Genzyme CEO Discusses Benefits of High-Priced Medications for Rare Diseases in NYT Letter to the Editor
"Rather than fear the cost impact" of the development of medications for rare diseases that "are among the first personalized medicines," as a March 16 article and a March 23 editorial in the New York Times indicated, "we should embrace its potential to redirect resources toward treatments that work more effectively and safely because they are prescribed only to those patients most likely to benefit," Genzyme Chair and CEO Henri Termeer writes in a Times letter to the editor (Termeer, New York Times, 3/31).
The article examined how the proper dosage of a medication "becomes a matter of public debate" when a treatment such as Cerezyme -- a medication manufactured by Genzyme to treat patients with Gaucher disease, a rare inherited enzyme deficiency -- can cost patients or health care providers hundreds of thousands of dollars annually. According to the article, Cerezyme can cost more than $300,000 annually (Kaiser Daily Health Policy Report, 3/17). The editorial stated that the high price for Cerezyme "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" (Kaiser Daily Health Policy Report, 3/25).
Termeer writes, "Our creation of a sustainable approach to developing treatments for rare disorders has helped transform the lives of people around the world with severe, debilitating and potentially fatal inherited diseases," adding, "From the beginning, we have openly explained the factors driving the cost of these treatments, primarily the extreme rarity of the disorders themselves." He concludes, "This transition from trial-and-error medicine will increase the value of truly innovative breakthrough drugs that society will demand for devastating diseases like Parkinson's disease, Alzheimer's disease and cancer" (New York Times, 3/31).
