CBI Scene Blog

Unique Challenges When Commercializing Specialty Drugs & How to Mitigate Risk

Posted by Sarah S on Nov 12, 2014 10:09:42 AM


It is understandable that drug companies wish to extend the use of a product they spent considerable time and money developing. Similarly, patients who have been unable to find answers to their medical issues can hardly be blamed for championing off-label use of a medication to help alleviate a problem.

Opening up a drug to uses other than the one explicitly stated on its label, however, brings significant challenges. Regulators must ensure that drug manufacturers aren’t pushing product or advertising it in misleading or untruthful ways. Medical affairs departments and physicians must remain compliant but still do their utmost to help patients.

Background of Off-Label Use

According to the Regulatory Affairs Professionals Society (RAPS), the FDA has “actively permitted” medical professionals to prescribe drugs for off-label uses since 1982, seeing it as “an essential part of the ecosystem of medical innovation.” Nevertheless, if off-label uses and promotion are not driven by consumer need and physician interest, or if requests for information about off-label uses receive artificially inflated or promotional responses, then drug companies are not following best practices, says the Medical Science Liaison Society.

Today’s restrictions on off-label promotion prohibit marketing off-label uses to physicians or medical professionals unless they ask, responding privately to solicited requests and presenting balanced information, adds RAPS.

Unfortunately, the medical community has seen a recent rash of failures to honor these practices. According to DMH BioPharm Advisors, this includes promotion of off-label uses, “failure to separate sales and medical affairs activities,” and selectively sharing study results, among other missteps, forcing increased scruity.

Consumer Impact of Off-Label Restrictions

Sadly, off-label drug use, while it may help a consumer deal with a medical issue, doesn’t always work out well for them financially. According to the American Cancer Society, “Many insurance companies will not pay for an expensive drug that’s used in a way that’s not listed in the approved drug label. They do this on the grounds that its use is ‘experimental’ or ‘investigational.’”

This presents a challenge both to medical professionals and to their patients. It also challenges medical affairs, who must then decide whether to seek FDA approval of the new use or uses for an already-approved drug, says the University of Pennsylvania Law Review. If they do not, however, they may face lesser likelihood of a drug being used in legitimate and helpful ways.

Moreover, restrictions about how drug companies can present the uses of their medications to doctors present the possibility of helpful off-label uses getting missed or buried. The Pennsylvania Law Review reports on the case of Pharmerica’s Ridaclot. In addition to its well-known on-label indications, third-party researchers determined that the drug also prevents mental decline in early Alzheimer’s patients. Because the study was published in a minimally circulated publication and not picked up by the press, however, these findings never attracted attention. Pharmerica is unable to tout these findings, according to the rules about off-label promotion. Rather than protecting consumers, it hurts them.

This example does make a compelling case for promoting off-label uses that might help alleviate consumer suffering. However, safety and responsibility must come first. Not just for the consumer’s sake, but for the company’s as well.

Engage Customers for Help While Remaining Compliant

It’s perfectly fine to engage customers for help in disseminating off-label information, so long as they solicit the information you are providing. Heavy penalties exist for sharing unsolicited information with customers like group purchasing organizations or specialty pharmacies, but if they ask, you are welcome to share all the data you have.
This includes information you have on file but that isn’t published, says RAPS. Anything that will help your customers understand the medication or technology and the way in which it might help patients is valid. Additionally, prohibitions about using a public forum to answer requests for information (social media, websites, etc.) do not extend to speaking about the drug at a conference or other open event. In that case, you should feel free to present fair, balanced information germane to the question.

Mitigating Risks

According to the FDA, off-label drug use “when the intent is the ‘practice of medicine’” does not require a new approval, but does place a burden on both physicians and pharmaceutical companies to ensure that medicine is at the heart of the endeavor.

Maintaining excellent records regarding whom you speak with about each off-label promotion, all questions you answer and all information you disseminate can help prove your intentions are pure and you are in compliance. Medical affairs compliance programs, moreover, must adapt to stringently adhere to guidelines about commercialization of specialty drugs so that they may take opportunities to promote their products lawfully while steering clear of legal pitfalls.

Want to better understand the risks and unique challenges associated with high-touch therapies and how your compliance program can adhere to new legislation of off-label promotion? Join us at CBI's 12th Annual Pharmaceutical Compliance Congress- PCC 2015, the flagship event where industry stakeholders come together to exchange ideas and strategies surrounding some of the most critical issues facing compliance officers and their programs across life sciences.

PCC 2015 button


Topics: Compliance