New bill moves to tighten accelerated approval regulation in US
US House Energy & Commerce Committee Chair Frank Pallone (D-NJ) has introduced a new bill to enhance the Food and Drug Administration’s (FDA) authority to ensure products that receive accelerated approval are providing a clinical benefit to patients.
Under the proposed Accelerated Approval Integrity Act, the FDA would be granted power to expedite the withdrawal of an accelerated approval status if the required post market studies are not completed within five years. However, trials may continue if the post marketing study has been completed and verified the clinical benefit, or the Secretary of Health and Human Services determines that adequate progress has been made.
The bill would also require drug companies to plan and initiate post market studies before a drug receives accelerated approval status.
“The Accelerated Approval Program allows patients to access innovative new drugs while the manufacturer and FDA conduct additional studies to confirm the product’s clinical benefit,” Pallone said. “However, under the current system, some products have been allowed to stay on the market for far too long without clinical trials that demonstrate a real clinical benefit for patients.”
Developed in 1992, largely in response to demand for new treatments during the HIV/AIDS epidemic, the Accelerated Approvals Programme has become a central feature of drug approvals in the US.
In its current form, the legislation grants the FDA power to expedite the approval process of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint.
Once a drug has been granted accelerated approval status, manufacturers are required to complete studies to confirm the anticipated clinical benefit. If the drug fails to demonstrate clinical benefit, the regulator has the power to remove the drug from the market.
However, the systems in place to remove drugs from the market have proven to be a slow and challenging process. Covis Pharma’s Makena, has been a notable example of this. The drug was granted accelerated approval in 2011 for the prevention of recurrent preterm birth in women. It took eight years to perform the required study to prove the drug’s benefits, a study which the FDA reports ultimately failed to demonstrate the benefit of Makena.
If passed, Pallone’s bill would modify existing legislation to give the FDA authority to accelerate the withdrawal process. With these powers in place, controversial issues such as Makena would be addressed and pulled from the market in a timely manner.
Pallone’s bill would also require more frequent updates from sponsors on post-approval studies, including updates on enrolment targets, milestones, and study design.
“I’m introducing the Accelerated Approval Integrity Act to protect patients and provide FDA with the authority it needs to ensure approved drugs provide a clinical benefit. The bill would also streamline the process for taking products off the market if no clinical benefit is proven in a timely manner,” said Pallone.