The long-term benefits of forcing clinical trials

Of course, banning off-label use would present new challenges for the healthcare industry. Most notably, lack of access to off-label drug treatments would put further separation between patients with no other approved medical options and breakthrough scientific discoveries. This assumes that the “discovery” is both medically valid, and the patient’s doctor is well-informed enough to know about the off-label use. A system that requires clinical trials for new uses, however, would ensure the treatment is valid and well-known. Although it might take a couple of years to gain approval, it’s arguably better (or at least not any worse) than thousands of doctors prescribing an off-label use that is completely ineffective, or even harmful, without any follow-up studies.

Another challenge would be the enormous increase in the need for clinical trials. Clinical trials are costly. A system that bans off-label use might collapse if patients who want to volunteer for a new clinical trial can’t work because of limited resources. Perhaps if the regulatory exclusivity period were tied to the cost of each clinical trial, more firms would be attracted to invest. Similarly, generics might be expected to step up and conduct their own clinical trials since the generic firm would receive the regulatory exclusivity for that indication, even against the pioneer. While a duopoly is currently the best market exclusivity a generic can enjoy, perhaps the promise of a monopoly would provide incentives for generic companies to fill the added need. Finally, government agencies could conduct clinical trials for indications that have high social value but low commercial value.

Even ignoring the long-term benefits of forcing clinical trials – thereby ensuring treatments are “safe and effective” – would likely balance out the loss of immediate access to off-label use options, the enormous benefits of discovering new uses would vastly outweigh the costs.

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