Best Practices and Emerging Trends for Market Access to Personalised Medicine in the US and EU: Learnings for Global Developed and Emerging Markets

While many would argue that personalised medicine has a long history, the era of modern deployment dates only to hormone-receptor guided tamoxifen therapy and growth-factor receptor guided Herceptin therapy in the 1990s and has been rather sporadic in the intervening years. From this uneven beginning, global adoption remains modest, with wide variations in availability of testing and associated therapy. Indeed, while over 120 U.S. Food and Drug Administration (FDA)-approved drugs have pharmacogenomics information in their labelling, most such labelling is informational, and there are today only 6 distinct “companion” markers cleared by FDA as in vitro companion diagnostic tests mandated for the safe and effective use of the corresponding therapy (HER2, CKIT, EGFR, KRAS, BRAF, ALK). Approval and availability of such companion tests with a 1:1 therapeutic relationship is only the beginning, however. “High value” diagnostic tests with indirect relationships to therapeutics and next generation multi-omic tests with higher predictive precision have even lower availability in global markets. Scientific and regulatory hurdles are only part of the underlying challenge. In an increasingly constrained global healthcare environment, heath-technology assessment and deployment models assume equal significance, and various models have emerged across the US and EU to accelerate market access. Some recent trends and best practices include use of adaptive clinical trial designs, pragmatic application of regulations, new Health Technology Assessment and reimbursement models, and emergent quality assurance practices. This article will review some of these prevailing global best practices for the deployment of personalised medicine, extract some general learnings, and draw some inferences for global markets, including emerging regions.