlemen. It was that bill, the Pharmacy Benefit Manager Transparency Act of 2022, and it was introduced by Chuck Grassley of Iowa, and Maria Cantwell, I think, of the state of Washington. She's a Democrat and Grassley is a Republican. They would eliminate the clawback fees. Clawback fees are when a PBM will issue a charge to the pharmacy, after the drug is given to the patient, saying, we need that cut as well. We need a little bit more money out from you guys, which not only inflates the price to the patient, but also damages the bottom line of independent pharmacies. The other aspects of the bill include banning spread pricing, which basically is, you buy low, and you sell high. It would make it a bit more difficult to do that and try to find more points along the way where the savings would be passed along to patients. All that's to the good and we'll be interested to see how that plays out in the months ahead.

Terry Wilcox (6s):
We can also link to this in the show notes. At Patients Rising, we submitted comments, and signed onto a large group letter of a lot of patient advocate organizations. I think it ended up being about 105 organizations asking the FTC to investigate PBM practices. We also sent our own letter. We'll link to those in the show notes. The PDM issue is really, really important. It's one that we've harped on for a long time, because we do not believe that just saying pharma, "do this", and drug prices will be lowered is an answer. A lot of people think that, and I'm not going to name names today, but there are folks out there who think that sticking it to big pharma is going to solve the patient prices at the pharmacy counter, and their out-of-pocket cost issues, and it really isn't. We do need this. We desperately need this type of investigation, and we need to have a lot of transparency for patients so that they can, as they say they would like to do in the PBM bill, ensure more savings are given to patients. As we mentioned at the top of the show, Kate's interview this week with Christine Von Raesfeld, focuses on access to genetic information and how that personalized information can improve outcomes. Bob, this is something you're involved in and you've just announced a collaborative project that would give cancer patients, and survivors, real-time access to their own data. Can you tell us a little bit about that?

Dr. Bob Goldberg (6s):
Mmmmmm.....I don't know. I don't think so. No, I will. I will. So you may know, and people in the audience may know, I co-founded a company, Prosper Digital Therapeutics, which uses technology that's now available under the 21st Century Cures Act. It allows people to access their data in real time and use that data to help customize, personalize, and predict treatment responses, and potential side effects related to diseases. Our initial project is with Lucille Adams Campbell.

Terry Wilcox (6s):
I'm so excited for her to come.

Dr. Bob Goldberg (6s):
She's great. We're partnering with the Director and Assistant Director of the Minority Health and Health Disparities Program, at Lombardi Cancer Center, to provide cancer survivors access to their own health records updated in real time. We then use that information to provide updates, but also predictive recommendations and resources, to avoid side effects. We can also use that information to optimize treatment selection to the extent that we're able to include genomic, and genetic data. Down the road, we hope that will be a repository for "real world evidence" so you won't have to reinvent the wheel and run randomized trials for everything. You would just go into the database, with patient consent, and say, we want to see how this medication would work and provide people the access to the medicine in real time, subject to FDA conditions, but it all starts, Terry, working directly with patients using the consent process to get that information and then provide guidance. Then, like what Patients Rising Concierge does, provide those supportive services. As you can imagine clinical outcomes are affected, certainly by genetics and mutations, and we've talked on the show for instance, if you don't have transportation to get to a follow-up visit, that can affect the outcome. That could affect your quality of life. It starts with the genomics, but also includes that kind of information about the everyday needs of patients to help make that outcome even better.

Terry Wilcox (7s):
I've got to tell you, and I think I've said this before many times and you know this from our conversations, the number one and number two issues that people call Patients Rising Concierge for is transportation and caregiving. Those are basic needs that can get in the way of patients sticking with their treatment, getting to their treatment, and taking their treatment. There's a variety of things that seem really simple, b,ut are huge roadblocks for patients.

Dr. Bob Goldberg (7s):
Yeah, and I know Christine will talk about it. It's one thing to be tested, for example, for a genetic marker or for tendencies. It's another to have it accessible in an understandable form, and still another, to figure-out what the logistics are to make sure that you can capitalize on that knowledge.

Terry Wilcox (7s):
Health data is truly the future of medicine. You and I both know this. To hear how this can transform care, Field Correspondent, Kate Pecora spoke with Christine Von Raesfeld. She's the Founder and CEO of People with Empathy. Take a listen.

Kate Pecora (7s):
Today I'm speaking with Christine Von Raesfeld. She is the CEO of People with Empathy and she's going to talk with us today about her experience as a patient advocate, and what she's doing within the world of genomic medicine, and access to health data. Great to talk with you today, Christine.

Christine Von Raesfeld (7s):
It's good to be here. I'm really excited about this!

Kate Pecora (7s):
Could you start off by telling me about yourself. A bit about what condition brought you into the world of advocacy and how that diagnosis really impacted your journey to where you are today?

