Outcomes data enters the 21st century
By Peter Weissberg, group director, market access, at Intouch Solutions
A lot can happen in 20 years. When the FDA Modernization Act (FDAMA) was passed in 1997, a mere 217 of its ~40,000 words were allocated to the presentation of healthcare economic information (HCEI). Those 217 words resided within Section 114 of the Act, giving birth to the abstract universe many of us now commonly refer to as FDAMA 114.
Fast-forward to January 2017, and the FDA’s release of its Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities – Questions and Answers – Draft Guidance for Industry and Review Staff. Now 7,000+ words, this document is likely to significantly alter the way pharmaceutical manufacturers engage with the audience who increasingly holds the keys to its commercial success … payers such as health plans, pharmacy benefits managers (PBMs) and hospital systems.
I encourage everyone to read the FDA’s 20-page Q&A document, closely monitor the industry’s feedback, submit your own if desired, and watch for the future final, “official” FDA guidance, if it is ever to come. Meanwhile, we have this very useful draft, and below are some key takeaways, as well as specific strategies; you can immediately employ to be prepared for the new world of economically focused discussions with market access stakeholders.
We Asked for Details; Now We Have Them
The most universal knock on the original FDAMA 114 guidance was its high degree of ambiguity. The agency likely didn’t originally anticipate how critical the communication of HCEI would prove to be. For if it had, it probably would have allocated more than 217 words to help manufacturers better understand the appropriate guardrails.
With the new guidance, we now have a much clearer understanding of the types of information that constitute HCEI; the specifics of who actually represents a formulary decision maker; and which information not specifically contained with the approved label is appropriate for communication. This should significantly assist field, regulatory, health economics outcomes research (HEOR), market access and brand teams in achieving a better understanding of who can, and should, deliver HCEI and with whom they are able to engage.
Market leaders in this space, such as Bristol-Myers Squibb and GlaxoSmithKline, have been establishing clear protocols in these areas for many years, while others have remained largely reactive. This reactive position has led to widespread inconsistency and confusion that compromises the ability to provide customers with the types of information they truly desire while also remaining compliant with internal regulations.
How You Present and Manage Just as Important as the Information Itself
It is now imperative that sponsors engage expert technology partners to help them adhere to the guidance or risk punitive action. Simply understanding how these new information sets are defined will be table stakes. The ability to successfully leverage these data sets will require the engagement of partners who are experts in user experience to guide the organization, presentation and archiving of information. Additionally, the new statue raises the bar on how HCEI will need to be stored.
Previously, the HCEI presented was required to be made available to the FDA only “upon request.” Now, such information will likely be considered “promotional” and subject to filing with the FDA via Form 2253.
For investigational/pre-approval treatments, manufacturers may be required to proactively disseminate updated data in the future upon changes to certain initial assumptions and data points. Examples of such instances may include failure to meet the efficacy endpoint in a Phase 3 clinical trial or a change to the review status, such as when a clinical hold has been implemented. Manufacturers will therefore need to engage expert content-strategy and information-management partners in order to ensure compliance with these requirements. The information itself, and the dates and identities of the organizations it was presented to, will need to be easily accessible and auditable to allow for proactive follow-up customer engagements.
Competent and Reliable Scientific Evidence (CARSE)
The CARSE threshold was included in the original statute to highlight the need to ensure that the information being communicated under Section 114 was highly credible. Back in 1997, there were only a limited number of methods and venues available to generate and disseminate such data (e.g., the International Society for Pharmacoeconomics and Outcomes Research), and the CARSE threshold was well understood by the tightly knit HEOR community.
However, the current availability of new and diverse data sets, coupled with increasingly innovative tools to manipulate and analyze the inputs, has led to an explosion of HEOR data. While many of these future analyses are very likely to meet or even exceed the CARSE threshold, it is almost certain that others will not. The current guidance applies CARSE to clinical and economic information yet does not go far enough to provide specific guidance on how a judgment on CARSE can actually be determined. And in our era of “fake news,” this lack of granularity may eventually undermine some of the statute’s good intentions.
Standing Still Is Not a Viable Option
The world of pharmaceutical commercialization and communications is extremely fast-paced, and change is often measured in days, not months. Twenty years is a long time to wait for updated guidance on a subject as complex and wide reaching as the dissemination of HCEI. Over the past two decades, innumerable internal processes have been put in place alongside the entrenchment of a multitude of human behaviors, which now collectively serve as barriers to our collective evolution.
But with the issuing of new draft guidance on HCEI, our world has been fundamentally changed. It is now up to us to adapt to these changes by collaborating with peers and partners who not only understand the nature of this change, but who also possess the acumen and expertise to facilitate a transformation in the way we communicate HCEI. While doing so is not a surefire means for long-term success, anything less is an approach better suited for the 20th century.