The evaluation of CFTR modulators involves rigorous preclinical and clinical testing. Preclinical studies assess pharmacokinetics, pharmacodynamics, and potential toxic effects in animal models. Clinical trials are conducted in phases, with Phase I focusing on safety and dosage, Phase II on efficacy and side effects, and Phase III on confirming effectiveness and monitoring adverse reactions in larger populations. Post-market surveillance is also critical to identify any long-term or rare toxicological effects that may not have been apparent during trials.
