Why Is the FDA So Frightened of Off-Label Communications?
Former HHS General Counsel and Deputy FDA Commissioner Bill Schultz recently expressed his belief that FDA policies developed 50 years ago remain sufficient to govern life in the 21st century. He’s wrong. Time marches on and regulatory practices must evolve to better serve the public health. Nowhere is this more urgent than in making sure physicians and patients have unencumbered access to accurate information about FDA-approved medicines.
Almost no one is against sharing valuable information about FDA-approved medicines. The discussion—often heated —is over how (or if) that conversation should be regulated by the FDA.
Off-label communication refers to information provided by pharmaceutical companies to health care professionals about uses for medication that are not mentioned in FDA-approved labeling. Often, a medicine that the FDA approves for one illness might also effectively treat other conditions not listed on the official label. Although off-label prescribing by physicians is common, the FDA currently prohibits pharmaceutical companies from sharing information about possible off-label uses of medications with physicians, which means doctors may be making off-label prescribing decisions without the best available information.
When the FDA considers a drug for approval, the agency does not base its decision on data for every possible indication. Initial approval is based on a ‘‘best foot forward’’ approach. But that does not mean there isn’t robust scientific evidence to support broader therapeutic uses. In fact, initial approvals, based on a narrow, randomized population, provide a window into future clinical possibilities.
In draft guidance, the FDA noted that ‘‘good medical practice and the best interests of the patient require that physicians use legally available drugs, biologics and devices according to their best knowledge and judgment.’’ And according to the House Energy & Commerce Committee’s 21st Century Cures Initiative,
"[These] conversations between and among doctors, patients, researchers, and scientists in academia and industry should be facilitated. This includes the free flow of data, research, and results related to what a therapy or combination of therapies does or does not do well and in what types of patients. "
New FDA Commissioner Scott Gottlieb recognized the importance of the availability of accurate information for physicians when he noted: “[P]atients and physicians make the best decisions when they have access to as much truthful, non-misleading, scientifically based information as possible.”
So what action should the FDA take?
The FDA’s first priority should be leadership though bold action and clarity when it comes to trustworthy and safe off-label communications. FDA leadership will be particularly important given the myriad of federal agencies – including FDA, FTC and DOJ – with differing views on pathways and jurisdiction, and the extreme danger in allowing federal judges to dictate regulatory policy. In the pursuit of improving public health, predictability – for manufacturers, for physicians, for patients – is paramount.
The end goal should be a regulatory process that augments FDA’s ability to review communication types that reflect rapidly emerging new evidence without changing the agency’s ability to pursue enforcement action.
Properly done, off-label communications advance precision medicine, deliver speedier positive patient outcomes, increase patient choice, and reduce costs to our health care system, Those who think that the argument over off-label communication is just about marketing and sales are looking at this issue through very narrow blinders.
Off-label communication is about getting the right medicine to the right patient in the right dose at the right time. The FDA should take steps to ensure that physicians and patients have the best available information when making decisions about treatment.
Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest. CMPI accepts funding from the biopharmaceutical industry, foundations and private individuals.