Comparative efficacy data should be considered before new drug approval

An endeavor to ensure that limited healthcare resources are invested wisely, and ascertain that the safest and most efficient treatments are provided to patients, has led researchers to emphasize on manufacturers revealing details of how their drug compares with established standard therapy.

Presently, manufacturers compare the benefits and risks of any new medicine against a placebo. However, it is only when placebo use is deemed unethical, that manufacturers are required to compare the new medications with existing drugs.

“This does not allow patients, clinicians, and other healthcare decision makers to determine whether a new drug is superior, equivalent, or inferior to its existing alternatives. This can result in the widespread use of potentially less efficacious and unsafe drugs,” according to investigators at the London School of Economics and European Observatory of Health Systems and Policies.

In several investigations, the authenticity of the true added benefit offered by new and generally expensive medications, in contrast to existing treatments, has been questioned.

The researchers write: “The European Medicines Agency (EMA) has long encouraged that, when possible, pre-market studies should be undertaken to establish comparative efficacy and risk, but has yet to set comparative assessments as the default evidentiary standard for market approval. Rather, requirements for comparative studies are made on a case by case basis.”

According to estimates, 50-70% of new molecular entities have comparative efficacy data at the time of approval. However, researchers argue that this variation is observed across various therapeutic areas and only limited information/evidence is often publicized during market authorization.

They add: “A further challenge is that no particular type of study is ideal for assessing comparative efficacy.”

Despite these obstacles, investigators opine that drug-licensing decisions should definitely take into consideration the comparative efficacy evidence. The investigators are seeking transparency and open communication between regulators, manufactures, and government agencies in an effort to establish enhanced correspondence among licensing and reimbursement requirements as well as improved public access to comparative data regarding the safety and efficacy of new drugs.

They conclude: “Numerous promising medicines have been developed and many more are on the way to initial clinical trials. With this success comes an equally important additional need to develop a systematic approach to evaluate the risks and benefits of these new therapies in the context of existing alternatives. An important initial step is to support a formal role for comparative efficacy evidence in drug licensing.”

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