Regulatory Focus™ > News Articles > 2019 > 4 > EMA Officials Debate Going Beyond Safe and Effective Determinations for New Drugs

EMA Officials Debate Going Beyond Safe and Effective Determinations for New Drugs

Posted 11 April 2019 | By Zachary Brennan 

EMA Officials Debate Going Beyond Safe and Effective Determinations for New Drugs

In the US and Europe, drug regulators approve new drugs based on whether the benefits of the new product outweigh the risks. But neither of the regulators consider if such new drugs are superior to drugs currently marketed.

Now, three senior officials at the European Medicines Agency (EMA) explain how an added therapeutic benefit criterion or head-to-head comparisons could help to drive more innovative therapies.

“The aim of these proposals is to contain the rising cost of what is often perceived as the nebulous concept of ‘innovation,’” EMA’s senior medical officer, Hans-Georg Eichler, chair of EMA’s human medicines committee Harald Enzmann and EMA's Executive Director Guido Rasi write in a commentary published in Nature Reviews Drug Discovery on Thursday.

The four proposals outlined in the commentary deal with: requiring added therapeutic benefit, requiring head-to-head comparisons, planning for indirect comparisons and focusing on comparative efficacy.

But the senior officials also explain some of the unintended consequences of requiring an added therapeutic benefit, particularly as so-called “me-too” drugs can reveal different safety or efficacy profiles for certain classes of medicines.

“Even when average or median effect sizes of products appear similar, treatment responses in individual patients may differ from one drug in a class to the next, owing to known or unknown individual patient characteristics. For example, this has been observed with tumor necrosis factor inhibitors and may become more important in the future,” they write.

As far as requiring head-to-head comparisons, the EMA officials note cases where active controls might not be feasible.

“Active comparator trials, including platform trials, should be encouraged where useful, but flexibility in the choice of direct or indirect comparators, including placebo, is needed to account for a range of different clinical scenarios,” they write.

In terms of contextualizing the effect of new medicines, the officials explain how they have been told by stakeholders to be more forthcoming with negative, neutral or positive added benefits where possible in relevant patient subgroups. And EMA is now discussing with health technology assessment bodies and payers about “how best to serve these information needs.”

Added therapeutic benefit and drug licensing
 

Tags: drugs, EMA, trials

Regulatory Focus newsletters

All the biggest regulatory news and happenings.

Subscribe