Christine Von Raesfeld (7s):
Of course. I actually have a little bit of a different story. I have been sick since I was a child. My first surgery was at the age of five, and at fourteen, I got a rare bleeding disorder. I did get a diagnosis for lupus, but also have multiple other rare and autoimmune conditions. I am a heart failure patient, as of two years ago. It was a genetic condition, so really, my whole life has kind of been pitted in advocacy. I like to tell people it wasn't really a choice, it was kind of just a battle I had to fight. I've used my experiences in healthcare, and everything that has gone wrong for me in my life, and really tried to use those experiences and turn them around and try to find solutions, or possible things that can help in these situations, that maybe other people aren't looking at. So really, that whole advocacy career started just because my life, and healthcare, has been a series of unfortunate events. Whatever could go wrong, did. Everything from toxic poisoning from medications, lost medical records, and I'm also on social security disability. I'm 46 years old, and I went out on permanent disability at 26, and it's been a long road. Just trying to use my experiences, and the things that I've been through, to really try and shape the industry with a different, more forward, patient-focused thinking.

Kate Pecora (7s):
When we had first discussed doing an interview, one of the things that you mentioned, that was important to you, is access to genomic medicine and health information. I wanted to see if you can talk about that. Why it's important to you and what are some of the efforts you're putting towards that?

Christine Von Raesfeld (7s):
Sure. Like I told you, I have multiple conditions. My entire life has been about treating symptoms and not about treating a disease. So, in this quest to find out what was wrong with me, I've done all my research. When I was 14 years old, I was sitting in Stanford's medical library, reading through books by myself, way before the internet. I just dated myself, but I've always had this fascination with things that were wrong with me. I was never a good student. Not a good student at all, but it wasn't until recently that I was enrolled in the Stanford Humanwide program, and I was also a member of, Patients Like Me, the digital me program. I was one of their advocates and really learning about those things, and getting involved in those, showed me the potential of what was in the future. I grew-up here, in Silicon Valley, so I have a very different way, I think, of thinking than a lot of people do. I focus a lot on the technology, the data, things that I've grown-up with, that are now sort-of new, to a lot of other people, right? So really, I've learned that through my own research and connections, I've found things that were wrong with me, that doctors weren't able to find. I was able to get a pharmacogenomics test through Stanford, which told me that I metabolize all of my drugs, and all of my compounds, on a different level. I do have damage. I have three joint replacements. I deal with toxic encephalopathy, but all of those things were due to, not the medication itself, but the way it was metabolized in my own body. Knowing that now, I've been able to tailor my own treatments. I've been able to speak better with my physicians, talk about my options, and really look at the bigger picture. Now I'm in discussions with talking to my own physicians about my own genomics, and looking at old DNA samples, comparing them to new. It's that technology, I think, that's going to bring us to answers. All of us. All of us patients. We deal with so many things, so many different medications, so many side effects, comorbidities, and I found that that simple test could help avoid some of those things. As I started learning more and more about things that were in the works, and things that were happening, it was just fascinating. I just got renewed with a whole sense of wanting to learn and really wanting to find answers, and not just for me, but I have nieces, and I have kids of my own, and my nieces are everything to me. My nieces, my kids, my friends, anyone after me. I want to make sure that the experiences we all go through now, can be shaped in a way, that they don't have to do this anymore and that they don't have to fight, because we've done the fight for them. So that's really, I think, where I'm at, but I do see the future of medicine. I see the value of our collective data and I think a lot of patients don't realize that. We like to look at ourselves, our own illness, and the journey that we're going through, but we're not looking how our information, and what we're going through, can actually help our entire community.

Christine Von Raesfeld (24m 19s):
I like to talk about the importance of the collective data and how that data can help come to solutions for our communities. Pulling together resources and being able to go to collaborate and really share with each other, things that are happening, instead of being competitive. I think we're in this together and we have to realize that and that the things we have to offer are valuable.

Kate Pecora (24m 48s):
As you said, things are moving really quickly, and that is great for families, for patients and for the future of medicine. At the same time, I also recognize that your experience wasn't necessarily the experience of a lot of other people who are going through this space right now. There is a great deal of, for lack of a better word, "privilege", to be able to live somewhere like California, in Silicon Valley, where all of these things are accessible. Two questions, what could be done to make sure that we are making every effort to make this available to people who need it the most and perhaps do not have the resources to get that data for themselves?

Kate Pecora (25m 27s):
Also, on the legislative piece, we seem to be really great at developing solutions to problems that exist, but we don't seem to be great at creating legislation that prevents that from being taken too far. I can think specifically around health data companies, who perhaps don't have great privacy policies, for example. With those two questions, what are you thinking about, as it relates to access to precision medicine?

Christine Von Raesfeld (25m 56s):
I think access to precision medicine is something that eventually will come to all of us patients, but I think one of the first steps is really educating ourselves. Educating our own physicians, because a lot of the physicians aren't aware of some of the technologies that are out there. I've talked to physicians about the pharmacogenomics test, and I've had physicians tell me they have never heard of it. I think first is awareness. Making sure that people know what options are out there and organizing our patient groups. Again, going back to that, "We're all in this together", and those of us who are in areas where we have this privilege to speak out, and bring together those other groups.

