Sen. Sam Brownback (R-Kans.) and Rep. Diane Watson (D-Calif.) held a press conference on May 21 to announce the introduction of the Access, Compassion, Care, and Ethics for Seriously Ill Patients Act. The ACCESS Act seeks to increase terminally ill patients’ access to promising treatments in the investigational phase of Food and Drug Administration (FDA) approval.
I had the chance to interview one of the speakers at the press conference, Emil J. Freireich, M.D., the director of the Adult Leukemia Research Program at M.D. Anderson Cancer Center at The University of Texas. His opinions are quite provocative.
Dr. Val: You’ve expressed frustration with the FDA’s cancer drug approval process, especially as it pertains to terminally ill patients and investigational drugs. What’s the source of your frustration?
Dr. Freireich: The problem with the FDA and the research process in this country is that it’s too risk-averse. Twenty-five thousand Americans die in car accidents each year, but we don’t ban cars. Five-hundred thousand Americans die of cancer each year, and the FDA makes it impossible for many of them to get the drugs they need. What’s the sense in that?
The excessive red tape [slowing down the research pipeline] is caused primarily by legislation created at the time of the thalidomide disaster [1957-1961]. They made a mistake in marketing a sedative to an untested population group of pregnant women, and that resulted in the alarming birth-defect consequences. However, because everybody panicked, now the FDA regulates the earliest development of a drug. The whole process of drug approval changed.
Treatment of human beings for disease is the only area of research where the scientists who know about it have to petition the government to begin to do research. I can do research in physics, chemistry, astronomy or any science. But if I’m going to do medical research, I have to petition the government first — even for animal studies.
The FDA should not have power over the Investigational New Drug (IND) process. Scientists should not have to petition nonscientists to do research. The FDA is always seeking more scientists to work for them — but they’ll never get enough because what kind of scientist wants to sit behind a desk and approve someone else’s research? If you’re creative and innovative, you want to do your own research.
Dr. Val: Let me ask you about funding. The health care system has a limited amount of money — how can you justify spending it on investigational drugs for the terminally ill?
Dr. Freireich: Of the $50 million used to bring a drug to market, $49.5 million is used to satisfy regulators. That’s where the money goes. If you are in a pharmaceutical company and you want to get approval for your drug, you have to hire people who used to work at the FDA to figure out what hoops they’re likely to require you to jump through.
And how does the FDA decide how the drug should be developed? By consulting the world’s leading scientists and researchers? No. Regulators make up the rules on a whim. They stipulate things like: “Before you can do human trials, you have to kill 1,000 monkeys in Africa, and then 4,000 rats in China. And if you bring home that data, we’ll be sure to approve the drug. Then pharma goes to venture capitalists to pay for the monkey and rat trials, and the FDA approves the drug for human trials. But to make sure that the maximal benefit is observed in the trial, participants have to be young Olympic athletes with normal kidneys, livers, a full head of hair, nice teeth and a small cancer. Meanwhile all my patients are dying as they’re ineligible to participate.
Take Gleevec for example. Chronic myelogenous leukemia (CML) used to have a median survival rate of 3 1/2 years (90 percent of people were dead in five to six years). Today 90 percent of people with CML have a 10-year survival rate. And that’s the result of just one drug. When we gave Gleevec to the first 10 patients, it was obvious how powerful it was. But we were required to do a randomized trial that took two years, and half the patients were given interferon — which we knew wouldn’t cure them. I had a patient who was on the board of directors of Novartis. He had CML, and he had all the data, and he knew he needed Gleevec. He entered the trial and happened to be randomized to the interferon arm and died. This shows you that the venture capitalists and administration of pharma are powerless. They can only do what these powerful FDA regulators allow.
Some of the regulators are 25-year-old college graduates, and they essentially control the lives of millions of people. All they have to do is sit at their desk and say “no” all day long to trials. Regulators have no incentive to approve drugs for trial because of risk aversion. I could create a cure for cancer, but if one person dies in a trial, then they’d fire the FDA guy who approved it. Then if they’re really smart, they go over to the industry side and get a tenfold pay raise and make more than the researchers and doctors who are trying to save the lives of cancer patients.
Dr. Val: What about all the research that is unregulated? The research in alternative medicines, for example?
Dr. Freireich: The tragedy is that the FDA can’t touch alternative medicine practices. FDA regulation only hinders the legitimate scientists, while the quacks get off scot-free. This is due to the “consequence of the unintended.” The legislation wasn’t intended to control quacks, but it controls legitimate scientists. Why? Because the government funds 30 percent of all research in this country. If I say to my research lab director: “I have a drug that can cure leukemia, but the FDA won’t let me test it — let’s just do test it without their approval.” He’ll respond: “Guess what? Thirty percent of our budget will disappear in five minutes.”
At the same time that I can’t get FDA approval to test promising drug therapies, there’s a quack in Houston who sells urine extracts to cure cancer. Why doesn’t the FDA touch him? He doesn’t accept federal funds, so he can do what he wants. In a sense, the quack movement is indirectly fed by the FDA. If patients could get legitimate treatments from doctors, they wouldn’t be turning to quacks. The problem is that they come to me and I have to tell them that they’re not qualified for clinical trials. The patients we turn away from M.D. Anderson go straight to the quacks. What else can they do? You can either pray, go to a quack or go on the Internet to look for miracle cures.
Tragically, if it takes 10 years to develop a cancer drug, 5 million people die while waiting for it to be approved. That same drug could be developed in one year, but we’re being regulated in areas where it’s not needed. The FDA should be worrying about the drugs we give to healthy people, not worrying about sick people — that’s the doctor’s job.
Dr. Val: Well, what do you suggest we do about this?
Dr. Freireich: The solution is legislation. Why does the public put up with the current IND process? Because most of the public is healthy. Healthy people never envision themselves getting sick. If you’re healthy, you don’t think about cancer happening to you. We need to wake up and support the ACCESS Act legislation.
Addendum: I spoke with Selma Schimmel, the CEO of Vital Options International, a cancer advocacy group, about her perception of the investigative drug process. She said that while she is sympathetic to cancer patients’ eagerness to gain access to drugs, she wouldn’t want them to be harmed by investigative drugs either. Schimmel says that the FDA is in a difficult position in which staff are held accountable for an incredibly high standard of safety — and yet the agency is being asked to push things through quickly.
This ACCESS Act has been a topic of debate for some time and was discussed here.
Another article of interest about FDA and Medicare joining forces to form an early warning drug network was published in the LA Times today.
What do you think of this issue?This post originally appeared on Dr. Val’s blog at RevolutionHealth.com.