Little did I know that way back in 1979 when Congressman Waxman’s office received a call from a constituent whos son had Tourette’s Syndrome, it sparked a chain of events that would culminate in the most important piece of legislation to affect me personally. Quite frankly, I’m sure Rep. Waxman didn’t either.
You see, this year marks the 30th anniversary of a little something called the Orphan Drug Act, a bill that made me and millions like me with rare and orphan disorders-ones that affect less than 200,00 people in the US—basically matter at all to the pharmaceutical industry. To us, the Orphan Drug Act is seminal legislation; it is our Americans with Disabilities Act. It’s our moment where we finally got people to notice us and care.
I wrote briefly on the issue of rare disorders for International Rare Disease Day, but I want to take the chance to elaborate, because the story of how the ODA came to be is fascinating and a tale of the good that can come when Congress and the media join forces. This 30-year anniversary is a huge moment in my own personal history as someone with an orphan disorder. But I want everyone to care and take note. I promise, this story is really cool.
Way back when the FDA was established, it did make immense strides for public health and safety for drugs going to market. It also had the unfortunate side effect of making the very act of developing a drug incredibly costly in terms of time and money. Thus, drug companies focused their efforts on medicines for large disease populations to justify a return on all that time and money needed to get FDA approval, such as heart disease, cancer, and diabetes. So when you have a disorder that virtually no one can spell or pronounce, and that affects, say, less than 200,00 people in the country, it’s kind of like drawing the short straw when it comes to incentives in the free market. Pharma says, “Why bother putting so much energy into such a small population?” and it leaves us without any treatments or drug development for our respective conditions. Hence, we’ve been “orphaned” –hence the term orphan disorder.
This was the case for Rep. Waxman’s constituent when she ran out of ideas to get access for a drug, Pimozide, to treat her son’s Tourettes. It was available in Canada, but not in the US because it wasn’t viable enough to treat such an obscure condition with such a minimal population. She even had a friend try to bring it back from Canada, only to have it confiscated at Customs. Rep Waxman realized that this one individual case was only a symptom of a much bigger problem that affected way more people across the country than anyone realized. A prompted hearing on the Health and Environment subcommittee, with the constituent’s son himself testifying, only proved the situation for people with rare and orphan diseases was dire. The ODA was well on its way to being drafted and pushed through Congress.
The ODA would be the first of its kind to put in place tax incentives for companies to invest in rare disease research. ODA also set up patient and market protections. It would provide tax breaks to companies that made treatments that qualified as “orphan” status, as well as made for an easier and more efficient path through the FDA approval process. For example, how on God’s green Earth can you expect a clinical trial to have 1,000 patients when the entire patient population is 2,000? Exactly, carry on. So that’s what the bill did, in a Reader’s Digest version. Now it was time to get it passed.
And here’s where Quincy, M.E., comes in and the story gets really good. A short aside for those of you who may not know, Quincy, M.E., was the CSI before its time. Jack Klugman, non other than everyone’s favorite medical examiner, Quincy himself, caught wind of the ODA and the fight to get it passed, and decided to turn his show into a bully pulpit for the cause. Not one, but a series of episodes aired featuring patients with rare disorders and the market-based obstacles they were up against. In the episode, “Seldom Silent, Never Heard”, a teenage boy with Tourettes dies and Dr. Arthur Ciotti asks Quincy if he can save the boy’s brain and use it for research toward Tourettes. Turns out Dr. Ciotti had been lobbying for years to get a green light for a treatment for Tourettes but there just wasn’t enough profit to justify the endeavor (sound familiar, Rep Waxman?). In swoops Quincy, with a grizzled face and determined eye, and he becomes a staunch vocal advocate for government backed rare disease research.
Quincy’s fictional crusade carried on as just as Rep. Waxman and Senator Hatch’s did in the chambers of the Congress and Senate. There were two more episodes in which the storyline followed the ODA, “Who Speaks for the Children”, and “Give me your Weak”. Jack Klugman appeared before another congressional hearing in 1981, making he and Congressman Waxman quite the odd couple (sorry, I couldn’t help myself). In essence, Quincy was Glenn Coco. Watch him go.
Thirty years after the Hollywood ending, we now have the National Organization for Rare Disorders—founded months after the bill’s passing. The passage of the ODA was the first step in recognizing that those of us with rare diseases are no less important simply because we offer no economic value or profit margin incentive. Aside from the fact that if you add up everyone who has a rare or orphan disorder we make nearly 30 million Americans (hardly something to sneeze at), the ODA with the help of Jack Klugman humanized us in the public eye and brought us out of the shadows. Mr. Klugman passed away this passed December, just before the 30th anniversary of the bill’s signing on January 4th. His legacy saw the bill through and the change it made for people just like me. As I said in my speech at the National Organization for Rare Disorder’s 30th anniversary gala, people he and Rep. Waxman decided who were worth it.