Nov. 6, 2023

Patients Need Treatments Fast

Patients Need Treatments Fast

There’s a new bill that could help many rare disease patients gaining traction, but it still has a long way to go. This week, we’re joined by Peter Pitts, a Former FDA Associate Commissioner, who gives us the lowdown on the Promising Pathways Act....

There’s a new bill that could help many rare disease patients gaining traction, but it still has a long way to go. This week, we’re joined by Peter Pitts, a Former FDA Associate Commissioner, who gives us the lowdown on the Promising Pathways Act. He tells us what types of diseases are likely to be affected by this bill, and why it will make a significant difference in the lives of patients. 

We also speak with Senator Mike Braun of Indiana, who introduced the bill alongside other legislators of both parties. 


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Peter Pitts  0:00  
The Promising Pathways Act is pretty straightforward and allows the FDA to provisionally approve applications that demonstrate substantial evidence of drug safety, along with relative evidence showing it improves therapeutic outcomes consistent with or greater than currently marketed on label therapies.

Terry Wilcox  0:21  
This week on the Patients Rising Podcast, a bill that can help many rare disease patients is gaining traction. But there's still quite a way to go before it's reality. How could this bill help you or someone you love? A former FDA Associate Commissioner tells us welcome to the Patients Rising Podcast. I'm your host, Terry Wilcox, CEO of Patients Rising, I'm joined by my co host, who just now finished removing all the makeup from his Grimace shake. All right, that is a really great visual. He's Bob Goldberg, co founder of the Center for medicine in the public interest. Now how much purple paint did it take?

Bob Goldberg  1:03  
Well, you know, I had this is the last time I'm doing Grimace. I think everybody knows Grimace. Grimace is a shake that McDonald's ran last summer, it's purple because there's blueberries in it. People thought I was a barium enema. So forget it. I'm not doing it anymore. But in addition to having a little congestion, I have a lot of helpful information for patients. And I know you do Terry as well.

Terry Wilcox  1:30  
while we always do, Bob, and today's episode gets into a piece of legislation that could literally be life changing and life saving for some patients. The Promising Pathway Act was introduced by bipartisan groups in both the Senate and the House. And will create a provisional approval pathway for promising treatments for fatal diseases that progress rapidly and have few or no treatment options. Now, what does that mean for patients Bob in plain English?

Bob Goldberg  2:03  
Well, I think in plain English, it means that if you have a disease that's rapidly progressing, and there's a drug that's supposed to treat it based upon the existing evidence, you could have access to that drug. And as more and more information about the drug comes in, you can either expand the use or adjust the dose or so on. So, Peter Pitts knows a lot about Promising Pathway Act. Now who is Peter? We've had him on the show. He's a former FDA Associate Commissioner, currently the president and my co founder of the Center for Medicine in the public interest. And we interviewed him and tells us what sort of diseases this bill would affect.

Peter Pitts  2:51  
These are for products that address series of life threatening conditions, conditions, like ALS, or Friedrichs ataxia, or a whole variety of orphan diseases that are oftentimes not pursued by developers, because the FDA is burdened is so heavy on the front end, that they can't afford to put the resources into it. What the promising pathways act shows is that there are other ways to bring drugs to market other than the traditional gold standard, randomized control, large scale long term expensive clinical trial proposition.

Bob Goldberg  3:26  
So Peter knows from his time at the FDA firsthand, just how complicated all the development processes are.

Peter Pitts  3:35  
This is an incredibly difficult process. You know, bringing a drug to market, even for simple disease, even for allergy medications, is very complicated and highly scientific. And what the Promising Pathways Act does is that it allows the FDA to use different types of evidence generated in different ways to be used for review, and hopefully, ultimately for approval. So these drugs can get given to patients.

Terry Wilcox  3:59  
But a treatment nobody is able to get isn't doing anyone any good.

Peter Pitts  4:04  
A drug that's not reached patients quickly is unhelpful. You want to make sure that new therapies get to patients as quickly as possible, so they can work as well as they possibly can. And by provisionally approving a drug, you're allowing it to come to market. And the FDA will have to say on the label of these drugs, again, according to the promising pathways act that these drugs had been approved, provisionally and explain what that means. Something else the Promising Pathways Act does that no other pathway in the FDA does is that it tells payers, both public CMS and private insurance companies that they must reimburse for these drugs just as they would for any other FDA approved product.

