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Forbes: Faster FDA Approvals Have Not Caused More Drug Safety Problems

August 12, 2014
In 1992, Congress, industry, and the FDA worked together to create the Prescription Drug User Fee Act (PDUFA). This program ensures that FDA has the ability to hire additional reviewers to expedite the drug review process by having industry pay “user fees.”

Recently, attention has been given to a flawed study in the August 2014 issue of Health Affairs, which has some critics claiming that PDUFA fees are biasing FDA review decisions. The study looked at drugs approved between 1975 and 2009, and found that drugs approved after PDUFA’s passage were more likely to be withdrawn or given a new black-box warning than drugs approved before its passage.

The study is problematic for a number of reasons. On Friday, John Graham at Forbes examined several of them:

First, the authors mixed black-box warnings in with withdrawals. The reason for this is that so few drugs are withdrawn that there are no useful conclusions to be made by examining withdrawals alone. As the authors note:

“This combined measure has a key advantage over the use of safety-related withdrawals alone, since withdrawals are rare (less than one per year) and hence analyses of this outcome alone have low statistical power.”

However, black-box warnings are a different outcome than withdrawals. While withdrawal indicates (to some) that a drug should never have been allowed on the market, a black-box warning indicates exactly the opposite. It is the FDA’s most strict labeling requirement, telling prescribers that the drug has severe side effects. New information has been discovered that increases the likelihood of a physician prescribing the medicine appropriately. This new information would not have been learned if the drugs had not been on the market.

Second, reporting the statistical results as percentages, instead of the raw figures, disguises an important fact: There were actually more black-box warnings and withdrawals in the pre-PDUFA 16-year sample than the post-PDUFA 16-year sample: 80 versus 76. (I had to go through the appendix with a pencil and paper to figure this out.)

So, patients in the post-PDUFA environment need not fear that more dangerous drugs are getting through the FDA. The opposite is true. While PDUFA improved the speed of FDA approvals after it was passed, normal bureaucratic inertia took over and the FDA returned to its previous, sluggish performance.

Read the full piece here.

PDUFA has been a tremendous success. With revenue generated by user fees, the FDA has been able to expand and modernize its post-market surveillance tools and capacity. FDA's standards for demonstrating the safety and effectiveness of new drugs and biologics are the most rigorous in the world, and the PDUFA program plays an important role in ensuring that patients have access to groundbreaking new therapies to treat unmet medical needs.