Michelle's Monday Minute: May 1, 2023

How are you? I texted my daughters who are away at university.?

Fine, it’s cold … oh … and I got projectile pooped-on by a cow.

How are you MMM readers? Hopefully not having to do an unexpected load of laundry during the middle of your day!?

Now, shifting gears over to non-bovine related topics –

EU Pharma Legislation Published

Last week’s publication of the proposed EU Pharma Legislation has major positive and negative implications. Importantly, there’s still ~3 years more of process before formal adoption and likely many amendments between now and then. There’s plenty of press on this, as well as EU Commission’s own Q&A. From my side, I’m pleased to see the proposed reduced review and adoption timelines, reduction in EMA committees and the regulatory sandbox set-up for innovative treatments. I advise bookmarking EFPIA’s Statements and Press Release webpage to stay abreast of developments as the process proceeds.??

Blinded Independent Central Review (BICR) for Oncology Trials

Roche scientists have published in The Oncologist March 2023 issue meta-analyses of 49 Roche oncology trials (both solid tumor and hematologic) completed between 2006 and 2020. The analyses showed BICR did not significantly impact the study interpretation nor drive regulatory submission decisions. BICR can be costly, resource intensive and time-consuming and possibly not needed to control bias in many settings. Indeed, these meta-analyses confirm local evaluation can be sufficient provided bias can be diminished by appropriate means which may be helpful to integrate into your study design decisions.

EMA’s Single Arm Study Reflection Paper

Recently, EMA published a reflection paper on important considerations for the use of single arm trials for the purposes of establishing efficacy for market authorizations. The paper covers a comprehensive set of parameters (endpoints, population, bias, statistical principles, external evidence) to consider when designing a single arm trial intended to constitute substantial evidence in regulatory decision making for approval.?

On vacation last week, I spent a lot of time in the garden pulling out maple tree seedlings. Somehow the weather conditions have created perfect soil conditions for maple tree seeds to bloom. While bending over and pulling little seedling after little seedling, I enjoyed listening to this Remarkable People podcast on linguistics. If you are fascinated by words or are curious to learn about language evolution, you will enjoy this conversation with Professor Valerie Fridland. As a teaser - did you know the use of “ah” and “um” in real conversation is signaling that we are thinking carefully? Or that the word “like” has 3 different purposes?

Happy May Day everyone and remember – stand back when near cows!

Michelle