Drug shortages have been of grave concern to cancer patients for whom the right drugs can be a matter of life or death. Now there is a published study that documents that a drug shortage can effect life expectancy of young cancer patients.
The study from St. Jude Children's Research Hospital found that estimated two-year cancer-free survival for patients with intermediate- or high-risk Hodgkin lymphoma fell to 75% from 88% when the drug cyclophosphamide was substituted for mechlorethamine, a drug that has been in short supply. The study's authors have pointed out that other treatment options were put into play and that no patients have died as a result but said, "those who relapsed received additional intensive therapy that is associated with higher odds for infertility and other health problems later." They said it is not yet known what the longer term impact of the drug substitution will be. The study was published recently in The New England Journal of Medicine.
"This is a devastating example of how drug shortages affect patients and why these shortages must be prevented," said Dr. Monika Metzger, the study's principal investigator. "Our results demonstrate that, for many chemotherapy drugs, there are no adequate substitute drugs available."
The study points out what the industry knows, that shortages, particularly of generic injectable drugs like mechlorethamine, is more than a financial or regulatory hurdle to be jumped. The drugs often bring thin margins so they may only have one supplier still making them. If the drugmaker has a plant problem or other interruption, then a shortage can quickly materialize. Mechlorethamine, which is again available, the study points out, has been used in cancer treatment since the 1960s. But cyclophosphamide also has been extensively prescribed for treating children and adults with Hodgkin lymphoma. Studies had shown it to be a safe and effective alternative to mechlorethamine.
The FDA in 2011 initiated new protocols for drugmakers to give it the earliest possible warning in case of potential shortages and has worked with companies to ease the impact of shortages. It has even granted temporary permission for some companies to import drugs that are not approved in the U.S. but are used in other countries as alternatives to drugs that are now in short supply. There have been fewer new drug shortages reported this year but the problem remains a big challenge for the industry and the agency.
The study's authors gave the FDA credit for working to solve the shortage problem but Dr. Michael Link, the senior author of the report, said, "This puts a face on the problem of drug shortages and shows that the problem is real, not theoretical. This is about a curative therapy that we were unable to administer because the drug we needed was not available."
- read the St. Jude release