Allowing Off-Label Data Would Do More to Protect Public Health

As we all know, there are a lot of products successfully prescribed for off-label use, including those used for pediatric purposes without pediatric indications, aspirin prophylaxis for coronary disease in high-risk patients, and almost half of all treatments for headache-related diagnoses. 

And since I'm going to be speaking at the FDA hearing, called Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products, I want to share some thoughts on the topic.

Before I tell you what I think the FDA should do, let me tell you why I'm so passionate about this topic. It affects me as a professional who has devoted my entire career to finding better ways to communicate between the pharmaceutical industry and the stakeholders it serves: healthcare professionals, patients and caregivers, and the healthcare system.

See also: Will drugmakers get what they're looking for at this week's FDA off-label hearing?

And, personally, I'm also a patient impacted by this very issue of using a product off-label. I've been a migraine patient all my life. As everyone in the industry knows, many managed care plans require a treatment protocol of on- and off-label products, tried one at a time, until failure — in the hopes of eventually finding the right solution. And, like most patients, I tried several PCPs and specialists. So that I could receive coverage (this is how managed care “keeps costs down”), they each proceeded through the same protocol to get to the most effective solution. One doctor recommended Norvasc, an antihypertensive. One recommended oral contraceptives. One told me to stop taking oral contraceptives. And so on, proving that migraine is a condition that is still mysterious to the scientific community. And extremely frustrating to me.

The problem is that there is some pretty extensive research on migraine medications – all of it done by pharmaceutical companies, since physicians don't sponsor clinical studies on medicine. But much of this research will never reach a physician's eyes since pharmaceutical companies can only reference those pivotal trials that relate to the labels they are submitting for a new drug application (NDA) or supplemental NDA.

But back to me: After suffering from migraines for 40 years, a physician finally suggested a generic drug called Elavil (amitriptyline). It was used as an antidepressant in the old days, before the newer classes of SSRIs or SNRIs became popular. She told me that at low doses, it could be a prophylaxis for migraines. I tried it, and for the first time in my life, my migraines were manageable. Now, nearly five years later, I am 80% migraine free — amazing!

So what do you think was the first thing I did when my doctor prescribed Elavil to me? If I have to tell you, then I invite you to reach out to me directly because we need to have a talk about the digital world that we live in.

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Want to guess what manufacturer-supported information is currently available online for Elavil or amitriptyline-related to migraines? None! AstraZeneca, the original manufacturer, no longer has any online content since it no longer manufactures the product. And the generic manufacturers don't have any online content related to migraines either.

Yes, there are lots of online forums and resources like Migraine.com that come up in the Google results. There is also the newish “people always ask” box on Google that recommends topics that align with the migraine therapeutic category as well other on and off-label products that can be used. And sites like WebMD and Everyday Health showed up in the results as well. But there was nothing from any manufacturers.

(Interestingly, one thing that was consistent is that no matter the resource, all highly, highly suggest that, on label or off label, you speak to your doctor.)

So what should the FDA do in absence of any information for off-label products that successfully treat conditions? Since all prescription products are already regulated and attaining them without a script isn't even possible, we need a way to continue to support our healthcare providers in making the right decisions. We should make off-label research publicly available in a non-promotional format to both healthcare professionals and patients. It will not only improve compliance, but also drive better overall health outcomes.

The best way to go about this? Encourage pharmaceutical companies to continue to collect data on off-label uses of their approved products and share it in a non-promotional way, making it available as a resource to everyone — reps, healthcare professionals, and consumers managing their healthcare.

There's already a strong model for this: GlaxoSmithKline has been openly sharing clinical research studies of approved and unapproved products and indications since 2004, when it started its Clinical Study Register, a publicly accessible place for clinical research it carries out on its existing medicines and the ones being developed.

Or if the FDA isn't comfortable with this information being housed on a pharmaceutical company's own website, perhaps the agency could create a database that it or one of its partners (like WebMD) could manage — with clear messaging to identify the information as off-label. These are just a couple of options — there might be others — but my point is that it's possible. And it should be done.

See also: Off-label promotion: By the numbers

In talking this over with a dear friend and industry colleague, he suggested that perhaps opening the Pandora's box of allowing for off-label communication directly to healthcare providers and consumers could impact the entire NDA and sNDA process. If pharmaceutical companies can publish clinical research for any indication, why would they even bother putting a product through the traditional long and expensive submission process?

It could also kill patent extension. If off-label data is available to doctors, then they could be writing for off-label indications already. Or, just maybe, instead of spending money on DTC, pharma companies will spend more money to discover new indications or on having an appropriate place at conferences to get the word out.

You decide: is allowing off-label data to be available in a non-promotional way bringing the FDA into the modern age, protecting public health while driving innovation forward in the industry? This is a clear (and already proven successful by GSK) opportunity for the pharmaceutical industry to continue developing new strategies to partner with healthcare providers and fulfill their commitment to being patient-centric.


Zoe Dunn is co-founder and principal of Hale Advisors.