Snake Oil & Enforcement
One of the unfortunate things about any new health concern is that the snake oil sales start immediately. There are far too many scumbags who see an opportunity to profit in a crisis. The COVID-19 outbreak is no exception.
These people represent a real danger to the public. People who promote unproven treatments can convince the public that they don't need to take the outbreak seriously, can lead to reckless behavior, and in the extreme can contribute directly to increased deaths as their victims rely on the snake oil instead of trusting healthcare providers.
In this setting, FDA and FTC enforcement is essential. We need vigorous enforcement from the authorities to stop the spread of misinformation. I'm glad to see FTC and FDA have already taken some action in this regard. I hope we'll see more.
Off-Label Promotion & Outbreaks
Researchers, practitioners, and industry are frantically searching for any effective treatments. Currently, the science is unclear about whether any treatment works.
As I write this, there has been a lot of buzz around the possible effectiveness of an anti-malarial treatment, hydroxychloroquine sulfate. This is an already-approved drug being used for an unapproved indication, i.e., this is a classic off-label use of a product.
I have seen some misinformation in the media about off-label uses of products, so I thought it might be helpful to set out the background to all of this.
To bring a new drug to market, a company must submit a New Drug Application (NDA) to the FDA. Among other information, the NDA includes proposed labeling. The proposed labeling provides directions to healthcare providers explaining how to use the drug safely. That labeling sets out the proposed uses of the drug. The uses of the drug are known as the indications. Indications typically include a population, a condition, and the stage/severity of the condition (if applicable).
The FDA evaluates the NDA to determine whether the sponsor has demonstrated that the drug is safe and effective for the proposed indications. Assuming the sponsor meets the evidentiary burden, the FDA approves the proposed indications for the product.
After being approved by the FDA, sponsors may only promote their drugs for the specific indications approved by the Agency. Any other use of the product is "off-label" use.
Off-label use is NOT inherently bad, wrong, or evil; and off-label use of a drug is also not prohibited. The other evening I heard a television news segment where the expert commentator spoke of using drugs in violation of FDA's rules. That's wrong.
FDA does not regulate the practice of medicine, and physicians are, for the most part, able to prescribe drugs as they believe is appropriate to treat the patients they see.
But the sponsors are not permitted to promote their drugs for any uses other than those approved by the FDA. Importantly, there is no explicit prohibition in law against the promotion of unapproved uses. You won't, for example, find off-label promotion listed as one of the items that causes a drug to be misbranded. Instead, there are multiple legal theories about this prohibition and what exactly makes such promotion illegal, including the failure to include adequate directions for use in the labeling.
FDA has made it clear that it sees the continued prohibition of off-label promotion as essential to protect the public, despite some adverse judgments by some courts.
If a company learns of a new off-label use for a drug and wants to promote the drug for that use, the sponsor must conduct additional clinical trials to demonstrate that the drug is also safe and effective for that new use. After completing the clinical trials, the sponsor submits an sNDA (supplemental New Drug Application) to the Agency.
Assuming the Agency approves the sNDA, the label is updated to include the new indication, clinical trial data, additional instructions for use, etc., and the company is then able to promote the new use of the drug.
So, FDA doesn't have to DO anything to make the possible off-label treatments available, despite several prominent people calling on FDA to take action. The drug being suggested for off-label use is already on the market and can be used for this off-label use.
Why doesn't FDA simply approve the new use?
Because FDA doesn't know if the drug works.
When FDA evaluates an NDA or sNDA it weighs the risks and benefits of the drug for each specific proposed use. As FDA has repeatedly made clear, each specific proposed use requires a separate risk-benefit determination.
It is possible (even likely) that a drug that has been proven to be safe and effective for one use might not be either safe or effective for another use.
Indeed, one frequent criticism of off-label use of drugs is that there simply isn't sufficient evidence of effectiveness for many off-label uses.
And that in turns leads directly to why FDA's prohibition on off-label promotion is so important to protect the public health. Using treatments without evidence and promoting such treatments can make things worse.
FDA has the tools to act rapidly to approve new uses of a drug, but it is unwise to short-circuit the approval process, and it's irresponsible to hype an off-label use without sufficient evidence to demonstrate its efficacy.
Why doesn't FDA simply approve the new use?
Because FDA doesn't know if the drug works.
When FDA evaluates an NDA or sNDA it weighs the risks and benefits of the drug for each specific proposed use. As FDA has repeatedly made clear, each specific proposed use requires a separate risk-benefit determination.
It is possible (even likely) that a drug that has been proven to be safe and effective for one use might not be either safe or effective for another use.
Indeed, one frequent criticism of off-label use of drugs is that there simply isn't sufficient evidence of effectiveness for many off-label uses.
And that in turns leads directly to why FDA's prohibition on off-label promotion is so important to protect the public health. Using treatments without evidence and promoting such treatments can make things worse.
FDA has the tools to act rapidly to approve new uses of a drug, but it is unwise to short-circuit the approval process, and it's irresponsible to hype an off-label use without sufficient evidence to demonstrate its efficacy.
