Getting expanded access to unproven therapies for rare diseases is more difficult than for more common illnesses, such as cancer, experts say.
, for the most common type of lung cancer, were similarly offered to large numbers of patients even as clinical trials were ongoing.Doctors also report that getting experimental drugs for cancer patients is relatively simple. More than 200 physicians around the country were surveyed, and among those who applied for access,
Companies base their decisions on whether to provide a therapy through expanded access on a number of factors, said Jess Rabourn, CEO of WideTrial, which helps pharmaceutical companies run compassionate use programs. In general, there should be evidence that patients can tolerate the treatment and an expectation that any benefit outweighs the risk, he said.
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