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Are FDA Regulators Preventing the Terminally Ill From Accessing Promising New Cancer Drugs?

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

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4 Responses to “Are FDA Regulators Preventing the Terminally Ill From Accessing Promising New Cancer Drugs?”

  1. Ivo Cerckel says:

    Myeloma and thalidomide?

    Human nature is so constituted

    that some individuals will always

    deliberately and unnoticeably cause the serious harm

    thalidomide can so easily cause.

    They do that precisely because the harm is so serious

    and because they can do that so easily and without being noticed.

    Yes, the FDA is terminally ill.

    The House of Representatives also.

    In Defense of OPEC

    May 22nd, 2008 by Ivo Cerckel


    The rule of reason, replacing the per se-approach, in oil-price-fixing cases is the first step towards the complete repeal of the immoral antitrust laws.

    Repeal the FDA!  – Repeal Antitrust Law!

    Ivo Cerckel

  2. Dr. Scherger says:

    Dr. Freireich means well, and many of his points are well taken.  Overall though, I find his comments supporting the expression, we should not let foxes guard the hen houses.  Way too one sided.  Under the current Bush administration, I believe the FDA has been way too influenced by corporate pressures.  Also, the FDA is not a policing agency.  Gullible Americans should be free to ingest animal urine if they want.  We need to be sure our drugs are safe and effective, and cancer patients should not be the experimental cases for the untested.

  3. MyChoice says:

    Thank you, Dr. Freireichand Dr. Jones.  We, prostate cancer advocates, support the ACCESS Act and have created a petition to send to U.S. Representatives and Senators.  Please sign the petition (below) and help us spread the word.  Thank you.

    To:  U.S. Congress and Senate

    Access, Compassion, Care, and Ethics for Seriously Ill Patients Act (S.3046 H.R.6270).

    Congress now has an opportunity to help save and extend the lives of cancer patients and others with serious life-threatening illnesses, thanks to the recently introduced Access, Compassion, Care, and Ethics for Seriously Ill Patients Act.

    The Access Act creates a new approval mechanism called Compassionate Investigational Access (CIA) for patients who cannot wait. Patients receiving a CIA drug must suffer from a serious or life-threatening disease, be out of approved options and unable to gain access to a clinical trial. Patients must also provide informed consent, and allow the collection of clinical data from their experience with the drug, so we will all know more about the safety and efficacy of new therapies before they are approved for wider use.

    House initiating sponsor Representative Dianne Watson (D-CA): “The activism of the AIDS community in the 1990s expedited the marketing to the general public of promising antiretroviral drugs. Sadly, the expedited approval of promising new drugs for cancer patients and patients with other life-threatening diseases does not receive the same attention or expedited approval.”

    A clear example and one of the poster children of “decelerated” approval at the FDA is Provenge, an immunotherapy for dying men with prostate cancer. Over 30,000 men have died since the FDA decision last May to delay Provenge, citing the need for more data. Provenge has shown activity and efficacy in every one of the six trials where it has been studied including three phase three trials. The FDA looked at the phase three trial that showed the median (midpoint) survival benefit was 4.5 months but the mean (average) survival benefit was much better: 34 percent of all men receiving Provenge were alive after three years compared to 11 percent of those who did not. In the last 45 years, chemotherapy is the only medicine approved to treat terminal prostate cancer.

    I urge Congress to support and pass the ACCESS Act (S.3046 H.R.6270).


    The Undersigned

    Click here to sign:

  4. TonyF says:

    Dr. Val,

    Thank you for publicizing the need for the ACCESS ACT! The public needs this information to make an informed decision about their choice to support it or not.

    Dr. Freireich’s example of a Novartis Director needing and being denied a treatment the patient “knew” would work and by the luck of the draw being given the control leading to his premature death borders on criminality.

    And, for a physician, Dr. Scherger’s comments imply a callousness of a patient and specifies high hurdles few treatments can surmount…. “safe and effective”.

