The US Food and Drug Administration (FDA) has published new for its Accelerated Approval Program, which provides a process for expediting new drugs that serve unmet medical needs for serious conditions.
The guidance outlines the requirements for the two types of endpoints considered as a basis for accelerated approval. These are either surrogate or clinical endpoints.
An acceptable surrogate endpoint is one 鈥渢hat is considered reasonably likely to predict clinical benefit鈥�, according to the guidance, while the clinical endpoint must be one 鈥渢hat can be measured earlier than irreversible morbidity or mortality (IMM) that is reasonably likely to predict an effect on IMM or other clinical benefit鈥�.
Surrogate endpoints are defined in the guidance as 鈥済enerally a biomarker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit鈥�.
The FDA offers examples of 鈥渞easonably likely鈥� surrogate endpoints used to support accelerated approval. These include the conversion of sputum culture from positive to negative during treatment of pulmonary tuberculosis, which has been considered 鈥渞easonably likely鈥� to predict the clinical benefit of resolution of infection.
The FDA also defines acceptable intermediate clinical endpoints that are considered in accelerated approval applications. It requires 鈥渁 measurement of a therapeutic effect that can be measured earlier than an effect on IMM and may support accelerated approval when it is considered reasonably likely to predict the drug鈥檚 effect on IMM or other clinical benefit鈥�.
How well do you really know your competitors?
Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.
Thank you!
Your download email will arrive shortly
Not ready to buy yet? Download a free sample
We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form
By GlobalDataIt offers the example of a study that demonstrates a short-term benefit in the treatment of chronic disease but that requires a longer duration of effect to be clinically meaningful. The FDA would consider whether it is 鈥渞easonably likely鈥� that the short-term benefit is indicative of this.
Drugs approved via the Accelerated Approval Program must be further tested through a postapproval confirmatory trial to be granted full approval. The trial must verify and describe the clinical benefit of the drug.
The new guidelines advise that 鈥渟ponsors must commit sufficient resources to conduct the trial(s) intended to verify the clinical benefit expeditiously so that a determination of whether the drug provides the expected clinical benefit can be made as soon as possible鈥�.
They also state the need for early consultation between reviewers and sponsors, explaining that additional preclinical or clinical data may be required by review teams. It is suggested that 鈥渟ponsors should seek early interactions with the FDA鈥� to avoid delays.
