99% Invisible - 329- Orphan Drugs

Episode Date: November 14, 2018

We chronicle the epic struggle to get drugs that treat very rare diseases on the market, and the unintended consequence of that fight, which affected the cost of all kinds of drugs. This is a strange story that involves a hit 70s TV show, a fake march on Washington, a courageous advocate, a carnival concessions wholesaler, and a new drug law that helped a lot of people, made drug companies billions of dollars, and opened a whole can of worms. Adapted from the new podcast An Arm and a Leg by Dan Weissmann Orphan Drugs

This is 99% invisible. I'm Roman Mars. Abby Mars grew up in Brooklyn and married a guy she'd known since they were kids. Their first son, David, was born in 1968. By the time David was two, Abby noticed something was different about him. David's face would twitch. He sometimes made involuntary noises. His arms flailed around out of control, so he could barely feed himself. He was very disturbed by the movements because other kids would make fun of him. David's teachers often got upset with him in class.

So you can imagine the torture that he went through. One Sunday morning, when David was eight, Abby watched him try to read the funnies, but he couldn't keep his head steady enough. It was heartbreaking. That's producer Dan Weissman, the host of a new podcast about the cost of healthcare called an arm and a leg. Watching David struggle with the comics,

Abby was ready to cry. She put off doing the dishes, sat down, and picked up another section of the paper. And through total coincidence, there was an article about a teenager who had symptoms just like David's, facial tics, weird noisems, flailing limbs. Abby and her husband brought the article to David's pediatrician, and the doctor agreed this story described exactly what David had. This condition will sound familiar to you, but at the time most people, even doctors, had never heard of it. It was called Tourette Syndrome.

Speaking to doctors, many doctors, after that, they said they never heard of it because they simply were not taught very much about rare diseases in medical school. And back then, when so few people had heard of it, Tourette's was considered super rare. It just seemed bizarre to me that I got a diagnosis out of a Sunday newspaper. From there, Abby found a specialist who eventually turned her on to a potential treatment for Tourette's syndrome that was being tested out in clinical trials. Well, it wasn't being tested for Tourette's. The drug company was mainly hoping to market this drug for schizophrenia. The particularly drug had been made available in Europe for a while and doctors knew it worked on turrets. So David got in on a trial for this drug with the hopes that it could be approved in the US and it turned out it worked really well for him.

But the drug trials didn't go as well for schizophrenia, which was supposed to be its more common use. So the drug company decided the market wasn't big enough. They just dropped the drug entirely. And so companies just said no, they wouldn't do it. They wouldn't make the drug, they wouldn't sell it. And this is how Abby learned a term that would define the next few decades of her life. Orphan drugs. Orphan drugs. As Abby learned, drug companies weren't interested in developing treatments for rare conditions, because the big money was in drugs with tons of customers, and biotics are drugs for stuff like diabetes, high cholesterol.

They just didn't think selling a drug for terats would be a big money maker. So promising treatments like the one that had been working for David became orphan drugs, with no pharma company to raise them. To keep herself from going nuts with frustration, Abby Myers joined the Tourette Syndrome Association. It was pretty small. They immediately invited her to join the board and asked her what she wanted to be in

charge of. I sure that that wanted to become the fundraiser or the arranger of special dinners or anything like that. And I wasn't a doctor, so I didn't want to do the research end of it. That left lobbying and advocacy, spreading awareness. Abby started figuring out how to do that. And she found a mentor. Her name was Marjorie Guthrie. Marjorie was the widow of Woody Guthrie, the iconic American folk singer who wrote the song, This Land Is Your Land.

Woody had died in the 1960s of Huntington's another rare disease. She was on a mission for her whole life, which was to find the answers for Huntington's disease. Marjorie was great at it. By the time Abby Myers started lobbying, everybody connected with rare diseases, new Marjorie Guthrie. Abby took note of one of Marjorie's best tricks.

Marjorie would be talking with scientists or farm executives, and they'd be using technical jargon. She's saying, please talk to me in plain English. I'm just a housewife. And they would. And when experts couldn't hide behind technical jargon, Marjorie Guthrie would pin them down, put them on the record, and use their words as ammunition.

