Streamlining drug approval

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By the time you are finished reading this article, 10 people will have died of cancer and seven people will have been diagnosed with Alzheimer's disease. Meanwhile, the FDA has approved the lowest number of new medicines in memory, and the success rate of drugs making it from the lab to patients has declined from 12 percent to 8 percent despite an increase in spending on drug development. At current rates -- and the number of people diagnosed with Alzheimer's will double in 20 years -- these two illnesses will cost patients' families, and the nation, $500 billion a year.

You would think that Congress would welcome an effort to close the gap between the development process and the promise of unprecedented progress against disease that advanced technologies such as genomics offer. Think again. Many members of Congress are pushing a battle plan to expand the regulatory reach of the FDA rather than waging a war against infectious diseases, Alzheimer's, cancer and other illnesses.

Worst of all, politics -- not science or suffering -- is playing the lead role in this controversy. Some want another level of control -- an independent Drug Safety Board that could intervene and demand additional safety studies of any size and quantity at virtually any time before, or after, approval.

Others want the FDA to have the power to demand additional clinical trials of any size and duration to ferret out possible safety problems and to yank drugs off the market, regardless of their benefit, if the studies are not completed within a specific time frame. Certainly, this would make the approval process far less efficient and limit a patient's access to new life-saving developments. But would it at least improve drug safety? Unfortunately, the answer is no.

It's simply not true that more studies make safer medicines. Over the past 40 years, the percent of medicines withdrawn from the market because of dangerous side effects has been essentially constant at about 2 or 3 percent, even as the number of required clinical studies -- a current average of 120 per drug approval -- has mushroomed.

In fact, improper use of otherwise-safe medicines remains the problem. Common over-the-counter painkillers such as aspirin and ibuprofen cause thousands of hospitalizations and hundreds of deaths each year.

There is an ever-widening disconnect between the safety show-trials being held in Congress and the advancing science of drug development. And the growing gap amounts to a valley of death for hundreds of thousands of patients who suffer from untreatable or incurable diseases. Consider the fact that only two drugs with new approaches to fighting infections have been approved in 40 years. The FDA is using computerized approaches to help determine which drugs work against which infections in an effort to beat back growing antibiotic resistance and speed drugs to market. But some in Congress want to micromanage how drug studies for antibiotics are conducted. They are insisting that the FDA revert back to evaluation methods scientists applied 50 years ago. Over the next decade, millions more will die because of an absence of new antibiotics, which will be caused by such "reforms."

Recently an Alzheimer's vaccine was shown to reverse the progress of the disease in the brain. But the FDA held up a similar vaccine for two years because it caused temporary swelling of brain tissue in about 5 percent of patients. What drug reviewer would take any risks if a Drug Safety Board agency was second-guessing its decisions?

The real problem with drug development is not the loudly proclaimed issue of drug safety but the growing shortage of newer, more effective medicines that can treat or prevent the most costly and devastating diseases. The problem, as the FDA's Dr. Scott Gottlieb notes, "Is... the development process itself is not keeping up at a fast enough pace to match the progress on the discovery end." This effort to adopt these approaches to drug, device and diagnostic development and increase the number of products hitting the market to improve health was dubbed the Critical Path Initiative by the FDA.

For instance, the use of biomarkers (a biological measure of response to a drug or disease progress) with links to health outcomes could shave years and billions of dollars off drug development. The new science produces medicines which are more personalized and better targeted to genetic profiles. Because biomarkers also screen out toxicities before medicines are marketed and identify who responds best, they are demonstrably safer and more effective. New computer models and imaging tools can predict not only the onset of illnesses such as Alzheimer's but also which drugs will work best for which patients.

The choice is this: Support the Critical Path and develop a new generation of safer, targeted medicines more quickly for the growing number of us who will be diagnosed with Alzheimer's, cancer and other diseases in the decades ahead. Or, slow down the current approach to drug development with paperwork and studies that discourage innovation and do nothing to make medicines safer and more effective. The battle over the future of the FDA is nothing less than a showdown over the future of medical progress and our ability to live longer, healthier lives.

Robert Goldberg is vice president for strategic initiatives for the Center for Medicine in the Public Interest. Peter Pitts is president of the Center for Medicine in the Public Interest and former associate commissioner for external affairs for the FDA.