"I'd be interested in finding out what the KOLs say about it on the investment day, what they're seeing in the real world. But, I bet they'll be singing the praises of Andexxa."
I think they are going to be careful enough about who they invite to speak that a negative review from an invited speaker is not very likely. A few things I want to hear about regarding real world use.
1. Are practitioners using a diagnostic blood test to guide necessity for A use and determine high vs low dose usage. If they are, how long from admittance to treatment?
2. When A was first rolled out there was a lot of grumbling about the high cost, the lack of availability until gen 2 came on line, and the lack of a RCT for approval. Has time and experience improved the drug's reputation at large?
3. Growth depends on adding hospitals and increased used by hospitals already stocking the drug. Back of the envelope says that in the 3rd quarter, on average, hospitals each used the drug twice. Looking at the hospitals that were early adopters, how much have those with the most experience increased their use of the drug over time?
4. I'd also like a phase 4 update.
Following is the detailed description from the clinicaltrials.gov website
This is a randomized, multicenter clinical trial designed to determine the efficacy and safety of andexanet compared to usual care in patients presenting with acute intracranial hemorrhage within 12 hours of symptom onset and within 15 hours of taking an oral factor Xa inhibitor. The study will use a prospective, randomized, open label (PROBE) design. The primary efficacy outcome will be adjudicated by a blinded Endpoint Adjudication Committee. To support the adjudication of hemostatic efficacy, a blinded Imaging Core Laboratory will review all available scans. Approximately 440 patients are planned to be enrolled in the study.
So how does this work? I assume there is a treatment arm (Andexanet) and a control arm (usual care). Is the randomization 1:1? Is there a planned interim? A lot rides on the outcome. A positive result pretty much guarantees universal adoption for ICH treatment and a negative or marginal result could crush sales and even threaten the approval. I still own the stock, so obviously I expect a positive outcome, but I still worry about it.