Elexacaftor/tezacaftor/ivacaftor

It was the very first drug to treat the underlying cause of the disease rather than just clearing the mucus.

But the real watershed moment, the one that changed everything, arrived in October 2019 with the launch of Trikafta.

The triple combination therapy, Alexicaftor, Tezacamptor, and Avicaftor.

Yes.

And the crash was deafening.

Let's talk about the price tag.

The U.S. list price for Trikafta is roughly $326,000 per patient per year.

per year, every year, for the rest of their lives.

But if you think about medications, they can often save money elsewhere in the health care budget.

So if you look at, for example, Trikafta.

I mean, taking aside the life-changing experience that many people with cystic fibrosis have had since that was funded, we've had almost no lung transplants in New Zealand over the last five years.

Now, I don't know what a lung transplant costs, but I would guess it's one of the most expensive procedures in the entire healthcare system.

And Tahir came to give a presentation and had a criticism of the agreement.

One, in terms of the scope of countries that were a part of the agreement that left out some key countries that had high prevalence of drug-resistant HIV-AIDS in particular that were left out.

and purposely so in many ways because of their market power, and also kind of a criticism about this idea of these sort of voluntary mechanisms that we were congratulating as being moving forward on access to medicines.

It led to a very loud and hot debate.

It was also kind of emblematic in terms of thinking about the role of civil society in terms of what we should be advocating for.

Should we be advocating for compromises that are much more reasonable or profitable? practical or feasible in the short term, or should we be thinking about long term sort of dynamic change, reimagining the system altogether? And so for me as someone who's kind of entering this space and understanding kind of the dynamics of pharmaceutical policy, it was a nice introduction in terms of where we should be and how we should be thinking about strategy.

I will say also, Tahir was also one of the instrumental folks that also brought the lens of the global south and what's happening in low-income countries in terms of drug pricing really here to the U.S. and really to the global north.

We obviously see kind of day in, day out on the news a lot of talk about lowering drug prices and different mechanisms to address that.

And over the years, we've done a lot with helping people, you know, pushing government to get drugs through, like with the kids for cystic fibrosis and that sort of thing.

Trikafta and all that sort of thing for, you know, kids under six.

And I, you know, there's been some great wins there that people really don't know about, but they're the ones that are really important.

Yeah.

hey never mind okay okay

crazy But anyway, CVS Caremark was calling me probably to talk to me about the fact of getting prior auths and going through this whole thing because Piper's getting on Trikafta.

Progress.

Progress, which is a gene modulator drug for cystic fibrosis.

So certainly we've seen, you know, lots of improvements.

Obviously, the life expectancy much, much improved with Trikafta.

highly effective modulators and growth and weight gain, hot topic last few years with the modulators and whatnot.

So yeah, so we've adapted.

vaccines.

therapies, meds, all this stuff.

I couldn't even fathom that they basically only had like one or two different kinds of enzymes, didn't even have Trikafta, didn't know about appetite stimulants.

It's just like, wow, really? Okay.

And 'cause just not to get to the end of the story, but I think it's important that the, the listeners understand that, you know, during that time at Mass Bio, I was there for 13 and a half years.

In 2019, after eighteen years and $13 billion invested, Vertex was able to invent a drug called Trikafta that's keeping my kid alive today.

And since he's been on the drug, he's grown eight inches, he's gained fifty pounds, and his lung function's back to what it was when he was five years old.

Three years ago I couldn't say this, but today I can say he's gonna die before me.

One was called Kalydeco that helped out some people.

Um, and this latest one is called Trikafta.

Uh, it came out a couple years ago, and it's really affected in a good way, um, a lot of these kids that are battling cystic fibrosis.

It's, their, their lifespan ha- has grown exponentially because of it, and their lifestyle, uh, feels a lot more normal, which is, is great.

You know, this isn't my life." And denial was a large part of it, which is why I was so non-compliant as a kid.

And even today, like, ever since Trikafta, I really haven't done many breathing treatments like I should.

I mean, like, the CF team recommends doing the vest at least once a day, and sometimes I don't even do that.

But non-compliance was a real big part of my life growing up.

Yeah.

And so I took those for a little bit, but I just, I kinda gave up on the enzymes.

And then after Trikafta, like, I haven't had to take a enzyme in eight years.

And, like, my stool, everything is pretty normal.

No, and you have, uh, so many unbelievable, uh, partners in this and people that every single year come back, um, to donate.

And that's what's so special about seeing how much progress the foundation has had, and now seeing the IVF fund and, of course, the many, many cystic fibros- patients, fibrosus, fi- cystic fibrosis patients who are using Trikafta that has unlocked futures for them.

Uh, it is, uh, really incredible to, uh, to see, and it's all thanks to guys like Mike Northseth and people who are here, uh, today.

So spectacular.