A proposal for a national program reporting beneficial drug responses, analogous to the existing program to detect adverse drug responses

Summary 

Many countries have regulatory agencies, such as the Food and Drug Administration in the United States, not only to determine approval of drugs into the marketplace, but also to monitor their safety following the approval process. The logic for the latter role of these agencies is that several important changes occur in the patient population after the approval to market. These changes include the tremendous expansion in the numbers of patients, the removal of many exclusion criteria of co-existing diseases that may have been included in the clinical trials, and the fact that the treatment time for agents used chronically will now exceed any previous exposure time. All of these factors may contribute to the development of adverse drug responses (ADRs) that were not predictable or observable during the clinical trial phases.

While the programs to monitor ADRs have important limitations such as the absence of a control group, they are extremely useful at determining possible associations between a drug and an adverse response that can now be used to generate hypotheses to be tested in a more proper and controlled set of trials.

A similar system could, in principle, be used to monitor non-predictable serendipitous beneficial drug responses (BDRs) that would otherwise not be seen due to the same limitations of clinical trials in detecting ADRs. The system would expand data collection beyond clinical trials – which often can pick up BDRs of drugs such as minoxidil and sildenafil. This system of monitoring for BDRs would have the tremendous advantages of generating hypotheses derived from data obtained in humans with the disease and a drug-able molecule. The data obtained from this system of monitoring for BDRs could lead to new uses for old drugs as well as provide starting molecules that could be modified to treat diseases unrelated to the original indicated disease for the parent drug.

The cost of creating and maintaining a BDR database are in-line with budgets of some of the projects funded by agencies such as the NIH or MRC. One possible feature is that the patient, rather than the doctor, does the majority of BDR reporting. Although lay-reporting would lead to false positives; repeated observations of a beneficial association would be a reasonable basis for further study in formal, professional clinical investigations.

It is time to create the largest stage ever for serendipity to appear in biomedical research. After all, her previous appearances have marked some of science’s most amazing discoveries.