The FDA’s Accelerated Approval Program began in 1992, and it is designed to allow drugs that treat serious conditions to be approved earlier, particularly if this earlier approval fills an unmet need when it comes to treating that condition. For example, if there is a disease that doesn’t have any drugs available to help slow the progression of disease, a promising drug that might slow disease progression could be approved via the Accelerated Approval Program. 

The accelerated pathway can increase the availability of life-saving drugs, but it also presents the issue that patients might take drugs that don’t have any real benefit for their condition. Below, learn how the Accelerated Approval Pathway works as well as the benefits and drawbacks. 

How drugs are granted Accelerated Approval 

Drugs that are approved via the accelerated program do not yet show a measure of clinical benefit. In other words, they haven’t yet been proven to actually change any outcomes when it comes to how a patient feels or how their disease progresses. Instead, the outcomes are measured via a surrogate endpoint, which could be a lab measurement or a radiographic image like an x-ray. 

The surrogate endpoint can help predict clinical benefit, which can shorten the time it takes the drug to get FDA approval because a surrogate endpoint can be reached much more quickly than the final endpoint. For example, it doesn’t take nearly as much time to prove that the drug caused a tumor to shrink as it does to prove that the drug increased the lifespan of cancer patients. 

Even though the drugs can be approved without showing any clinical benefit, companies still need to submit studies that show the anticipated clinical benefit. They also still need to prove that the medication is safe. 

Benefits of the FDA’s Accelerated Approval Pathway

This Accelerated Approval Pathway can be very helpful for people with diseases that don’t have many beneficial treatments available. It can allow them to have access to promising drugs that can greatly improve their quality of life or even survival rate. It can take up to 15 years for a new drug or treatment to be developed and approved. Under the accelerated pathway, however, drugs can be available to patients sooner, which can save their lives. Once a drug is approved, the company still needs to follow up to confirm that the medication is effective, which means these drugs aren’t just thrown out there quickly and then forgotten about. 

One study showed that it made cancer treatments available an average of 3-4 years sooner. “For patients with serious or life-threatening oncologic diseases, AA brings products to the market years before confirmatory trials are typically completed,” the authors concluded. For someone who is living with a chronic disease and waiting on a cure or treatment, accelerated approval provides hope. 

Drawbacks of the FDA’s Accelerated Approval Pathway 

However, this faster approval can sometimes prove dangerous. Though drugs still have to meet the same safety standards when approved via the accelerated pathway, there is always the risk of side effects when taking a drug. If the benefits outweigh the risks, then it’s worthwhile to take the drug. However, if a drug eventually proves to not have clinical benefit, then people may be suffering from side effects without realizing a benefit at the end. 

In one review, the Office of Inspector General (OIG) found that there were concerns for 3 of the 24 drugs approved via the Accelerated Approval Pathway in the review. Concerns were raised due to the FDA approving the drugs despite concerns from reviewers and advisory committees. 

For example, in the case of Oxbryta, a drug used to treat sickle cell disease, the drug was withdrawn after previously being granted accelerated approval. Once the researchers were able to evaluate the clinical outcome, they found that the drug didn’t provide the benefits they were hoping for. For the many people who took Oxbryta, the risks of the drug did not outweigh the benefits. Some people who took Oxbryta experienced VOCs (vaso-occlusive crises), which is a very serious complication of sickle cell disease. It happens when sickled red blood cells block blood flow; these events can be incredibly painful and even fatal in some cases. 

Because of these dangerous events with the drug, some people have begun to file lawsuits against Pfizer and Global Blood Therapeutics, claiming that they did not properly warn patients of the dangers of the drug. If you’ve been affected by this concern with Oxbryta, you may be eligible to file a lawsuit, and we can help determine your eligibility.