Fast Tracking the FDA

Bart Madden and James Pinkerton suggest a new “free to choose” track for pharmaceuticals. Pharmaceuticals which showed initial effectiveness would be available for early sale but all treatment information under the early-sale program would have to be reported to an open-access database.

After a drug successfully passes safety trials and shows initial effectiveness in clinical trials—that is, the early steps—a drug developer could request that their drug be available for sale on a “free to choose” track (the developer could elect also to continue on the FDA clinical trial track). As a result, patients such as Matt Bellina would be able to access innovative new drugs up to seven years earlier than waiting for a final FDA decision. For patients given only a few years—or months—to live, seven years sooner could spell life, not death.

Under our proposal, a patient’s doctor would be required to submit treatment results and medical information such as a patient’s genetic data to the open-access database. Doctors and patients would get real-time updates about the benefits and side effects of any “free to choose” drug and be able to make informed decisions about an early use of these new drugs versus approved drugs.

We might bear in mind that clinical trials involve patients who are mostly similar. On the other hand, because the “free to choose” option would be available to everyone, new insights would be obtained about how a drug performs for a far broader range of patients. These insights would better inform the biopharmaceutical industry, leading, in turn, to better allocation of research funds and faster innovation.

Bart’s excellent book Free to Choose Medicine has more on the proposal, which I think would speed drugs to patients and increase pharmaceutical research and development. Do note that I hold the Bartley J. Madden chair in economics at Mercatus at GMU and I have my biases.


Well, in theory this is a great idea for everyone: speeding the process in this way would be of potentially great benefit. But as usual the "economists pro-market view" is oh so naive. Small trials are usually far from enough to make an informed reimbursement decision, and dont get me talking about all the statistical issues with small samples which combined with the american tendency to over-hype anything new and well industry wants to make money (and trust me usually NEW = EXPENSIVE)... well you get the picture. From my understanding this argument "it will be available for a broad range of patients" does not stick... most new medicines will likely be cost-effective in a few subgroups of patients, not for the general patient population. In most areas we talk about incremental benefit, not spectacular improvements so the lives saved argument, is also not as strong as it might intuitively seem. In summary: For an outsider it might look like the FDA is very very innefficient (and it is so) but it is certainly not an easy task to replace it. There are lots of trade-offs to consider!

'Small trials are usually far from enough to make an informed reimbursement decision'

Of course they are - just look at Vioxx. It only was only after the large scale trial - fully reimbursed from Merck's perspective - that adverse effects were not only noted, but actually taken into account.

'There are lots of trade-offs to consider!'

Well, if one could just reduce that pesky American liability frameword AND the FDA as market barrier for any product that a pharmaceutical company wishes to sell in the U.S. Luckily, the Heartland Institute is all over this - for example, any newspaper was likely more than welcome to use this text, without attribution, as 'reporting' (after all, this is most definitely the sort of thing any PR dept does - provides lots of essentially ready to print text to any outlet that will print them -

Vioxx was never taken off the market by the FDA: it was taken off the market by a jury. The FDA still thinks that the benefit/side-effect tradeoff is worth it for Vioxx. The increase in heart attack risk is minor (it took large scale studies to be discovered) and the alternative long-term pain management drugs all have even worse issues, so it may be better to manage the heart risk and take Vioxx.

So, when you actually look at the evidence, Vioxx is an excellent example for the 'free to choose' camp and for the camp that argues that FDA's incentives are overly weighed towards caution (there is a large overlap between the people who hold these two positions, but are logically distinct).

And will the manufacturer be free to advertise this "free to choose" drug in thousands of spots on CNN? And will it be required to state in such ads that "effectiveness has not been validated in controlled trials against either a placebo or another medication"? Will it ever be required to sponsor controlled trials? Will it be allowed to create a spurious and possibly long-lasting public impression of effectiveness?

This is a great first step, but like many proposals doesn't go nearly far enough. If there is a chemical I would like to try to cure myself, and with full knowledge I want to make it or pay someone to make it for me, why put either of us in jail? Why make someone wait an unnecessary three years even if it is better than ten?

I support the reporting requirements, that is a minor burden and in the public interest. There is no reason to interfere with liberty after basic safety testing is completed, and even then since the test needs to be on someone why not let people opt-in? In any event, basic safety testing need to be adjusted so it takes no more than one month and costs no more than 10k, opening medical innovation to the kind of new ideas and competition seen in every other sector.

You have no idea what is going on here, otherwise, you would not state such a naive "pro-liberty" position. It is much more complicated than that c'mon...

OOh now i see where u come from (website).... well never mind...

The real question--what do experts in medical trials and pharmacology think the downside of this approach is? Just because it satisfy a political philosophy does not guarantee the benefits outweigh the risks.

The major potential downside to this is that making experimental treatments available to all can make it harder to recruit patients for the clinical trials necessary to rigorously test the treatments, since patients in a clinical trial will not all get the treatment. It's sort of a Gresham's Law of clinical trials, bad experiments taking all the patients away from the good experiments. This has been seen before, with autologous bone marrow transplants for cancer. From The Emperor of all Maladies:

"But as autologous transplantation for cancer exploded into a major enterprise, the scientific evaluation of the protocol fell further and further behind. Indeed the trials were caught in an old and perverse quagmire. Everyone — patients, doctors, HMOs, advocacy groups — wanted trials in principle. But no one wanted to be in trials, in practice. The more health insurance plans opened their floodgates for bone marrow transplantation, the more women fled from clinical trials, fearing that they might be assigned to the nontreatment arm by what amounted to a coin flip. Between 1991 and 1999, roughly forty thousand women around the world underwent marrow transplantation for breast cancer...Meanwhile, patient accrual for the clinical trials, including Peters's trial at Duke, nearly trickled to a halt. The disjunction was poignant. Even as clinics overflowed with women being treated with high-dose chemotherapy and wards filled their beds with transplanted patients, the seminal measure to test the efficacy of that regimen was pushed aside, almost as if it were an afterthought."

The data from a large and diverse treatment population could indeed be useful, but if I have to choose between having that data or having a rigorously controlled clinical trial, I'm taking the trial every time.

Oh, and autologous bone marrow transplants turned out to be [useless.](

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