Is 4% a lot or a little? Well – it depends whether you’re one of the 4%.
As the AP reports, “Pfizer Inc.'s just-approved drug Xalkori, the first new medicine in more than six years for deadly lung cancer, proves the value of precisely targeting rare diseases linked to gene variants
The drug was approved Friday in the U.S. along with a companion diagnostic test for just a small subset of lung cancer patients. It is the poster-child for the 21st century strategy of developing medicines for relatively few patients to replace (as the AP puts it) “blockbusters for the masses.”
"This is a paradigm shift," Dr. Paul A. Bunn Jr., a University of Colorado professor and cancer researcher involved in testing Xalkori, told journalists during a conference call hosted by Pfizer. "It used to be that everybody with cancer was treated the same," with surgery and chemotherapy.
Xalkori, a pill with relatively minor side effects compared to the hair loss and nausea that chemotherapy can cause, was approved for the roughly 4 percent of patients with advanced nonsmall cell lung cancer who have what's called the ALK fusion gene.
About 6,000 Americans a year develop this cancer, Pfizer said. Those patients, called ALK positive, now can be identified with a $250 molecular diagnostic test developed by Pfizer's partner, Abbott Molecular Oncology. The test was also approved Friday.
According to Dr. Mark Kris, head of the thoracic oncology service at Memorial Sloan-Kettering Cancer Center in New York, "You're going to be sparing individuals side effects (and) the waste of resources, time and drug that really isn't going to help them."
Cost efficient and patient-centric. Bingo.
Personalized medicine, targeted therapy – call it what you will. It’s real. And it’s a giant step towards achieving the 4 rights – the right medicine in the right dose for the right patient at the right time.
