The pharmaceutical market is complicated: companies invest in researching and developing treatments and cures for diseases. It can cost millions of dollars to create something that could earn them billions in profits – or that doesn’t work at all. So it makes no financial sense to invest in curing and treating diseases that don’t affect very many people because the company will never recoup its investment. Even if those could make all the different for people with those diseases.

But then came the Orphan Dug Act of 1983. It changed the pharmaceutical market, kickstarting the research and development of drugs for rare diseases that has saved thousands of American lives. Made so many more livable. And it was quite a story. Thanks, gov.

So what’s an orphan disease?

Orphan diseases are just very rare diseases that affect fewer than 200,000 people in America. That means that it affects 1 in 1,500 people. But there are nearly 7,000 diseases considered rare in the United States: so 1 in 10 Americans are impacted by a rare disease. You probably haven’t heard of most of them, but you’ve heard of some, including Tourette’s syndrome, muscular dystrophy, cystic fibrosis, spina bifida, and ALS.


So talk to me about the pharmaceutical companies again?

When few people have a disease, there is no financial incentive for pharmaceutical companies to invest money in curing or treating them. It doesn’t make them bad or unfeeling – they have responsibilities to their shareholders to make a profit. And the reality is pharmaceutical companies could never make back their research and development investment in these orphan drugs: there just aren’t enough people sick with the disease to buy it. So millions of Americans continued to suffer and die of diseases because the market simply didn’t make it possible, or profitable, for drug companies to investigate them.

There were really no treatments found for rare diseases?

Nearly. Before 1983, only 34 orphan drugs came to market to treat any of these thousands of diseases. It doesn’t mean that scientists had no leads or promising treatments. They just couldn’t find drugmakers to take a chance on expensive testing and promotion – there was no potential for profit. Something had to change.

OK, so how did we get the Orphan Drug Act?

It began with a woman who imagined a better world for her children. Abbey Meyers knew that only through government could we rebalance the market to make it possible, and profitable, for them to invest resources in researching diseases and treatments for orphan diseases. So she ImagineGov’d, founded the National Organization for Rare Diseases, and made her vision a reality.

Abbey Meyers’ son had Tourette syndrome and there were no treatments for him. She founded NORD to be an umbrella organization for all rare diseases. The logic is simple: when patients and parents banded together around a single disease that by definition affects few people, their voices are still too quiet. But as a movement for all rare diseases, they could make a giant roar.

They enlisted the help of Representative Henry Waxman (CA – D) to write what became the Orphan Drug Act to provide substantial financial incentives to pharmaceutical companies that invest in orphan diseases: it reduced the regulatory burden for developing new orphan drugs, offered drug companies a seven-year monopoly so they could recoup their investment, and gave companies a 90-percent tax credit for the cost of clinical trials. It also sought to create an Office of Rare Diseases at the National Institutes of Health.

What did they do to get the law passed?

Everything they could think of. Through NORD, Meyers rallied affected patients, parents and organization to lobby representatives and testify before Congress.

She even convinced actor Jack Klugman, who played the medical examiner on Quincy, M.E., a “wildly popular 1980s medical drama” to use his celebrity to get the bill passed. Klugman’s brother had seen the only article covering initial hearings about the bill and having a personal connection to the issue (he suffered from a rare cancer), wrote an episode of Quincy about Tourette’s and the orphan drug problem. It was a sensation. So much so, that Rep. Waxman asked Jack Klugman to testify before the subcommittee – this never happened at the time. It was an even bigger sensation, and the bill flew through the House of Representatives.

But Senator Orrin Hatch (UT – R) was blocking the bill in the Senate, removing most of the incentives, including the tax credit, and that version passed the Senate. Without them, the bill would have little or no effect on the problem.

So Klugman wrote a second Quincy episode in which Quincy confronts a heartless senator who is holding up an orphan drug bill with a huge crowd of protestors outside his window calling for its passage. The crowd in the show was populated with extras who suffered from rare diseases in reality. (Read his extraordinary obituary from The Washington Post for the whole story.)

The bill was passed  as the Waxman-Hatch Orphan Drug Act of 1983 and signed into law by President Ronald Reagan immediately. He wrote: “Over the past century, the United States—largely through innovative pioneering by private industry and medical researchers in universities—has led the world in developing new drugs that have saved millions of lives. That is a gift to mankind we can be very proud of. Yet the sad fact remains that many diseases still cripple or kill hundreds of thousands of Americans, as well as citizens of other countries, because no drugs have yet been developed… The bill that I am signing today helps to cure that problem and consequently, we hope, some of the diseases as well.”

