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If you were dying, surely you would grasp at any chance medicine might offer you. Recently, there has been a lot of effort at the state and federal levels to try to increase patient access to experimental drugs. The approach has been to cut the FDA out of what is known as “compassionate use”; i.e., access to investigational medicines outside of clinical trials. This approach is misguided and will fail. To help patients, you must understand what is standing in the way of them accessing the investigational drugs they wish to try.

We have reviewed the cases of over a hundred patients with incurable cancer, each of whom wished to try an investigational drug that might prolong their life or relieve some of their symptoms. These patients had been unlucky: their case histories showed them having tried drug after drug after drug for their disease. Some were still relatively healthy, while others were hospitalized due to severe pain or failing kidneys, but none of them were able to participate in clinical trials. They were just too sick.

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Forbes

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