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Drug Safety Benefits From Off-Label Promotion


If Amarin's lawsuit is successful, and FDA reverses its current stance, I believe that data and information will be exchanged more freely leading to an overall benefit to drug safety

On May 7, 2015, Amarin Corporation, Plc filed a lawsuit against FDA, looking to lift restrictions in place on marketing products based on claims that are currently not on a drug’s FDA-approved label. If Amarin’s lawsuit is successful, and FDA reverses its current stance, I believe that data and information will be exchanged more freely leading to an overall benefit to drug safety.

As my writing for the AdverseEvents Blog can attest, I’m not usually one who takes the side of pharmaceutical companies. In my view, their monopolistic control over important information limits the ability for healthcare decision makers to perform unbiased, objective comparative effectiveness research (CER). As such, it seems counterintuitive for me to side with Amarin and the rest of the pharmaceutical industry when it comes to off-label promotion. In this case however, the Amarin lawsuit, and the proposed change in FDA guidelines, are simply about opening the door to the unhindered flow of information. It is, and has always been, my belief (and the very foundation of our company, AdverseEvents) that the better informed healthcare decision makers are, the better treatment decisions will be made.

Unless there is a dramatic reversal by FDA, all off-label promotions will be required to be based on published studies. Pharmaceutical companies are the chief sponsor for most late stage and post-marketing drug research and they have the most to gainand more importantly, loseif their data fails to meet healthcare decision maker and market demands. Allowing for off-label promotion, supported by published studies, will require more research to be sponsored by the drug manufacturer. This will also cause their competitors to respond in-kind, providing for more balanced data.

Off-label promotion will also allow for more differentiated and value-based research. Even though payers are demanding more real world data studies and health economics and outcomes research (HEOR), only 43% of respondents from a recent EY survey, agreed with the statement that “pharmaceutical companies have data that is credible for measuring and improving outcomes.” There are good reasons why the amount of research in this area, and the acquisition of independent data sources by manufacturer sponsors have been limited. The results of such studies, and the use of independent data carry risks of coming to conclusions that can’t be readily commercially used. If a claim that is derived from the study is not in-line with the approved label, then the time, effort, and money spent on the study has effectively been wasted. By allowing off-label promotion, FDA effectively incentivizes market access and brand teams to fund differentiated/alternative research and seek out independent data sources. Thus providing healthcare decision makers with types of data that they are asking for to make fully informed CER decisions.

Even with more, differentiated research being conducted skeptics could argue that publication bias will skew data releases to only show the positive. Historically speaking, they have a point. In a large study conducted in 2004, of over 100 clinical trials, 65% of harms and outcomes were incompletely reported. However, in the past couple of years, there has been a marketed transformation by pharmaceutical companies to be completely transparent in publishing the results of all research conducted. Just searching Google for “pharmaceutical company transparency,” page 1 results show the public transparency policies of major manufacturers such as Janssen, UCB, and Takeda. This movement shows no signs of stopping and I believe it will be the rule, rather than the exception, in short time. With more research being conducted as a result of restrictions being lifted on off-label promotion, the transparency push will lead to more data being published, both positive and negative, leading to a less biased data pool.

All of the potential benefits from off-label promotion are contingent on FDA making smart decisions in the coming months that balance the need for a centralized authority on drug safety with the need for increased flow of data and information. It will be interesting to see the results of the open meeting they hold this summer on the topic.

In the meantime, we at AdverseEvents will continue to provide healthcare decision makers the independent, unbiased comparative safety research they demand. Click here for a Cost Comparison and Safety Analysis of Eylea vs. Lucentis for Diabetic Retinopathy.

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