Nine-year-old Emily Whitehead is a cancer survivor. She owes her life, in part, to her doctors’ decision to administer a pharmaceutical for a use different than that approved by the federal Food and Drug Administration (FDA). Paradoxically, the FDA would consider it a crime for the drug’s manufacturer to tell doctors about the treatment that saved Emily’s life.
After suffering a relapse in her drug-resistant leukemia in 2011, Emily entered a clinical trial at the University of Pennsylvania testing a new cancer fighting strategy genetically re-engineering her body’s T-Cells to attack her cancer. Her body responded, but the response was so powerful it nearly killed her.
As Emily lay unconscious in the ICU, her immune system raging out of control, her doctors ran blood tests and found that her body was overproducing an inflammatory protein associated with rheumatoid arthritis. So they gave her a drug, tocilizumab, which was known to switch off production of that protein in arthritis suffers. The experimental therapy killed her cancer, but it was the arthritis drug that kept the cure from killing her. Today, the drug is used to help patients in clinical trials who have the same reaction as Emily, saving even more lives and helping advance her cancer therapy from the clinic to (hopefully) eventual FDA approval.
Emily’s story is repeated in some variant tens of thousands of times across the U.S. every day, as doctors treat patients with rare or hard to treat disorders with medicines that are FDA-approved to treat other ailments. Studies have estimated that about 20 percent of pharmaceuticals used in the U.S. today are used “off label,” and many are actually considered standards of care for cancer, autoimmune disorders, and neurological disorders.
This is in some respects the future of medicine, because diseases with very different clinical symptoms can be driven by the same or very similar molecular pathways. Instead of talking about breast or lung cancer, as the science advances, doctors are increasingly referring to cancers by their genetic and molecular signatures: HER2Neu, ALK, or CD19. Oncologists are using the treatments for those cancers accordingly, regardless of what the FDA’s label says about the initial site of the cancer.
But even as medicine is evolving at a staggering rate, our regulatory system is frozen like a deer in the headlights. The FDA’s system for approving new drug indications can take years and cost millions of dollars. When patients like Emily need answers in minutes and when some of the newest treatments benefit small patient populations, it’s little wonder that off-label drug applications have become so commonplace.
Unfortunately, FDA regulations are not only pushing many drugs off-label but also keeping cutting-edge information from doctors that could save lives, even when that information is truthful and supported by sound science. Under current FDA rules, companies aren’t permitted to communicate information about off-label uses directly to doctors, with a few narrow exceptions. To do otherwise is considered unlawful drug promotion, and has led to billions of dollars in fines and prosecution of companies by the Department of Justice—$3 billion for GlaxoSmithKline, $2.3 billion for Pfizer, $1.5 billion for Abbott Labs, and $1.4 billion for Eli Lilly—even when the underlying uses are considered standards of care by public and private insurers.
The FDA’s approach makes little sense, and costs lives. Contra critics, permitting companies to alert doctors to the newest therapeutic uses of already-approved pharmaceuticals wouldn’t lead to a “Wild West” where snake-oil cures were sold to desperate patients. Doctors are not gullible dupes but highly trained professionals capable of making judgments about a drug’s potential benefits and side-effects, treating any novel drug information not subjected to FDA review with an appropriate level of skepticism. Rather than prohibiting all speech from companies that goes beyond a drug’s FDA-approved label, the agency should create a safe harbor for truthful, scientific information that is communicated to physicians, along with comprehensive information about the known risks and benefits associated with those uses.
The exact parameters of such a safe harbor are open to debate. Expert medical societies and patients’ groups could work with industry and the FDA to develop standards for science-based off-label communications, along with protocols for evaluating that information and validating it over time.
A safe harbor for communicating novel uses of FDA-approved medicines to doctors would encourage robust discussion and exploration of emerging indications. Such a rule could spur significant investment in novel research for medicines that have already run the FDA gauntlet and would free up scarce agency resources to aggressively police the true “snake-oil” cures that don’t meet the standards of sound science.
Paul Howard, Ph.D., is a Manhattan Institute senior fellow and director of the Manhattan Institute’s Center for Medical Progress. James R. Copland is a senior fellow with and the director of the Manhattan Institute’s Center for Legal Policy, which seeks to develop and communicate thoughtful ideas on how to improve the civil and criminal justice system.