Peer review journals are a powerful tool to efficiently filter the gold from the dirt... there is so much data generated and noise in the medical community that the key decision makers and industry leaders need a credible, high quality 'feed' of material that has been critiqued by respected peers within their own field, thus ensures integrity of analysis performed and conclusions formed.
Put simply, if you are time poor but need to be aware of any important advances in your field .. you want to be able to get that information efficiently from a source you can trust.
The NEJM is a weekly medical journal that is one of the most prestigious peer review journals and will be followed by anyone that matters in the field of medicine. If something important happens, it will be published in the NEJM.
It is worth noting that not all trial results are peer reviewed and published in a peer review journal... fewer still get a slot in the NEJM. You need to have some pretty compelling data to share if you publish your trial in the NEJM.
It is known as best practice to get trial results peer reviewed and published, and I believe there is good reason why Mesoblast and Dr Perin decided to do this before meeting with the FDA. It is best practice for a reason, it raises awareness of the results and that generates interest and industry momentum and buy-in, which is very important when you plan to get a treatment approved through the FDA.
As we saw through the Ryoncil FDA approval process, the industry plays a role in the approval process... not only behind the scenes, but they can represent your company, or even sit on the advisory committee panel. Having a peer review journal effectively puts you through a more in-depth advisory committee before you even start talking to the FDA with your trial results.
It is very important in this case because as many have said before... we were drilling for silver, and we hit gold. Stem cell technology is such a new industry that there is very little credible data out there... so any credible data generated such as large-scale well controlled and conducted randomised controlled studies is going to get the attention of the medical industry. However not everyone in the industry checks ASX or Nasdaq announcements, or has the time to attend of watch online seminars ... but they will find the time to read the NEJM.
For many the NEJM publication will be the first time they have read about the results, and when they see a RCT data showing a 60% reduction in non-fatal MACE events (due to heart attack or stroke) with 537 patients across Class II and III with a p value of 0.002 (0.05 or lower is considered clinically significant),.. 60% reduction in cardiac death in class II patients (p=0.037)... they are not going to care if it was a primary end-point, secondary end-point or post hoc analysis... they are going to need to wipe their laptop screen after they spit their morning coffee out.
The peer review journal will dive a lot deeper in the data and probably focus a lot more on the benefit of treating patients early (much more effective on class II patients), as there was strong evidence of significantly slowing disease progression from Class II to Class III (p=0.004) over a mean of 20 months disease stability. It will look at many facets that all interact with each other, but ultimately point back to the proposed mechanism of action. To achieve the results MSB have achieved in the phase 3 trial... Mesoblast need only convince the FDA that they (and their peers) have a reasonable idea of how their treatment/cells are achieving the incredible outcomes.
This is why is why I believe Mesoblast have not rushed with the FDA meeting for the CHF results.. they want the NEJM to be published, and quite possibly submitted the Type C request just before, or will submit the request shortly after the publication. Allowing it sufficient time to stew and filter through the medical community before meeting with the FDA.
Mesoblast are going about this the right way, and we have seen it play out with the COVID trial results as well... the peer reviewed COPD medical journal (
Respiratory Research MSC COPD medical journal ). The focus was on the mechanism of action, in the COPD case the focus was on the MSC's effectiveness when compared to high or low CRP levels (measure of inflammation). The COPD journal was published in late May, and the COVID ARDS FDA meeting is scheduled for mid July (I believe it is some time this week). And again with the CLBP results where the results published in the spine journal (
spine journal MSC CLBP) .. and that meeting with the FDA should take place in August.
Goodluck all
(20min delay)