A proposal under discussion in Congress would allow pharmaceutical companies to legally share what is considered off-label product information for FDA-approved drugs.
The plan would essentially permit manufacturers to communicate new “scientific and medical developments” for drugs beyond what is included on FDA-approved labels in communications with physicians, insurers and researchers.
According to the drug industry, doing this would improve patient care by increasing healthcare providers’ access to clinical data that manufacturers cannot currently share with physicians who already prescribe some drugs off-label. Other experts say that sharing such data would allow pharma companies to better market their drugs to the insurers and pharmacy benefit managers that are increasingly demanding more information about medical products before putting them on a formulary.
“It’s intended to get at the issue of how manufacturers [can] communicate evidence around economic value of the products when it’s not on the label,” said Craig Burton, director of life sciences for advisory firm Avalere Health.
The proposal is included in 21st Century Cures, an extensive Congressional initiative spearheaded by two House lawmakers. Lawmakers plan to introduce legislation this year as part of a broader effort to modernize medical discoveries, development and treatments, in part by requiring the FDA to update some aspects of the current regulatory system.
Little information about the off-label proposal has been made public. The House Energy and Commerce Committee released a 393-page discussion draft in January, with so-called placeholder language briefly describing the proposal. The second discussion draft is expected to be released in April.
“The committee is working on a proposal that would clarify and rationalize these rules of the road so that scientific and medical developments can be shared with physicians, insurers, and researchers, with appropriate safeguards, in order to optimize patient care,” said a committee white paper also released in January.
PhRMA, which supports the proposal, believes drugmakers should be able to share a wider range of medical information about their products with healthcare professionals. This could include observation and comparative data gathered from outside of a randomized clinical trial, information from trials pertaining to the effects of a drug on a patient’s race or gender and pharmacoeconomic data that can be used to inform treatment decisions, according to PhRMA’s response to the committee’s white paper.
“The FDA’s regulations regarding companies’ ability to share truthful, non-misleading medical and scientific information are outdated,” PhRMA said.
Allowing drugmakers to share information that is not included on the label, such as data gathered from observational studies, would be helpful for physicians as well as payers, which are increasingly demanding more information about products before putting them on formularies, Avalere Health’s Burton said.
“Payers want to have value conversations,” he added. “How can manufacturers engage in those value conversations short of doing head-to-head clinical studies?
As new drugs come to market, insurers and PBMs are seeking more information about how drugs perform and how they compare to alternative treatments. The entry of more costly drugs in recent years has kicked off a national discussion about how much is too much to spend on a medication.
Express Scripts, the nation’s largest PBM, said in a statement it looks forward to seeing the legislation once it is drafted. “In the meantime, we will continue to advocate for our plan sponsors and make the use of prescription drugs safer and more affordable,” a company spokesman said.
A study published in JAMA in 2006 estimates that 20% of US prescriptions are off-label. Other studies suggest that 80% of prescriptions for children are off-label and that off-label prescriptions tend to be written more in certain categories, such as oncology and pain management, according to an abstract published in the Duke Law Journal in December. The authors of the Duke study cited other research that found “15% of all drug uses lack scientific support for efficacy and more than 70% of off-label uses lack significant scientific support.”
“On the other hand, off-label drug use is a vital tool for patient care,” they wrote, further noting that some off-label prescriptions are supported by scientific data. “It allows physicians to treat patients for whom off-label drug use may be the only therapy available, including patients for whom on-label use has failed.”
“It’s a real moral dilemma,” said Michael Walsh, a partner with law firm Strasburger & Price. “There are a high number of prescriptions being used with no scientific evidence and a regulatory scheme that prevents a manufacturer from disseminating information.”
One concern that may be driving attention to this issue is that some payers are no longer reimbursing for certain drugs that are prescribed off-label, according to the National Organization for Rare Disorders. “The government severely restricts what drug companies can say about new research and about off-label uses, thus cutting off information from the most knowledgeable sources,” NORD President and CEO Peter Saltonstall said in a May 30 comment letter.
Despite industry support for the proposal, it’s unclear whether it will make it into legislation given the sheer volume of proposals presented in the January discussion draft. “We expect policymakers to start narrowing down their priorities,” Burton said.