U.S. docs know scarily little about the FDA process for new drugs

Print 15 April 2016
Ben Adams / FierceBiotech

The very people charged with prescribing medicines to patients don’t, as it turns out, know very much at all about how they are approved--and this may be leading them to over-prescribe new drugs.

This is according to a new survey and report, published this week in JAMA, which saw Aaron Kesselheim of Brigham and Women's Hospital in Boston and his colleagues ask internists and specialists to look at just how well they know the FDA’s approval standards, and what they think its “breakthrough therapy” designation means for new meds.

Of the 1,148 physicians contacted, 60% responded, and each was asked 3 questions about FDA approval and 5 about breakthrough therapies.

The results were poor: As the paper notes, “respondents showed limited knowledge of FDA approval” as a staggering 73% incorrectly believed an FDA stamp meant comparable effectiveness to other approved drugs, while 70% thought that approval required both a statistically significant and clinically important effect.

In fact, FDA approval can be based on a new drug besting just a sugar pill, but not to a comparable drug, or even the standard of care. Safety is often a large factor for the regulator when assessing new drugs.

And among the 3 “breakthrough” knowledge questions, just over half (52%) thought that strong evidence (i.e., randomized trials) is needed to earn the breakthrough designation--but this isn’t the case either.

Since 2012, the FDA has been able to designate a drug as a breakthrough therapy in order to speed up its review time and get drugs to patients more quickly.

The official word from the FDA is that a drugmaker will get a breakthrough designation if preliminary clinical evidence--such as an improvement in a pharmacodynamic biomarker--suggests an advantage over existing options. This might not prove as ground-breaking as the tag “breakthrough” might suggest.  

The new designation was created as part of the FDA Safety and Innovation Act, which re-authorized prescription drug user fees that pharma pay to FDA to support its budget--although it did not formally alter the approval standard.

The authors note that: “Although the term breakthrough leads consumers to overly optimistic beliefs about drug effectiveness, it [had not been] known how physicians understand this term, or more generally, what FDA approval means.”

But armed with the data from this new survey, the authors said that: “The misconceptions identified may lead physicians to overprescribe newly approved drugs--particularly breakthrough therapies--and inadequately communicate how well these drugs work to the patients who will use them.”

The FDA received nearly 250 applications for breakthrough therapy status in the first two years of its life, of which it granted 68--even though its sponsoring legislators intended it to apply to only a handful of drugs each year. In fact, the FDA predicted back in 2012 that just 2 - 4 drug candidates each year would be granted breakthrough drug designation. 

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