It’s Time We Talk—The Importance of Open Communication on Off-Label Use

Physicians and drug companies know drugs work for conditions other than what the FDA approved them for. When a doctor uses a drug for a condition not approved by the FDA, the practice is called off-label prescribing.  To stay current on off-label prescribing, physicians share their experiences with each other, and this informal knowledge transfer is pretty much the only evidence of safety and efficacy for the off-label prescribing decision.

Many of these off-label uses result in better patient outcomes, especially with mental health or oncology drugs where the majority of off-label use occurs, according to a story in the December issue of the journal Pharmacotherapy. The problem is that there is no uniformity in conveying this information, or science to back up the drug choice.

Unfortunately, the institutions with the most knowledge and information about the therapies are the biopharmaceutical companies that developed them, but they are legally muzzled from conversations outside clinical trial-substantiated claims which appear in the prescribing information of the products.  As a result, we’re left with an environment in which only the most networked and connected physicians have access to this information about off-label drug use.

This reality is even more puzzling when you consider that there are virtually no restrictions on the types of communication any other individual, company, academia, or insurer can have with the medical community or patients.

This can have real and lasting negative consequences for patients. In this rapidly evolving era of medical advancements, what’s happening in clinical practice often outpaces the FDA process for formally approving new indications and labeling changes. For example, more than 30 million Americans, their families, and caregivers are dealing with one or more of the roughly 7,000 known rare diseases—the vast majority of which lack a specific FDA-approved treatment. Despite this reality, many therapies are widely accepted as safe, effective, and medically-beneficial to rare disease patients. But the companies that created these drugs cannot share new information about their products being used in these situations.

I want to be clear, I am not advocating for the unraveling of the safety structure that is built into the FDA approval process. We can’t let drug companies avoid clinical trials, but—at the same time—we can’t force drug companies to withhold information that can clearly help patients and physicians.

Rather, the Global Healthy Living Foundation proposes biopharmaceutical companies be permitted to discuss, with informed decision-makers, off-label use that is truthful, and only in specific forums or publications such as medical association conferences or medical journals.

Patients have a right to know about and use the best medicine for their condition, and their doctor should not have to rely on hearsay evidence when prescribing.

Congress and the FDA should begin implementing regulations that allow scientifically grounded communication between physicians, insurers, patients, and biopharmaceutical manufacturers.  In July of this year the House of Representatives took a step forward with the recent passage H.R. 6 — the 21st Century Cures Act. The nonpartisan legislation seeks to bring our health care system into the 21st Century by investing in science and medical innovation, incorporating the patient perspective, and modernizing clinical trials to deliver better, faster cures to more patients.

In addition, the legislation touches on the off-label prescribing issue by requiring FDA to issue draft guidance to clarify how drug and device manufacturers can permissibly disseminate truthful and non-misleading scientific and medical information about a drug or device that is not included in the approved labeling for the product. Collectively, we need to encourage Congress and the FDA to continue pursuing solutions that acknowledge the reality of common medical practice, protect patient safety, and enable applications with proven outcomes.

It is important that advocacy organizations across the country continue the conversation about how we best ensure patients get truthful and scientifically accurate information we need from our physicians, insurers, and the researchers and manufacturers behind their treatments.


Louis Tharp is the Executive Director of the Global Healthy Living Foundation. GHLF advocates for improved access to care at the community level, amplifying education and awareness efforts within its social media framework.

Morning Consult