There should be a movie about that.

There should. Let’s write one.

Did the ODA work?

Let’s put it this way: In the 10 years before the ODA was passed, only 34 orphan drugs were brought to market. However, from 1983 until 2015, the Food and Drug Administration approved 486 orphan products and over 200 new drugs. Many of these compounds are hopeful first steps to cures to many of these orphan diseases.

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We’re only just beginning: as of 2010, only 200 of the roughly 7,000 officially designated orphan diseases have become treatable. But AZT was developed under the ODA.

More examples please.

Ok. One of the best-known orphan diseases in America is cystic fibrosis, a genetic disease that affects about 30,000 Americans today, with 1000 new cases diagnosed each year. Because of genetic mutations, people with cystic fibrosis have a thick, sticky mucus in their lungs, pancreas, and other organs. It can make it harder for them to absorb food and cause lung infections and livery disease.

Unfortunately, it is very difficult to find cures and treatments for cystic fibrosis. It is caused by over 1,800 known mutations of a single gene and there are few patients in which to perform drug trials to determine if a drug is even effective. Thus, until the passage of the Orphan Drug Act, there were few treatments for those suffering from CF.

So, in the 1950s, children “with CF rarely lived long enough to attend elementary school.”


But, with the passage of the Orphan Drug Act in 1983, grants from the FDA supported drug research and development for CF. One of the first drugs produced under the ODA was Tobramycin, an inhaler that breaks up mucus. Known as Tobi, this drug became available in 1992, a remarkably fast development and approval time for a new drug. Novartis, which developed Tobi, received substantial financial incentives to encourage it to create this drug. With the help of the ODA, new therapies and drugs continue to be developed, most notably Dornase Alfa, or Pulmozyme in 2005, the most widespread and effective treatment for the most common mutation causing cystic fibrosus.

The emergence of new drug therapies for CF patients has led to an overall improvement in quality of life. In 1988, just 5 years after the ODAs passage, the median life expectancy for a CF patient was 28. Today, it is 38 years. People with CF can attend college, have careers, have their own children. It’s a new world, almost completely because of effective drugs developed under the ODA.

What do pharmeceutical companies think about the ODA?

It makes pharmaceutical companies more profitable and makes it much more cost effective to bring an orphan drug to market. Drug companies didn’t want to not research and find cures for rare diseases – it was simply unaffordable. This law makes it possible for them to meet their shareholders’ needs (profits) and to help save the lives of people with rare diseases. It paves the way for them to improve their companies bottom line and communities. Win win win.

Is the problem of orphan drugs and diseases solved? What would happen if the ODA was repealed?

It would be kind of a disaster. According to NORD, “Based on historical growth rates, if the ODTC were repealed, it is estimated that 57 fewer new orphan drugs would be approved over the next 10 years.” There’s a chart:

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It would raise the price of researching, developing, and getting approved any orphan drug. It’s likely that the pipeline would largely shut down. So we really couldn’t do without the ODA.

Are there problems?

As with anything, of course. In 2014, “41 percent of all FDA approvals were for orphan drugs. And sales of orphan medicines, which carry high price tags, are forecast this year to total $107 billion.”

It looks like drug makers may be exploiting loopholes in the law to get the incentives designed for rare diseases for blockbuster drugs. They submit a drug for approval for a limited population, knowing that it has a much larger and broader use. Then, they can receive the longer monopoly, tax incentives, and lower regulatory burden imposed on orphan drugs and then seek additional usages afterward.

Precision medicine makes it easy to describe a disease as affecting fewer than 200,000 people, such that anything can be described as an orphan disease.

So some scientists are suggesting that drug companies be required to pay back taxpayer subsidies if their drug becomes a blockbuster, as that was not what this bill was trying to address.

So what’s the upshot?

The Orphan Drug Act is just about the perfect ImagineGov story. Abbey Meyers saw a problem and realized that only if people came together through our government could we change market incentives and save lives. So she got the Orphan Drug Act passed and it has been helping us save lives and improve the lives of thousands of Americans every year. And it’s paving the way for our pharmaceutical industry to thrive and do research that helps not only those with rare diseases, but potentially everyone.

Basically, we came together through our government to help create cures and treatments for rare diseases. Thanks, Abbey for ImagineGov’ing so we could make the Orphan Drug Act a reality.

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