Back in 2010 when European regulators announced plans to overhaul legislation on how the European Union oversees medical devices, the industry knew that it would take time for changes to take effect. With the EU reaching agreement on the regulation, it appears that the final Medical Device Regulation (EU MDR) may take effect as early as January 2017.
Advisory Committee meetings are an incredibly intense and high stakes endeavor within the drug development process. Millions in R&D costs, potential revenue, and patients served can come down to an eight hour meeting. When the FDA ultimately sides with the Advisory Committee’s recommendation approximately 85% of the time, these meetings have to go well.
Over the course of my 25+ year regulatory career, I’ve participated in numerous Advisory Committee meetings across a variety of therapeutic areas. Some went smoothly, and others didn’t. In my experience, when things don’t go well, it’s usually because of three main problems.
As you well know, off label communications is on my short list of regulatory policy and public health issues that keep me up at night.
Why? There is a leadership vacuum on the topic, and absent clear leadership, federal judges are starting to dictate regulatory policy through the Amarin case and others. If existing policy has evolved to protect the public from snake oil, the recent Amarin decision is precarious precedent for communications about fish oil – and beyond.
To help find better solutions to this important issue, on February 18th the new Duke-Margolis Center for Health Policy held a conference titled, “Off Label Communication in 2016: Meeting Information Needs through New Policy Options.”
Those new options are detailed in an important new paper:
Policy Options for Off-Label Communication: Supporting Better Information, Better Evidence, and Better Care
I am honored to be one of the co-authors and to have had the opportunity to speak at the event.
Just about every speaker pointed to the need for FDA leadership though bold action and … clarity. The paper lays out what we refer to as Guiding Principles for Lasting Solutions. They are:
It is often said that the FDA loves ambiguity – because it gives the agency unlimited authority. As a former regulator, I know that’s true. However, what’s more important to the men and women of the FDA is to be an innovation accelerator. And that means the agency has to lead through … clarity.
That wish is apparent when you study the FDA’s recently released 2016 Guidance Agenda, which outlines what the agency views as its top priorities for 2016.
The document is worth perusing in its entirety, but to save you some time, I have assembled what I view as some of the more interesting items for biotechs to watch.
To protect sustainable innovation, it is vital to look beyond present conditions and develop proactive interventions for anticipated environmental changes. With development times for new biomedical medicines and devices now spanning years (and sometimes over a decade), regular audits (at least yearly) are essential in recognizing and adapting to key "climate changes."
Given the importance of both identifying new trends and their impact on the development and commercialization of every product and process, YourEncore gathered an eclectic entourage of experts with deep experience in regulatory and industry leadership after the recent RAPS meeting in Baltimore. We call these sessions R.A.N.T.s - Relevant Assessments, New Trends. We turned the panel discussion into a white paper, full of insider insights on the changing face of biomarkers, pediatric studies and benefit-risk, all of which are integral factors in development planning that exist in a state of constant flux.
The discussion is particularly timely if you've read the FDA's recently released 2016 Guidance Agenda. We trust you will find this material useful in preparing for the upcoming guidance season as well as informing your product/portfolio management and regulatory strategies.
During my 18 years at a major pharmaceutical company I initiated and managed the introduction of many new technologies into both pre-clinical and clinical project teams. One of my greatest challenges was translating new biomarker discoveries into assays that could be used successfully to support clinical trials.
Discovery of new clinical biomarkers is occurring at a furious pace, which is encouraging to both drug developers and patients hoping to shorten clinical study and approval timelines. However, qualification of these new biomarkers has just begun and, while the FDA has recently outlined a proscriptive qualification process, it is unclear what biomarkers will ultimately receive regulatory approval (or how long it will take).
So, you may find yourself in the situation where you are aware of a new biomarker that could be a significant improvement over existing assays used to support clinical trials but the data to support that contention is not yet available. Under these circumstances, what is the best way to proceed?
I recommend you answer the following six questions before adding a new biomarker to your clinical trial protocol:
Most people agree that hindsight can provide a valuable perspective. Marty McFly in the movie Back to the Future certainly learned a lot about his parents by going back in time. However, in the case of regulatory approvals, hindsight can be downright confusing when trying to predict the future.
The biopharma industry is having a Jerry McGuire moment.
I’m referring to the scene when Jerry is on the phone with Rod Tidwell and they are shouting, “Show me the money!”
Short for Relevant Assessments, New Trends, YourEncore’s R.A.N.T. offers a refreshingly pragmatic perspective of regulatory policy strategy from people who understand it from the inside out.