Large-scale randomized clinical trials (RCTs) have been the long-established ‘gold standard’ for testing new drugs for safety and efficacy. While this model works well for diseases affecting large numbers of people, it neglects individual variations that may make the difference between recovery and chronic illness. RCTs are also biased toward drug companies able to finance costs running to the millions of dollars. This entire methodological apparatus having exclusive hegemony over validation of new drugs crowds out other methods of validation that may cure many individual illnesses. While assuredly less profitable than mass-market blockbuster drugs, other methods that focus on individualized treatment could effect superior treatment in those cases. Furthermore, discovering the reasons for individual variations in diseases and treatment responses could advance scientific understanding of how the body heals itself. Analyzing individual variation is a fundamental principle of scientific discovery that the RCT mass-market model suppresses.

As Dr John Catanzaro puts it, ‘An N-of-1 trial is a personalized, single-patient clinical study that aligns treatment based on the individual’s unique biomolecular response. Instead of relying on population-based statistics, this method uses real-time molecular surveillance, patient-specific peptide therapeutics, and adaptive treatment adjustments to achieve true precision medicine.’ His company, Neo7bioscience, develops ‘N-of-1’ treatments based on ‘the precise molecular disruptions driving [each patient’s] disease. We create precision-targeted peptides that restore faulty signaling, neutralize inflammatory cascades, and support immune recalibration. Unlike mass-market drugs, these peptides are designed specifically for the individual’s biomolecular landscape.’ More here: petermcculloughmd.subst…

There should be room in medical practice today for more than one method of validating treatment, especially in cases of statistically rare diseases, and maladies that have eluded precise enough characterization to interest blockbuster-seeking drug companies.