For instance, she could get experts to explain exactly how promising a proposed drug might be, and why they wouldn't sell it. And all of that would be in there, in plain English. So when Abby started meeting with government officials, she introduced herself the same way, just a housewife. She figured it wouldn't be a bad thing to let them underestimate her a little.

I found that people wanted to think about a woman as somebody who's just trying to raise her kids. She's just a housewife. By June 1980, Abby and Marjorie and their allies got a California congressman to hold a hearing to call attention to the problem of orphan drugs. This was their big chance to finally have a national audience to put pharma companies on the spot to make them face the patients who were waiting for treatments. Maybe this hearing could really change things.

Well, nobody from the pharmaceutical industry showed up. Also, hardly any elected representatives showed up. Well, no Republicans at all showed up. Maybe one or two congressmen stuck their head in and sat down for five minutes and then left. The gallery was pretty empty too. It was like nobody was there except the very, very last row in the room.

A young man was sitting there and I had no idea who he was. And it turned out that he was a reporter for the Los Angeles Times. That reporter was there because for him, it was a local story. One of the people testifying a teenage boy with Tourette's was from Los Angeles. It resulted in a very short story buried in the middle of the paper where nobody was likely to see it.

Fix up somebody dead. If you're old enough to have watched TV in the 1970s, the name Jack Klugeman will instantly conjure a raspy voice, slightly pudgy New Yorker. The sitcom The Odd Couple was huge. Jack Klugeman played a middle-aged slob whose roommates with a neat freak. What's this, Joe? Christmas present. I forgot to give it to you.

Merry Christmas. But is it? Assorted Jesus. When that show ended, Jack Klugman got his own brand new TV show, a kind of detective series. He played a medical examiner called Quincy. The Quincy Show, which was nice and entertainment about a doctor who looked at dead bodies and decided who the murderer was.

It was like a precursor to CSI, and it was kind of a big deal at the time. Gentlemen, you are about to enter the most fascinating sphere of police work, the world of forensic medicine. And after that little story, the Los Angeles Times came out. Abby got a call from some of the few said he was a producer on the Quincy Show. She was skeptical. I said yes, and I'm Mrs. Santa Claus. But after a few minutes on the phone, she realized the guy was telling the truth.

He was a producer for Quincy, and he happened to have a fairly rare disease himself. In fact, he was dying of bone cancer. And so he said this whole thing with orphan drugs is a beautiful story and I'd really like to do an episode of Quincy about it. And this producer had the pull to make it happen. His name was Maurice Klugeman. He was Jack Klugeman's brother. I mean these things happen in the movies. They don't happen to real people, but that's what happens. The episode actually got made. It happened. And in the episode, a teenager with Tourette dies under suspicious circumstances. Turns out it's not exactly a murder, but by the time Quincy figures that out, he meets another

young man with Tourette's and gets obsessed with the problem of orphan drugs. Here's Quincy, talking with that kid's dad. So if you've got a disease that happens to have a low incidence, don't count on the pharmaceutical companies for any help. That's why they call them orphan drugs. Miracles, the cures for rare diseases that no one wants to adopt. Well, isn't there a bill before Congress

that's supposed to deal with all this? The orphan drug bill, it's still being considered. The show suddenly goes from on mystery to a policy drama. Quincy goes all in on the orphan drugs issue. He lobbies, farmer companies, the FDA, congressman becomes a total policy nerd. At the big finish, the kid with Tourette's, after a pep talk by Quincy, gives a blockbuster speech at a congressional hearing.

I'm thinking about the other victims. Not just of Tourette's, but anyone who is unlucky enough to have a disease that never made it to the top 40. This congressional hearing was obviously fictional, but it got a real world response. When that show was broadcast, Jack Klugman received thousands and thousands of letters. And they didn't even open up. They just had these big sacks, they were like, Santa Claus sacks, and they stuffed all the mail into them, and they sent all of those sacks to me.

