Less than half of new drugs add therapeutic value over existing treatments

Experts are calling on pharmaceutical companies to create innovative and better treatments for patients after a study found that less than half of supplemental treatment indications approved in the last decade provide substantial therapeutic value compared with first approvals.

The study also found that the proportion of approved supplemental indications rated as having high therapeutic value was substantially lower than for approved first indications, and in the US, third indication approvals were 45% less likely to have a higher therapeutic rating than the first.

There are now calls for pharmaceutical companies to clearly communicate to patients about which drugs have the highest therapeutic benefit, and for the price of drugs to be aligned to their clinical benefit.

Researchers compared therapeutic benefit using ratings from HTA bodies

The study, which is published in The BMJ, looked at new drugs in the UK and Europe between 2011 and 2020. Researchers identified 124 first and 335 supplemental indications approved by the US Food and Drug Administration (FDA) and 88 first and 215 supplemental indications approved by the European Medicines Agency (EMA).

The researchers then looked at how many supplemental indications the drugs had. These are summarised in the table below:

The researchers then compared the therapeutic ratings of each drug and its supplements. Therapeutic ratings from French and German health technology assessment (HTA) bodies were available for 107 (86%) first and 179 (53%) supplemental indications in the US and for 87 (99%) first and 184 (86%) supplemental indications in Europe.

In the US, 41% of first indications had a high therapeutic value compared to 34% of supplemental indications. In Europe, a similar pattern was seen, with 47% of first indications rated as having high therapeutic value compared with just 36% of supplemental indications.

Among FDA approvals, when the sample was restricted to the first three approved indications, second indication approvals were 36% less likely to have a high value rating and third indication approvals were 45% less likely when compared to the first indication approval. Similar findings were observed for Europe.

Public health goals should be aligned with drug development

The authors acknowledge some limits to the study, including that these findings are only observational and that therapeutic value ratings were not available for all indications.

However, they performed a sensitivity analyses with the value scores of each authority separately, which confirmed the initial results.

As such, they conclude: “Fewer than half of approved first indications in the US and Europe were rated as having high therapeutic value, and the proportion of approved supplemental indications rated as having high therapeutic value was substantially lower than for approved first indications.

“When indications do not offer added therapeutic benefit over other available treatments, that information should be clearly communicated to patients and reflected in the price of the drugs.”

In a linked editorial, Beate Wieseler at the German Institute for Quality and Efficiency in Health Care said there now needs to be a move towards aligning “current legislation on drug development with defined public health goals”.