Excerpted from the booklet More Choices, Better Health: Free to Choose Experimental Drugs, by Bartley J. Madden–the last in a several-part series. The complete booklet is available at http://www.heartland.org.
In the current Food and Drug Administration (FDA) environment, information from highly specified and lengthy clinical trials is almost exclusively sought based on its relevance to FDA statistical milestones. This surely is not a broad, open feedback environment conducive to learning, evolving, and speedy allocation/reallocation of drug developers’ resources.
By contrast, dual tracking would involve a diverse group of patients. In this environment, doctors are knowledge resources, empowered to use their medical experience and problem-solving skills to focus exclusively on helping their patients, yet benefit other patients and society as well by sharing information.
Every American family would have Internet access to the Tradeoff Evaluation Database for real-time, continuous updates about the safety and efficacy of all experimental drugs. In a dual-tracking system, patients and their doctors could choose whether to use an experimental drug now, wait for more information, or rely on only FDA-approved treatments.
Dual tracking offers unique opportunities to small drug-development firms with enormous scientific skill but lacking financial resources and/or skill in dealing with the FDA bureaucracy. Such entrepreneurial firms would be able to generate significant revenues and stock market gains if their new drugs are highly effective for early users.
Although some would object, drug developers should be free to set prices as they do for approved drugs. The benefits from obtaining a number of positive-outcome early users would likely be a major factor in initial pricing decisions, which should encourage developers to hold prices down.
Further, scientific skill in discovering breakthrough medical treatments would become more valuable than skill in dealing with the FDA bureaucracy–a skill large drug companies possess far more often than small companies.
Importantly, as for long-term drug prices, if early drug access after Phase I safety trials is successful, that would launch a fundamental evaluation of the enormously costly and time-consuming requirements for Phase II and III clinical trials. Such an evaluation could well lead to streamlined clinical trials, large-scale cost reductions for drug developers, and greatly reduced consumer drug prices.
To get to a world of patient/doctor control, legislation must be designed that will enable the flow of information to allow freedom of choice in medical treatments. It is important as well to facilitate learning and continuous improvement.
An environment of learning and continuous improvement requires an information system that will:
(1) help patients and doctors by providing up-to-date summaries of ongoing clinical trial results;
(2) orchestrate the processing of up-to-date results of experimental drug usage by patients (non-clinical trial data), including adverse side effects;
(3) document that patients, collaborating with their doctors, are informed and capable of assuming responsibility for the use of drugs still in clinical trials, so that good-faith drug developers are protected from lawsuits; and
(4) promote more choice and competition, not only for patients and their doctors but also for drug developers and FDA.
Tradeoff Evaluation Database
It is noteworthy that a treasure trove of continuously updated data would now be in the public domain. For example, insight into why drugs work or do not work for specific patients is extraordinarily useful. Scientists would gain insights and increasingly be able to determine, at an early stage, whether a research approach is likely to be productive.
This speaks directly to the concern about the recent slowdown in drug approvals, especially for drugs that are not “me too” drugs but instead offer a new standard of care.
This essay has described the Dual Tracking-Tradeoff Evaluation Database model in broad strokes. Certainly there are many issues concerning implementation that need to be addressed. For example, should the government construct TED from the ground up by using the private sector and the competitive bidding process? How might the existing infrastructure for tabulating and communicating the results of clinical trials and off-label drug usage fit into a dual-tracking environment?
Issues such as these can be debated after the critical design is accepted.
Dual tracking would enable drug development firms to achieve an economically viable solution to the problems related to personalized medicine. The same characteristics of personalized medicine that offer the prospect of dramatic strides in health for individuals undermine the FDA’s insistence on large-population, lengthy clinical trials.
Society would benefit if pharmaceutical firms could implement a personalized medicine business model linking profits to successful innovation in four steps:
(1) develop a genetically targeted drug with exceptional effectiveness in early usage;
(2) after Phase I safety evaluations are successfully passed, achieve near-term revenues from sales to consumers who choose not to wait for final FDA approval;
(3) on one track, produce a documented record of outstanding drug performance from patients who meet the stringent genetic patient profile and make an informed decision to use the experimental drug; and
(4) on another track, meet a greatly reduced burden of FDA clinical testing for Phase II and III trials. Dual tracking would facilitate such a business model and accelerate the delivery of drug advances stemming from expanding genetic knowledge.
Doctor, Patient Control
We should expect our elected representatives to seek a better world in which patients and doctors control medical treatments and priority is given to existing patients. A dual-tracking system would achieve that end.
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 ought to 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 it calls out for genuine reform.