Abstract
In addition to being an example of unsubstantiated hype about regenerative medicine, the controversy around the Italy-based Stamina Foundation's unproven stem cell therapy represents another chapter in a continuing debate about how to balance patients' requests for early access to experimental medicines with requirements for demonstrating safety and effectiveness. Compassionate use of the Stamina therapy arguably should not have been permitted under Italy's laws, but public pressure was intense and judges ultimately granted access. One lesson from these events is that expert regulatory agencies may be the institutions most competent to make compassionate use decisions and that policies should include more specific criteria for authorizing compassionate use. But even where regulatory agencies make decisions based on clear rules, difficult questions will arise.
Highlights
In addition to being an example of unsubstantiated hype about regenerative medicine, the controversy around the Italybased Stamina Foundation’s unproven stem cell therapy represents another chapter in a continuing debate about how to balance patients’ requests for early access to experimental medicines with requirements for demonstrating safety and effectiveness
The Stamina controversy represents another chapter in a longstanding debate about how to balance seriously ill patients’ desire to use experimental medicines—what the European Medicines Agency (EMA) calls “compassionate use,” and the US Food and Drug Administration (FDA) calls “expanded access”—with legal requirements for demonstrating safety and effectiveness before clinicians can use these medicines (Zettler & Greely, 2014)
During the 1980s, AIDS patients mounted a highly publicized effort to gain early access to experimental antiretroviral drugs. In another well-publicized controversy in the 2000s, an advocacy group called the Abigail Alliance unsuccessfully sued the FDA for broader compassionate use after the founder’s daughter was unable to enroll in clinical trials of two unapproved medicines for cancer, including a trial of a drug that was later approved for her particular diagnosis
Summary
In addition to being an example of unsubstantiated hype about regenerative medicine, the controversy around the Italybased Stamina Foundation’s unproven stem cell therapy represents another chapter in a continuing debate about how to balance patients’ requests for early access to experimental medicines with requirements for demonstrating safety and effectiveness. The Stamina controversy represents another chapter in a longstanding debate about how to balance seriously ill patients’ desire to use experimental medicines—what the European Medicines Agency (EMA) calls “compassionate use,” and the US Food and Drug Administration (FDA) calls “expanded access”—with legal requirements for demonstrating safety and effectiveness before clinicians can use these medicines (Zettler & Greely, 2014).
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