State legislatures jump into the thorny compassionate-use debate

Currently, just about everyone would agree that the program in place for the compassionate use of experimental drugs is deeply flawed. The FDA typically approves all the requests that come through and the companies are left to decide whether they can afford to hand over a supply--if they even have a program in place. In some high-profile cases, like the recent example at Chimerix, a dying patient becomes a cause célèbre as public forces pressure developers to buckle after initially rejecting their request.

Now, some states are getting into the act. Colorado recently passed a "right to try" bill that allows people to use an experimental drug when the company agrees to give it, without federal approval, according to a report from NBC News. And now other states are beginning to discuss similar legislation, as The Goldwater Institute presses for such laws from coast to coast.

In Colorado, the bill was passed unanimously and signed into law over the weekend. But skeptics say the bill has no teeth, providing dying patients none of the leverage they're looking for in obtaining drugs.

The FDA, meanwhile, is not happy to see states treading on federal authority in these matters. And the trade association PhRMA isn't very pleased either.

PhRMA VP of Scientific and Reg. Affairs Sascha Haverfield

"Successful completion of the clinical trial process is necessary to demonstrate that an investigational medicine is safe and effective, which is required to obtain FDA approval, so that companies may make the medicine available to a broader patient population when clinically appropriate," Sascha Haverfield, vice president of scientific and regulatory affairs at PhRMA, told the Wall Street Journal.

In the end, these new state laws may make no one happy. Or, in other words, nothing much changes. -- John Carroll (email | Twitter)

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