The letters were from people with rare diseases, and people who just wanted to do something to help solve the orphan drug problem. With this kind of publicity and support, Abby and her friends got another congressional hearing. On March 10, 1981, real-life elected officials heard testimony about a real-life proposal to create financial incentives for pharma companies to take on orphan drugs. On hand to give that proposal a boost of star power was Jack Klegman himself. The actor flew to Washington and testified in this new hearing in Congress.

This time it was packed. People just sat down on the floor in the aisles. It was pednimonium because Jack Klegman was in the room. I'm telling you, Jack Klegman was a big deal in those days. An orphan drug bill passed the house, but it got stalled in the Senate, where Abby heard a single senator was secretly blocking it. It was rumored to be orange hatch. So Jack Clogman and his crew swing into action.

They do another episode of Quincy, one where a single senator is holding up an orphan drug bill. In the climactic scene, Quincy confronts that senator in his DC office. If I choose to keep voting against this bill it's something because I think it's in the best interest of the election. Then you should be ready for the reaction. There won't be any reaction. I play this course too many times.

Well, maybe the rules have been changed. Why don't you take a look at the window, Senator? The Senator glances incredulously toward the window. Then, back to Quincy. Then, both guys turn and walk solemnly over this huge picture window. And out in the street, there's a big demonstration heading straight toward the

senator's office. The camera gently zooms past the two men and focuses on the action outside. There are hundreds of people, lots of them in wheelchairs, and they've got colorful signs with slogans like, now is the time, vote for the orphan drug bill and in a direct pitch to the fictional senator watching through the window, we need you, Senator Reeves. This sequence lingers and lingers and lingers. These extras were all actual people with rare diseases that Abbey Myers and her colleagues recruited, except not actually in Washington. They shot it in Pasadena where the weather was better. The fake march on Washington totally worked. The fake senator on the show chaved.

I cannot support this bill, but I want that in its way. And so did the real life senator. Or an hatch. There would not be an orphan drug act if it was not for that episode of Quincy. The orphan drug act passed the Senate. President Ronald Reagan signed it into law and the fake march on Washington became, in some people's minds, a real life event. People would say to me that March on March, it was wonderful. There wasn't a March on March, it was.

But you know, it was on TV, so. Abby Myers went on to help start a new advocacy group, the National Organization for Rare Disorders, and she ran it for more than 20 years. But these days, when people ask her about her legacy, this is what she tells them. Be careful what you wish for. It might come true. The Orphan Drug Act of 1983 created financial incentives

for pharma companies to develop drugs to treat orphan conditions. The Orphan Drug Act basically says once the FDA agrees proposed treatment will address a rare condition under current definitions that means it affects fewer than 200,000 people in the US, then the developer qualifies for special goodies. That includes subsidies like actual tax payer cash for clinical trials and a big prize at the end. The first drug company to get FDA approval for a certain kind of treatment gets market exclusivity. Basically, I'm an OPLI. No one else can market an equivalent drug in the US for seven years. Which means that during that seven-year period, the company gets a chance to make money off

this drug even if relatively few people use, by setting a higher than usual price. You know, if you have a drug for high blood pressure, it's probably half of the United States needs blood pressure medicine, but with a rare disease. You're not going to make money if you charge a normal price for those. And then drug companies realized that if they had a monopoly, they didn't have to charge anything that would be remotely considered a normal price. They could charge pretty much whatever they wanted, especially if the insurance companies were paying for it. It took pharma companies a few years to catch on to how good a deal the orphan drug act

could be, but by the 1990s, things were definitely rolling. One rare disease drug came out in 1991 with a price tag of $150,000 a year. It was huge money. I've been involved in the formation and financing of biotechnology and healthcare companies for almost 30 years. This is Josh Washine, and he's one of the many entrepreneurs who took advantage of the orphan drug policy. His path to the big farm of money led through Las Vegas. It illustrates just how wild west this new world could be.

In 2003 Joshua Shine started a company called Lev Pharmaceuticals and this company had just one mission, a single thing it was going to do. Lev Pharmaceuticals was going to market a drug for a rare disorder called hereditary angiodeima. Actually, the drug already existed. It had been used successfully in Europe for 30 years, but nobody had taken it through clinical trials in the US. As shine recalls, that looked like a good setup. It seemed like a very good opportunity from its inception.

