Missouri bill would ease access to unapproved drugs

Personal experience fuels legislator's mission

KANSAS CITY, Mo. - A bill expected to pass easily out of a house committee in Jefferson City on Thursday would ease access to potentially lifesaving, albeit unapproved, drugs for terminally ill patients desperate to try anything that might help them after other treatments have failed.

Republican Jim Neely, a former physician who represents Missouri’s 8th district (just north of Kansas City), proposed the bill in the house in February and testified in an emotional house committee meeting about its merits just last week. It would allow patients and doctors to bypass the FDA and go straight to drug companies to receive unapproved treatments when other measures have failed.

“If a person is in a situation where the current therapy out there is not working, why not try some of these investigational medications that have already passed phase one,” Neely said.

Investigational medicines include drugs, biologics and implantable medical devices. Phase one refers to the first phase of FDA approval in which a drug has been proven to be safe for human consumption, but not thoroughly tested for overall efficacy, appropriate doses or possible side effects – a process that could take years.

An FDA spokesperson said the agency does not comment on legislation. The agency does allow some so-called “compassionate use” or expanded access to investigational drugs, but Neely said that process can be incredibly burdensome for doctors and patients and still requires FDA approval and oversight.

Neely’s bill follows a similar bill proposed in Arizona last month which was authored by the libertarian Goldwater Institute, which also helped on the Missouri bill. Legislatures in Louisiana and Colorado are considering their own versions of the bill and a spokesperson for the Goldwater Institute said the group has received interest from lawmakers in California and Utah about helping develop bills for those states as well.

Neely’s interest in this legislation is deeply personal. He said his daughter Christina was denied access to a clinical trial of a drug to treat her liver cancer because she was pregnant. Now, her cancer is stage four and metastatic

“She’s keeping up a good fight,” Neely said.

As a physician, Neely also treated AIDS patients in the 1980s and said he struggled with the hopelessness of knowing there were drugs in development, but nothing available to help his dying patients.

“You were scrambling to find a way to help and you saw that there was none, and you just kept moving forward,” Neely said.

The plight of those patients struggling to get access to drugs in development has drawn new attention of late. Actors Matthew McConaughey and Jared Leto won Academy Awards last Sunday for their portrayals of AIDS patients in Texas who faced off against the FDA over unapproved medicines.

Neely said Wednesday he hadn’t seen the movie yet, but was grateful for any additional attention to his cause.

“When you're in the legislature, you sometimes wonder if you can make a difference,” Neely said. “This bill would make a difference in people's lives.”

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