PDUFA Reauthorization is critical to millions of Americans with chronic diseases

Ten years ago, most Americans had never heard of lupus - a chronic autoimmune disease. Today, through education and outreach, more people are becoming familiar with the serious impact lupus has on the health of an estimated 1.5 million Americans. With Congress actively considering reauthorization of the Prescription Drug User Fee Act (PDUFA), it has never been more important to focus on the future development of lupus drugs and what is needed to help people with lupus overcome their life-diminishing and life-threatening disease.
 
Consider the following: it is doubtful that more than 1,000 new critical treatments would have moved through the Food and Drug Administration (FDA) drug approval process in the last 20 years had PDUFA not been in place. Since its 1992 passage, PDUFA-provided user fees have helped the FDA fulfill its core function of ensuring safe and effective drugs are available to improve the health and well-being of Americans. PDUFA offers hope for new treatment for people who suffer from underserved and chronic diseases such as lupus by helping expedite the drug approval process, resulting in new drugs getting to patients in a more timely and efficient manner.

Lupus causes inflammation and tissue damage to virtually any organ system in the body. The disease can cause organ failure, disfiguring skin lesions, strokes, heart attacks, and seizures. Ninety percent of people with lupus are women, and the disease is two to three times more common among women of color. An accurate diagnosis of lupus can take as long as four years and visits to more than three physicians. Physicians prescribe a wide variety of medications to treat lupus because the manifestations of lupus vary dramatically from one person to another. Many medications are prescribed off-label, are intolerable, and have harsh side effects.
 

While there is no cure for lupus, researchers are actively studying the underlying science of the immune system. Armed with a better understanding of the disease, researchers will be able to develop more effective and personalized treatments. Certainly, PDUFA has had a salient effect on advancing treatments for this unpredictable, life-diminishing, and life-threatening autoimmune disease, as well as many others.
 
In 2011, the FDA approved 35 new breakthrough treatments, including the first lupus-specific treatment in more than 50 years. This treatment received a fast track designation by the FDA, was evaluated under the priority review program, and approved for use in 9 months. Nevertheless, because each individual’s experience with lupus is so different, one treatment will never suit all, and people with lupus need and deserve a full arsenal of therapies.
 
Biopharmaceutical researchers understand the complexity of lupus and are making great strides in the quest for more effective treatments. There are more than two dozen potential drugs for lupus in the development pipeline. People with lupus should not have to wait another 50 years for the approval of another drug, and are anxious for enhancements to the FDA expedited review process.
 
While an acronym that few will understand or hear directly about, Americans look to Congress to reauthorize PDUFA in a timely and efficient manner. Passing this essential legislation will help ensure that innovators and researchers are able to translate medical breakthroughs into FDA-approved treatments that will save lives and improve the quality of life for many Americans. Stated simply, a fully funded FDA, with enough staff to safely and efficiently accelerate the approval process, is critical to the health of lupus patients and Americans overall.
 
Raymond is president and CEO of the Lupus Foundation of America, Inc.

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