We saw a serious unmet clinical need in the US. To bring this treatment from Europe to the point where it could be sold in the US, meant that Joshua Shine would have to raise enough money for lab pharmaceuticals to run clinical trials for FDA approval, which would be a lot easier to do as a publicly traded company. And that's how they found Fun City Popcorn. created company. And that's how they found Fun City Popcorn. Right, it's not a typical path

and that's really not a typical name for it. A biotech company. Fun City Popcorn sold popcorn and nacho cheese sauce to concessionaires all around Las Vegas. They had hundreds of clients, malls, ball parks, skating rinks, unit. But of course, Lev didn't pursue fun city for their nacho cheese sauce recipe.

It turned out Fun City's owners were getting out of the popcorn and concession business, and they were game to try something new. Fun City agreed to actually merge with Lev Pharmaceuticals. Thus, bestowing on Lev, their status as a publicly traded company, which made it easier for Lev to attract even more investors. But for a few months until they could get the paperwork sorted out, they had to go by the name, Fun City Popcorn.

We felt we could overcome the man in Fun City Popcorn and had the jokes that might bring up. Did people kid you about it? Sure. Still do it, darling. Shine could laugh all the way to the bank. Over the next few years, Lev Pharmaceuticals raised about $75 million

to pay for the clinical trials that would win FDA approval. Of course, Joshua Shine still had to get the trials done and do it before anyone else. After all, the orphan drug act is set up to give a monopoly to whoever gets to the treatment first. Finishing second, can mean losing the whole investment. It was a real horse race to see you could finish the trials first.

And Shine won this particular race. Yeah, I'm very pleased with how this worked out. It worked out most importantly for patients. And of course, he's also glad it worked out for the shareholders. Most companies don't pan out. And so, you know, I'm pleased that as managers of a company that we were able to provide a return for our shareholders. It was a big return. In 2008, a couple weeks before the FDA gave the new drug its final approval, Lev Pharmaceuticals sold for $442 million.

for $442 million. And within a few years, the new owner was making $400 million back from this one drug every year, thanks to a price tag that made this one of the world's most expensive drugs, more than half a million bucks a year per patient. And then, that owner sold itself to a still bigger company for $4.2 billion. That company CEO says that the company's entire strategy is to make money from the treatment of rare diseases. The Orphan Drug Act and the crazy pricing that is its legacy is this company's whole vision for making tons of money.

This is why Abbey Myers has mixed feelings about her legacy. Thirty-five years later, the orphan drug act has been responsible for the most miraculous treatments and many, many diseases. But some of the companies that make these drugs have put prices on them that make the drugs absolutely unaffordable. In other words, it's like a ransom note for thousands of desperately ill people. Joshua Shine from Lebpharmaceuticals has a different perspective. He says it is hard enough to get money to develop these drugs, even with the orphan drug acts incentive, but massive payout. These drugs are hugely expensive to develop, and the failures far, far outnumber the successes,

and without a system in place like the orphan drug act, the drugs would not come to market. Of course, it doesn't have to be this way. I'd be Myers and others have their own proposals for how to balance incentives with reasonable prices. It's a giant debate. If you look at a list of the most expensive drugs, you'll notice two things. They are unbelievably expensive, tens of thousands of dollars a month,

and they are generally for rare conditions. Whoever pays an individual bill insurance companies patients The whole thing contributes to the spiraling cost of health care And it's gonna take more than I've faked TV march on Washington to fix the problem If you'd like to know more about how the cost of healthcare has gotten so messed up and what we may be able to do about it, or if you just like really good storytelling, check out Dan Weissman's new podcast. It's called The Arm in a Leg.

We'll have more about that show after the break. Dan Weissman is a journalist based in Chicago. You may remember that he reported a couple stories with us over the years about the reversing of the Chicago River and how the popularity of Billi billions drove the development of plastics. And as we mentioned, he now has a show about healthcare called an arm and a leg. I asked him why he was compelled to create a podcast about this particular subject. I've kind of been interested in this topic for a long time and partly for personal reasons.

