Accelerated Approval provides patient access to promising new medicines faster. The treatment is approved based on limited data, based on surrogate measures that aims to predict meaningful clinical benefits for the patients. This means an increased uncertainty of the products profile and confirmatory studies must verify the expected clinical outcome.  

When the FDA issued the Accelerated Approvals in 1992, it was a response to the AIDS epidemic and enabled the FDA to approve these drugs faster. Accelerated approval allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint, a prediction of a clinical benefit. Between 1992 and 2021, the FDA has granted 278 accelerated approvals*.

Accelerated Approvals is reserved for drugs:

• Intended to treat a serious condition.
• Show meaningful advantage over available therapy.
• Demonstrates an effect on an endpoint that is reasonably likely to predict clinical benefit.

Accelerate Approval allows drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint or an intermediate clinical endpoint. The FDA has determined that an effect on the endpoint used to justify approval can reasonably predict clinical benefit at the time of approval.

Surrogate endpoints are markers, such as laboratory measurements, ages, physical signs, radiographic signs, or other measures that are generally accepted to predict a clinical benefit, but they do not directly measure the clinical benefit itself. FDA bases its decision on whether to accept proposed surrogate endpoints on the available scientific support for that endpoint. 

A surrogate endpoint may correlate with a real clinical effect but does not necessarily guarantee causality. Choosing the right surrogate endpoint and proving that it can predict the intended clinical benefit within the scope of the targeted disease is not always straightforward. The surrogate marker must show sufficient evidence for being reasonably likely to predict a drug’s intended clinical benefit in order to be used for accelerated approval. 

An Intermediate Clinical Endpoint is a measurement of a therapeutic effect that can be measured earlier than an effect considered reasonably likely to predict the clinical benefit - such as an effect on irreversible morbidity and mortality (IMM) – of the drug. In this approach, patients may be exposed to a drug that ultimately does not provide an actual clinical benefit. There may also be fewer, smaller, or shorter clinical trials than for a traditional approval, which may mean there is less information about rare or delayed adverse events. For these reasons, accelerated approval is reserved for drugs that appear to provide a meaningful advantage over available treatments for serious conditions.

Applicants must submit copies of all promotional materials, including promotional labelling and advertisements, intended for dissemination or publication within 120 days of marketing approval to the Agency for consideration during the preapproval review period unless otherwise informed by the Agency. An applicant must submit promotional materials at least 30 days before the intended date for the first dissemination of labelling or the first publication of advertisement after 120 days after marketing approval.

The anticipated effect on IMM or other clinical benefits of drugs granted accelerated approval must be verified and described in post-marketing confirmatory trials. Due diligence must be exercised in the completion of these trials.