The following is a guest post by author Kira Peikoff, a former guest blogger here at NELB, and author of the new book, No Time to Die:
In my new book NO TIME TO DIE (Kensington), a young woman has inexplicably stopped aging, and researchers believe that the key to unlocking the genetic fountain of youth lies in her DNA. The story is ripped from the headlines—in an unprecedented new study, scientists are currently comparing the genomes of six people who appear not to age. If researchers do manage to devise a drug that turns off genes underlying the aging process, we could theoretically stay forever young. It’s the ultimate dream, but don’t get your hopes up. The FDA is bound to get in the way, even if the drug could save untold millions of lives. Here’s how I think the scenario might unfold:
Imagine that you are a person suffering from old age. You’ve heard of a new medication being used in Europe to biologically halt aging and are desperate to try it, but you can’t legally get a prescription—it’s not FDA-approved. But you’re dying; you don’t have the time to wait for a regulatory agency to tell you what’s safe.
You’re briefly heartened to learn from PhrMA, an advocacy organization for the pharmaceutical industry, that there are 3,400 medicines in development today in the United States alone, an increase of 40 percent since 2005. You feel hopeful that a drug to help you must be on the verge of coming onto the domestic market--but then you learn that only 82 drugs are undergoing regulatory review at the moment. You learn that it takes an average of 14.2 years to go from discovery to approval, at a cost of more than $1 billion per single indication of a drug. And even then, only 8 percent of drugs reach the stage of filing for approval.
But the FDA’s slogan reads: “Promoting and Protecting Your Health.” The italics on the word ‘your’ strike you as ironic. If they would just let you decide for yourself how much risk you wanted to take, then you could try whatever experimental drugs you want. You might have a chance.
You contact the FDA for help and are relieved to discover that there is a “fast track” program, an expedited process to approve drugs for life-threatening conditions; no one could deny that aging belongs in this category. The number of deaths it causes every year is the equivalent of six Holocausts. But then you learn that the FDA bestowed this fast track designation on a mere 23 drugs in the last fiscal year, none the one you need, and none guaranteed to meet with eventual approval anyway.
Losing hope, you investigate further and learn that an off-label use of an FDA-approved drug has helped buy time for other patients with your condition. You rush to your doctor in a frenzy. Can he find out more information about this from the pharmaceutical company that developed the drug?
No, he tells you. It’s illegal for them to talk to me about anything other than the approved use. If they do, I’m supposed to report them to the FDA through the “Truthful Prescription Drug Advertising and Promotion.”
What is that, you ask?
It’s a program started in 2010 to recruit doctors into being FDA informants, so the latter can collect billions of dollars in fines from companies that tell doctors about off-label uses of their drugs—despite the fact that such uses are a recognized, appropriate reality in medical practice, especially in oncology, HIV treatment, and dermatology.
You’re quite frustrated now. You want to decide for yourself what treatment is best for you, with your doctor’s advisement and his expertise in reviewing the data. You demand to know why don’t you have that right, as a rational, autonomous adult with full capacity?
We need some protective umbrella for the good of society, your doctor replies. The FDA weighs the risks and benefits of drugs based on the clinical trials and then decides what’s safe for the general population.
But risk versus benefit is an inherently subjective judgment, differing from person to person, you point out. How can a government bureaucracy possibly make decisions that are right for all individuals, all the time?
They have to be very conservative, he replies. They have to employ the highest standards so that people don’t get hurt.
Right, you retort. Their standards are so ridiculously high that in May of 2010 they rejected the drug pixantrone for treating non-Hodgkin lymphoma, a blood cancer that kills almost 12,000 Americans a year—even though the drug was a qualified success in clinical trials. Why? Because the trial was not “flawlessly executed” according to their regulations—as if perfection ought to be the standard for a severely needed cancer drug.
Okay, your doctor concedes, their standards can be too conservative. But that’s because the political pressure is intense.
You nod. You are reminded of American economist Walter E. Williams’ explanation of the agency’s modus operandi: “If FDA officials mistakenly approve a device that has unanticipated harmful effects, their necks are on the chopping block because the victims are highly visible. Career-minded FDA officials don’t like that kind of exposure. They prefer the hidden mistake, erring on the side of over-caution by needlessly delaying approval. When FDA officials err on the side of over-caution, their victims are invisible.”
