Nothing wrong with looking at ranges, of course. Were I responsible for ensuring adequate funding of the company however, I would err on the side of caution, and plan with a conservative mindset.
As for the likely penetration rates, I'm not sure how the Japanese government would "get behind this measure" to promote uptake. With the exception of vaccines, I'm unaware of any government promoted medicine. My suggestions may well be conservative, but particularly in the absence of any efficacy data with which to develop more bullish forecasts, they're not unreasonable numbers.
You say that the product has been successful so far. Has it?
Leaving aside the issue of whether the MSB cells have been "successful" - which for the sake of discussion, we'll say means clinically effective to a meaningful degree - which you and I have differed on, and would no doubt continue to differ on, the product under consideration in Japan is the Osiris product, correct?
If that is the case, then, no, to date, it has not been successful. The Phase 3 programme failed to achieve its primary end points, and the paediatric indication was only developed courtesy of post-hoc data-mining.
Maybe the Japanese application is only for a paediatric indication, in which case, the chances of success may be higher. However, then factor down the sales substantially, for the lower number of target patients.
So in summary, if it's the Osiris product that's under study, then my points are highly relevant to the MSB study. As I said, I'm sure MSB will have considered the trial design, but if the product is inherently flawed, that may not help. Looking at the MSB website, all references to GVHD seem to be to the Osiris product - Prochymal.
People with refractory GVHD die quickly. It's an acute condition, and if they don't respond adequately to first line therapy, the prognosis is very poor.
If they respond well to the MSB/Osiris product - fantastic, though my understanding of these patients - particularly older ones - is that they are likely to require long term therapy anyway of other drugs - anti-inflammatories, immunosuppressants and specific symptom relief.
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Speculation on Japanese revenue stream, page-8
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