Aug. 03, 2015

Regulations for Off-Label Drugs Must Change

Lupus Foundation of America President and CEO Sandra C. Raymond recently participated in a briefing for U.S. Senate staff discussing the use of off-label medications in the treatment of lupus and the importance of physicians having vital information about the impact of these off-label medications on lupus patients. 

When a drug is used in a way that is different from that described in the Food and Drug Administration (FDA) approved drug label, it's said to be an “off-label” use. FDA regulations permit physicians to prescribe approved medications for other than their intended indications, but do not allow the companies that make the drugs to provide information on that type of usage to physicians.

The following is an abbreviated version of Sandra’s remarks:

If any topic is a shade of gray, the education of physicians about the use of off-label drugs is one. I would posit that lupus is the poster child for the position that we must educate and promote education directly to physicians to ensure the medications for individuals with lupus are appropriately prescribed and monitored.

The FDA’s current regulations prohibit the pharmaceutical industry from sharing information about a medically accepted alternative use of an FDA-approved medication. We need to ensure that physicians are knowledgeable and are able to access the knowledge about the potential uses of a medication and the significant side effects a medication can have on complicated patients with complicated diseases.

Many medications used to treat lupus symptoms represents the standard of care (SOC’s) for people with lupus. We have strong, anecdotal evidence that when a person with lupus goes to the emergency room they may receive high doses of prednisone to knock down an organ threatening flare. Yet, when the rheumatologist sees that patient after the episode it is common to see they have been give far too high a dose and it may take many months to stabilize the patient.

Two SOC’s used to treat one of the more serious aspects of lupus, lupus nephritis, are cellcept, an organ anti-rejection drug, and cyclophosphamide, a chemotherapy. These drugs have never been fully tested or approved for use in lupus by the FDA.

We need to ensure that physicians are able to access the knowledge about potential uses of medications and the significant side effects these therapies can have on individuals with complicated diseases. To address this, the FDA should modify its existing regulatory framework. Through the process of revising regulation and updating guidance, the FDA should ensure:

  • All communications about medicines should be truthful and non-misleading.
  • A balance between patient benefit and potential risk to determine appropriate limitations on healthcare communications.
  • Companies are allowed to provide adequate directions for use for both approved and medically accepted alternative uses.

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