May 20 is International Clinical Trials Day, an occasion for honoring clinical trials professionals. The limits of sample size prevent clinical trials from answering all questions about the effects of new medical products in broad populations. However, clinical research professionals ensure that we learn as much as possible while also limiting patient exposure to investigational products.
On this date, it is important to reflect on what healthcare could be like before the advent of professionalism in clinical research. The story of Sanocrysin illustrates. Sanocrysin, sodium aurothiosulfate, was a gold compound widely used in the 1920s and early 1930s to treat tuberculosis. Development and testing of Sanocrysin took place before definition and refinement of the modern randomized controlled clinical trial and before the emergence of design, management and monitoring of clinical trials as professions.
Holger C. Møllgaard developed Sanocrysin in a series of experiments in animals. He was convinced that he had observed a treatment effect against tuberculosis and that he had developed a method for managing the toxicity of the compound. Investigators conducting case-control trials in tuberculosis patients also came to believe that Sanocrysin was effective against tuberculosis. As a result, Sanocrysin was used widely in Europe and the USA to treat tuberculosis. Sanocrysin won such high praise in some circles that Møllgaard was nominated for a Nobel Prize in Medicine in 1925 for chemotherapy of tuberculosis with Sanocrysin.
Randomization and independent oversight were necessary to establish the truth. That did not happen until three researchers in the United States, J. Burns Amberson, B.T. McMahon and Max Pinner, ran a trial in 1931 that was a milestone in development of modern clinical trial methodology. Among other things, the trial utilized randomization by coin flip. The trial failed to demonstrate the efficacy of Sanocrysin against tuberculosis but did document serious adverse events in the liver and especially the kidneys of trial subjects. Adverse events included deaths.
This anecdote shows the importance of sound methodology and professionalism in clinical trials. Let’s take the occasion of Clinical Trials Day to praise the professionals who deliver important services that advance the availability of new medical products for patients with unmet medical needs as well as ensuring safety of subjects in clinical trials. Such professionals include statisticians who design clinical trials and the project managers, clinical research associates and quality assurance professionals who manage, monitor and audit them. It takes true professionalism in all of these areas to ensure that trials ensure safety of test subjects, exclude bias and collect accurate safety and efficacy data. Because of such professionalism, physicians and patients have the information they need to decide whether new drugs and medical devices offer a favorable balance of efficacy and safety.
In addition to this blog post, Health Decisions has issued a press release to celebrate this day and honor clinical research professionals.