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Science in Mind

Doctors question high drug prices for rare diseases

Dr. Brian P. O’Sullivan, a pediatrics professor at the University of Massachusetts Medical School, said the system cannot sustain such expensive drug treatments.University of Massachusetts Medical School

It was a scientific victory, a triumph of a different model of funding drug development, and a boon to patients when Vertex Pharmaceuticals’ cystic fibrosis drug, ivacaftor, was approved in early 2012. It was the first drug to treat one of the genetic defects that cause the fatal, inherited lung disease — and gave hope for a longer life to a small fraction of cystic fibrosis patients.

Then, the price tag was unveiled: $294,000 a year, for a drug that would be taken for the rest of a patient’s life. Many doctors balked, sending a letter to the company pointing out their conflicted feelings.


Dr. Brian P. O’Sullivan, a professor of pediatrics at the University of Massachusetts Medical School who coauthored that July 2012 letter, said the more he thought about it, the broader the problem seemed. It wasn’t just one pricey cystic fibrosis drug.

As chairman of a board that oversees drug trials at UMass Medical School, O’Sullivan said he was beginning to see more of the same — drugs being tested for increasingly small fractions and subpopulations of the sick.

“If they all cost a lot to develop and there’s only a small number of people able to use them they’re all going to cost $300,000 a year — and can our system really sustain that?” O’ Sullivan said. “I would argue that it can’t.”

In an attempt to trigger a larger discussion about drug pricing, O’Sullivan and colleagues published a commentary in the Journal of the American Medical Association Tuesday, pointedly asking whether the trend of developing drugs for small, niche populations and selling them for often whopping sums can continue.

There is some evidence of pushback on high-priced medical treatments, but the price of ivacaftor has only increased since the doctors sent their letter.


A Vertex spokesman said in a statement that the company is continuing to do research in cystic fibrosis that will benefit a larger group of patients, and the price “reflects how well this medicine works, the time and cost it took to develop, and our commitment to reinvest to help many more people with [cystic fibrosis] — work that is highly expensive, risky, and takes the dedication of hundreds of people over decades.”

Carolyn Y. Johnson can be reached at cjohnson@globe.com. Follow her on Twitter @carolynyjohnson.