Today on bmj.com researchers argue that, before approval, manufactures should have to reveal how their medicine compares to treatments that already exist, in order to make sure that the most effective and safest treatments reach patients and that limited healthcare resources are invested wisely.

At present, the risks and benefits of any new medicine must be compared against a placebo by the manufactures. However, manufactures are only required to compare the new drug with existing therapies when the use of a placebo is deemed unethical.

The investigators at the London School of Economics and European Observatory of Health Systems and Policies argue:

“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.”

Numerous investigations have also questioned the real added value offered by new (and usually more expensive) medications in comparison with existing treatments.

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.”

Although estimates indicate that at the time of approval comparative efficacy data are available for 50% to 70% of new molecular entities, the researchers claim that across therapeutic areas this varies and only a small percentage of evidence is often available at the time of market authorization.

They add:

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

In spite of these restraints, the investigators believe that comparative efficacy evidence should have a crucial role in the decisions of drug licensing.

In order to achieve improved congruence among licensing and reimbursement requirements as well as improved public access to comparative data of the efficiency and safety of new drugs, the investigators ask for open communication between regulators, manufactures and government agencies.

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.”

Written by Grace Rattue