Health Affairs November 1, 2019
Five years ago, we wrote about a seminal event in “compassionate use”: the use of social media by the family of a critically ill child to gain preapproval access to an experimental medicine. This was not the first time that social media was used for this purpose, nor was it the last. But it was one of the key coalescing events that has led to increased societal focus on providing access to experimental medicines—treatments that are still “unproven” for their target disease condition and unlicensed by a regulatory agency such as the Food and Drug Administration (FDA).
Since that event, there have been meaningful changes regarding preapproval access across many if not all segments of the US health care...
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