High Prices for Medications for Rare Diseases Raise Concerns for Health Care System, Editorial States
The "extraordinarily high prices of some drugs used to treat ultrarare diseases raise troubling questions for the American health care system" because, when "prices mount into the hundreds of thousands of dollars a year, they impose a burden on patients and sometimes on employers and insurance programs that must pay most of the bill," a New York Times editorial states.
The editorial cites the case of Cerezyme, an injectable treatment for the symptoms of Gaucher disease, a rare genetic disorder that can cause enlarged livers and spleens, anemia, bone deterioration and death. According to the editorial, Genzyme charges each of the 5,000 Gaucher patients worldwide more than $300,000 annually for and "justifies the high price as necessary to sustain a business that develops drugs for remarkably small groups of patients."
By charging "very high prices for a few thousand patients," Genzyme has made Cerezyme a "blockbuster, with sales of $1.1 billion last year," and that "could bode ill for efforts to curb health care costs if, as expected, the future of medicine lies in targeting treatments to limited numbers of patients most likely to benefit from them," the editorial states. Genzyme is "essentially exploiting a monopoly position to charge what the market will bear to treat desperate patients with no other option," a practice that is "hard to take, given that the federal government did much of the scientific work that led to development of the drug and provided contract money that got the company started," according to the editorial.
The editorial concludes, "For the future, it would be wise to foster generic competition for biological drugs and allow government programs to negotiate for lower prices on drugs with no competitors" (New York Times, 3/23).
