A new research and development paradigm for pattern-based new traditional Chinese medicine compound preparations: Integrating human use experience with patient-centered principles

The development of pattern-based traditional Chinese medicine (TCM) compound preparations constitutes a core domain that represents the principle of pattern differentiation-based treatment, a hallmark of TCM. However, the field has long been constrained by scientific and regulatory challenges in animal modeling, efficacy evaluation, and clinical positioning. This article proposes a new research and development (R&D) paradigm strategically based on human use experience (HUE) and centered on the patient as the key to overcoming these bottlenecks and achieving high-quality progress. We systematically dissect the traditional problems in this field and demonstrate the pivotal role of high-quality HUE in enabling precise clinical positioning and optimizing R&D pathways (e.g., applying for exemptions from non-clinical studies). HUE guides the implementation of the “pattern-symptom integration” model. Furthermore, we detail the implementation of the patient-centered concept throughout the process of clinical trial design, collection of patients’ experience data, clinical outcome assessment, and benefit-risk assessment. The integrative application of artificial intelligence in the R&D of pattern-based TCM drugs is also specifically explored. By synthesizing the “TCM theory, HUE, and clinical trials” evidence system, this article aims to provide a systematic strategic framework for establishing an R&D pathway that adheres to the intrinsic principles of TCM while simultaneously meeting modern scientific standards.