By Robert Goldberg
Published March 29, 2007
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This week there were two bits of news that reminded most of us that when it comes to medicine, the real crisis is not safety but the lack of treatments to prevent or at least battle disease effectively.
First, the Alzheimer's Association released revised estimates showing that the number of Americans with Alzheimer's jumped from 4 million to 5 million over the past decade, a trend which, if left unabated by new medicines, will triple to 16 million by 2030.
Second, Elizabeth Edwards and Tony Snow announced that their respective breast and colon cancers had come back and spread. The likelihood of each's survival depends on a complex combination of factors that are increasingly understood, which has translated into better drugs and longer lives. As Allan Lichter, president of the American Society of Clinical Oncologists, said recently, "in the years ahead, doctors will be able to detect cancer early with a blood test, a blood signature, if you will." The same knowledge used to detect cancers at their earliest stage will be used to develop drugs to treat them at that nascent and intimate level, making them increasingly curable. The same goes for Alzheimer's. If caught early, it can possibly be delayed for years.
Don't count on these breakthroughs anytime soon. It is taking longer than ever to bring new medicines to market. The Enhancing Drug Safety and Innovation Act currently under consideration in Congress will ensure that millions of Americans will die waiting for medicines that politicians purport to protect them from.
The act requires that before any drug is approved, a risk-management program (RiskMap) be created. As former FDA Deputy Commissioner Scott Gottlieb points out, this "legislative proposal extends the FDA's ability to restrict which physicians can prescribe a medicine, and which pharmacies can dispense it." Mr. Gottlieb notes that the FDA now limits RiskMaps to about two dozen drugs with very serious or poorly understood side effects. Getting these drugs is burdensome for patients who want them. Severe penalties await doctors and physicians who prescribe and dispense them outside of FDA guidelines.
But Congress wants to extend RiskMAPs in order to prevent possible rare side effects by limiting who can prescribe new drugs, for what uses and to whom. Under the current legislation, doctors and pharmacists who violate a RiskMap are subject to civil penalties. No doubt being accused will lead to criminal proceedings and personal injury cases. All of this means that the current legislation takes the off-label prescribing of new drugs -- though such prescribing is standard for breast cancer, Alzheimer's and mental illness -- and virtually makes it a criminal activity.
I once supported the act, known as Kennedy-Enzi. That was before its authors decided that the best way to protect the public from adverse drug events is to replicate clinical trial conditions in the real world. RiskMap could create the equivalent of a prescription-drug police state, complete with computerized monitoring by the FDA of who gets what. My friend's father was locked out -- literally -- by a computer from receiving a cancer drug on a clinical trial because his pulse was less than a tenth of second off the interval set by the FDA. No access. No treatment. If the doctor had tried giving him the drug, it would have triggered an investigation and closed down the entire trial.
This approach could be applied to millions of Americans as doctors, hospitals and pharmacists, terrified by the threat of FDA oversight, congressional hearings and lawsuits, are bludgeoned into practicing cookbook medicine. Which health plan would dare to pay for a drug that doesn't meet RiskMap mettle? The legislative push is already slowing drug approvals because FDA drug reviewers are afraid of second-guessing by the media and Congress. A new drug for diabetes-related blindness to be approved this year may now be delayed for three more years of study. A drug to treat gram-negative sepsis that was supposed to be tested against the current medicine must now be tested with a group of patients that get a placebo (a sugar pill) to satisfy the demands of Rep. Henry Waxman. Yet, in many cases, gram-negative sepsis is 100 percent fatal.
The FDA is already delaying accelerated approvals of cancer drugs until dying patients are enrolled in double-blind, randomized, placebo-controlled, post-market studies. Kennedy-Enzi wants to make this practice mandatory. That means no accelerated approval at all for cancer or Alzheimer's.
These restrictions will choke off hope, scare off innovation, give trial attorneys control over the health-care system and deny many of us longer life. The congressional assault on medical innovation is not a presidential campaign issue yet. Perhaps, thanks to the courage of Mr. Snow and Mrs. Edwards, it will be.
Robert Goldberg is vice president of the Center for Medicine in the Public Interest..