The history of the U.S. Food and Drug Administration (FDA) is one of relentless growth in regulatory power. Growth spurts typically occur on the heels of well-publicized safety issues, such as the recent Vioxx situation.
No one really knows what the optimum structure and level of FDA regulatory power are, but problems in the system make it clear the current level is not it.
The optimum level of regulation depends on the tradeoff decisions (risk versus benefits) individuals are willing to make.
If Congress were to debate the FDA’s regulatory role seriously, a major focus should be the common-sense principle that medical decisions rightly belong first and foremost with patients and their doctors.
Lengthy Approval Process
A second focus should be on the harm done by the long delay before therapeutic innovations reach the public because of the FDA’s lengthy drug-approval process. In Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach (May 2006), a divided panel of the U.S. Court of Appeals for the District of Columbia Circuit supported this principle by affirming a dying individual’s right to access to drugs not yet approved by the FDA.
Streamlining the number of clinical trials required by the FDA could reduce drug-development costs, leading to lower prices. Undoubtedly, some in Congress may notice the FDA’s one-size-fits-all approval procedure is not attuned to the fast pace of twenty-first century medical innovations.
A third focus should be on solving an emerging dilemma facing pharmaceutical companies that are gaining insights into how diseases (often rare diseases) relate to patients’ genetic profiles. The dilemma is that the greater the gain in personalizing medicine, the smaller the target population for such drugs, the smaller the prospective revenues, and the less likely there will be a worthwhile return on investment, due to the high costs imposed by the full set of FDA clinical trials.
Individual Preferences
An alternative that deserves debate is that existing patients, not future patients, should be the first consideration. Allowing individuals to express their preferences regarding whether to take drugs not yet approved by the FDA would undermine the agency’s drug-access monopoly.
In its defense, the FDA says it needs total control in order to benefit society–meaning future patients–by applying rigorous statistical evaluations to its extensive clinical trial data. According to the FDA, patient/doctor freedom to use not-yet-approved drugs would interfere with clinical trial enrollment. Finally, there is an unspoken FDA assumption that patients and their doctors are not capable of making decisions about experimental drugs.
Breaking the FDA’s monopoly through competition would compel the FDA to develop new ways of analyzing a broad spectrum of information.
For Congress to push forward on the core principle of patient/doctor control of medical decisions, it will be helpful to understand how the current system could be changed to let the optimum level of regulation surface. Details of how this transition might occur are presented in my Heartland Institute white paper, “A Visible Hand for Market-Based FDA Reform.” The most critical points are summarized below.
Optimum Regulation
The task is to create legislation that enables patients and their doctors, drug developers, and the FDA to evaluate continuously what best meets their needs and to develop better ways of doing things. In this way, a process will evolve for accelerating medical solutions in an increasingly effective manner.
The key to success or failure in empowering individual preferences is information–the type of information, how it is disseminated, and to whom.
Approaching the regulation problem from the perspective of patient/doctor choice leads us to the need for a government-operated Tradeoff Evaluation Database (TED). In the TED would reside both clinical trial data and non-clinical trial data of not-yet-FDA-approved drug results and side effects. Importantly, just as auditors are independent of the firms they audit, the TED must operate independently of the FDA.
Patients and doctors could use the TED’s continuously updated information to decide whether to try an experimental drug that has successfully passed the FDA’s Phase I safety trial.
Liability Issues
A troublesome obstacle for drug developers who want to provide non-FDA-approved drugs is the fear of being sued by people who experience adverse side effects. To prevent this problem, legislation needs to lay out explicitly the minimum acceptable information about an experimental drug deemed adequate to inform patients about risks and potential benefits.
Although the construction and operation of the TED would most likely be contracted to a private-sector company, the government would have oversight to ensure adequate information is available publicly. To avoid lawsuits, drug developers would have to promptly and honestly report all not-yet-FDA-approved drug use outcomes, including all adverse side effects.
Because it would remain unchanged, the traditional FDA clinical trial process would be a counterbalance to this new system. Patients who preferred the least risk could await FDA drug approvals. Those who are on death’s doorstep, by contrast, could access the TED to determine the most promising experimental drug and most likely would choose to use it.
Anyone in the gray area between these two poles could access the TED to help inform their tradeoff decisions on risk versus potential health improvement.
More Information
A TED Web site would receive details from doctors about patient treatments, and this information then becomes available to both drug developers and the public. This would help their patients, and likely other doctors, in making better medical treatment more widely available.
Implementation of this TED proposal would reveal how well or poorly patients fare when they choose to bypass the FDA’s advanced clinical testing process. Other patients would quickly learn about the outcomes and be able to make more informed choices for either experimental drugs or approved drugs. As a result, the total use of approved versus not-yet-approved drugs would be the aggregate of individual patients’ decisions on what is in their own best interest.
Unfortunately, the FDA would certainly strenuously oppose this opportunity for greater patient/doctor control, even though the results would be uniquely useful for improving its own testing and approval procedures.
However, greater access to information and freedom of choice to try to improve or save lives is an extraordinarily powerful rallying call with genuine potential for cooperation across political parties.
Bartley J. Madden ([email protected]) is an independent researcher in Naperville, Illinois.
For more information …
“A Visible Hand for Market-Based FDA Reform,” by Bartley J. Madden, December 2006, is available through PolicyBot™, The Heartland Institute’s free online research database. Point your Web browser to http://www.policybot.org and search for document #20546.
“A Clinical Trial for the FDA’s Clinical Trial Process,” by Bartley J. Madden, December 2005, http://www.LearningWhatWorks.com
