FDA publishes ICH guidelines as FDA guidances. This guidance reflects just one element in FDA’s work with regulatory authorities and industry associations from around the world to promote international harmonization of regulatory requirements under the ICH. One of the goals of harmonization is to identify and reduce differences in technical requirements for drug development among regulatory agencies. FDA is committed to seeking scientifically based harmonized technical procedures for the development and manufacture of pharmaceuticals.
- ICH (2019) E19 Optimisation of Safety Data Collection
- FDA Guidance for Industry (2016) "Determining the Extentof Safety DataCollection Needed in Late-Stage Premarket and Post approval Clinical Investigations"
Duke-Margolis Center for Health Policy organized a two-day workshop "Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes"
Dr Ellis Unger from FDA discussed the topic "Safety Monitoring in Clinical Trials for Generation of RWE" (see the video @1:10)
"Once a drug has been approved, its risks/harms are well-characterized; safety monitoring in studies for new indications can be less intensive (in may circumstances)"
"...about the defensive research, which is really a huge problem, which is that let's collect everything because some regulators might ask us in a year, ho, well, what were the CBCs? all these nitpicking questions that require mountains of data.
We put on a guidance in 2016. it's called, 'Determining the Extentof Safety DataCollection Needed in Late-Stage Premarket and Post approval Clinical Investigations' What it basically talks about collecting less than usual safety data in those situations where the safety data are well characterized. My particular office oversees the Division of Cardiovascular and Renal products, and we talk with many sponsors. Look, don't collect all this stuff in your next trial. You want to do a cardiovascular outcome trial, that's great. But we already know how many headaches and hangnails a drug causes. Just collect serious adverse events. and they thank us and they shake their heads and they walk out of the door. and then when they send in the final protocol, they are collecting everything. This happens time and time again and it dawned on us. well, maybe the problem is regulators across the pond and so about 4 years ago, I think, we proposed this topic to ICH collecting less than full safety data collection and it was adopted and I'm glad to say that the step 2 guideline is out and open for public comment. it is called "optimization of safety data collection".
And the interesting thing was that a lot of the resistance to this approach was not coming from the regulators across the pond or in Asian. It was coming from companies who were just firmly entrenched in the idea that they better collet it because they may need it. I would like someone who works for a company that has done one of these outcome studies for an anti-diabetic drug could tell me exactly what they learned by getting vital signs every, lab everything, every nonserious adverse event. Tell me what you learned and tell me what it costed you. I suspect that you didn't get much bang for your buck.
So this guideline is out there and I really do hope most of the people who have a vested interest take a look at it and there's a docket in the US and other countries where you can submit your comments Through official Pathways to get them considered."