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 The laws, enacted in at least 24 states, have been promoted as a way
to help dying patients get hold of medicines and devices that
haven’t been approved by the U.S. Food and Drug Administration
(FDA).
But the laws haven’t yet helped patients gain access to experimental
therapies, Alison Bateman-House, an ethicist at New York University
Langone Medical Center, and colleagues argue in an article in Annals
of Internal Medicine.
The state laws may also cover access only for terminally ill
patients, excluding people with serious or rare diseases with
limited treatment options that are not immediately life-threatening,
the authors note.
And the laws may disrupt the current system for granting so-called
“compassionate use” of unproven treatments to dying or seriously ill
patients, which includes FDA oversight to track any safety issues,
Bateman-House said by email.
“The FDA’s participation in this process helps protect patients from
what may be an even worse outcome than what they are facing,”
Bateman-House said. “By seeking to remove the FDA from approving
compassionate use attempts, right to try laws remove an essential
safeguard.”
Under current federal policy, when terminally ill patients want to
use drugs and devices that are too early in their development to be
approved by the FDA, their doctors typically ask for the drug or
device companies to grant expanded access to the experimental
therapies.
If the company agrees, the FDA and a review board where the
physician works would also vet the request. Among other things,
reviewers might assess whether the patient has exhausted other
options and understands the potential risks and benefits of
therapies that generally haven’t yet been tested in large-scale
human trials.
The FDA has not taken a position on right to try legislation, Sarah
Peddicord, an FDA spokeswoman, said by email. The FDA approved more
than 99 percent of compassionate use applications, also known as
expanded access requests, received during the 2010 to 2014 fiscal
years, she said. The FDA is required to respond to these requests
within 30 days, but often does so much more quickly, she added.
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States have approved right to try laws, however, because the current
system doesn’t move quickly enough for terminally ill patients who
seek experimental treatments as a last resort, said Kurt Altman,
director of national affairs at the Goldwater Institute, which has
drafted model legislation and helped promote the laws.
He doesn’t dispute the FDA’s 99 percent approval rate for
compassionate use requests. Instead, Altman argues that these
requests represent only a fraction of the patients who might have
benefited from getting experimental treatments.
“What the 99 percent figure does not tell us is how many terminal
patients didn’t know about expanded access, had a doctor with
insufficient time to complete the process, etc.,” Altman said by
email.
The application can take doctors more than 100 hours to complete, he
said. Though the FDA announced plans to streamline this process so
it takes only 45 minutes, Altman said he isn’t aware of this being
implemented yet.
“Overall, I think right to try is a first step that will lead to
reforms that not only speed some FDA processes but also make their
processes more transparent and understandable,” Altman said.
SOURCE: http://bit.ly/1jrpS5Z Annals of Internal Medicine, online
September 28, 2015.
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