US signals need for shorter salvage trials - but still out of step with Europe

Keith Alcorn
Estimated reading time 2 minutes
This article is more than 24 years old.

The US Food and Drug Administration has signalled that it is willing to move in the direction of shorter trials to license drugs for salvage therapy, following the lead of the European drug regulatory agency, the EMEA. However, significant differences in emphasis still exist between the European approach and the emerging consensus in the US.

Earlier this year a group appointed by the EMEA's scientific committee recommended that approval should be granted for drugs proven effective after 12 to 16 weeks in treatment-experienced patients - before 48 week studies are completed in treatment naïve patients. But the committee also emphasised that phenotypic and genotypic data would be necessary, and that provisional licenses would last for one year, renewable on the basis of safety data submitted.

Glossary

Food and Drug Administration (FDA)

Regulatory agency that evaluates and approves medicines and medical devices for safety and efficacy in the United States. The FDA regulates over-the-counter and prescription drugs, including generic drugs. The European Medicines Agency performs a similar role in the European Union.

treatment-experienced

A person who has previously taken treatment for a condition. Treatment-experienced people may have taken several different regimens before and may have a strain of HIV that is resistant to multiple drug classes.

naive

In HIV, an individual who is ‘treatment naive’ has never taken anti-HIV treatment before.

salvage therapy

Any treatment regimen used after a number of earlier regimens have failed. People with HIV who have experienced side-effects and/or developed resistance to many HIV drugs receive salvage therapy, sometimes consisting of a large number of medications.

treatment-naive

A person who has never taken treatment for a condition.

The European guidelines were partly influenced by strong lobbying from the European AIDS Treatment Group, which has been recommending that the EMEA needs to take a pan-European view of the substantial differences in compassionate access to new antiretrovirals between European countries. While France, Germany and the UK have regulations which allow early access, Spain, Italy and Portugal do not have similar mechanisms.

Last week, a public hearing called by the FDA's Antiviral Advisory Committee heard that the FDA may prefer future license applications to consist of studies in treatment naïve and highly treatment-experienced patients, suggesting that studies testing an agent in people still naïve to one class of drug will not be considered adequate evidence of effectiveness in highly treatment-experienced patients.

However, the FDA gave no hint that it is willing to uncouple the licensing timelines for the use of drugs in naïve and highly treatment experienced patients, which means that companies could obtain licenses more quickly in Europe for salvage products. They may also be forced to design different types of studies if Europe and the US do not agree on their requirements.

A full report on the FDA hearing, written by Mark Mascolini, is available at the NATAP website.

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