How Effective Are FDA Approvals?

How Effective Are FDA Approvals?

February 11th, 2014 // 6:55 pm @

FDA is effectively the gatekeeper in the US for access to many types of pharmaceuticals. That is why it gets so much attention from the media, as well as from Congress. Some will say that FDA is too lenient in making drug approvals, and that it subjects people to unproven drugs. But others say FDA is too conservative and FDA is delaying critical access to new drugs.

So, researchers at Yale last month published a study that attempted to bring more transparency to drug approval decisions by FDA. The name of the study is Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 20-05-12.

According to the authors of the study, many doctors and patients assume that a newly approved drug is safe and effective. However, the strength of the evidence used by FDA from clinical trials in making such calls has not been truly tested.

The researchers used information from FDA’s website to obtain data from 188 drugs that were approved by FDA over the 7 year period. The results of the study showed that not all of the approvals by FDA had used the same type and amount of evidence to make their drug approval decisions.

The lead author if the study found that about 1/3 of the approvals were based upon results from a single clinical trial without replication. And some of the other trials were short, small and focused just on lab values or another less valuable effect metric.There also was not a uniformity in the evidence that FDA used. Based upon the study, the authors noted that they could not be certain that FDA approval means that safety and efficacy of the drug has been proven.

Of course, this information caused some parties to criticize FDA harshly. But for people who know how the drug approval process works, this information is not a surprise. FDA has for years had a very flexible approach to approving all kinds of new drugs. The reason for this is that different situations mandate different standards. If a new drug is being created that attempts to treat heart disease, then FDA rules require the drug to be studies with two major clinical trials with 10000+ patients showing safety and efficacy, PLUS they need to have shown improvement in patient survival.

These types of studies can take over five years, and only really big pharma companies have the money to do them.

But how about a drug that is being developed to treat a fairly rare disease, and there is no current therapy for it? Clinical studies for this kind of drug can’t be like those done for a chronic disease drug. And there are no current drugs to compare to, and there are not that many patients to use for a trial. And these patients are demanding a new treatment. So it seems that FDA is probably right to approve an experimental drug based upon one clinical trial with small numbers of patients.


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