Study: Few new drugs have high therapeutic value

Regulatory NewsRegulatory News | 12 October 2020 |  By 

A new study published in The BMJ finds that only a third of new drugs approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the decade from 2007-2017 have high therapeutic value, according to appraisal by one of five independent organizations.
However, the analysis found that drugs approved through expedited programs – and especially EMA’s programs – were more likely to have high therapeutic value.
“Most of the increase in the number of new drug approvals over the past decade was driven by drugs rated as having low therapeutic value, which calls into question the common practice of using simple counts of new drug approvals as a measure of innovation,” the authors write, suggesting instead that a “more nuanced view of innovation is needed that takes into account the clinical benefits and relevance to patients.”
For the study, the researchers looked at 320 new drugs approved by FDA and 268 recommended by EMA during the study period. More than half (n = 181; 57%) of the drugs approved by FDA qualified for at least one expedited program compared to less than one-sixth (n = 39; 15%) of the drugs recommended by EMA.
To determine the value of the drugs, the researchers looked at therapeutic value ratings published by Canada’s Human Drug Advisory Panel, France’s Ministry of Health, Germany’s Federal Joint Commission, the Italian Medicines Agency and the non-profit organization Prescrire.
The researchers were able to identify therapeutic value ratings for 267 of the drugs, 84 (31%) of which were rated as having a high therapeutic value by at least one organization.
The researchers found that drugs that qualified for one or more expedited programs, including FDA’s fast track, priority review, breakthrough therapy and accelerated approval programs and EMA’s accelerated assessment and conditional marketing authorization, were more likely than those that did not qualify for any expedited programs to be highly rated.
“Few non-expedited drugs were highly rated. However, in absolute terms, most drugs in the FDA’s expedited programs were rated as having low therapeutic value—even for breakthrough designated therapies and those that qualified for priority review, which is intended for drugs that provide ‘significant improvement,’” the authors write. The authors also point out that far fewer drugs qualified for EMA’s expedited programs, and in contrast to FDA, most that qualified for accelerated assessment were highly rated.
The researchers write that these findings “suggest a widening gap between regulatory approval and the clinical and public health priorities of health systems, payers, and patients after approval.”
A greater proportion of FDA approved drugs that qualified for one or more expedited programs were found to have high therapeutic value compared to those that did not qualify for any expedited programs (45% vs. 13%). The association between high therapeutic value and expedited programs increased in relation to the number of expedited programs a drug qualified for.
“The number of qualifying expedited programs was also associated with the proportion of drugs rated as having high therapeutic value: 49% (29/59) of drugs qualifying for two and 65% (26/40) of drugs qualifying for three or more expedited programs were rated as having high therapeutic value, compared with 26% (14/54) qualifying for only one expedited program,” the authors write.
However, when looking at the different expedited programs, the authors found that priority review, fast track and breakthrough therapy designation were more strongly associated with having a high therapeutic value, while accelerated approval did not share that association.
Looking at drugs approved by EMA, the authors similarly found that drugs that qualified for an expedited assessment were more likely than not to be rated as having a high therapeutic value, while conditional marketing authorization was not associated with being more likely to be rated as having a high therapeutic value.
The authors also note that, “Drugs that were approved by the FDA but not the EMA were less likely than drugs approved by both regulators to be rated as having high therapeutic value.”


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Tags: approvals, EMA, EU, FDA, US

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