A spokesman for Celgene, which makes Revlimid, said that Revlimid, developed as a treatment for rare blood and bone marrow cancers, is used nearly exclusively in its approved orphan drug indications.
Makary said that what pharmaceutical companies say their drugs are used for is different from how doctors use them in the real world. Off-label uses can be lifesaving, and we doctors need the ability to prescribe off-label, but their comments on uses are legal, not data-driven.
"No one tracks the indications for 99 percent of prescriptions in America. From the perspective of Celegene's executive suite, Revlimid is only used for narrow orphan indications, but on the front lines of medicine, it's used broadly off-label as a second-line agent for a variety of conditions, including chronic lymphocytic leukemia and Hodgkins lymphoma," Makary said.
A Genentech spokeswoman said that the orphan drug designations for its medicines, including Herceptin, referenced in the study were for patient populations considered sufficiently small enough to meet the definitions within the Orphan Drug Act. She said the company, whose parent corporation is Roche, does not promote off-label uses of its medicines. Of the approved indications for Herceptin, only gastric cancer has an orphan drug designation. She did not comment specifically on Rituxan.
Designation refers specifically to the track the drug goes in for the FDA approval process. Indication is the specific medical condition the company lists on their FDA approval application, and if approved, it's the indication for "on-label" use as opposed to the "off-label" use.
The Genentech spokeswoman said, "We are committed to discovering new medicines for people who have few treatment options, and we welcome updates to regulations around orphan drugs to ensure that pathways and incentives lead to more medicines for rare diseases."