Terry Wilcox  4:49  
So Bob, on this Promising Pathway Act, what are some of your initial thoughts on this bill? I mean, I've read it, it's pretty straightforward. 

Bob Goldberg  4:58  
Yeah. So, you know, there's probably some concern that, you know, if it ain't broke, don't fix it. I would say, however, that the Promising Pathway Act has an educational value in that many people on the Hill, many staffers, they're young, and they don't know the drug industry, they don't know if this has been a way to sort of educate people about the complexities and the barriers that patients face. 

Terry Wilcox  5:29  
The diseases, and we're talking about many of them here. We listened here at Patients Rising to several stories, many parents over the past several weeks, and diseases are progressing faster than the FDA approved treatments. And a lot of the things that come out from Nord and others is we need to just keep pushing on the FDA. There's already levers in place that the FDA Well, what are they?

Bob Goldberg  5:57  
Yeah, one thing that is problematic at least in the bill, I think it's been left out, is there was supposed to be a office of patient advocates. So you could take your concerns directly to a designated officer at the FDA that was stripped out of the bill. But it underscores the fact that I've just heard too many people, they just they need a medicine, they can't get it, they're going to die waiting.

Terry Wilcox  6:24  
The worst part, Bob, to me is when they're on a medicine, and they've been on a medicine many years that's keeping them stable. Yes. And that becomes threatened. That is the most problematic piece of this to me. And that needs to be resolved. So Bob, from Peter's opinion, who would most benefit from the Promising Pathway Act?

Bob Goldberg  6:46  
So we wanted to make sure that we directly asked Peter that question, because that was the $64,000 question. And he said it would be many of the diseases and disease organizations that Patients Rising represent.

Unknown Speaker  7:00  
Clearly, you have the entire orphan disease population, many diseases have no treatments, despite the tremendous success of the Orphan Drug Act, there are still many orphan diseases without any labeled therapies. And this would help I think those people first but even larger disease states, which will bury us as a nation, unless we really give the thumbs up in the green light to developers to help move very exciting things forward through new, very solidly science based evidence generation techniques.

Terry Wilcox  7:33  
All of these things sound like real positives. Now, Peter says there's a fallacy that this disregards safety, I want him to clear that up for us. People

Peter Pitts  7:45  
People say well throw safety to the wind, you know, it doesn't. And the FDA says we don't need new authorities. I believe that it does. And I believe that this new pathway, the Promising Pathways Act, will add provisional approval and make a significant difference not just to the lives of patients, which always has to be first. But it'll also show companies who invest billions of dollars of venture capitalists who invest in startup companies with very exciting new medical technologies, that it's important to keep these companies afloat to make sure these programs get green lighted, and do the right thing. It's a partnership between developers and investors, and the FDA and patient that's that's more than promising.

Terry Wilcox  8:22  
I agree with him. I mean, after the the few weeks, I've spent learning more about some of the patients and families who are advocating for this pathway very strongly, and why they are and what their situation is, I have definitely come to see this in a new light. And to really take a look at it as a way to move the dial forward. And especially the conversation forward.

Bob Goldberg  8:49  
Well that raises the you know, the ultimate question, Terry, and that is what can patients do to make this legislation law. And Peter had some suggestions about how we have Patients Rising, can make it happen by doing what it always is done, and that's, you know, courageously amplifying the patient voice.

Peter Pitts  9:11  
Well, I think from a drug development perspective, you know, individual patients and patient organizations more broadly need to become a more equal partner with both drug developers and the FDA in the development and review and ultimately, the launching of these products into the market. And that includes not just better labels and more aggressive types of pharmacovigilance, but also making sure that payers again, both public and private, aggressively reimburse these projects, based on the therapeutic outcomes they provide, versus some bizarre, self interested mathematics that nobody can understand.

Terry Wilcox  9:49  
That's my new Kabuki math bizarre self interested mathematics. That's it bizarre self interested  mathematics.

Bob Goldberg  10:01  
Yeah. Your calculations are always tell you how beautiful you are and that you've actually lost weight. Yeah, exactly.

Terry Wilcox  10:10  
So this is a bipartisan bill, everyone. It is it is a bipartisan bill, what a thought, something that can still happen here in Washington DC. Now look, it makes sense for those who need it. Um, it's not the perfect bill. It's not for every situation. It's not for every drug. But there are people out there that need something like this, they need something. And the Promising Pathway is the most promising pathway, so to speak, out there right now.

