Facing an urgent need to treat patients with HIV/AIDS in the 1980s, advocates called on the FDA to streamline drug approval requirements.
The result was the creation of Accelerated Approval, an FDA program that allowed earlier approval of drugs that filled an unmet medical need for serious or incurable diseases.
Valuable for Many Diseases
Even though the HIV epidemic was the impetus, a majority of the 270 drugs that have received approval via the pathway are cancer treatments. Oncology products brought to market via Accelerated Approval were made available 4.7 years faster than medications approved through the traditional process, according to one study.
The pathway has also brought drugs to market faster for a host of other conditions including many rare diseases and, more recently, neurological disorders.
Ensuring Safety and Speed
Despite Accelerated Approval’s proven track record, public understanding of the pathway’s approval process is still limited, which has led to some concerns about safety.
In fact, every drug – whether it is approved via the traditional or accelerated pathway – must meet the same safety and efficacy requirements. What’s different about those in Accelerated Approval is the endpoint.
Drugs in the traditional pathway achieve a clinical endpoint – a measure of how a patient feels, functions or survives. In contrast, Accelerated Approval drugs meet a surrogate or intermediate endpoint – one that is reasonably likely to predict a clinical benefit. Using this surrogate endpoint can save valuable time and allow drugs to get to patients faster.
As a backend check, the FDA requires drugs that get Accelerated Approval to undergo post market studies. These Phase 4 studies are used to confirm that the drug delivers its intended benefit. Not validating the surrogate endpoint is grounds for the FDA to remove the drug’s approval.
Threats to Patient Access
There have been occasional disputes over the handling of some drugs that were considered via Accelerated Approval, but for the most part, the process works well. And, as one expert noted, “without it, many patients would have few options.”
But that’s not stopping state governments and groups like the Institute for Clinical and Economic Review from trying to limit coverage for Accelerated Approval drugs. ICER, for example, is on record advocating for pricing mechanisms that would discourage usage of Accelerated Approval drugs. And Oregon recently requested a federal waiver that would allow the state Medicaid program to exclude coverage for drugs approved via the pathway.
Quicker approval of safe therapies can be life-changing for patients, which is why access to Accelerated Approval drugs is worth protecting.