21st Century Cures and Off-Label Promotion Benefit HEOR and Drug Safety

Jim Davis

By Jim Davis

As my writing for the AdverseEvents Blog can attest, I’m in favor of data transparency. In my view, no organization should have monopolistic control over important information that limits the ability for healthcare decision makers to perform unbiased, objective comparative effectiveness research (CER). Furthermore, transparency leads to data and information being exchanged more freely, which will lead to an overall benefit to drug safety.

Pharmaceutical companies have historically had a bad reputation when it comes to transparency. 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 marked transformation by pharmaceutical companies to be completely transparent in publishing the results of all research conducted. Just searching Google for “pharmaceutical company transparency”, page one 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.

It is now time for FDA to lift the remaining obstacles to transparency they have put in pharma’s way. And several ongoing initiatives may do just that.

The 21st Century Cures legislation attempts to provide a clear picture of what manufacturers can, and cannot, communicate with healthcare decision makers. New research from Avalere Health suggest that if approved, this could result in better evidence on cost-effectiveness, comparative benefit, and real-world outcomes for payers determining patient access to these medications. In addition to 21st Century Cures, on May 7 2015 Amarin Corporation, Plc filed a lawsuit against FDA, looking to lift restrictions  on marketing products based on claims that are currently not on a drug’s FDA approved label.

Pharmaceutical companies are the chief sponsor for most late stage and post-marketing drug research and they have the most to gain, and more importantly, lose, if their data fails to meet healthcare decision maker and market demands. 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 providing guidance and allowing for off-label promotion, FDA effectively incentivizes market access and brand teams to fund differentiated and alternative research, as well as 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. 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.

 

Written by Jim Davis

jim@adverseevents.com

EVP, AdverseEvents

As Executive Vice President, Jim manages the company’s global sales and business development efforts. Jim brings over 12 years of experience in commercial strategy, global sales management and execution, business development, and product development. He has over 9 years of specific domain expertise in biopharma market research, intelligence, and data.