Bringing Medical Advances to Patients Sooner

Commentary by Pete du Pont

Some 70 Americans will die today because the Food and Drug Administration doesn't allow existing medical technology to be used to save their lives.

If the FDA approves a product that eventually causes a problem in the marketplace, it gets into trouble. But if the product isn't available in the United States and isn't generally known to exist, the FDA doesn't get into trouble even if thousands of people die for lack of it.

Thus there was no outcry even though 119,000 people died of hypertension or heart disease during an eight-year wait for beta blockers to be approved. Or when more than 2,000 died from heart disease during three years of waiting for Mevacor. Or when 3,500 died of kidney cancer during the three-year wait for Interleukin-2.

The procedures for bringing new products to use in the United States are among the slowest and most expensive in the developed world. In the 1960s it took six years for the FDA to approve a new drug. Now it takes almost 16 years, even with the recently heralded reduction in the time required for final review to just over a year and a half.

It's time to help the elderly and the ill by bringing drugs and medical devices to market more quickly. The Progress & Freedom Foundation has a proposal to do it. It would separate the development and product review process from the FDA's role of enforcing mandated safety and effectiveness standards.

The FDA would license private entities called Drug or Device Certifying Bodies (DCBs), which drug or device manufacturers would retain to oversee their product's development. Since manufacturers could choose among DCBs, they could compete to produce a lower-cost, faster, and higher quality process for drug and device development and review.

More important, the FDA would have 90 days to approve a positive recommendation by a DCB. If it did not act, the use of the drug or device would be allowed. This would be far different from now, when the FDA raises hurdle after hurdle, requires more and more tests, never saying yes, but rarely daring to just say no. The current bureaucratic gridlock would be broken, so that more effective treatments can get to the ill.

There's no reason private entities should not be used to review a drug application. Private entities already perform all functions involved in this process except the final approval step. Currently, the FDA requires sponsors of new drugs to obtain approval to begin clinical trials, and then continues to oversee the sponsor's clinical program through regular reports and other mechanisms. After the clinical studies are completed, sponsors apply for FDA approval. The FDA does no testing, but only reviews the data collected through clinical trials. The FDA would review the DCB's analysis of the data, and could veto approval if it found the new drug unsafe or ineffective.

The approval process for medical devices needs to be changed, too. Currently, if a device is not substantially equivalent to one already on the market, it must go through a complex clinical trial process even if it's a simple device that carries little or no risk. The breast examination pad, for example, is two pieces of plastic with a layer of silicone in between. It has no moving parts, is non-toxic, and in use does not invade the body. But it is illegal in the United States because there is no analogous product on the market. So years of testing loom while women whose lives could be saved by it are forbidden to use it.

Finally, there is the question of liability. Where the treatment of patients with serious medical problems is involved, there are going to be adverse outcomes from the use of drugs and even death on occasion, no matter how responsible the manufacturer and how strict the regulation. U.S. companies are not permitted to defend themselves from lawsuits simply by pleading that they have complied with regulations. As a result, manufacturers of some pharmaceuticals have simply quit making them. Here, we might look to Europe, where regulatory standards protect the patient, but a company is free of tort liability if it can show that the state of scientific and technical knowledge when the product went into circulation was not such as to enable the existence of the defect to be discovered.

The FDA was designed for the 1930s and is bureaucratically incapable of handling the exploding technological advances of medicine in the 1990s. We must change the current system for bringing medical products to the marketplace so that our citizens will receive the full benefits of the dramatic scientific discoveries and medical breakthroughs our nation's vast research establishment is producing.

And they need those benefits while they are still alive.