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Do Medications Actually Work? An Ode to Phase IV2026-04-28

This question may seem provocative. After all, medications can be salutary and have transformed medical practice, increased life expectancy, and so on. Medications that are FDA approved have cleared a series of hurdles. Evidence of safety, (some) evidence of efficacy, overall favorable risk/benefit ratio.

A closer look at medications for different areas of medicine in general, and for psychiatry and pain in particular, paints a less rosy picture. The studies that led to approval were done for limited durations, in curated populations that do not necessarily reflect the real world, and in locations that may not enforce the highest standard of scientific integrity.

What matters to individual patients is if that medication works for them, in the long-term, given their real-world complexities and co-morbidities, with a favorable risk/benefit ratio and net positive impact on their life goals. This is where things get murky.

As a clinician, I evaluate at every visit if a patient should stay on a medication, whether it should be increased, decreased, or discontinued. All that data from individual patients using a medication should be collected proactively and programmatically into Phase IV continuous evaluation mechanisms by the FDA. The FDA could decide then whether a medication should stay on the market, increase its usage to other conditions, restrict its usage, or pull it from the market.

With the advent of precision medicine and AI, this is feasible and could be win-win-win for everybody. Patients, society, and pharma companies- which would see their drugs prescribed on merits, and avoid costly lawsuits.

At MindX Sciences, we have created the infrastructure to make that possible for psychiatry and for pain (https://mindxsciences.com/ ).

Live. Happier. Longer.

Alexander B. Niculescu, MD, PhD

Founder +CEO| MindX Sciences