Accelerated Approval Program

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The U.S. Food and Drug Administration (FDA) instituted the Accelerated Approval Program in 1992. The program is in place to expedite the approval of life-saving drugs. During the standard FDA approval process, the FDA must evaluate the clinical outcome, or end result, of using a new drug or device. A positive clinical outcome can be seen through improvement in the patient’s condition, such as feeling better or living longer. If the clinical outcome shows a positive impact on the patient’s condition, the drug becomes one step closer to FDA approval.

However, clinical trials used to determine the clinical outcome can take years before a drug shows positive endpoint results on a deadly condition. The FDA Accelerated Approval Program focuses on a measurement called a surrogate endpoint. Surrogate endpoints can help the FDA to evaluate the clinical outcome of a drug or device in significantly less time than a standard clinical trial. This way, patients with aggressive and life-threatening conditions can receive treatment more quickly.

What Is a Surrogate Endpoint?

The FDA Accelerated Approval Program is based on the evaluation of a surrogate endpoint. A surrogate endpoint is a marker that indicates the potential for a positive long-term clinical outcome, or endpoint. This endpoint is referred to as “surrogate” because it is a suggested short-term alternative. A surrogate endpoint typically suggests that the long-term goal will be met.

For example, a drug used to treat liver cancer is intended to cure the cancer and increase the patient’s life span. In order to definitively reach this endpoint, the clinical study would require years of ongoing monitoring and evaluation. However, early evidence may show that the drug shrinks liver tumors. Tumor shrinkage is an indicator of an improved cancer condition. This evidence can be used as a surrogate endpoint because it shows that the drug is reasonably likely to produce a real clinical benefit for liver cancer patients.

FDA Accelerated Approval Program Controversy

The FDA Accelerated Approval Program has garnered a great deal of controversy since it was adopted. The program is often criticized for rushing certain drugs and devices to the market without sufficient proof of their efficacy. It is estimated that 10 percent of accelerated approvals in the oncology market fail in post-marketing studies. This translates to ineffective drugs and devices being used by patients who are in critical need of effective treatment. Additionally, approval through the FDA Accelerated Approval Program may fail to identify deadly side effects that result from use of the product.

Delay in Post-marketing Studies

After a product is approved under the FDA Accelerated Approval Program, manufacturers are still required to conduct post-marketing studies. These studies are referred to as phase 4 confirmatory trials. If the confirmatory trial shows clinical benefit, the product receives traditional FDA approval. If it does not, FDA regulatory procedures may be used to remove the drug from the market. The manufacturer of a liver cancer drug must conduct testing that confirms the suggestion that shrunken liver tumors will help patients live longer. However, several manufacturers fail to provide timely post-marketing study results.

In 2000, Pfizer received accelerated approval for a treatment for acute myeloid leukemia (AML). Unfortunately, the study was not conducted until four years after the drug was approved under the FDA Accelerated Approval Program. Pfizer withdrew the drug in 2010. The withdrawal took place because clinical studies showed a lack of clinical benefit from the drug. Additionally, an unexpected number of deaths occurred in patients using the drug.

FDA Accelerated Approval Program Debate

In 2011, the FDA discussed changes to the FDA Accelerated Approval Program. Several FDA officials suggested using an existing provision within a 2007 law. This provision would result in a fine of up to $10 million for companies that failed to conduct timely post-marketing studies. Additionally, the FDA discussed that post-marketing studies began before accelerated approval is granted to a product. This way, manufacturers would be unable to postpone clinical studies.