Patient Power
CALFEE, JOHN E.
Patient Power The FDA doesn't have to decide everything. by JOHN E. CALFEE THOUSANDS OF multiple sclerosis (MS) patients got an all-too-familiar message recently: Their lives are ruled by...
...the rest agreed "somewhat...
...Patients want FDA expertise, but not FDA fiat putting valuable drugs out of reach...
...by JOHN E. CALFEE THOUSANDS OF multiple sclerosis (MS) patients got an all-too-familiar message recently: Their lives are ruled by experts...
...In order to discover the preferences of MS patients, I worked with survey researchers at Roper Public Affairs to conduct a representative survey funded by Biogen Idec, one of the manufacturers of Tysabri...
...What we found is spectacular...
...No one thinks they will keep the drug off the market—but the FDA still has to decide which MS patients it will allow to use Tysabri and which patients it will keep from using Tysabri...
...The experts on these questions are the patients...
...It convened an advisory committee of experts...
...The issue was their access to Tysabri, a breakthrough biotech drug with a unique ability to slow down the debilitating progression so feared by Ms patients...
...Far from it...
...But as a cable news anchor said, the FDA decides on the basis of science, not emotion...
...Critics of patient power can correctly point out that people who testify at FDA hearings are not representative...
...Seventy-one percent agreed with the statements, "If a drug has safety concerns, the FDA should warn people, but I should be free to decide with my doctor whether to use those drugs or not," and "I am capable of making my own treatment choices, based on the information and advice I get from my doctor...
...In addition to the usual clinical trial evidence on safety and efficacy, we have exceptionally sound survey data...
...Well, a bunch of them testified at that advisory committee meeting, shufJohn E. Calfee is a resident scholar at the American Enterprise Institute...
...There is some very good news here...
...Nor is it a good idea to burden them with sweeping controls over exactly when the drug is used, although the plan to keep close tabs on PML, the fatal condition that triggered Tysabri's withdrawal, certainly makes sense...
...MS sufferers who are happy enough without Tysabri, for example, are unlikely to travel to suburban Maryland to talk for three minutes before a panel of silent doctors and bureaucrats...
...They unanimously recommended putting the drug back on the market...
...Now the FDA has to decide whether to let the drug come back after new research has verified its potency against MS while also suggesting a 1 in 1,000 chance of the fatal side effect...
...Their views should be absolutely paramount...
...They are expected to reach a decision in the next month or so...
...But they split on the crucial question of whether to recommend using the drug as a first-line therapy instead of waiting until patients fail on the current crop of drugs...
...This is no time to second-guess patients and neurologists about when to start using the most effective method to slow down the otherwise relentless progression of MS...
...He was compensated by Biogen Idec for designing the survey discussed here, but not for analyzing its results or testifying before the FDA...
...The FDA needs to know what patients want, and it should give those preferences very great weight indeed...
...Three-fourths of our sample had seen their neurologist at least four times in the previous two years and usually talked about drug side effects...
...Finally, this is not just about Tysabri and MS...
...But, as it happens, we know quite a lot about what MS patients in general think of Tysabri...
...The issue, after all, is not whether the drug works or whether it sometimes kills people...
...Eighty-one percent said they would be willing to visit their neurologist more often in order to get a better but riskier drug (only 2 percent disagreed...
...In other words, patients who are still early in the progression towards severe disability are about as interested in Tysabri as those who are already using canes and wheelchairs...
...The patients also have strong feelings about FDA regulation...
...The manufacturers pulled Tysabri from the market after two patients died from an unexpected side effect...
...fling about with canes and wheelchairs, and almost all of them begged the FDA to bring Tysabri back...
...In this instance, however, science doesn't have the answer...
...But if it pays enough attention to what patients want, its job looks a lot easier...
...They got it half right...
...What about the patients, you ask...
...Roughly half of MS patients want to take their chances with Tysabri and its potentially fatal side effects...
...This feeling is consistent throughout the MS population regardless of level of disability...
...The FDA should tread lightly in its risk-management plan for Tysabri...
...The FDA has been under so much pressure in the past year and a half to crack down on risky drugs that you might think it would have a tough time with a drug like Tysabri...
...This is not to say the patients want to decide everything all by themselves...
...Experts only, please...
...The FDA did what it usually does in these situations...
...And the committee strongly suggested that the FDA put together a potent "risk management" scheme to make sure Tysabri is used the way the FDA thinks it should be used...
...Then the FDA's own experts took over...
...They are the only ones who can balance quality-of-life against the risk of death...
...When it comes to trading off the risks and benefits of most drugs, the best experts are the patients themselves, while medical experts are just well-informed amateurs...
...It is whether the trade-off is worth it: Is a 1 in 1,000 chance of dying a risk worth running in order to reduce the much-feared "relapses" associated with MS by two-thirds or to diminish the formation of brain lesions by 83 percent...
Vol. 11 • May 2006 • No. 32
