The FDA is a bottleneck in America's health-care system, creating an enormous invisible graveyard of those who were, are, and will be denied access to new drugs.
Consumer choice would break FDA’s monopoly on access to new drugs. That is the way to turn this problem into an opportunity. The core idea, which should appeal to Republicans, Democrats, and Independents alike, is that we need to be free to make our own informed decisions about whether to use not-yet-FDA-approved therapeutic drugs – that is, new drugs that have successfully passed safety trials, generated preliminary efficacy data, and may offer us the opportunity to improve our health or even save our life. I call this “Free To Choose Medicine.”
Consider how Free To Choose Medicine would have helped those with advanced prostate cancer, which kills 30,000 men every year. Many of those patients literally marched to Washington to rally against FDA’s excruciatingly slow process to approve Provenge, a strikingly innovative cancer vaccine that triggers the body’s natural immune defenses. Patient advocacy groups called for immediate access to Provenge. One memorable advertisement by the advocacy groups was headlined, “Dysfunction at the FDA: Prostate Cancer Victims Face Needless Suffering and Premature Death.”
FDA, with its habitual arrogance and seeming invincibility, is accustomed to ignoring the voices of patients, and it ignored, for many years, the demands of these men.
FDA Will Never Reform Itself
Henry Miller, M.D., a physician, molecular biologist, and public policy analyst who formerly held high-level positions within FDA, gives us his first-hand account of FDA pressures and incentives:
In the early 1980s, when I headed the team at FDA that was reviewing the NDA [New Drug Application] for recombinant human insulin ... we were ready to recommend approval a mere four months after the application was submitted. ... With quintessential bureaucratic reasoning, my supervisor refused to sign off on the approval – even though he agreed that the data provided compelling evidence of the drug’s safety and effectiveness. “If anything goes wrong,” he argued, “think how bad it will look that we approved the drug so quickly.”1
Dr. Miller’s devastating account of FDA decision-making is Exhibit One for the case that FDA is a dysfunctional system. The consensus opinion of economists who have studied the impact of the steady trend of increasing FDA demands for ever-more expensive and time-consuming clinical trials is that the cost to society has far exceeded the benefit. (See www.fdareview.org.)
A system that involves people is ineffective when incentives are misaligned with goals. Consider, for example, FDA’s “compassionate use” program. This is a program for people who have a life-threatening illness and for whom approved drugs are of little use. The more promising a new developmental drug is and the more serious an illness is, the more motivated patients are to gain access via compassionate use and avoid clinical trials. But there is little incentive for FDA to advertise or expand this program, since it competes with FDA’s primary motive, which is to encourage patients to enroll in clinical trials.
What FDA does want is for the public to accept without question that FDA’s role is to provide “safe and effective drugs.” But no drug is 100 percent safe. Even aspirin can lead, especially in elderly people, to hospitalization and death due to gastrointestinal bleeding and perforation. The simplistic slogan of safe and effective drugs diverts attention away from the basic tradeoff issue facing FDA: More extensive and hugely expensive testing may reduce the probability of unanticipated adverse side effects from an approved drug, but at the same time also greatly increases drug costs to consumers and, most importantly, causes suffering and premature deaths from delayed access. That’s the unavoidable tradeoff situation that is better handled by Free To Choose Medicine.
My proposal for Free to Choose Medicine is illustrated in Figure 1.
Figure 1 Dual Tracking
On one track, a new drug continues with conventional FDA clinical trials with the goal of securing FDA approval. On a new, separate track – the Free To Choose Track, which is independent of FDA – patients advised by their doctors make informed decisions to contract with a drug developer to buy a not-yet-approved drug. The drug developer would need to elect to place the drug on the Free To Choose Track, and that drug must have passed the midpoint of its Phase II testing so there would be an early indication of risk and effectiveness. Patients who opt for the Free To Choose Track could gain quicker access to new drugs – by five years or so – compared to waiting for possible FDA approval.
Instead of the current one-size-fits-all regulatory straitjacket that assumes everyone is equally risk-averse, patients could express their own unique preferences for risk, guided by their doctors’ and their own judgments about pain, the limited ability to work or perform daily chores, and the opportunity for health improvement – decisions only they can make. Patients could elect to use only approved drugs, which are very safe. No one is forced to accept less-safe drugs.
Creating a Tradeoff Evaluation Database
For us to be able to judge if the benefit from a new drug exceeds the benefit from an approved drug and is worth the risk – i.e., for us to be informed well enough to be able to evaluate the tradeoff – we and our doctors would need relevant, up-to-date information. Under my proposal, that information would be accessible on the Internet through a Tradeoff Evaluation Database (TED), as shown in Figure 2.
