Archive for category #RWE

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

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.

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Real-World Evidence Studies: Checking it Once, Checking it Twice…

By now, either through posts on this tHEORetically Speaking blog, the official blog of HealthEconomics.Com, or through your own personal experience, it is apparent that real world evidence (RWE) is a hefty subject to tackle. Synthesizing evidence into something meaningful that meets the needs of the 5 Ps – Pharma, Payers, Providers, Patients, and Policy-Makers, as well as any other pertinent stakeholder, is no small task. Add to that the fact that this is real world evidence –  and not your run-of-the-mill clinical data where everything is carefully planned and controlled for – and you have a potential recipe for disaster!  So how do you navigate the waters and keep your boat headed in the right direction?

The first place to start is by attending the first ever virtual summit on RWE being held THIS THURSDAY (Dec. 11, 2014 from 8 a.m. – 5 p.m. ET; for more information and to register for this FREE summit, visit  The Summit is entirely virtual…meaning you can attend from anywhere on the globe, as long as you have an internet connection. Join us by clicking on the graphic below.

Thanks for downloading your copy of the Final Findings from the Survey 2014 on Real World Evidence_image001


ship      Amy Smalarz, President of Strategic Market Insight, will discuss the tools you need for successfully navigating the RWE boat. Specifically, Amy will describe how she has helped otherschecklist structure and maximize their RWE. Amy has developed her own RWE Checklist for Success that she will share with the audience (and even allow for audience feedback- after all, we  are all in this together!). This Checklist will visually present how to ensure the correct stakeholders have been incorporated and included. It will also provide a map to use throughout the  RWE process, to make sure the proper stakeholders are engaged at the right time, and detail just how exactly they should be engaged. RWE doesn’t happen overnight, and it shouldn’t  be an afterthought. Amy will highlight the importance of having discussions around RWE before project development.

How do you make this happen? Amy knows, and she will discuss strategies to facilitate conversations among the key players to get things moving and plans in place before moving forward. Recognizing that not all projects are created equal, the Checklist allows the user to customize the tool so that it meets the needs of individual projects.

Like everything else in life (including clinical trials), RWE is an evolving process. Amy will share her stories on how she sees things evolving, and what this may mean for RWE. You won’t want to miss out on this Virtual Summit (virtual = you can participate from the comfort of your office or home!) this Thursday. It’s not too late to register for this meeting (did we mention it is FREE, and more than 300+ of your colleagues have already signed up??? Learn how others are successfully navigating the RWE waters, and how to use your Checklist for success to charter your own boat!

Join us on December 11th at this free, day-long Virtual event and find out!   See you Thursday!!!


More about the Speakers

amysmalarz Amy Smalarz, PhD, is President of Strategic Market Insight.  Amy has a PhD in Social Policy and Health Services Research from Brandeis University. She also has a Masters in Health Care Administration from Simmons College and BA in Biology and Sociology from Brandeis University.  She has worked for various payer groups and consulting/data analytics companies conducting HEOR and Market Access research for more than 13 years.  Awards and honors include the Tom Crossman Prize for Public Policy (Simmons College, 2002), the best paper based on a dissertation (Academy of Management, Health Care Division, 2006) and being an Agency for Healthcare Research and Quality (AHRQ) Fellow (Brandeis University, 2002 – 2005).


This blog posting was written by Meg Franklin, PharmD, PhD, Owner and President Franklin Pharmaceutical Consulting, LLC, on behalf of HealthEconomics.Com. Dr. Franklin may be reached at or

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