I had thought about going out of my own as a reporter instead of having working for somebody else for a while, something I wanted to do, but who's going to buy the health insurance for our family? My wife has her own business, and so it just seemed unthinkable. I found myself kind of having left one job thinking like, crap, crap, I might need a new career, which was really, you know, sad for me, that I was like, well, there is this one thing that's kind of running my life, and I know I'm not the only person,

which is, you know, the cost of healthcare and the need for health insurance and the expense of that, you know, just drives so many decisions for so many people. I don't see a conversation about that or a kind of accessible conversation about, like how do we get here? And it's like, is there anything any of us can do about it?

And is how did we get here the driving question or is it how do we solve it? Like where do you place the emphasis in the show? That is a super good question. It's a good question. I think because of my disposition, there are some good stories in how we got here. I think that there's two good things about that. One is that understanding how we got here might help us understand a little bit about how we could make things a little better. And also that like one of the things,

in some way the thing that I most want the show to do is that it allows us to be company for each other, right? That like we're all kind of scared and angry and confused and telling some interesting maybe fun stories is also just a way to pass the time and bring our blood pressure down to the point we actually could like see our way clear to doing something else about it. As you've been gathering the stories, is there an underlying theme that's emerged that

surprised you? The thing that surprised me first was just from talking to people about the idea of doing this show that like every single person I would talk to him. This is I'd say pretty much remained true. Well, either right off the bat or as we get to talking, be like, oh yeah, well, this is a story that happened to me. But that was probably the single biggest surprise was like everybody, everybody. So you have one of these examples in your pilot

with your friend Peter. Could you describe the situation with Peter? Yeah, Peter is a real estate developer and he makes a perfectly good salary. And when I tell him I'm doing this show, he's like, oh, yeah, you know, the health insurance at my job just keeps getting worse and worse and last year my deductible was like $13,000

in this year, it's worse. And yeah, and he's like, oh, wait, and did I tell you about when I had the kidney stone last year? I like no, he's like well, you know I Put off going to get help for as long as I possibly could because he was like they're just gonna charge him money And not people to do anything for him and he finally breaks down and his wife makes him go to the hospital and they give him an MRI and He is kicking himself and they told me exactly what I knew that I had a kidney stone and It was passing and I'm like thank you very much and then I get the bill Well, it was $6500. Oh my god, and you had it was all on you

It was all on me. It didn't help me in any way shape or form and then I'm left with this bill Peter one of the things that I find helped me in any way shape or form. And then I'm left with this bill. Peter, one of the things that I find you know, awful and delicious about this story is like it's not just your real estate developer, you know, you make a good salary, your real estate developer like negotiating with people like say contractors who most people would never think

they were slick enough to do is what you actually do for a living. Right. And I can't negotiate this system. And so if you want to hear that story and it's entirety, you have to subscribe to an arm and a leg. Thank you Dan, I appreciate it. Oh Roman, thank you so much. 99% invisible with produces weak by Dan Weissman and edited by Avery Trollfoman.

Mix and tech production by Shereef Yusuf, Music by Sean Riel. Katie Mingle is the senior producer Kurt Colstead is the digital director. The rest of the team includes Emmett Fitzgerald, senior editor Delaney Hall, Joe Rosenberg, Vivian Lee, Taren Mazza, and me Roman Mars. We are a project of 91.7 K-A-L-W in San Francisco and produced on Radio Row in Beautiful, Downtown, Oakland, California. 99% of visible is a member of Radio Topia from PRX, a fiercely independent collective of the most innovative shows in all of podcasting. Find more at radiotopia.fm.

You can find the show and join discussions about the show on Facebook. You can tweet at me at Roman Mars and the show at 99PI Orc. Or on Instagram, Tumblr, and Reddit too. But this week, I think it's a good idea to check out Dan Weissman's other 99PI stories on our website. Just use the handy search function at 99pi.org. Radio Tapio.