- The Washington Times - Monday, June 13, 2005

Benjamin Disraeli observed, “In politics nothingiscontemptible.” He obviously never had the chance to watch Rep. Edward Markey in action.

Mr. Markey has made a career of over-regulating fairly well-functioning private enterprises. Indeed, he once proposed (and I am not making this up) to establish a national G-force limit on roller coaster rides. Now he has decided that the process that has allowed drugs for AIDS, cancer and Alzheimer’s to reach patients faster — called accelerated approval — is “now broken and fails to protect patients.”

How did Mr. Markey come to this conclusion? By claiming that the FDA and companies have failed to complete studies that look at the impact of these medicines after they are on the market, in a timely fashion. And to rectify what Mr. Markey calls the “Conspiracy of Silence” he would impose a fine of a million dollars for every day a study is late. He would impose penalties for any safety problems that crop up before the studies are complete. In addition, he would require drug companies to state that the safety and efficacy of these products have not been proven.

The Markey proposals are — like his national G-force limit initiative — a solution in search of a problem. Accelerated approval by definition is only used for life-threatening disorders. Starting with AIDS drugs, medicines for such illnesses have been approved without long-term studies if there is evidence that the biological change brought about by the drug is linked to improving or extending lives. For example, HIV drugs were never shown to increase life expectancy outright. Rather, they reduced the amount of HIV virus present in blood tests, which was considered a surrogate indication for being able to keep people healthy and live. For cancer or rare diseases, the shrinkage of tumors or estimates of how much better off people would be compared to giving them no drug at all or a placebo is the endpoint. Thereafter, companies are expected to conduct full studies comparing the drug to a placebo to actually measure survival or other benefits.

Mr. Markey uses fuzzy math to conclude that 50 percent of follow-up studies have not been completed. Actually, only 4 percent of the nearly 80 studies for drugs that have received accelerated approval are delayed, though they do take longer than anticipated in many cases.

To be sure, once breakthroughs for life-threatening diseases are on the market, it is hard to get patients to enroll in formal studies. As former FDA official Scott Gottlieb notes in his blog (fdainsider.com),”The biggest reason why companies are unable to complete post-market studies after drugs are approved through the accelerated pathway, is that all of these drugs treat life-threatening disorders (they have to in order to qualify for accelerated approval) and dying patients are unwilling to roll the dice on a placebo controlled trial — raking a chance they may get little more than a sugar pill — when the drugs are readily available on the market.” Indeed, there are questions if it is ethical to deliberately deny a potentially effective treatment to a patient for study purposes. But Mr. Markey would make it a crime not to run such trials.

In fact, there are dozens of studies being conducted independent of the FDA. Mr. Markey attacks Novartis for not completing FDA research on time; there have been 30 randomized controlled clinical trials using the medicine. In addition, Bristol Myers Squibb is working on a drug that is tailored to people whose cancer is Gleevec resistant. However, in Mr. Markey’s mind, Gleevec would be labeled as not being proven effective and could be withdrawn from the market. Similarly, Mr. Markey singles out the cancer drug Iressa as an example of a drug that was allowed to stay on the market without studies showing it provided a benefit. In fact, 46 clinical trials using Iressa are underway, as is development of a genetic test to determine who will benefit most from the drug.

Accelerated approval has stimulated an explosion of research into therapies for cancer and rare diseases. Moreover, it has stimulated investment in practical clinical studies to see where these drugs work best. As economist Frank Lichtenberg has shown, people suffering from these diseases have seen the largest increase in life expectancy of any other population in the United States.

Nevertheless, Mr. Markey would change all that. He would suffocate medical progress in order to make a few headlines. He would fine the inventors of innovative medicines for life-threatening illnesses millions of dollars and would deny millions of patients accelerated access to medicines because federal paperwork is not filled out. Maybe we need that national G-force standard after all. Mr. Markey obviously got his idea to punish companies for faster cures from another dimension where political calculations come before compassion.

Robert Goldberg is director of the Manhattan Institute’s Center for Medical Progress.

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