Cystic Fibrosis and the Cost of Medicine
It’s
difficult not to get excited about the prospect of genetic medicine, but as the
results of research into genetics and personalized medicine continue to soar,
so do the prices of disease-treating drugs. A recent
story on NPR detailed the history behind Kalydeco, a newly released treatment
for cystic fibrosis (CF). Released
twenty years after the discovery of “the CF gene,” Kalydeco is the result of a “long,
hard slog” in the development of genetic medicine.
“We tested
over 600,000 chemicals in cells with the defective protein that causes cystic
fibrosis,” says Fred Van Goor, leader of the team that developed Kalydeco at
Vertex Pharmaceuticals.
Cystic fibrosis is the “most common genetic disease in Caucasians,” causing a
defective gene to produce a malfunctioning protein. Kalydeco works on a
specific set of patients with a “particular mutation in the CF gene,” but for
those without the mutation, an effective treatment still remains to be seen. The
NPR article notes that, while the time from discovery to successful drug trials
is becoming shorter, the production of new drugs stands to remain a slow
process in the meantime. Of course, this story has a happy ending. Emily Schaller, an early Kalydeco study, might
not be cured, but she does finally have her disease under “control” – a silver
lining she greatly appreciates.
“Everyone
talks about curing a disease,” she says. “Cure CF, cure all these other
diseases. [But] Kalydeco controls CF at
the basic defect, so I’m OK with the other ‘c’ word, control, because I’m
living it and I've never felt better in my life.”
Despite an optimistic
hope for faster, more accurate drug releases in the future, the price of
medication remains a cause for concern. The NPR story went on to state that “Kalydeco
costs in the neighborhood of $300,000 per year." Meanwhile, the cost of genetic sequencing and analytics are decreasing. When will the shrinking cost of laboratory
research into genetics start to reflect in the price of effective genetic drug
production and treatment for patients and consumers? Is an affordable genetic drug the next big thing on the horizon?