Probably like many on here I’ve had a few sleepless nights....

Probably like many on here I’ve had a few sleepless nights.

@Celestial I can totally relate to what you are going through.

Given the recent announcement I have been evaluating and giving thought not only of the financial implications but the implications and impact on thepoor kids affected by this horrendous illness.

My cousin sent me the following video the other day (while very funny), it struck me that this epitomizesthe innocence of children and the blind faith they have in adults.

As adults (most of us) are genetically programmed to look after children to the absolute best of our ability.

This brings me to the CRL:

With the FDA basically saying:

• They want to see more scientific proof that the treatment works (by doing a further trial on children or adults).
• There is buckley’s chance that anyone will agree to do a placebo orientated trial on kids, so it must be done with adults.
• Thus an additional trial, MSB clearly stated in the ODAC presentation they were preparing for this exact trial.
• Secondlythe FDA stated they wanted to be able to determine the potency of Rem-l (Ryoncil).Now this may well eventuate from the current FDA/NIH sponsored gold standarddouble blind trial (for ARDS) currently going on (same product).

I recognise that I am repeatingwhat others have far more eloquently put, but this brings me to the point.

• There isnot one suggestion as to the safety of the treatment!

• Nothingreferring to side effects!

Looking at it crudely we are talking about a commercial issue:

i.e. are the people (insurers, health depts., or individuals) getting what they pay for or are they getting the equivalent of expensive saline solution?

Certainly, the ODAC committeethought so!

With 90% voting to approve the product (BTW this is not just bunchof GP's pulled out of a medical centre....this is a panel of experts selected by the FDA themselves!)

Secondly, the expertsin the field of AGVHD certainly feel (Dr Kurtzberg, and others) Ryocil is doing the job!


Finally, thisbrings me to a point (perhaps naively) if this is a purely commercial issue howabout MSB at the 30 day Type A meeting offer the FDA a commercial solution alongthe lines of:

MSB to FDA: lookall your experts and ours agree that the treatment works, we have alreadystated we are commencing a trail on adults (to meet your stated objectives), ifit is value for money you are questioning , give us approval and MSB will bill onlyi.e. 50% of the normal rate until it is proven one way or another (ifsuccessful you pay the full amount if not we refund you)!

Put the money into somesort of escrow account!

This at least enablesthe sick kids (who are relying on us as adults to protect them) some options!

Let’s face it,if it turns out (which I doubt) that Ryoncil is no better that saline solution,then then children are no worse off (the children in a position to be given this treatment have an 80%chance of dying anyway).

If however, it turns out that the results arespectacular (and children’s lives are saved- which I truly believe), then everyoneis a winner.

Lives saved, and usergets what they paid for!

I know this suggestionis purely commercial, but it potentially saves children’s lives while we adultswork on dotting the “I’s” and crossing the “t’s”!


Finally, I know that(on paper) I with many others have lost a fortune, I would however, like to spare a thought for those whohave lost substantially more, who were drooling in anticipation of a CGTwindfall!


I’m referring of courseto the poor ATO (IRS in the USA)