May 23rd, 2008 by Dr. Val Jones in Expert Interviews, Health Policy
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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.
May 20th, 2008 by Dr. Val Jones in Medblogger Shout Outs
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Blogging is a social hobby that has brought me many new friends. I suppose one could say that blogging is a modern form of “pen pals” – only more public, and with faster responses. The medical blogosphere (comprised of people who blog primarily about health topics) is small but growing rapidly. Those who have been blogging for 3 years or more have achieved a kind of patriarchal/matriarchal status within our peer group, and they can be depended upon to serve up interesting facts, commentary, and true life stories on a nearly daily basis.
Although I read about 70 medical blogs regularly, I have met only 2 of these bloggers in person. I met Ramona Bates, physicist, plastic surgery blogger, and quilter extraordinaire in Little Rock, Arkansas last year and even observed one of her surgeries in the O.R. She took me to a fried catfish bar with her hubby and I met her affable chocolate lab, Rusty – a sizeable dog who doesn’t take direction from strangers.
I have been a big fan of Dr. Rob Lamberts’ Musings of a Distractible Mind. Of all the blogs I read, his is probably the least predictable. Interspersed between insightful commentary of health IT and helpful clinical pearls, one will find diatribes about accordians, dancing-goat exposés, and warnings about mutant cucumbers. For those who arrive at his blog looking for health content, yet unaware of Dr. Rob’s tendencies, I can imagine that certain posts might leave them with a quizzical feeling.
So I wasn’t terribly surprised when Dr. Rob arrived at Revolution Health late one Friday afternoon carrying a small grocery bag containing a plastic lobster. My astute colleagues realized at once that this was the sign of a man who needed a beer – and he was immediately offered one at our in-office happy hour. I waited a few minutes for the beverage to take effect before I inquired as to the purpose of the lobster.
Dr. Rob has named his lobster “Zippy,” and plans to have him sent around to various medical bloggers to be photographed in interesting places. He has created a website for these photos, and has a strategy for raising money for brain cancer associated with Zippy’s travels. I was honored to be one of Zippy’s first stops on the blog trail and will need to find some interesting photo settings for him in DC.
Currently dear Zippy is sitting on my desk top, confirming to colleagues that I must indeed be a woman of rather ecclectic decorative office tastes. I certainly hope that Zippy will develop a cult following for his good cause.
The rest of my time with Dr. Rob was most enjoyable – I had no idea that he was a Rochesterian (i.e. from Rochester, New York) as I have a secret fondness for people from that part of the world (hey, I married one!) We had dinner at a local Inn and Dr. Rob and my hubby gleefully discussed health IT interoperability initiatives. I listened with interest, and toasted to a couple of guys trying to make healthcare better for Americans – lobsters and all.This post originally appeared on Dr. Val’s blog at RevolutionHealth.com.
May 9th, 2008 by Dr. Val Jones in Uncategorized
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Revolution Health is always looking for creative ways to bring important health information to the public. This week, in honor of Mother’s Day, we’ve created a virtual women’s health expo – and we’ll donate to the featured non-profit organizations for every click you make.
So why not do some good for the following non-profits by visiting their booths at the virtual expo? This is the easiest way for you to support your favorite non-profit – you can even visit them in your pajamas. 
Y-Me National Breast Cancer Association
Depression and Bipolar Support Alliance (DBSA)
National Healthy Mothers, Healthy Babies Coalition
National Alliance on Mental Illness (NAMI)
National Women’s Health Resource Center
The Wellness Community
Women’s Sports Foundation
Go Red For Women
Healthy Weight For Life
Society for Women’s Health Research
*Go to the Women’s Health Expo*
This post originally appeared on Dr. Val’s blog at RevolutionHealth.com.
May 6th, 2008 by Dr. Val Jones in Patient Interviews
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Cameron J. Mitchell was diagnosed with a glioblastoma multiforme (or GBM, an aggressive form of brain cancer) over four years ago. The prognosis for this tumor is usually grim, as most people succumb to it within 4 to 18 months of diagnosis. However, Cameron got involved in a clinical trial for a promising new therapy and has beaten the odds. Since Revolution Health was founded partly in response to Dan Case’s battle with a GBM (and I had the opportunity to participate in the Race for Hope to benefit Accelerate Brain Cancer Cure last weekend), I thought it would be great to highlight Cameron’s story. Cameron also happens to be a close friend of one of our staff at Revolution Health.
