The FDA is often criticized for the length and ardor of its drug approval process. Its critics often point to lives that could have been saved by drugs approved more quickly, which although quite possibly true, does not necessarily warrant significant reduction of the entire process, as well as the administrative costs of trials that last approximately ten years on average. While these concerns are valid, they address the effects of the process and lose sight of the FDA’s self-proclaimed mission: “protecting the public health by assuring the safety, efficacy, and security of human and veterinary drugs.”
The second part of the mission statement, “efficacy,” is a more recent change in policy, dating back to the 1962 thalidomide tragedy. Perhaps there are some who believe this mission should never have been changed, that the FDA should solely seek to determine drugs that do no harm and not concern themselves with drug efficacy. While this is a reflection of a general, understandable disinclination to larger government, the case for small government and consumer discretion simply does not hold for drugs given the gravity of the issue. Even if all approved drugs were safe, lives would still be in the balance because selecting one over another would affect the treatment and prognosis of the patient. There are so many treatment choices to be made already that most physicians would probably agree having more alternatives would not be beneficial, but would rather overload the patient with information and increase the risk of suboptimal choice. Perhaps it would make more sense from a strictly economic standpoint to widen the drug market even further and place more responsibility on patients to inform themselves, but the medical reality is that treatment would be more useful overall if physicians and patients made choices from a limited number of highly effective drugs rather than from a large pool of drugs of unknown efficacy.
As unappealing as the huge amounts of time and money required to approve drugs may be, these elements are essential to the utility of the drug approval process. The effect of time that drugs have on patients simply cannot be simulated, and side effects of drugs can take years to manifest themselves. If drugs are going to be tested, it is unreasonable and misleading to obtain approval on the basis of results from an overly restricted time course, and would certainly result in more undesired side effects and drug recalls. In a similar vein, reproducible results in large samples must be obtained if the drug is to be approved with the intention of reliable, general efficacy. Naturally, however, no great number of people volunteer for an entirely untested drug, so only those few who have limited other options will choose to do so initially. Evidently not only is a long time course necessary, more than one trial is needed if effective, reproducible effects are to be achieved.
It seems clear that the FDA drug approval process is not too long or too stringent as many of its critics profess, but rather a necessary measure to ensure the safety and efficacy of its drugs. The real problem is not an excessively lenient system that limits new effective drugs, but rather an insufficiently selective one that allows the development of only marginally more effective drugs. As the British Medical Journal reports, “more relevant than the absolute number of new drugs brought to the market is the number that represent a therapeutic advance.” The mission statement of the FDA aspires to efficacy, but not explicitly to progress in efficacy. “Covering a roughly similar time period (1974-94), the industry’s Barral report on all internationally marketed new drugs concluded that only 11% were therapeutically and pharmacologically innovative. Since the mid-1990s, independent reviews have also concluded that about 85-90% of all new drugs provide few or no clinical advantages for patients.” The question is why so much time and effort are poured into researching, producing, and marketing drugs that are only marginally more effective. The answer? Pharmaceutical “companies use “’clinical trial administration, research publication, regulatory lobbying, physician and patient education, drug pricing, advertising, and point-of-use promotion’ to create distinct marketing profiles and brand loyalty for their therapeutically similar products. Sales from these drugs generate steady profits throughout the ups and downs of blockbusters coming off patents.” This type of behavior that ostensibly has profit and not the patient as the priority is propagated by “the low bars of being better than placebo, using surrogate endpoints instead of hard clinical outcomes, or being non-inferior to a comparator, allowing approval of medicines that may even be less effective or less safe than existing ones. Notable examples include rofecoxib (Vioxx), rosiglitazone (Avandia), gatifloxacin (Tequin), and drotrecogin alfa (Xigris).”
The main problem with the FDA drug approval process appears to be not its length, but rather the loosely interpreted mission statement of efficacy and the excessively lenient implementation concerning it. The question of how this cycle of enormous investment in creating marginally superior drugs in order to ensure profit arose is perhaps linked to the Prescription Drug User Free Act or other legislation that in essence has companies pay fees to the FDA for their own regulation, which seems like a dangerous conflict of interest. However this problem was created, the mission of the FDA must be clarified as one that provides for drug safety and efficacy not only for immediate success, but for longitudinal progress, if patient health is to be held as the chief objective.