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02/03/2017

A Solution In Search of A Problem: Streamlining the FDA

by Craig Klugman, Ph.D.

A professional association for regulatory affairs posted an article on Wednesday reporting Trump’s comments “calling for a massive overhaul of US Food and Drug Administration (FDA) regulations.” Trump issued an executive that called for reducing the number of federal regulations (for each new one created, two must be retired). And he met with the heads of pharmaceutical companies, where he said that he plans to cut taxes, FDA regulations and approval times. He also criticized the companies for selling drugs that were too expensive. The two events combined suggest that Trump will try to reduce the FDA and its power.

What does the FDA do anyway? Aren’t they just an obstacle to innovation and getting drugs to the marketplace?

In 1906, Congress passed the Pure Food and Drugs Act to provide oversight, regulation, and consumer protection to a cowboy market where anybody could sell anything. Tonics and “cures” were regularly sold with no indication of their ingredients and no proof that they worked. The drug marketplace was ruled by “caveat emptor.” The actual organization that we know as the FDA grew out of a chemistry lab in the Department of Agriculture in 1862, becoming the Bureau of Chemistry in 1901, the Food Drug and Insecticide Administration in 1927, and the FDA in 1930. Today, “the Food and Drug Administration is responsible for protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices; and by ensuring the safety of our nation’s food supply, cosmetics, and products that emit radiation.”

Although the FDA has responsibility for safe food and cosmetics, my focus here is on pharmaceuticals. The FDA makes sure that new drugs are safe, effective, and that advertisements are supported by empirical studies. Remember thalidomide a current cancer drug that was marketed in the 1950s and 1960s for morning sickness? As it turned out, the drug has a host of effects that damaged the development of the fetus. Children born of mothers who took the drug often had lifelong health issues including anatomical deformities and undeveloped limbs. In Germany alone, over 10,000 children were victims of this drug. In the UK, over 2000. Estimates are that worldwide, 24,000 babies were injured. Although it was widely tested in the US, the FDA never approved it, believing that the drug had not been adequately tested. Thus the US only saw 20 thalidomide babies born. That was the FDA at work. Today Thalidomide is used as a cancer treatment, but before a woman can begin using it, she must have two negative pregnancy tests and take action to prevent becoming pregnant.

Who has problems with the FDA and its speed?

Allegedly, advisors close to the Trump administration have been arguing that the FDA is an obstacle to drug innovation. They believe that the many regulations mean that drugs take years to get approved and that such drugs could more quickly reach market and help people. Certainly the flurry of calls for expanded compassionate use follow this line of thinking.

About 10% of drugs that begin a Phase I trial are ever approved for patient use. The 90% that fall out do so because they have a high risk-to-benefit ration, unacceptable side effects, or cannot prove efficacy.

Some of those who have worked at the FDA do not believe that the approval process could get any faster and still offer protections. Consumer groups have complained that both the FDA and European drug agency are approving some drugs too quickly, perhaps influenced by the $7 billion in fees companies paid to get their drugs “fast tracked.”

Though the pharmaceutical industry may complain about the FDA and its rigorous testing requirements, they are not calling for a change. In fact, the FDA keeps everybody honest and playing by the same rules, thus making sure that competition is somewhat fair in the market. And over the last several decades the FDA has responded to concerns about long time lines for drug development by speeding up the approval process. The recently passed 21st Century Cures Act under President Obama slated money to help the FDA speed up even more.

Certainly there are problems with the FDA. Relying on companies to run clinical trials on their own products creates an incentive for manipulating data and not reporting outcomes that do not mesh with the company’s interests. An independent testing body would be a better, more objective system. Pharmaceutical start up companies are priced out of the current system since they cannot possibly afford the millions of dollars that testing requires: Independent testing would help alleviate this barrier to entry. And of course, the conflicts of interest between the FDA and industry (with industry sitting on many of the FDA approval committees) are legendary.

What alternatives are being proposed?

Several proposals are being floated to replace the FDA or a new system to bring drugs to market. All three proposals could be adopted since they complement one another. But with one exception, none of them rely on empirical evidence or clinical testing.

The first is to harness the power of the internet and consumer reviews. Using a Yelp-like model, patients could post their experiences with various drugs and then other patients can decide what drugs to take based on the reviews. There would not be efficacy testing. There might not even be safety testing. But every single person who used any particular drug would be offering his or her own reviews. This would be the end of the clinical trial and drugs would only be reviewed under case studies. The problem, of course, is that case studies are not usually generalizable to a large audience.

A second proposal is to let the market decide. All drugs that a company wants to release would be put onto the market. Then doctors and patients would decide what is best for them. Considering there be no formal, large-scale studies, it is difficult to know what data to help make those choices.

The third proposal is to bring all drugs to market once they pass a Phase I trial, meaning the drugs would be relatively safe but would not be tested for efficacy.

But insurance only pays for approved drugs, not experimental ones

Insurance currently, when it covers drugs, only covers FDA approved drugs. Experimental and unproven drugs are not covered. So what happens when no drug is proven to work? Would insurance start covering everything or nothing? The decision would be up to insurance companies or any health laws passed by Congress.

This can’t really happen, can it?

Putting aside the issue of drug safety, a reduced FDA would mean that food safety would not be assured. Time to build a small laboratory in your kitchen so you can check all of the food entering your home for safety. Of course this is ridiculous, there are simply some things that we have to depend on others for—like knowing what makes for safe food and whether a drug is efficacious and safe. No internet bulletin board or individual playing with drugs and doses can do this. A reduction in the scope or powers of the FDA puts the lives of every single person in the U.S. at risk.

The FDA needs some reform to help alleviate the conflicts of interest and barriers to entry for new companies. But throwing out the baby with the bathwater is not the solution.

One way that the administration could change the FDA is to issue executive order or to have Congress introduce some new bills. Another way is to simply starve it—reduce funding or personnel to the point where the agency cannot fulfill its functions. A combination of approaches might be in the pipeline as streamlining efforts are already underway. An executive order froze hiring at the agency, leaving nearly 1,000 positions unfilled.

This entry was posted in Clinical Trials & Studies, Featured Posts, Pharmaceuticals, Politics and tagged . Posted by Craig Klugman. Bookmark the permalink.

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