Dr. Val: Tell me about how you were diagnosed with a brain tumor. What symptoms did you have? What led up to the diagnosis?
First of all as you can imagine getting the news is like being hit by a truck. You think, “this just can’t be happening to me.” And of course the “why me?” scenario comes into play.
My initial diagnosis came after having a bout of seizures, both grand mal and partial. After a series of tests and procedures they determined that the ongoing problem was the result of a brain tumor. The typical protocol requires 6 weeks of radiation along with oral chemotherapy followed up with a visit to your oncologist to determine a plan of action.
Dr. Val: How did you find out about the Duke clinical trial?
Being that the “plan of action” did not have a very favorable outlook I asked my oncologist, “What would you do in this case?” He suggested I contact the Duke Brain Tumor Center. He actually made the initial call. Later that night I received a call from Dr. Henry Friedman saying that they could help. By the end of the week I received a call from Dr. John Sampson telling me that he was working on a specific clinical trial. As long as I could meet some prerequisites I would be eligible to participate. A lot of thought and pondering goes into that decision: “Is it the right one?” “What if it does not work?” But at that point nothing else had been proven, so my wife and I decided that I have nothing to lose to give it a chance.
Dr. Val: What have the treatments been like for you? Do you have any advice for other patients with GBM?
The treatments are an ongoing, once a month procedure, and I travel from Michigan to North Carolina for them. So it definitely is a huge sacrifice but at for very worthy cause. Having MRIs every two months can create a lot of anxiety not knowing if the tumor is back or not.
The four main pieces of advice I could give to new GBM patients are: 1) Get as much as information as you can. Ask lots of questions. 2) Keep a positive attitude (SO IMPORTANT). 3) Keep a strong group of supporters like family, friends and especially spouse. 4) Remember that this is NOT your fault.
There are a lot of very good sites out there on the internet for advice. But one thing to keep in mind, some of them are a little outdated. The better sites allow you to ask questions.
Dr. Val: What advice do you have for family members and loved ones of those diagnosed with a GBM?
Family members must be informed about what is going on. Under absolutely no circumstances should they be left out in the cold. Most patients find out early that their spouse has at least as much (if not more) difficulty dealing with this disease as the patient.This post originally appeared on Dr. Val’s blog at RevolutionHealth.com.
April 30th, 2008 by Dr. Val Jones in Health Tips
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May is skin cancer awareness month, and Revolution Health has created an awareness campaign to help people become more educated about their risks. In a unanimous vote, I was selected as the blogger/spokesperson for skin cancer awareness – probably because I’m “the fairest in the land.” Well, the truth is I’m so white I’m actually closer to light blue – couple that with a high freckle count and green eyes and you’ve got one very high risk lady.
So I’ve decided to see a dermatologist once a year for a full skin check. I must admit that the first year I went I was convinced that I’d be biopsied into oblivion. The only way to be sure that a mole is not cancerous is to take a sample and check it under the microscope. So any doctor with a conservative eye would need to do a lot of “rule out melanoma” testing, right? Wrong. I was happily wrong. Dermatologists are trained to recognize individual freckle and mole patterns, and don’t do a biopsy unless they see an “ugly duckling” mole – one that stands out from all the others. I was so excited to escape the office with my skin in tact that I vowed to be obedient and return for a yearly check up.
If you are fair skinned and/or have had a significant amount of sun exposure in your life, or if people in your family have had skin cancer, you should definitely check in with a dermatologist to make sure you don’t have any suspicious moles. The doctor will tell you how frequently you should have follow up exams.
Here are some things you can do right now:
Find out if you’re at risk for skin cancer and learn what you can do to prevent it.
See what skin cancer looks like.
Check out my recent interview with Dr. Stephen Stone, past president of the American Academy of Dermatology, about skin cancer and about tanning salons.
Coming soon: the true story of my blogger friend who had a basal cell carcinoma removed from the side of her nose. She required plastic surgery to fill the gap, but it looks great now!This post originally appeared on Dr. Val’s blog at RevolutionHealth.com.
