Commercial opportunity implies peak sales of $500m or better, page-3

A very interesting question @ViperX1. I'm not an expert on the letter of the FDA law but can try and fumble around and draw conclusions from what we have seen in other clinical areas. So with that in mind the following are my best guesses and may ver well prove to be wrong:

1) The drug label will reflect the fact that VF-001 was proven to have efficacy in moderate VLU patients only.
2) Reimbursement will likely be restricted for easy to treat ulcers because the health economics is not compelling.
3) Reimbursement will be automatically approved for moderate ulcers because of evidence of efficacy and compelling health economics.
4) Reimbursement will be available on application (i.e. not automatic but possible) for difficult to treat ulcers because the health economics is compelling even if efficacy is lower than demonstrated in moderate patients.

I think the most controversial guess is #4. The reason why I believe reimbursement will be ultimately available to this group is because the costs to the healthcare system for these patients is many multiples of moderate and easy ulcers. We have plenty of precedents of approved therapies that don't have evidence of efficiency in hard to treat diseases being approved by payers because i) the cost of the therapy is so small relative to the life time costs of the disease; and ii) patients and doctors are often fairly desperate and able to present a compelling case for reimbursement to the payer. Sirtex is a prime example of a therapy that has done very well as a last-line therapy without clinical trial data supporting its application for this specific patient population.

That said if VF-001 is successful in Margolis 1 patients I fully expect them to run a subsequent trial that would either be an "all-comers trial" or at least a trial that includes difficult to treat patients to eliminate any friction in reimbursement.

But these are just guesses so likely to be wrong in some aspects. To their credit the company is collecting a full suite of health economics data as part of the trial which will be used to show the benefit to payers.