AIDS fight led to streamlined review process for lifesaving drugs

In 1992, Congress passed the Prescription Drug User Fee Act (PDUFA) to streamline the drug approval review process and make it more predictable. Under PDUFA, the FDA sets a standard goal of reviewing new drug applications within 10 months. For drugs that promise major advancement in treatments, the priority-review goal is six months.

To ensure these reviews are carried out with maximum regard for patient safety, PDUFA also authorized an expansion of the FDA’s review staff. Furthermore, the law launched a new system of user fees, funded by the pharmaceutical industry, which supplements the FDA’s congressional funding.

These user fees go directly to improving and speeding up the drug review process. In fact, in a time of tight budgets, PDUFA represents an outstanding example of how the public and private sectors can work together to advance patients’ interests. User fees paid by the pharmaceutical industry now account for two-thirds of the FDA’s review budget, with one-third coming from taxpayers.

The results? Since PDUFA first passed, more than 1,100 new medicines have reached patients faster than they would have under the old regime, providing needed relief to countless Americans.

PDUFA has been remarkably effective, but progress has been slipping. In PDUFA’s first decade, drug-review times decreased dramatically – by upwards of 60%. Over the past few years, however, review times have begun creeping up again, as applications for new drugs have swelled and Congress has mandated new requirements during and after review that are slowing the process.

Among these new requirements, all applications must be reviewed by an advisory council of outside experts – a step previously only required for drugs with active ingredients that hadn’t already been approved for use in the U.S. In addition, applicants may be asked to submit paperwork such as communications plans for health care providers and package inserts for patients that can take months to produce and are sometimes only requested well after the review process has started.

These measures are well-intentioned, but doctors, hospitals, and health care associations have noted that they can delay access to vital drugs and therapies. For instance, in 2008, the FDA missed its six-month review goal on 40% of priority-review applications, versus just 14% in 2007. A wait of even a few additional months can literally mean the difference between life and death for a patient with a disease for which few treatments exist.

It’s important to understand that, under PDUFA, patient safety has never been compromised. Applications still go through the same close scrutiny that existed when it used to take two and a half years or more for drugs to be approved. Before PDUFA, 2-3% of drug approvals were later withdrawn because of safety concerns discovered when the drugs were on the market. Under PDUFA, that rate has not changed.

PDUFA’s goal is to approve safe, effective medicines as swiftly as possible. We need to get the law back on track, and Congress has an opportunity to do that through the upcoming re-authorization of PDUFA.

In reauthorizing PDUFA, Congress should ensure that the drug review process is science-based and transparent, with a clear definition of how the FDA will judge risks and benefits. The agency should also communicate clearly and regularly with applicants, so that they can be full partners in getting drugs to market and monitoring them when they’re on the market. Well-intentioned requirements that may be holding up the process without contributing additional benefits should be reconsidered.

This is one issue on which there are no “sides.” An FDA that is fully funded, with enough staff to review new drug applications swiftly and carefully is in the interest of all Americans, particularly those who are living with serious illnesses.

The stakes are extremely high. People with AIDS, Parkinson’s and other diseases for which hope is high but treatments are limited need to know that the pharmaceutical industry and regulators are working hand-in-hand to deliver the latest breakthroughs as quickly as possible.

Frank Oldham, Jr. is the president and CEO of the National Association of People With AIDS.