You are one of the invisible victims. You hate to wonder how many more like you are out there.
Just because a drug is granted the seal of approval, you say aloud, doesn’t mean it is actually safe. Just look at Vioxx and Avandia.
The approval process at the FDA is fraught with problems, your doctor admits: There are conflicts of interest galore. Take the birth control pills Yaz and Yasmin, for example, which contain a drug called drospirenone. This drug made women who took it seven times more likely to develop a thromboembolism. It was endorsed by an FDA advisory committee in December 2011, and the FDA approved the drug—even though the agency knew that four members of the committee had received money from the drug’s manufacturer. The FDA not only failed to publicly disclose these ties, but also failed to stop the members from voting; unsurprisingly, all four voted to approve the drug. Now more than 11,000 lawsuits have been filed against Bayer, the manufacturer, accusing it of negligence, strict product liability, fraud and deceit.
You raise your eyebrows. Exactly what the FDA is supposedly there to prevent.
Yes, but think if it wasn’t there at all, your doctor counters. We can’t just have a free-for-all of unregulated drugs and devices. People would get hurt.
You look at him pointedly.
He glances away, mumbling that class action lawsuits are an inefficient way to regulate the system.
First of all, you say, people don’t take any responsibility now for checking into the safety of what they’re taking because the FDA approval lures them into a sense of false complacency. In a better system, people would have more freedom and likewise more responsibility for their own health. Companies would furnish the data on their products for consumers and doctors to examine—not by government force, but out of free market incentive: a company would only be able to sell its drug if consumers elect to buy it, based on their doctor’s advice and on neutral third-party certifications that are not politically motivated like the FDA’s. If a company were found to be dishonest in omitting or distorting information, it ought to be punished in court to the fullest extent of the law. That is the point of the tort system. It’s the only fair way to allow consumers to have freedom of choice, to encourage transparency and innovation, and to lift the burden of ineffective, exorbitantly expensive oversight.
Expensive to whom? the doctor asks.
Everyone, you say. The taxpayers, whose money helps to fund the $4.65 billion budget for fiscal year 2014. The drug companies, who are forced to pay sky-high fees for Investigational New Drug applications, New Drug Applications, expedited reviews, and more--if they can even afford it. Who knows how many companies, hospitals, physicians, investors, and researchers are prevented from pursuing innovations that could save lives due to the prohibitively expensive regulatory barriers? And finally, the consumers—that massive cost of putting a drug on the U.S. market is inevitably passed on to them.
Your doctor says nothing.
Time is running out. You turn to leave.
Wait, he says. I did hear of one experimental treatment that could help you. It was developed by a couple of doctors here in the U.S. and involves adult stem cells extracted from a specific patient, then sterilized and grown in a lab and re-injected into the same patient, which eliminates the risk of rejection.
Sounds great, you say, your hope rising. Why didn’t you tell me sooner?
It was shut down, he says sadly. The FDA decided the stem cells were a “drug” subject to regulation. The doctors fought back with a lawsuit, Regenerative Sciences, LLC v. United States, but lost in July 2012. The U.S. District Court for the District of Columbia concluded that the stem cell procedure was a “drug” because a solution that the stem cells were mixed in contained a drug shipped through interstate commerce, rendering the unapproved procedure subject to federal enforcement. An appeal is pending.
So now the FDA wants to regulate cells from our own bodies? you say, horrified. What does that mean for any future cell-based therapies? Will they have to go through the whole drug approval process, too?
Your doctor shrugs. The plaintiffs have moved some of their labs to the Cayman Islands. Investors are taking note, he adds. According to Henry Miller, a fellow in Scientific Philosophy and Public Policy at Stanford University, a recent survey of venture capital firms indicates that 31% plan to decrease investment in life science companies in the U.S., compared to 7% that intend to decrease investment in Europe. The primary reason given was the United States’ hurdle of regulatory challenges, more specifically the “dysfunction, unpredictability, and risk-aversion at Obama’s FDA.”
You sit down slowly, defeated. Your doctor takes your hand.
I’m sorry, he says. I wish there was something I could do.
But it doesn’t matter. His hands are tied, and so are yours.
It’s too late. Time’s up.