[schoonder]

Yes, this ruling can be appealed.
FDA, our wonderful beaurocratic agency chose to literally interpret rules and regulations dealing with new drug acceptance process.
They opted to issue a letter of Refusal to File eventhough guidelines in execution of RTF gave the agency freedom to forgo this decision if and here I insert an excerpt from this regulation:

"The agency may, for particularly critical drugs, not use the RTF procedure, even where it could be invoked or might review parts of a refused application if it believes that initiating the full review at the earliest possible time will better advance the public health"

This exclusion to go the RTF route clearly shows the "spirit" in which this regulation was written. It basicly informs FDA leaders that circumstances may arise that are so critical to well being of its citizenry, to provide requestor with sufficient leeway to see a particular drug become available to public at earliest possible time. Yes, that was the only reason to insert excerpt's paragraph, to better advance the public's health, no more, no less, but FDA chose to ignore criticallity of their act in keeping this drug from extremely ill patients for at least two years.

Would it be surprising if Roche, in view of FDA's decision that T-DM1 is not a particular critical drug, discontinues their recently initiated compassionate use trial? I don't really have an answer to that.