Figure 2 Tradeoff Evaluation Database (TED)
Legislation to implement Dual Tracking should specify that participation in the Free To Choose Track requires not only that doctors input treatment results to TED, but also that patients permit doctors to transmit the patient’s genetic and biomarker information to TED. Over time, this would create a treasure trove of public data that would greatly benefit pharmaceutical research.
Drug developers would be unlikely to make new drugs in clinical testing available through a Free To Choose Track if they could be held liable for all side effects even if they were not negligent in developing, testing, or manufacturing. Consequently, as part of a Free To Choose Medicine Act, Congress would adopt legislation permitting patients to waive their right to sue drug developers under strict product liability as long as developers do not provide false or misleading information.
At no charge, a government-operated TED would provide the information needed to make informed decisions about what is in patients’ best interests. Private-sector companies (e.g., Google, Microsoft, IBM, and such) would have a profit incentive to sell customized “consumer reports” that would further help patients and doctors. Consumer reports could pinpoint subsets of patients who are most and least likely to benefit, forecast the probability of FDA approval, and provide head-to-head comparisons of Free to Choose drugs against relevant FDA-approved drugs.
TED would transfer knowledge (and power) to doctors and patients. This could disrupt enrollment in those clinical trials where knowledgeable doctors judge the drug to be tested as superior to approved treatments. The proper response from FDA should be to develop more innovative testing procedures that avoid unethical clinical trials and give top priority to today’s patient needs.
Before and during Phase III testing, TED would make public a vast amount of clinical trial and observational data. Some drugs would show strikingly effective treatment results compared to FDA-approved drugs. Then, many knowledgeable doctors may object to enrolling their patients in Phase III randomized control trials on the ethical grounds of not wanting to subject their patients to the risk of receiving an inferior drug or useless placebo.
In this new environment, the formerly invisible costs of suffering and death due to delayed access to new drugs would become much more visible. Heightened public awareness of the opportunity costs would make things increasingly uncomfortable for FDA. Contributing to this pressure would be the lack of insurance coverage for the new drugs that are in high demand via the Free To Choose Track when they appear to be delivering significant health improvements.
Most private health insurance contracts cover only FDA-approved drugs. Lack of FDA approval could keep people with less financial means from obtaining the life-improving or life-saving drugs accessible via the Free To Choose Track. This is not a situation Americans would want, and we would pressure FDA to get more engaged with the needs of all patients.
One way to help FDA respond is for the Free To Choose Medicine Act to allow the agency to grant conditional approval for a new drug based on a combination of results from clinical trials and Free To Choose Track use. To maintain conditional approval, the developer would have to agree to complete Phase III trials and obtain conventional FDA approval within a reasonable time.
Change Attuned to the Future
There already is a segment of the medical marketplace where consumer choice is much less restricted. Off-label drug use occurs when doctors write prescriptions to be used in ways for which the drugs were not FDA-approved. A leading researcher in the treatment of breast cancer noted, “If I had to use drugs for their approved uses only, half my patients would be dead.”
Off-label drug use is a window into an environment where things happen to best serve today’s patients. The widely acknowledged success of unregulated, off-label prescription use is consistent with expectations that doctors would actively use TED in the future for the benefit of their patients.
We need a drugs-to-patients system that can adapt to a future with an accelerated pace of medical innovation, coupled to the widespread advancement of personalized medicine. Diagnostic testing will match patients according to their genetic makeup with drugs that are much more likely to work and to have fewer adverse side effects. In this environment, early access becomes more and more beneficial over time. Dual Tracking would accommodate early access as well as dramatically speed up the delivery of medical advancements.
With the Free To Choose Track, a new drug showing strongly positive results would lead to a surge in use by patients with diverse characteristics that more accurately reflect the general patient population of drug users than do the patients enrolled in FDA’s randomized controlled trials. Consequently, upon receiving FDA approval, drugs would have a more reliable safety profile if they were used by patients on the Free To Choose Track. Consumers who choose to use only approved drugs would benefit from the voluntary decisions of those willing to accept more risk in exchange for early access.
A quite plausible forecast is that prescription drug prices would drop substantially after feedback about how well patients do when they opt to make their own choices for approved versus not-yet-approved drugs. Such feedback would be expected to compel FDA to radically streamline its testing process, thereby providing a major reduction in regulatory costs for drug developers.
To sum it all up, Free To Choose Medicine would provide faster access to new drugs, save countless lives, and end needless suffering. It would put us on a competitive path that both lowers prescription drug prices and advances innovation – exactly opposite of a price control path to lower drug prices that some health care reform advocates are pushing. Their option would seriously reduce long-term innovation.
Passage of a Free To Choose Medicine Act would be a defining moment for America – a directional change from today’s trend of increasing litigation and regulation – a stake in the ground that control of medical decisions belongs, first and foremost, with individual patients and their doctors, and not the government. It deserves a chance.