Regulatory Science for Companion Diagnostics in Precision Medicine: Harmonizing Approval, Integration, and Innovation

Companion Diagnostics (CDx) have emerged as a cornerstone in the rapidly evolving field of precision medicine, enabling clinicians to tailor therapies based on molecular and biological patient profiles. The integration of CDx into clinical practice has redefined patient stratification and optimized outcomes, especially in oncology, where molecularly guided therapies are now standard for diseases like HER2 positive breast cancer and EGFR mutated lung cancer. Recent advances have expanded CDx applications to a wider range of malignancies and have prompted the development of innovative testing platforms such as liquid biopsy and multi omics assays. Despite these successes, mounting challenges persist in biomarker validation, regulatory harmonization, laboratory accreditation, and sustainable healthcare financing. Regulatory frameworks like the European IVDR and updated FDA guidelines have elevated CDx to high risk devices, intensifying requirements for clinical evidence, post market surveillance, and cross disciplinary collaboration.Ongoing scientific debates now focus on synchronizing drug and diagnostic approvals, ensuring laboratory quality, integrating real world evidence, and navigating the economic complexities of health technology assessment (HTA) and reimbursement. Studies show that while co development of drugs and diagnostics enhances treatment efficacy and reduces healthcare waste, existing models often struggle to accommodate the interconnected value of diagnostic and therapeutic pairs. Moreover, methodological innovation is needed as new paradigms driven by artificial intelligence, digital health solutions, and adaptive regulatory models transform the scope of companion diagnostics beyond single analyte testing. Consequently, there is a pressing need for research that bridges disciplinary divides and catalyzes integrated approaches to regulatory science, clinical deployment, and economic evaluation.This Research Topic aims to foster global, interdisciplinary dialogue at the intersection of scientific validation, regulatory transformation, clinical integration, and sustainable healthcare innovation for companion diagnostics in precision medicine. The objective is to clarify current challenges, stimulate novel approaches to regulatory and economic appraisal, and promote solutions that harmonize the roles of clinicians, scientists, policymakers, and health system leaders. Research efforts should address regulatory synchrony, clinical evidence generation, digital transformation, value based reimbursement models, and the evolution of laboratory and health system governance in CDx adoption.To gather further insights within the boundaries of companion diagnostics development, implementation, and impact across diverse healthcare contexts, we welcome articles addressing, but not limited to, the following themes:• Biomarker discovery, analytical and clinical validation, and multi omics integration• Drug diagnostic co development, trial design, and synchronized approval pathways• National and international regulatory frameworks (for example, IVDR, FDA, EMA, IMDRF)• Clinical integration in oncology, rare diseases, and advanced therapies (for example, CAR T, gene therapy)• Laboratory accreditation, digital pathology, AI based interpretation, and interoperability• Economic modeling (cost effectiveness, budget impact, and integrated HTA on CDx drug combinations)• Future perspectives on AI driven diagnostics, real world evidence, and decentralized testingInterdisciplinary and cross sector contributions are particularly encouraged. Article types welcomed: Original Research, Systematic Reviews, Narrative Reviews, Policy Brief, Policy and Practice Reviews, Clinical Trial, Perspective and Opinion Articles. Topic editors: Valentina Pecoraro, Beatriz S Lima, Tommaso Trenti Source: https://www.frontiersin.org/research-topics/78532/regulatory-science-for-companion-diagnostics-in-precision-medicine-harmonizing-approval-integration-and-innovation