Thirty years ago, Congress acted to spur research on rare diseases. Today, we have hundreds of new drugs — along with runaway pricing and market manipulation, as pharmaceutical companies turn a law with good intentions into a profit engine. Times analysis tracked about 2,000 rare diseases before the Orphan Drug Act. There are now at least 6,432 diseases — a profit-driven strategy known as salami slicing, dividing one disease into many to create more drug markets. Drugs for rare diseases commonly begin in the six figures for a year of treatment. In 2013, one reached $440,000. The family of Violet O’Dell, a 10-year-old girl diagnosed with a deadly cancer, was told that an orphan drug might extend Violet’s life by a few weeks. But the drug would cost about $50,000, more than the family makes in a year. Gina Dichter, a mother of two, was told that her lung disease was so rare – and for the drug industry, so unprofitable – that if any research was to be done, patients must take the lead. While dealing with a disease that could kill her, she raised more than $1.7 million, to help make a clinical trial possible. For pharmaceutical companies, the biggest profits from orphan drugs typically trace to off-label sales. In the series’ second story, the Times detailed how one Seattle biotech used marketing tactics condemned by the U.S. Department of Justice to ring up tens of millions of dollars in sales from a drug approved for only 400 patients a year. The paper also mapped these tactics in a three-minute video that illustrated their reach and impact.
