Romania's Pioneering Law: Establishing the Right to Personalized Medicine.

This analysis and commentary discuss Romania's landmark law, the first globally, acknowledging the right of citizens and patients to personalized medicine. Initiated following the EU Council's 2015 policy on personalized medicine, the law is a result of intersectoral collaborative efforts led by the Centre for Innovation in Medicine in Romania using a quadruple (later evolved to penta) helix model involving academia, public, private, and civil society sectors. Promulgated on May 24, 2023, the law legally entitles patients to personalized health care and in ways informed by individual genetic and phenotypic consideration. The law mandates informed consent for medical interventions and ensures data protection in accordance with the General Data Protection Regulation. We suggest that this pioneering legislation paves the way for integrating personalized medicine into Romania's health care system, shaping clinical practice, research, and health policy. In all, it marks a significant step in redefining health care delivery, emphasizing individualized treatment and the political determinants of personalized medicine, and setting a precedent for future health care innovations worldwide.

A framework for validating AI in precision medicine: considerations from the European ITFoC consortium.

BACKGROUND: Artificial intelligence (AI) has the potential to transform our healthcare systems significantly. New AI technologies based on machine learning approaches should play a key role in clinical decision-making in the future. However, their implementation in health care settings remains limited, mostly due to a lack of robust validation procedures. There is a need to develop reliable assessment frameworks for the clinical validation of AI. We present here an approach for assessing AI for predicting treatment response in triple-negative breast cancer (TNBC), using real-world data and molecular -omics data from clinical data warehouses and biobanks. METHODS: The European "ITFoC (Information Technology for the Future Of Cancer)" consortium designed a framework for the clinical validation of AI technologies for predicting treatment response in oncology. RESULTS: This framework is based on seven key steps specifying: (1) the intended use of AI, (2) the target population, (3) the timing of AI evaluation, (4) the datasets used for evaluation, (5) the procedures used for ensuring data safety (including data quality, privacy and security), (6) the metrics used for measuring performance, and (7) the procedures used to ensure that the AI is explainable. This framework forms the basis of a validation platform that we are building for the "ITFoC Challenge". This community-wide competition will make it possible to assess and compare AI algorithms for predicting the response to TNBC treatments with external real-world datasets. CONCLUSIONS: The predictive performance and safety of AI technologies must be assessed in a robust, unbiased and transparent manner before their implementation in healthcare settings. We believe that the consideration of the ITFoC consortium will contribute to the safe transfer and implementation of AI in clinical settings, in the context of precision oncology and personalized care.

The FDA Changed Everything.

On May 23, 2017, the FDA approved the first cancer treatment (pembrolizumab) for any solid tumor with a specific genetic biomarker: microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR). For the first time in history, a solid cancer treatment was approved based on the genetic makeup of tumor not on the location in the body where the cancer originated, for example lung or breast cancer (TNM staging). This indication covers patients with solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options and patients with colorectal cancer that has progressed following treatment with certain chemotherapeutics. All cancer drug approvals in the last 30 years were grounded on TNM staging independent of the therapy type (chemotherapy, monoclonal antibodies, TKI inhibitors, immune therapies or targeted therapies) and despite the huge and fast advances in understanding tumor biology. In fact, the FDA previously approved pembrolizumab taking into consideration the TNM staging, for the treatment of certain patients with metastatic melanoma, metastatic non-small cell lung cancer, recurrent or metastatic head and neck cancer, refractory classical Hodgkin lymphoma, and urothelial carcinoma. The archaic TNM staging will probably be changed under the disruptive wave of molecular biology. The recent FDA approval could be considered the certificate of birth for a truly new dimension of personalized medicine in cancer. We recommend European Union to follow the FDA approach of tissue-agnostic cancer drugs in order to speed up the development of next-generation oncologic therapies and to increase the access of patients to truly personalized medicine.