In 1983, the year before Henry Waxman got the Waxman-Hatch Act on the Statute Books he got the Orphan Drugs Act passed. Here’s how it happened.
In a wonderful 2016 self-publication, Abbey Meyer, a self-styled Connecticut housewife, outlines a story that began when her eldest son David was born in 1968. The book is downloadable for free and is well written and compelling.
When David was only a few years old he began to show signs of what her doctor called tics. As problems grew at school, she became increasingly concerned for him.
Then one Sunday in a newspaper she read an article about Tourette Syndrome. This led to clinical appointments and treatment. The standard drug, haloperidol, turned David into a zombie. He was enrolled in a clinical trial of a new drug pimozide, which was better, but supplies were cut off in 1976 when McNeil pharmaceuticals opted not to develop it further. Her doctor used the term orphan drug to explain what was going on.
An Orphan Drug was one that was likely to end up being used by less than 200,000 people – which the pharmaceutical industry figured was their break even point. Unless that many people took the drug, they claimed they were unlikely to make a return on their investment. Around 1980 concern was growing that this might mean no new drugs for Multiple Sclerosis, or Epilepsy or Lou-Gehrig’s Disease (Amyotrophic Lateral Sclerosis – ALS).
Meyer was astonished. She became active. Among the first people she tied up with was Marjorie Guthrie, the wife of Woody Guthrie who was campaigning for orphan diseases. A group of women mobilized pretty quickly. Muriel Seligman from Los Angeles had a son Adam with severe Tourette’s. She approached her Congressman, Henry Waxman. Waxman convened a hearing in June 1980 inviting all pharmaceutical companies. None turned up. There was no-one at the hearing apart from a few families with rare diseases and a reporter from the Los Angeles Times.
On the back of an LA Times article, Meyer was contacted by Jack Klugman who ran the Quincy MD show a widely watched medical series on TV. Klugman bought the idea of an episode that would have a boy (an actor playing Adam Seligman) testify at a Congressional Hearing in very emotional terms about the damage caused by lack of treatment.
The show Seldom Silent, Never Heard ran in March 1981. Its a must watch. There are 5 minute clips out there showing the key scene when Adam Seligman makes a pitch to Congress – but I can’t find the link at the moment.
Thousands of letters flooded in. Waxman convened a follow up hearing at which Quincy (Klugman) testified in person. There was standing room only. The Wall Street Journal laughed at Waxman and the idea that Congress might take advice from actors. But Nancy Kassebaum introduced a bill in the Senate, which Waxman introduced in the house that passed both houses in December 1982 and was signed into law by Ronald Reagan in January 1983.
A few days later, Meyer and others formed the National Organization for Rare Diseases (NORD) and began cataloguing all the conditions that might qualify – there were over 6000.
By this stage she had met Bill Haddad. Haddad, as Chair of the Generic Pharmaceutical Industry Association, was chasing a Bill to regularize the position of generic drugs – that later became Hatch-Waxman. Among other things he had made it clear that if the branded industry didn’t bring orphan drugs to the market, the generic companies would.
Haddad provided funds for an office for NORD in Danbury Connecticut. When the landlord heard what NORD was up to, he asked whether this meant AIDS patients might be visiting the premises. Meyer hadn’t thought of this possibility. The landlord told her she would have to go elsewhere but after a later “meal” with one of her supporters he made a several thousand dollar donation.
AZT turned out to be the first drug approved under Orphan Drug provisions, with almost all other drugs for AIDS and its associated infections being approved in the same way.
A key contact Meyer got from Haddad was Agnes Varis, the owner of AgVar a generic company. Varis was connected to everyone in the generic industry and the Democratic party.
At this stage rare diseases were assuming a shape fronted by ALS, multiple sclerosis (MS) and hemophilia. A drug called copaxone had shown benefit for MS but Meyer couldn’t persuade a branded company to pick it up. Among other things the patent status was obscure. It had been discovered in the Weizman Institute in Israel.
Varis strong-armed Teva, a small generic company based in Israel and the US to pick it up. It was approved under the Orphan Drugs Act and went on to blockbuster status, earning Teva over $1 Billion per year, and making Teva the leading generic company.
Meyer’s book is a compelling read. But its difficult to avoid a slight feeling of deliverance from a ghetto on trains that turn up to transport us East to safety.
As of 2016, the branded drug industry is a $1,275 Billion dollar industry half of whose profits ($638 Billion) come from drugs brought to market in the standard way. The other half ($637 Billion) comes from drugs availing of the Orphan Drug provisions which allow for an easier ride through FDA and a longer period of monopoly.
The orphans have included Merck’s Vioxx, Lilly’s Cialis, Astra’s Crestor, Bristol Myers Squibb’s Abilify, Botox and many others among the best-selling drugs of recent years.
While it is possible to close some loopholes that lead to blockbusters being named orphans, the precedent of allowing high prices for drugs that will supposedly only ever occupy niche markets has laid a business model for personalized medicines. If genetic tests ever permit a tailoring of medicines to specific patient groups, the orphan argument will apply.
For most of this period, the Wall Street Journal had Henry Waxman as one of its prize targets claiming that he was to progress what the Troll under the bridge was in the Billy Goats Gruff.
But without mentioning him, as early as 2005 WSJ was trumpeting the fact that Orphan Drugs Act had been the savior of the pharmaceutical industry – not that an industry earning $638 billion from other sources really needs saving.