More Research Needed
"The outcome for many children with cancer is much more favorable than [it used to be], so 80% of children with cancer are long-term survivors, but [cancer] remains the lead cause of death from diseases in this country; the price many of our children pay for survivorship is quite high. It's a relatively small population that we're dealing with when we're talking about relapsed or cancer that's refractory to treatment," Dr Adamson said.
Many of these children participate in clinical trials, but there are times when that is not an option; there may not be an appropriate clinical trial, or the child may not be eligible. For these children, expanded access may be their best hope for survival.
"One of our challenges is we don't have as many clinical trials as we're able to do and support, because the economics are not there for industry; for very ultra-rare diseases, it's hard to make an economic model to develop a cancer drug," he said.
"Our biggest challenge as a field, is we want earlier access to do the research…there is an eagerness to not only provide new drugs for children with cancer, but to learn from that experience as far as whether the drugs are effective or not."
Dr Caplan and Dr Adamson have disclosed no relevant financial relationships.
More Research Needed"The outcome for many children with cancer is much more favorable than [it used to be], so 80% of children with cancer are long-term survivors, but [cancer] remains the lead cause of death from diseases in this country; the price many of our children pay for survivorship is quite high. It's a relatively small population that we're dealing with when we're talking about relapsed or cancer that's refractory to treatment," Dr Adamson said.Many of these children participate in clinical trials, but there are times when that is not an option; there may not be an appropriate clinical trial, or the child may not be eligible. For these children, expanded access may be their best hope for survival."One of our challenges is we don't have as many clinical trials as we're able to do and support, because the economics are not there for industry; for very ultra-rare diseases, it's hard to make an economic model to develop a cancer drug," he said."Our biggest challenge as a field, is we want earlier access to do the research…there is an eagerness to not only provide new drugs for children with cancer, but to learn from that experience as far as whether the drugs are effective or not."Dr Caplan and Dr Adamson have disclosed no relevant financial relationships.
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