Accelerated Approval allows for early access to drugs and biologics based on initial evidence of safety and effectiveness, while confirmatory studies required to verify clinical benefits are ongoing. Do patients care? No.

Accelerated Approval was developed in 1992 in response to the HIV/AIDS crisis and has led to expedited drug and biologic approvals in several disease areas across the FDA. The program was codified into law under the Food and Drug Safety and Innovation Act (FDASIA) in 2012.

Accelerated Approvals are granted on the basis that products have an effect on “a surrogate endpoint that is reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefits, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments” (FDASIA, 21 USC §301).

Do patients care? The answer to that would be “no.” Over my twenty-plus years of DTC experience, one aspect has remained constant; patients trust their physician to prescribe drugs that treat their health problem”.

According to an analysis of CDER data conducted by the National Organization for Rare Disorders, from 1992 through 2010, the pathway was primarily used to approve drugs to treat HIV (39.7% of approvals), cancer (35.6%), and other rare diseases (24.7%). Since then, accelerated Approval has shifted focus to oncology drugs. 85% of accelerated approvals from 2010 to 2020 were for oncology indications.

If you’re a cancer patient and your oncologist is prescribing a drug approved via accelerated Approval, are you going to refuse treatment? Of course not. Should patients be made aware that the drug they are being prescribed is an “accelerated approval drug”? Of course, they should, but in the end, it’s going to come down to trust in their doctor and the FDA.

But…

Elizabeth Mahase writes for The BMJ wrote, “nearly half of the 253 drugs authorized via FDA’s accelerated approval pathway since 1992 have never been confirmed as clinically effective—leading some experts to suggest the approval process has been “exploited, to the detriment of patients … and of taxpayers”.

Accelerated approvals carry the condition that manufacturers must conduct so-called Phase IV studies to ensure they provide a clinical benefit post-approval. And according to The BMJ, 112 of the 253 drugs approved through the pathway thus far still haven’t been confirmed as clinically effective in post-approval studies—including 24 medicines that have been on the market for more than five years.

The FDA has also been revisiting cancer therapy approvals made through the program. In April 2021, the agency convened the Oncologic Drugs Advisory Committee (ODAC) to review six so-called dangling accelerated approvals. The confirmatory trials had failed, but the drug remained on the market. Of the six checkpoint inhibitor indications reviewed, ODAC recommended that four be maintained (RELATED: ODAC recommends pulling 2 of 6 accelerated approvals, Regulatory Focus 30 April 2021).

There is no doubt that the accelerated approval program will undergo some modifications as the FDA gets feedback from the medical community. Still, the idea that accelerated approvals should be communicated to patients in DTC (websites) is needed but won’t make a difference. Patients trust their doctors, so the accelerated approval process must be modified to increase their trust with physicians and explain to patients precisely what accelerated drug approval means.