Washington – There are no shortcuts when developing safe and effective medicines. On average, it can take 10-15 years to bring a new medicine from early-stage research through clinical trials. The reality is, despite recent scientific advances, there are many serious and life-threatening diseases that lack meaningful treatments, therapies, or cures. Waiting a decade could be the difference between life and death. Fortunately, regulatory agencies from around the world, like the US FDA, have found ways to help bring innovation to patients sooner through pathways like the Accelerated Approval Program.
What is accelerated approval?
The Accelerated Approval Program is a drug development pathway that offers an approval based on a “surrogate” marker in a clinical trial. A surrogate marker is a biomarker or clinical marker that can be measured at an earlier point in a trial than the type of endpoints that may be used in a traditional approval.
For example, a company might submit an application based on a cancer therapy’s impact on tumor shrinkage, or an HIV medicine’s effect on T-cell count. Under accelerated approval, the surrogate marker must be reasonably likely to predict clinical benefit. Additional or continued studies are then conducted to verify the clinical benefit of the therapy. If the clinical benefit is not verified in follow-up confirmatory studies, FDA can seek withdrawal of the drug from the market.
What’s the benefit of going this route?
By permitting earlier access to novel therapies, the accelerated approval pathway has helped extend, and even save patients’ lives. One study found that cancer treatments approved under the pathway were available to patients an average of three to four years earlier than a traditional approval would have allowed. Our own accelerated approval of Alimta® (pemetrexed) was among five medicines highlighted in a recent analysis which found that accelerated approval of these medicines allowed for earlier access by over 9 million people (that’s more than the population of the state of Indiana). Since its inception, more than 290 new drugs and biologics to treat serious or life-threatening illnesses have been approved through the accelerated approval program.
We firmly believe in the goal of the accelerated approval pathway — namely ensuring people living with unmet medical needs have timely access to safe and effective therapies. But to have confidence in the medicines approved under the Accelerated Approval Program, there must be confidence in the underlying pathway. We recognize that stakeholders have raised concerns, mainly related to the time it takes some companies to complete confirmatory studies, and what happens when those studies fail or are not completed.
To address these concerns, Congress passed the Food and Drug Omnibus Reform Act in December 2022. Among other things, the act gave FDA additional authority to require confirmatory trials be underway at the time of approval. It also offered an additional enforcement mechanism if sponsors do not complete these studies with due diligence. Pharmaceutical companies have a key role to play in that. We’re committed to doing our part and have developed a set of guiding principles that transparently communicate how we approach the process. Since the early 1990s the accelerated approval pathway has helped provide earlier access to safe and effective therapies. It’s a critical tool for delivering promising therapies to patients facing the greatest need.