RAPS recognizes that the current situation in Ukraine impacts our members and customers on many levels. If you are directly impacted by the current situation in the region and are challenged to meet your deadlines or obligations to RAPS, please reach out to raps@raps.org so that we can defer those challenges. Your health and safety are paramount to us.

Regulatory Focus™ > News Articles > 12 > Drugmakers Call on FDA to Expand the Scope of Master Protocols Guidance

Drugmakers Call on FDA to Expand the Scope of Master Protocols Guidance

Posted 21 December 2018 | By Zachary Brennan 

Drugmakers Call on FDA to Expand the Scope of Master Protocols Guidance

Novartis, Regeneron, Pfizer and industry group BIO called on the US Food and Drug Administration (FDA) to expand a recently released draft guidance on master protocols to make it applicable beyond oncology.

The 21-page draft guidance on master protocols, also known as “umbrella,” “basket” or “platform” trial designs, is meant to help sponsors of cancer drugs or biologics regarding the design and conduct of clinical trials intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure in adult and pediatric cancers.

While praising the document’s creation, Regeneron noted, “This draft guidance also presents an opportunity to be expanded whereby the FDA can provide advice regarding the design and conduct of late-stage clinical trials using master protocols across multiple therapeutic areas... For example, utilization of master protocols in rare diseases presents a good opportunity to expediate orphan drug development.”

Similarly, Novartis encouraged FDA to work across its various offices to identify areas where master protocols could be used in other therapeutic areas.

And Pfizer said it thinks there may be other therapeutic contexts, for example with autoimmune diseases, in which a similar approach to that suggested in this draft guidance could be used.

Meanwhile, though BIO welcomed the draft, the group also sought more clarity in the statistical sections of the guidance.

“For example, information critical for controlling for type I error in Master Protocols appears to be missing from this draft guidance. The full potential of a confirmatory master protocol trial can only be realized with consensus on type I error control,” BIO said.

BIO also said FDA’s thinking and expectations for master protocols used in early stage trials versus late stage trials are not fully clear. “It would be helpful if FDA would provide further explanation for what should be considered for an early stage trial and, similarly, for a late stage trial, and what would apply to both,” the group said.



© 2022 Regulatory Affairs Professionals Society.