What do you do when dangerous drugs can’t be sold legally? Change the rules.

We may get to see this –  nationally.

The FDA gets lots of brickbats, and has a tough job.  Much of its funding comes from the drug companies it’s supposed to regulate.  The process of drug approval is arduous, highly bureaucratic, and generally ends in failure.  If you’re a drug company that wants to get its drugs approved, those obstacles can knock you out of business.

Things have changed in recent years.  Most drugs now go through “expedited” FDA approval. It’s still very expensive, but faster.   But thanks to recent political changes and the application of Citizen’s United, FDA drug approval may soon change radically.  Here are some of the options:

The Free for All Model

Libertarians, and a few makers of over-the-counter drugs,  feel that government often unfairly limits individual choice.  The argument goes like this: if we can show you our drugs are “safe,” shouldn’t you be able to choose what drugs you want to take?  Why can’t prescription drugs be any other product, like soap or tires?

Because people’s lives are at stake.

Safety has many meanings.  Many drugs, like vioxx, pass through rigorous hurdles to FDA approval, only to be found to be dangerous following years of treatment (or if the drug companies deliberately suppress data, which they did far more easily in the past when they could hide “negative” clinical trials.)  Thousands of people can die using even rigorously tested “safe” drugs.

And efficacy really is always an issue. If you take something, given all the potential side effects, you want it to help you.

The University of Essex has been studying the many different non-prescription medications available to the public that have not been considered by government agencies.  They look at drugs they think will work, and put them through clinical trials.  So far, the results are that 95% of these substances are either harmful or no better than placebo.

So when checking out those new supplements at the drug store, there’s perhaps a  one in 20 chance that they will do something useful.  Better than lottery odds, but strangely they’re the same odds, one in 20, used to determine whether “clinical significance” is achieved in statistically accepted clinical trials.  And the University of Essex studies are not looking longterm, where many of the negative side effects of drugs and devices fall out.  It took well over a decade before lithotripsy treatment of gallstones was found to cause of diabetes.

So the Free for All model fits certain ideologies.  It just doesn’t do the public much good.

Nor does it aid the established pharmaceutical industry.   The public does not realize that many medications that are passed by the FDA are better than placebo – but not necessarily by much.  Something that is 43% effective when a placebo is 40% effective may not be a great deal.  But it fits legal definitions of efficacy.

And people trust the FDA.  They know it’s hard to get FDA approval.  That approval is worth a lot of money.

The “Soft Endpoints” Model

One of the potential candidates for soon to be announced FDA commissioner believes that “hard endpoints,” stuff like preventing heart attack and stroke, are indeed too hard to obtain.  He wants drugs approved that change known “risk factors” like cholesterol.

Be careful what you wish for.

One of the most anticipated drugs of the last ten years were the HDL modifiers.  These drugs would increase the “good” cholesterol, or HDL.  Many of them also concomitantly decreased the “bad” cholesterol, LDL.  The combination looked like a Big Pharma “slam dunk.”

But when drugs like torcetrapib were finally tested, the results were not as expected.  Yes, HDLs went up.  Yes, LDLs went down.  But the overall result was a marked increase in cardiac deaths – 58% in the case of torceptarib.  Billions of dollars in research and development costs from multiple companies went down the drain.

But people were not harmed.

For those who looked carefully, cardiovascular epidemiology had been warning this might be the result.  The effects of HDL were non-linear – more was not necessarily better.  At high levels of HDL, cardiovascular deaths went up.  Such drugs also have many ancillary effects that cannot be predicted, and can only be thoroughly tested with randomized clinical trials.

Using the  soft end point, “shifting the goal posts” argument, may provoke irreparable harm.

What To Do

The FDA has many problems.  It needs more support staff and inspectors if useful drugs are to get quicker  approval. It needs to try new ways of doing clinical trials, and through its funding model remains too captive of the companies whose products it tests.

Yet we have not had thalidomide disasters in the country for quite some time, because we do rigorously test most medications – though medical devices, OTC herbs and supplements represent a blatant exception.  People want to know what works, and whether the inevitable side effects and downsides are worth it.

Which is why you have the FDA of today.  We don’t want poison drugs.  We don’t want poisoned water.  We don’t want poisoned foods.

When you hear the word “regulation,” think protection.  That’s one of the things governments are supposed to do – protect the public.  And what many who tell you they will “reform” and “bust” regulations want to see is  the end of protections that might save your life.

You have to keep watching.