Christine Von Raesfeld (26m 37s):
Making sure that those in our communities aren't forgotten as other ones of us get treatment. I think it's an interesting thing, as we come up with new technologies, I see a lot of people often being left behind as well. If you're on a treatment that's currently working, and it's good enough for your doctors, you may not get those options. So really connecting with the community, finding out what everybody else is doing, building those bonds, and building those bonds with our pharmaceutical and our payers, everybody, right? We need to have those relationships with them, because right now, in our industry, it doesn't always feel like, as a patient, we're equal stakeholders.

Christine Von Raesfeld (27m 20s):
I do think that that's going to change. I think as we educate more and more people around the value of their collective data, we become stronger. That's when we can help our other communities and when we are stronger together, when we are taking care of ourselves and really looking out for our communities, instead of looking out for ourselves individually. I think that when technology hit, we kind of took the wrong turn. Now we're going so fast-forward into the future of medicine, that we are also forgetting the people in medicine. We're at a really strange collision-course of all this great technology, that people think can take over and we won't need people anymore, but that's what we do need.

Christine Von Raesfeld (28m 9s):
It's the patient communities that are going to keep that connection with those people going. Building relationships with physicians and our care teams. Working with non-profits on options for us, but really reaching out and helping each other as a community. I think that that's what is missing. We get siloed, even as patients, into our own disease spaces and we fail to realize that we're all the same. We may not have the same diseases, but we deal with the same symptoms. We have, a lot of us, the same quality of life, the same issues that we deal with, but we tend to pull back and stick with our one community and not go out, not look, not find the options that are there for us.

Christine Von Raesfeld (28m 55s):
I think it is the responsibility of all of us patients, those who do have a little bit more privilege, and I say "privilege" loosely, because I do live on social security disability in one of the most expensive areas in the world. I've been lucky, extremely lucky to have what I have, but I also see potential for other people to have that. I think as we're moving forward with decentralized clinical trials, and the option to really go back to community and engage physicians at a community level, and get them to know their patients again, we'll be able to get to those levels.

Christine Von Raesfeld (29m 36s):
I think we have to care about each other first.

Kate Pecora (29m 39s):
Thank you so much, Christine, for sharing your thoughts today. I really appreciate it.

Christine Von Raesfeld (29m 45s):
I appreciate it too. Thank you for having me.

Dr. Bob Goldberg (29m 48s):
Thank you, Kate. To mark the start of Scleroderma Awareness Month, this week's patient correspondent, Amy, shares why more research in this space is necessary, and here's Amy.

Amy Gietzen (29m 60s):
Hi, my name is Amy. I'm from upstate New York, which is Congressional District 26. I was diagnosed with systemic stomach scleroderma, in 2001, at the age of nineteen. To say it was difficult would be putting it very lightly. From day one, I've had difficulty finding local care. Because of the rareness of scleroderma, finding proper treatment in innovative practitioners was extremely difficult. Along my journey, I realized the complexity of scleroderma required a collaborative care team of multiple specialists. Having little, to no knowledge of the disease I was actually living with, I made a decision to become an advocate for my care, and along the way, that included being an advocate for others as well.

Amy Gietzen (30m 44s):
Scleroderma is a disease like no other. It has no known cause and currently no cure, and because of that, it makes it extremely hard to treat, and find, appropriately skilled professionals to manage your disease. If I could ask for one thing, it would be for the government to fund research for scleroderma. Give scientists, and research coordinators, the opportunity to deep-dive into this disease and try to find long-term treatments, medications that are effective, and ultimately and hopefully, a cure. In doing that, it would help our community of patients living with scleroderma, have the best quality of life, the most appropriate care, and the best medication to treat their disease possible.

Amy Gietzen (31m 29s):
It would also give us hope and hope is a beautiful thing.

Dr. Bob Goldberg (31m 32s):
Thanks, Amy, for sharing your story with us. Now, other people out there, if you're listening and want to be our next correspondent, we'd love to have you on the show to discuss healthcare policies that impact you. To get started, it's really simple. Send an email to Terry and me at podcast@patientsrising.org.

Terry Wilcox (31m 53s):
We're so glad you joined us for today's episode. Now, if you learned something new, or found a story interesting, please do us a favor and pass the episode along to a friend. This helps us grow our audience and raise awareness of the challenges that face the chronic and rare disease community.

Dr. Bob Goldberg (32m 11s):
Make sure to follow the podcast on your favorite podcast app, that way you won't miss out on any future episodes. If you can't be there to listen, I will serve as your stunt double, to listen to those future episodes. How about that?

Terry Wilcox (32m 25s):
Thank you, Bob.

Dr. Bob Goldberg (32m 27s):
Oh, no problem.

Terry Wilcox (32m 28s):
We'll be back next week with more patient stories, news and insights. Until then, for Dr. Bob and everyone at Patients Rising, I'm Terry Wilcox. Stay healthy!