After the Trial Ends…

Our December 2019 issue featured a look at the unforeseen costs for patients who participate in clinical trials, including eventually paying for post-trial access to the drug that they helped to bring to market (“Calculating the Costs of Clinical Trials”). Here, one reader writes in about another important piece of the clinical trial participation puzzle – the volunteers who never received the study drug.

The “Calculating the Costs of Clinical Trials” feature article in the December issue made a plea for the continued coverage of experimental trial drugs after the end of the trial. However, no mention is made of the patients in the control arm of the trial who never received the new experimental drug. If the new drug is found to be effective, the control-arm patients, who never received it, should be able to get it when their therapy (without the new drug) fails. It seems unfair and cruel, in a trial showing a drug to be highly effective, for half the patients to be unable to benefit.

In multiple myeloma, for example, several new drugs have been proven beneficial for progression-free survival when given as induction and maintenance therapy. This disease is still said to be incurable, so the new drug is likely to become ineffective eventually.

However, if unexposed patients received the new drug only upon a disease relapse, it could still be effective, thus avoiding years of medical and financial toxicity.

Herbert Goldman, MD, DSc
University of Puerto Rico