In many ways, 2017 marked a turning point for biomedical innovation. The FDA approved the first U.S. gene therapy, the first CAR-T cellular immunotherapy, the first cancer drug based on a genetic signature, and the first smartphone app to treat substance abuse. Add to that the first successful Phase 3 trial for an RNA interference drug, and 2017 was a year in which medicine began to change significantly.
Yet with those changes come significant questions. How will the U.S. healthcare system handle these newer, longer-lasting treatments? Will there finally be a drug price reckoning as new therapies push costs higher—and will biopharma adapt with innovative payment models? Elsewhere, will setbacks emerge as CRISPR-Cas9 gene editing speeds towards human testing? Will combination treatments expand the reach of cancer immunotherapy, or pose new obstacles?