    On first reading Scherger’s comments, I actually thought they were from Dr. Howard Isadore Scher of Sloan Kettering. Scher sat on the FDA Advisory Committee which overwhelming recommended Provenge, an immunotherapy for late stage prostate cancer, by a 17-0 vote on safety and 13-4 that it’s efficacious. (Scher voted YES on safety & NO on efficacy).

    To sit in judgment of Provenge, Scher certified to the FDA he had 3 Conflict of Intersts; net research shows he had 17… note items 1 & 17, in particular.

    1. NOVACEA:

    … Grants & Research support;

    … STUDY CHAIR of DN-101, Asentar-since HALTED as ineffective

    … a Direct competitor to Provenge

    2. GPB BIOTECH: Financial conflict of interest per Scher in

    3. PHARMION: Financial conflict of interest per Scher in

    4. SANOFI-AVENTIS: Grants & Research support

    SQUIBB: Consultant, Grants & Research

    6. MILLENNIUM PHARMCEUTICALS: grant of Research support

    7. COUGAR BIOTECHNOLOGY: Principal Investigator; Advisory Board;

    8. INNOVIVE PHARMACEUTICALS: Principal Investigator,
    Scientific Advisory Board

    9. INFINITY PHARMACEUTICALS: Principal Investigator

    10. BIOGEN-IDEC: jointly held stock with spouse

    11. PFIZER: jointly held stock with spouse

    12. GENTA: Scientific Advisory Board (March 6, 2007; since removed , but cached)

    13. CONFOMA THERAPEUTICS: Scientific Advisory Board

    14. DEPARTMENT of DEFENSE: Principal Investigator PC
    Clinical Trials-P1 and P2

    15. AMBRILIA
    BIOPHARMA INC: Principal Investigator PCK3145, Phase I/II

    16. MEDIVATION, INC: Principal Investigator MDV3100

    17. PROQUEST

    … Consultant;

    … Scientific Advisory Board;

    … Limited Partner FINANCIAL interest.

    3 weeks after the FDA issued Provenge an “approvable” letter–meaning no treatment outside clincial trials for late stage (a pleasantry for “terminal”) cancer victims, Novacea inked a multi-million dollar deal with a big pharma; Scher was the Lead Investigator of Novacea’s Asentar clinical trial.

    Ironically, several months later, Asentar’s clinical trial were halted by the medical oversight board… more patients were dying on Asentar than those on placebo and Scher’s clincial trials were halted.

    Yet, Provenge whose side effects are mild, flu-like symptoms for a day or two–unlike the only approved treatment for this class of patients–Taxetore–which kills 1 or 2% of the patients from the treatement not the disease–, has to continue with a 3rd PIII clinical trail which will not finish until late 2009 at the earliest.

    Dr. Scherger, the FDA’s own AC highly recommended Provenge for approval as safe and effective–YOUR stated requirements–and tens of thousands will die awaiting Provenge’s approval.

    Frank Burroughs of the Abigail Alliance sought Eubitrix for his 21 year old daughter who had head/neck cancer. Because it was in clinical trails, Abigail was refused and she died. Yet, several years later, the FDA did, indeed, approve Eubitrix–too late for Abigail. How do you square this?

    The Abigail Alliance has fought for a number of drugs to have “early access” for patients and EVERY drug/treatment they have championed has eventually been FDA “Approved”… not one miss by this advocacy group yet thousands upon thousands have died “hoping” a treatment they need and desire will be allowed to them, but it isn’t.

    83 men die DAILY from prostate cancer totaling some 30,000 men each year, a tragic loss of life for family and society.

    The United States Constitution grants all of us the right to “life, liberty and the pursuit of happiness”; if my physician and I decide a treatment should be tried to extend my life–with good quality of life–I already have that right granted by our Fore-Fathers… so let me have it!

    I proudly support the ACCESS ACT and I hope Dr. Val with continue to follow this story; it cries out to be told honestly and fairly; and, the patients need it NOW!

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