New Efforts Taking Root to Ease Off-Label Drug Promotion

Kerry Dooley Young

May 15, 2018

Legislative campaigns to ease restrictions on the off-label promotion of medicines could put patients' health at risk and leave them paying for costly drugs for which there is little evidence in support of their use, researchers said in a recent article.

Current federal regulations help physicians and patients in making choices about prescribing, leaving it to the US Food and Drug Administration (FDA) to define what new uses of already approved medicines outweigh their risks, write Michael S. Sinha, MD, JD, MPH, and Aaron S. Kesselheim, MD, JD, MPH, both of Harvard Medical School, Boston, Massachusetts, in an editorial published online May 8 in PLOS Medicine.

The FDA often requires pharmaceutical companies to conduct significant testing before it grants approval of expanded uses of their drugs. The resulting body of research shows which patients may benefit from a product, while revealing more information about potential side effects.

"These distinctions are crucial for individual physicians — who do not have the time or expertise to perform the same critical data evaluation conducted by the scores of highly trained scientists at the FDA — and for patients, who could be exposed to more non-evidence-based and potentially dangerous off-label uses of high-cost drugs," Sinha and Kesselheim write in support of current federal policy.

Law enforcement officials, legislators, insurers, and pharmaceutical companies have been wrestling for years with questions about how early and how aggressively to disseminate preliminary information about potentially expanded uses of approved drugs. Sinha and Kesselheim note that "tens of billions of dollars in civil and criminal penalties" have been paid in connection with off-label promotion. They cite widespread use of antipsychotics in elderly patients with dementia as an example of the risk patients face from off-label promotion.

The conservative Goldwater Institute is working to remove what it sees as barriers to "the sharing of truthful, scientific information about off-label uses" of medicines, according to an initiative called Restoring Free Speech in Medicine. The Phoenix-based group last year applauded its home state of Arizona for being the first to pass a law that permits greater communication about off-label use of drugs. At this point, the Arizona statute is essentially symbolic, owing to the fact that the statute is preempted by the federal Food, Drug, and Cosmetic Act (FDCA), which gives the FDA its authority.

"However, invoking such a challenge may be one of the goals of the law's key proponent, the Goldwater Institute, in its effort to limit the FDA's ability to regulate off-label promotion," Sinha and Kesselheim write.

Taking It to the States

The Goldwater Institute has been encouraging more states to take on the FDA's off-label marketing restrictions. "A number of states have introduced legislation this year, and we expect additional efforts to be made in several of the states with year-long sessions," wrote Jennifer Tiedemann, the Goldwater Institute's communications manager, in an email to Medscape Medical News.

In outlining its initiative, the institute argues that the current FDA rules are "increasingly inconsistent with the rapid availability of healthcare information in the 21st century." It also depicts those rules as being "at odds with constitutionally protected speech."

"Unfortunately, physicians and other healthcare providers often lack the most up-to-date information on potential treatments for their patients," wrote Naomi Lopez Bauman of Goldwater in a Web posting. "That is because current FDA rules and regulations have essentially criminalized the sharing of truthful and nonmisleading information between manufacturers and healthcare providers and payers."

Through the 21st Century Cures Act of 2016, Congress allows drugmakers to provide certain economic information about off-label drug uses to insurers' formulary committees. These kinds of exchanges will become more important as insurers increasingly seek to peg their payments to the benefit that medicines provide to patients, according to the Pharmaceutical Research and Manufacturers of America (PhRMA).

"As the health system moves towards value-based care, payers and providers are seeking more information about treatments, including information that may be outside the four corners of US Food and Drug Administration approved labeling," said Andrew Powaleny, a PhRMA spokesman, in a statement issued to Medscape Medical News. "A regulatory framework that allows biopharmaceutical manufacturers to responsibly share such information is essential to fostering innovative approaches such as value-based arrangements and other efforts to improve patient care."

Research Is Important

Yet, physicians and other healthcare professionals have ready access to medical journals and other sources of information about the testing of new uses for medicines.

The current restriction on the broad promotion of drugs for unapproved uses serves as an important driver of medical research, Christopher Robertson, JD, PhD, a law professor at the University of Arizona, in Tucson, told Medscape Medical News in an interview. Physicians, the FDA, and drugmakers often make only educated guesses about how a medicine works before conducting robust studies. If companies did not need the FDA's permission for expanding labels, they likely would not invest in costly and risky tests of already approved drugs.

"We want to incentivize the companies to do that research," Robertson said. "This Goldwater effort actually stands for ignorance."

Research for article was supported by the Laura and John Arnold Foundation. Dr Kesselheim has received grants from the FDA Office of Generic Drugs and the Division of Health Communication for unrelated work. Dr Sinha has disclosed relevant financial relationships.

PloS Medicine. Published online May 8, 2018. Full text

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