If you or a loved one were to receive a diagnosis of a serious or life-threatening disease, you would likely want access to a U.S. Food and Drug Administration (FDA) approved safe and effective medicine as soon as possible. That’s precisely why the accelerated approval pathway was created back in 1992 at the urging of patient advocates. Since then, it has been a lifeline for millions of patients. Primarily utilized for cancers, rare diseases and HIV/AIDS, the pathway allows patients earlier access to potentially lifesaving medicines – often when no other treatment is available and when patients are facing difficult odds.
Here are three key things to know about this patient need-focused pathway and its impact over the last 30 years:
The accelerated approval pathway was developed by the FDA in 1992 as a response to the growing HIV/AIDS epidemic and calls by patient advocates for the agency to expedite access to promising medicines in development.
- Developing an innovative medicine is a lengthy and complex process that takes an average of 10 to 15 years. But patients fighting serious or life-threatening diseases – including hard-to-treat cancers – often don’t have time on their side. Amid the growing HIV/AIDS epidemic in the early 1990s and in response to the urging of patient advocates, the FDA designed the accelerated approval pathway to allow patients earlier access to potentially lifesaving treatments — an action that, no doubt, saved lives then and has been improving and extending lives for the last 30 years. For example, accelerated approval awarded to two therapies for non-small cell lung cancer allowed more than 500,000 patients to get those treatments sooner, allowing the combined patient population to live nearly 200,000 additional years compared with expected survival had the medicines not been available.
Medicines granted accelerated approval must adhere to the same standards for establishing safety and effectiveness as medicines receiving a traditional FDA approval.
- Medicines granted accelerated approval must provide substantial evidence of effectiveness per the FDA based on “adequate and well controlled clinical investigations.” The pathway specifically allows FDA to approve drugs based on a determination that the product has an effect on certain surrogate endpoints or intermediate clinical endpoints. Surrogate or intermediate endpoint can be based on a laboratory measurement (like blood glucose level for diabetes), radiographic image (like tumor size reduction), physical sign (like blood pressure for cardiovascular disease) or other measure. For instance, reduction of viral load in HIV patients, reduced tumor size in cancer patients or alleviation of symptoms can all be used to show a treatment’s benefit to patients. Congress has clarified, through statute, that the accelerated approval pathway does not alter the standards of evidence required for approval.
After a medicine is granted accelerated approval, companies are required to conduct, and provide updates on, confirmatory trials and the FDA is required to make this information publicly available.
- Companies are required to conduct confirmatory studies of medicines granted accelerated approval and are subject to reporting requirements on the status of these studies. The FDA is required to track, and make publicly available, progress of confirmatory trials.
- It is important to understand the challenges of conducting confirmatory trials. There are a number of reasons that a confirmatory trial may take longer than originally planned, including the inability to enroll patients as quickly as anticipated, for example due to patients enrolling in other studies aimed at the same population, patients being less willing to volunteer for studies of FDA-approved medicines or small patient populations.
The accelerated approval pathway has demonstrated considerable success for patients facing serious or life-threatening diseases and provided earlier access to treatments for illnesses with unmet medical needs. As policymakers examine the pathway, it is vital any changes do not hinder the significant benefits stemming from the program expediting patient access to lifesaving medicines.
To learn more about the importance of the accelerated approval pathway, click here.
Topics: FDA, Prescription Drug Safety