In an Oct. 2 commentary for the Journal of the American Medical Association, Brian P. O’Sullivan, MD, professor of pediatrics, voices concern about the unsustainable pricing model pharmaceutical companies are using to develop and market personalized therapeutics and drugs targeting rare diseases. He and his colleagues fear that price is based on what the market will bear without taking into account moral and ethical consequences of such pricing.
“Pharmaceutical companies need to be more transparent about how the price for a new drug is determined,” writes O’Sullivan and colleagues. “[They] have an implicit obligation to put patient well-being and resource utilization on equal footing with return on investment. A model of reduced profitability, particularly for lifelong therapies, is ethically responsible and institutionally plausible but will require pharmaceutical companies to develop new models and educated investors about the long-term advantage of regaining the trust of the public.”
In the case of the drug ivacaftor, which treats the underlying molecular defect of a subset of patients with cystic fibrosis (CF) caused by a very specific genetic mutation, Vertex Pharmaceuticals has placed that cost at more than $300,000 annually. Cystic fibrosis is a rare and inherited disease that affects approximately 30,000 individuals in the United States. Only a small fraction (estimated at less than 5 percent) of CF patients have the genetic mutation, G551D, that ivacaftor treats. Unlike some short-term, high-cost therapies, patients will need to take ivacaftor for decades at a potential cost of many millions of dollars.
The issue of drug pricing goes beyond just one disease, however, writes Dr. O’Sullivan and colleagues David M. Orenstein, MD, MA, professor of pediatrics at the University of Pittsburgh School of Medicine, and Carlos E. Milla, MD, associate professor of pediatrics at Stanford University School of Medicine. It is a problem that will only intensify as advances in personalized therapeutics based on individual and disease-specific genetic information move forward. As the subset of patients being treated by a single drug become smaller and smaller, the corresponding price will become higher and higher.
Adding to the unacceptability of this pricing model is that most of these therapies, including ivacaftor, are not developed by pharmaceutical companies alone, O’Sullivan said. Drug development relies on basic biological discoveries supported by the National Institutes of Health, as well as contributions from non-profits, philanthropic organizations and patient advocacy groups. The Cystic Fibrosis Foundation contributed substantial resources to the development of ivacaftor including $75 million in research support for Vertex.