Enrollment numbers vary by phase; it is not uncommon for a drug to be approved after studying fewer than how many subjects, especially for a very specific disease?

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In drug development, how many people are studied can vary a lot depending on the disease and the available patient population. When a disease is very specific or rare, there aren’t enough patients to recruit thousands for a trial. Regulators recognize this reality and may grant approval after pivotal studies that include a smaller number of participants, as long as the data show a meaningful and consistent treatment effect, a favorable safety profile, and clinically meaningful benefit. This approach is common with orphan or highly targeted diseases, where the potential benefit to patients is weighed against the feasibility of enrolling large numbers. Safeguards often include confirmatory post-marketing studies and robust risk management to continue evaluating safety as the drug reaches a broader patient population.