Patient Safety Advocates, Industry Spar Over Off-Label Promotion

On Nov. 9-10, 2016, the Food and Drug Administration (FDA) convened a highly contentious two-day public hearing to obtain input on the agency’s rules limiting off-label promotion — the pharmaceutical and medical device industry practice of promoting products for unapproved uses.[1]

Doctors are permitted to prescribe medicines and devices for uses not approved by the FDA, but drug and device makers are prohibited from peddling their products for those uses. However, the industry in recent years has claimed a First Amendment right to distribute materials about unapproved uses of medical products and is pushing the FDA to relax restrictions on such practices. The FDA already has proposed policy guidance that would permit additional off-label promotion by manufacturers, but industry wants the agency to go even further.

Lining up on one side of the debate at the FDA hearing were more than two dozen industry representatives and allies who argued for weakening limits on off-label promotion. On the other side were three Public Citizen experts, including me, and more than two dozen other patient safety advocates who emphasized the dangers posed to public health if the FDA were to grant the industry’s wishes.

Dr. Sidney Wolfe, founder and senior adviser of Public Citizen’s Health Research Group, presented data showing that for 80 percent of cases in which a drug is prescribed for an unapproved use, there is a lack of strong evidence that the drug is effective for that use.[2] Moreover, patients who receive a prescription drug for an unapproved use lacking such evidence are more likely to experience an adverse drug reaction that resulted in stopping the drug than those who are prescribed a drug for an approved use.

In my testimony, I explained why a manufacturer’s claim that a drug is safe and effective for an unapproved use is no substitute for the FDA’s rigorous, independent evaluation of the evidence for each proposed use.[3] I highlighted the many ways medical journal articles — which the industry wants to use to tout off-label uses — can be flawed, including by outright fraud, biased study design and incomplete reporting of safety and effectiveness data.

Finally, Robert Weissman, president of Public Citizen, emphasizing the FDA’s public health imperative, urged the FDA to reject out of hand manufacturers’ arguments that the First Amendment permits them to promote drugs and devices for unapproved uses.

Where the FDA comes down in this debate will have profound consequences for patient safety and public health. Opening the door to the promotion of potentially dangerous products for unapproved uses would fundamentally undermine the entire FDA approval process for drugs and devices. The FDA needs to slam this door shut.

References

[1] Food and Drug Administration. Manufacturer communications regarding unapproved uses of approved or cleared medical products. http://www.fda.gov/newsevents/meetingsconferencesworkshops/ucm489499.htm. Accessed November 16, 2016.

[2] Wolfe SM. Testimony for FDA meeting on manufacturer communications regarding unapproved uses of approved or cleared medical products. November 9, 2016. http://www.citizen.org/documents/2347a.pdf. Accessed November 16, 2016.

[3] Carome MA. Presentation at the Food and Drug Administration’s November 9-10, 2016, public hearing on manufacturer communications regarding unapproved uses of approved or cleared medical products. http://www.citizen.org/documents/2347b.pdf. Accessed November 16, 2016.