Bob Goldberg  10:40  
Well it makes sense. And that may be that's, it's, it's a bipartisan bill. That is common sense, Terry, we should celebrate these rare moments.

Terry Wilcox  10:52  
And I also think, Bob, I think it also brings to light several things. It brings to light the importance of the accelerated approval pathway. It brings to light the importance of all the work that's being done in the space and sort of what the Orphan Drug Act has been able to create from an innovation standpoint. And we need to want to continue that. Well, thank you so much to Peter Pitts, for not only coming on this podcast, but for voicing his support for the legislation, with letters to Congress as well.

Bob Goldberg  11:26  
So if you want more information on Peter or me, you can use the link in the show notes or just contact me through the show.

Terry Wilcox  11:34  
We also had the pleasure of speaking with Senator Mike Braun of Indiana about the Promising Pathway Act, which he introduced alongside other senators of both parties. Senator Braun had a family member who passed away from ALS and is a member of the ALS caucus, those experiences opened his eyes to the issues those with rare diseases face.

Mike Braun  11:54  
Now that I learned more about it, it's in a category kind of by itself, still considered having probably too few people affected by it, to get a lot of the attention that larger drug companies give to efforts to try to find a cure or therapy, something working. And it was very frustrating because the prognosis is so short, in terms of timeframe, people are in clinical trials, and it just looked like nothing was happening there to give hope.

Terry Wilcox  12:30  
When we asked Senator Brown what he sees the Promising Pathway Act accomplishing, he said this:

Mike Braun  12:36  
So what it's doing is just putting some practicality into the whole process of anything that enters into the FDA process. If it's showing promising results, rather than having to wait 15 years to the endpoint that is typical on getting full approval, it creates provisional approval. So once you've gone through every process, and it's out there in the field, showing benefits, it gets provisional approval to match it up with some of the dire prognosis that many conditions have, especially neurological ones of having just a few years to live.

Terry Wilcox  13:23  
We are working with Senator Braun on this bill and hope to see it continue to move forward. More information on the Promising Pathway Act is in the shownotes.

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Bob Goldberg  14:16  
A few episodes ago we told you that the Centers for Medicare and Medicaid services would be hosting a series of patient focused listening sessions as part of the Medicare drug price negotiation program.

Terry Wilcox  14:32  
Those sessions are now underway. Now there's some big medications in there that many, many people are on things that you're all going to recognize like Eliquis and Embro, and others. There's diabetes medications, we have a full link to all the medications that are being reviewed in the show notes. I know Bob that you listened in on some of the Eliquis listening session which was one of the first I think it was the first. So what did you hear? And what are your thoughts on the listening sessions in general?

Bob Goldberg  15:06  
What do you mean, we pretend to listen session? Yeah. Well, the best statement, I thought was from our friends through passion, who made it very clear that this is not a price that whatever price cut they have, is still not going to be done transparently. Secondly, they're still using QUALYS as a way of making these determinations. You can't ration but you can  set a price based upon the QUALY, which is the same thing. So if the QUALY says this drug is not cost effective, you can't use the QUALY to say, well, because senior citizens won't have as much QUALY, you can't ration it based upon that. But you can say, Oh, we're just not going to pay for it, or we're going to reduce the price of the the price of what we're going to pay based upon the QUALY, which is, you know, winds up being the same thing.

Terry Wilcox  16:01  
And this is why we need to pass the QUALY ban bill, the quality adjusted life year, ban bill before the end of the year, to ban this everywhere, yeah, in all levers. For price and access. I guess we'll see how it all wraps out. We are actually going to be starting a page on the patient's rising website called IRA watch, to go along with our ICER watch, CMS watch, we're going to be watching. You know, it's not that I don't I don't understand the need to lower health care costs, you know, societally, like I do understand that. But the important part for patients rising is obviously are we lowering cost for patients because that's what you're on TV with millions of dollars selling every right. Yeah, and the sessions will wrap up on November 15. So we will be chiming in about this and seeing where these listening sessions go and how much the patient voice really does weigh in on the final outcome. We'll continue to track these and as they continue, we will put a link to them in the show notes each week. 

Bob Goldberg  17:08  
That's all for today. But we have another episode right here next Monday. So make sure to continue to follow the Patients Rising Podcast on your favorite podcast player. And that way you can be notified as soon as a new episode is live.

Terry Wilcox  17:25  
Until next week, from Dr. Bob and everyone at Patients Rising, I'm Terry Wilcox, stay healthy.