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New drugs: investing in more informative clinical trials

More informative clinical trials are more expensive, but that is the price of better care.

Researchers have estimated the cost of the 138 trials submitted by pharmaceutical companies applying for marketing authorisations for the 59 drugs authorised by the U.S. Food and Drug Administration (FDA) in 2015-16.

The lowest spend was  2.1 million US dollars for a trial on 4 patients for an orphan drug, the highest was 347 million US dollars for a non-inferiority trial evaluating a drug versus a reference comparator on a clinical endpoint. Fifty percent of the trials each cost between 12 million and 33 million US dollars.

Incidentally, these figures show that the frequent claim that the total cost of research and development for new drugs runs into billions of dollars appears improbable and questionable.

Unsurprisingly, the mean estimated cost of these trials increased with their duration and size. It was also influenced by the nature of the comparator and the use of clinical endpoints. More informative trials are more expensive. It is the price that must be paid to help patients and healthcare professionals make better treatment decisions.

In a context in which most drugs introduced to the market do not constitute a real therapeutic advance for patients, it is unlikely that pharmaceutical companies will, of their own volition, choose to conduct costlier but more informative trials that may well provide results that are unfavourable to the drug they are seeking to market. The system has been broken for decades.

It is high time regulators start putting it right.

©Prescrire 1 September 2019

"Investing in informative trials" Prescrire Int 2019; 28 (207): 199. (Pdf, free).

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