Posts Tagged FDAMA 114

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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Playing in the Real World Evidence Sandbox: Sharing, Teamwork, and Transparency

SandboxIs just playing in the same sandbox together an example of Real World Evidence (RWE) collaboration? Not really. So how exactly do you play nicely in the RWE ‘sandbox’, but really collaborate and actually get a project off the ground, into the field, and into publication?

Nathan White has some secrets to success. Nathan is the Director for NucleusX Market Access, a global healthcare consulting firm specializing in market access. On December 11, 2014 Nathan will share some of his knowledge and experience at the RWE Virtual Summit hosted by HealthEconomics.Com (for more information or to register, click on the graphic below or visit this link:

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

Nathan will discuss findings from research he’s conducted and pull back the covers on what Payer-Pharma nathan-white-quoteCollaboration really means.  What works? What doesn’t work?

He’ll lay out some ideas of what to discuss, once you get the stakeholders to the table.  And even when everyone thinks they have a deal, there’s the dreaded contract! How do you survive the contracting process, and what are your barriers to getting through it? What does it take besides money?

Lastly, transparency of data is an obviously important component for all parties involved in research. Nathan will address transparency head-on, and discuss the need for consistent data in outcomes research. He’ll also provide some case studies of RWE collaboration, and the reasons why identifying a good example is difficult.

You won’t want to miss Nathan White’s presentation on Payer-Pharma RWE Partnerships at 11:25am on December 11, so click here to register!

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Dr. Korn Quote

Transparency – there’s more there than meets the eye!  And there’s more on this topic.  Adding to the discussion on payers and partnerships (and transparency!) is Allan Korn, Chief Medical Officer Emeritus for Blue Cross and Blue Shield Association (BCBSA). As Allan says, “Health care is dangerous. Transparency and an understanding of the impact of innovations and discoveries on real patients in their own “real worlds” will make it safer. Much safer.”

Allan will focus more on metrics (outcomes) beyond those of a clinical trial, and begin to hone in on aspects of the patient side of RWE. He’ll discuss the pace of scientific discovery and how it has potentially outstripped the ability of a randomized, controlled trial to meet the needs of patients. How, on the date of FDA or EMA approval, can you assume a product tested in only 200-300 ideal patients in a controlled setting is safe and effective for all patients in all circumstances? He’ll also discuss the symbiotic relationship between a Quality Adjusted Life Year and a Patient Adjusted Product Year. We asked Allan about his opinion on expectations that payers have for RWE. His response? “Question everything with respect to the clinical research enterprise.”

Hear Allan’s talk at 11:45am on December 11 at the first-ever Real World Evidence (RWE) Virtual Summit: Practical Strategies for Addressing Multiple Stakeholder Needs.


So if you are able to get everybody into the same sandbox (to continue the metaphor) for collaboration, and you convince the parties involved to extend themselves beyond the confines of a clinical trial…what happens next? What does it mean? Can you influence policy with your results?


Neumann_PeterPeter Neumann, Director of the Center for the Evaluation of Value and Risk in Health at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center and Professor of Medicine at Tufts will discuss evidentiary standards for evidence. He’ll discuss what and how much evidence you need to be sufficient. He’ll talk about the way things are currently being done, and how they should be done in the future. The existing standards (Guidelines?) are vague, so there needs to be some clarity from the FDA, and some flexibility from a regulatory standpoint about the things pharmaceutical companies are allowed to say. Alas, no discussion would be complete without the mention of FDAMA Section 114, so he’ll be sure to incorporate that into the virtual summit as well.

You won’t want to miss this, so if you haven’t already, please register for the FREE, virtual (you don’t have to leave your home/office/sandbox) event now!

Real World Evidence (RWE) Virtual Summit: Practical Strategies for Addressing Multiple Stakeholder Needs.


Our next few blog posts will highlight even more speakers from the RWE virtual Summit!  Stay tuned to learn about the stakeholders involved in RWE and incorporating RWE over the product life cycle!

What issues would you like for our speakers to address?  We’d like to hear from you!  (The cartoon is just for giggles…we REALLY do want to hear from you.) Contact us here or leave a comment on this blog.

Dilbert Cartoon

A little more about the speakers featured in this post:

Nathan White, CPC is the Director for Market Access at NucleusX.  Nathan has experience in the pharmaceutical and biotech arena, with a focus in managed markets, market access, HEOR, product commercialization, specialty pharmacy operations, account management, business development, patient program design, and consulting operations.  He also has global experience and a vast understanding of healthcare reform and HTA in the U.K., Australia, France, and Germany.
Allan Korn, MD, FACP,  is Chief Medical Officer Emeritus, Blue Cross and Blue Shield Association (BCBSA). He has extensive experience in patient care, managed care policy, procedure and program development and customer service as well as operational responsibility for the development and enhancement of programs supporting the many clinical and business requirements of a sophisticated health insurance enterprise. This includes technology assessment/management, claims data reporting, physician-hospital integration, and creation of policies to support claims adjudication quality improvement programs, and managed care initiatives. He has also worked extensively to improve relationships between physicians and payers.
Peter J. Neumann, Sc.D. is the Director of the Center for the Evaluation of Value and Risk in Health at the Institute for Clinical Research and Health at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and Professor of Medicine at Tufts University School of Medicine, in Boston, Massachusetts. He has conducted numerous economic evaluations of medical technologies, the use of willingness to pay and quality-adjusted life years (QALYs) in valuing health benefits, and the FDA’s regulation of health economic information, and the role of clinical and economic information in healthcare decision making. He has served as President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), and as a trustee of the Society for Medical Decision Making. He has also held policy positions in Washington, including Special Assistant to the Administrator at the Health Care Financing Administration.

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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