The Most Important New Drug Of 2012 - Kalydeco???

Exactly, it is not a joke, according to Forbes:

Kalydeco, for cystic fibrosis, is a triumph of genetics and drug development, the first medicine to directly affect the genetic defect that causes the disease. It will only help 4% of the 70,000 people who suffer from declining lung function, damaged pancreases, and shortened lives due to CF worldwide, but in those few it has a dramatic effect. It makes medical history for three reasons:

•It’s a genomics triumph: Francis Collins, later famous for heading the Human Genome Project and then the National Institutes of Health, discovered the gene that, when mutated, causes cystic fibrosis 23 years ago. Kalydeco is the first drug to directly counteract this gene, leading to improvements in patients’ lung function.

•A patient group powered its development: Kalydeco would probably not exist were it not for the Cystic Fibrosis Foundation, which funded its early development at Vertex and gets a royalty on the drug. This success paved the way for other disease foundations including the Michael J. Fox Foundation, Myelin Repair, and the Multiple Myeloma Research Foundation.

•Its price: Kalydeco, given alone, will only help a few thousand patients the world over. Like other drugs for very rare diseases, its price is very high: $294,000 per patient per year.

Well, I think that this drug is really illustrative! The key words here are: “ It will only help 4% of the 70,000 people” and “$294,000 per patient per year”. In this perspective the drug is a real triumph of ineffectiveness and price escalation.