Have to very careful comparing response across trials and eras. The complete response rates in the 80s would be in a much better cohort of patients than more recent studies. You have to remember that a significant proportion of AML is now cured. The response rate in heavily pretreated high risk disease is likely to be substantially lower. Moreover you have neglected to mention newer, more promising therapies such as venetoclax.
Wrt a named patient program. It is very difficult to get patients to pay for new highly effective drugs, let alone drugs that are 30years old and basically chemotherapy. It will be tough work to convince physicians to prescribe this without seeing some really good responses first hand. Most special access schemes do not generate revenue and are in fact essentially marketing activities to get physicians used to drugs whilst awaiting funding approval. It is also a substantial amount of work to apply for licensing to prescribe off label drugs- Australia which has a relatively streamlined online process is substantially easier than Europe where most physicians wouldn’t even know where to start to prescribe not EMA or Nice approved therapies.
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Ann: June 2018 Quarterly Report, page-11
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