CDER Director, Dr. Janet Woodcock gave the opening keynote at yesterday’s third annual Risk Management and Drug Safety Summit. And it was an address to reckon with.
Some selected comments:
“Safety issues aren’t headlines – they’re biology; a scientific puzzle.”
“The CDER mission is to promote and protect the public health by assuring that safe and effective drugs are available to Americans.”
“Safe does not mean risk-free. Effective does not mean equally for all. We do not have a legislative mandate to consider pricing issues and I doubt we ever will.”
Her four-part definition of “safety”: (1) risks are managed, (2) quality is assured, (3) health fraud is pursued, and (4) advertising is appropriate.
She announced that in “the Fall,” FDA would release several FDAAA-related guidance documents:
– Guidance that should reduce burdens on the healthcare system of “Medication Guide-only” REMS, while preserving use of Medication Guides to present important information to patients as part of patent labeling
– Guidance on safety related labeling changes
– Guidance on Postmarketing Studies and Clinical Trials
Relative to advancing the science of drug safety Janet discussed:
• In silico modeling
• Systems biology
• Animal models
• In-vitro models
• Benefit-risk assessment
She also announced that the agency was creating a new group within CDER’s existing biostatistician office to focus on safety.
She discussed failure modes and effects analysis (FMEA). Some in the audience whispered that, hopefully, FMEA would do a better job than FEMA.
Janet also shared that morale within CDER “despite what you may read in the press,” is high and has improved since more staff has been hired to more fairly address the workload.
She addressed the FDA’s participation in the Observational Medical Outcomes Partnership, and many more issues -- all which can all be found in her PowerPoint here.
And it’s definitely worth a look to any and all interested and/or concerned about the future of REMS specifically and risk management more generally.
Next up was Sir Alasdair, who shared the MHRA’s “pharmacovigilance toolkit. (Note, this toolkit can be found as part of his PowerPoint presentation here.) It’s elegance and simplicity is in stark contrast to the FDA’s approach, which seems to be “let 1000 initiatives bloom.”
He made an interesting observation that, in the 1950s, thalidomide was drug safety’s first historical moment and that Vioxx was it’s “second coming.”
He spoke to the Avandia issue by stating that EMA and FDA had reached the same conclusion by reviewing the same data simultaneously. However, since the regulatory tools of MHRA (as part of EMA) and FDA are different, the results were different. To wit, while Avandia remains on the market in the US with a REMS, in the EU the drugs license has been suspended.
Sir Alasdair also adroitly addressed the need to move from data management to risk management – a finesse that many in the audience also viewed in contrast to the current FDA approach.
Specifically, he discussed the need to move from reactive to proactive methodology and harness more robust sources of data, moving “up the evidence hierarchy. And that “regulatory actions should be linked with measurable public health benefit.”
It was an information-packed presentation that is worth careful examination by those interested in how the EU is approaching various risk management issues.
The highlight of the morning was a brief, heated (but friendly) Q&A debate Drs Woodcock and Breckenridge on the topic of HbA1c as a legitimate biomarker for Avandia. (Sir Alasdair’s point was that, if Avandia was being reviewed for approval today, HbA1c would not be an acceptable marker.) It was a collegial exchange that should only slightly impact the Special Relationship.
A video of this point/counterpoint will soon be available on www.drugwonks.com.
Juergen Schmider (Corporate Safety Officer and Vice President of Global Pharmacovigilance & Epidemiology at Cephalon) was up next and made some very interesting points about, among other things, off-label promotion – about which he commented, “It used to be a gentleman’s offense, but no longer.
Indeed.
Herr Schmider’s presentation can be found here.
There were a series of other presentations, all of which can be found at www.fdanews.com/RMDSSpresentations.
The day ended on a hopeful and thoughtful note with the following comment from Gary Appio (US Safety Director, Novartis): “We need to advance towards thinking not about risk management, but benefit/risk management.
Amen.
