There is a growing market of niche biopharmaceuticals that are treating rare diseases, and every year more appear on the market.
There was an editorial in March in the New York Times talking about the extraordinarily high price of Cerezyme. Cerezyme, a drug manufactured by Massachusetts-based Genzyme eases the symptoms for Gaucher disease, a rare, sometimes fatal, inherited disorder that can cause enlarged livers and spleens, anemia, and bone deterioration.
The editorial goes on to say:
The company justifies the high price as necessary to sustain a business that develops drugs for remarkably small groups of patients. Only about 5,000 people around the world are taking Cerezyme, including about 1,500 in the United States. The company makes other very high-priced drugs for patient pools that are even smaller.
The experience with Cerezyme and other biological drugs defies conventional wisdom on drug marketing, which holds that blockbuster drugs — generating revenues of a billion dollars a year or more — are generally those that can be sold to vast numbers of people. But Genzyme has made Cerezyme a blockbuster, with sales of $1.1 billion last year, by charging very high prices for a few thousand patients.
Cerezyme is not alone. There is a growing market of niche biopharmaceuticals that are treating rare diseases, and every year more appear on the market. For diseases that used to kill people there is now hope — in many cases — of maintaining a higher quality of life. This is a good thing. It does require new thinking, however, in how we insure patients, how we market to them, and how we support them.
(Image courtesy of booleansplit via Flickr)