A Common-Sense Solution to Drug Approval Delays

Published May 23, 2007

Clearly, people have different preferences for risk-taking and unique health conditions–yet we are stuck with a one-size-fits-all Food and Drug Administration regulatory system. Why not give patients and doctors the right to evaluate for themselves the risk and potential health improvement from experimental drugs and the right to choose whether to use these drugs?

A dual tracking system would be a common-sense solution to the problem of excessive government control of medical treatment. On one track, a new drug would continue along conventional FDA clinical testing procedures. On a new track (only after successful completion of FDA Phase I, toxicity and safety evaluations), drug development firms would have the option to legally contract with consumers (individual patients advised by their doctors) to sell them a not-yet-FDA-approved drug.

To function successfully, dual tracking requires consumers be fully informed of the possible risks of using not-yet-FDA-approved drugs. This would be the function of a Tradeoff Evaluation Database (TED). The database would contain clinical and non-clinical trial results (including side effects) of not-yet-FDA-approved drugs. TED’s continuously updated, online information could be accessed by patients and their doctors to decide whether to try an experimental drug that has passed FDA Phase I safety trials.

Implementation of dual tracking would reveal how well or poorly patients fare who choose immediate access to experimental drugs. Other patients would soon learn about the outcomes and 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 decisions.

Under dual tracking, the traditional FDA clinical trial track would remain unchanged, enabling patients who prefer the least risk from unknown side effects of a developmental drug to await FDA drug approvals. Those who are on death’s doorstep, however, could access TED to determine the most promising experimental drug and most likely would choose to use it. Anyone in the grey area between these two poles could access TED to help make their tradeoff decision on risk versus potential health improvement.

To maintain its regulatory monopoly, FDA would probably oppose this opportunity for patient/doctor control, even though the results would be uniquely useful for improving its own testing and approval procedures.

A troublesome obstacle for drug developers who want to provide not-yet-FDA-approved drugs is their fear of lawsuits from people who experience adverse side effects. If left unchanged, the threat of litigation would undermine dual tracking. To prevent this problem, legislation needs to define and require the minimum acceptable information about experimental drugs deemed adequate to inform patients and doctors about risks and potential benefits.

To avoid lawsuits, drug developers would have to promptly and honestly report all outcomes, including all adverse side effects, from not-yet-approved drug treatment. Although the construction and operation of TED would likely be contracted out to a private-sector company, the government would have oversight to ensure adequate information is available publicly. Moreover, just as auditors are independent of the firms they audit, TED must operate independent of FDA.

It is reasonable for us to expect our elected representatives to seek a better world in which patients and doctors control medical treatments and priority is given to existing patients. The dual tracking system would achieve this end. Specifically, dual tracking would bring about:

  • greater freedom of choice for medical patients;
  • faster feedback on the safety and effectiveness of new drugs;
  • a higher rate of new drugs made available to doctors and their patients;
  • access to unregulated comments and ideas from doctors, which can lead to insights with immediate, practical benefit;
  • a fundamental shift in the pharmaceutical industry, wherein skill in developing drugs that deliver a new standard of care is valued to a far greater extent than skill in navigating the FDA bureaucracy; and
  • the possibility of more streamlined FDA clinical trials, resulting in a huge decrease in costs to drug developers, dramatically lower drug prices for consumers, and ultimately healthier and longer lives.

The most powerful argument for dual tracking, one that has appeal across political affiliations and every other possible source of disagreement, is that individuals and families should be free to improve or save a life, even if doing so incurs some risk. The current regulatory regime is profoundly at odds with this simple and compelling idea, and that calls out for genuine reform.

Bart Madden ([email protected]) is an independent researcher in Naperville, Illinois and a policy advisor for The Heartland Institute. His Web site is http://www.learningwhatworks.com.