- - Tuesday, May 8, 2012


In late April, at the annual meeting for the American Academy of Neurology, the American drug developer Genzyme announced a breakthrough for one of its new multiple sclerosis drugs. The company revealed that a late-stage trial of an experimental therapy found that an astounding 65 percent of participating patients had gone without an MS relapse two years after starting treatment.

The best of current MS medications registers a 47 percent relapse-free mark. If further trials go as planned, this medicine could soon be submitted to the Food and Drug Administration for approval, providing new hope for thousands of people now suffering from this debilitating disease, including Ann Romney, wife of Republican presidential hopeful Mitt Romney.

That expeditiousness - between a stunning in-trial discovery and the release of a life-changing new medication - is almost wholly a function of an obscure-sounding but profoundly important piece of legislation called the Prescription Drug User Fee Act (PDUFA).

That act will soon expire. It’s vital that Congress reauthorize the PDUFA with all deliberate speed. The lives and health of millions of American patients depends on it.

The PDUFA was enacted in 1992. It was meant to address a huge problem then plaguing the federal review process for new drugs. Pharmaceutical firms would come up with treatments, submit them to the FDA for evaluation and wind up waiting at least two years before being given the green light to begin trials.

The reason for the delay was simple: The FDA didn’t have enough money. It was understaffed and couldn’t afford to hire more reviewers.

Congress fixed the problem by passing the PDUFA. The bill established fees for drug companies utilizing the FDA review process. Those fees provided an influx of cash used to hire additional FDA staff and finance other logistical resources needed to make the review process more efficient, predictable and timely.

The results of the PDUFA have been remarkable and since 1993, the bill has been renewed on four separate occasions. The FDA now has 200 additional staffers. The review period is 60 percent shorter and currently leads the world in speed. In fact, nearly 70 percent of the 35 drugs approved by the FDA last year hit the U.S. market before being sold anywhere else in the world.

Since the PDUFA passed, roughly 1,500 new medicines have been introduced in the United States, including treatments for cancer, diabetes, cardiovascular disease and neurological disorders.

The PDUFA reauthorization bill currently being considered in Congress would extend the life of the bill another five years, beginning in 2013. This newest version would do more than just maintain the status quo, though. It also makes targeted improvements to parts of the FDA review process that are still weak.

For instance, the PDUFA would update the review process for new high-tech medicines such as biologics. This class of drugs tends to be significantly more complex than traditional chemical medications. They require special procedures and staff to ensure they get through federal review in a timely fashion.

The new PDUFA would also establish new protocols for employing complicated medical tools such as “pharmacogenomics” and “biomarkers.” These tools would help determine the therapeutic benefits of a submitted drug more rapidly and help the FDA better identify patients who could benefit from a new treatment.

The reauthorization bill would also create a special, accelerated approval avenue for “orphan” drugs - so-called because they treat rare diseases. The diseases in question affect so few patients that the economic incentive to develop drugs to treat them is much lower.

By creating a fast, low-cost review process, the PDUFA would reduce the financial barriers for creating orphan disease therapies. Pharmaceutical firms will accordingly have a greater incentive to invest resources in therapies for those suffering from rare conditions.

Reauthorizing the PDUFA with the improvements described here will enhance the predictability and efficiency of the federal drug review system. Patients would see more and better drugs introduced to market.

But the benefits aren’t just in health care. The American pharmaceutical industry directly supports more than 674,000 American jobs. Its total economic output every year tops $918 billion.

A robust and efficient FDA review process will help these companies create new jobs and boost the American economy. Failure to reauthorize the PDUFA would introduce catastrophic uncertainty for the industry.

Congress must provide the FDA with the resources it needs to keep the drug review process timely, efficient, transparent and predictable.

Reauthorizing the PDUFA would ensure that American drug innovation stays vibrant and productive, that patients continue to have access to the best medicines in the world, and that this country’s pharmaceutical industry continues to create jobs.

Sally C. Pipes is president of the Pacific Research Institute. Her latest book is “The Pipes Plan: The Top Ten Ways to Dismantle and Replace Obamacare” (Regnery, 2012).



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