Preview
When people talk about expensive drugs, they usually are referring to drugs for high cholesterol ($1,500 a year), for schizophrenia ($7,000 a year) or for cancer ($50,000 a year). But none of these medicines come close to making of the most expensive medicines on the planet exclusive survey by Forbes.
Most of them treat rare genetic diseases that afflict fewer than 10,000 patients. For these diseases, there are few if any other treatments. So biotech companies can charge pretty much whatever they want.
Soliris
At $409,500 a year, is the world's single most expensive drug. This monoclonal antibody drug treats a rare disorder in which the immune system destroys red blood cells at night. The disorder, paroxysymal nocturnal hemoglobinuria (PNH), hits 8,000 Americans.
In the inverted world of drug pricing, the fewer patients a drug helps, the more it costs. Before testing Soliris for PNH, They tested the drug for rheumatoid arthritis, which afflicts 1 million Americans. The trials failed. But if it had worked for arthritis and would likely have had to charge a much a lower price for this use, as would have to compete against drugs that cost a mere $20,000.
Elaprase
Three other drugs cost more than $350,000 per year. Elaprase ($375,000 per year) treats an ultra-rare metabolic disorder called Hunter's syndrome. Just 500 Americans suffer from the disease, which causes infections, breathing problems and brain damage. Last year domestic sales of Elaprase were $353 million.
Naglazyme treats another rare metabolic disorder and costs $365,000 a year, according to investment bank Robert W. Baird. Viropharma predicts that sales of its Cinryze, a treatment to prevent a dangerous swelling of the face, will increase from $95 million last year to $350 million several years from now. The drug costs an estimated $350,000 a year.
Pills
Unlike pills that come in standard doses, all the most expensive drugs are injected biotech drugs whose dosing varies by weight or other factors.
Amazingly, many brutally expensive cancer drugs don't make the cut. Targeted cancer drugs only help a small minority of patients for a few months. This reduces their average cost. Folotyn treats a rare type of lymphoma and costs $30,000 per month. But the average patient is only on the drug for just a few months, so it doesn't make the list.
Nonetheless, the price of each new rare-disease drug seems to get higher each year. It used to be that pricing a drug at $100,000 per year raised eyebrows. Now that price level has become routine.
Review
Selling drugs for rare diseases has become immensely profitable. There are so few patients that companies don't have to invest as heavily in marketing. The medicines usually get paid for by insurers or governments. The manufacturers give their drugs for free to uninsured patients.
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