NEW YORK – 2023 was a dynamic year for precision medicine.
In a tough economic climate, scores of life sciences companies in the precision medicine space announced they were cutting staff and restructuring this year. The US Food and Drug Administration approved of a slew of new gene therapies, including two for sickle cell disease, approved a second beta-amyloid-targeted Alzheimer's drug, and on the diagnostics side dropped a controversial rule in September proposing to bring laboratory-developed tests under its oversight.
The year was also full of debate about whether the high price tags attached to precision medicines are worsening existing healthcare disparities. Drugmakers pushed back against government price controls in the US, arguing that the framework of a law allowing Medicare to negotiate the prices of the most expensive drugs will hinder their ability to keep developing innovative precision medicines for cancer and rare diseases. At the same time, drugmakers continued to struggle to keep up with demand for CAR T-cell therapies and radiopharmaceuticals.
Precision Medicine Online was on top of these trends and debates, but we also brought you stories that you won't find elsewhere, like our deep dive into drugmakers' divergent views on how to develop less toxic PI3Kα Inhibitors; our article exploring why the field of psychiatric genetic counseling has been hard to establish; and our scoop on a PGx software company's regulatory struggles. In case you missed them, here are 12 stories showcasing our best reporting in 2023:
Race for Better Mutant PI3Kα Inhibitors Hinges on Disputed Science, Dec. 20, 2023
Vertex, Bluebird Bio Ready for Competition as Docs Weigh Pros, Cons of Sickle Cell Gene Therapies, Dec. 14, 2023
Collaboratives, Companies Mobilize to Build Momentum for 'N-of-1' ASO Therapies, Oct. 30, 2023
PGx Labs Searching for New Result Reporting Options After Translational Software's Failed 510(k) Bid, Oct. 12, 2023
Precision Oncology Drugmakers Are the IRA's Staunchest Adversaries—And They're Not Backing Down, Oct. 4, 2023
Canadian Clinic's Struggles Highlight Challenge of Establishing Psychiatric Genetic Counseling, Sept. 14, 2023
Drugmakers Strive to Bring Precision to Small Cell Lung Cancer Treatment Despite Challenges, July 14, 2023
Price of Precision: Patients, Oncologists Highlight Costs of Fighting for Access to Newest Drugs, June 1, 2023
FDA, Industry Taking Steps to Streamline Cell Therapy Regulation, Though Uncertainty Remains, May 24, 2023
FDA Guiding Industry to Optimize Cancer Drug Dosing in Precision Oncology Era, March 28, 2023
Patients Are Dying Waiting for Pluvicto, but Novartis Can't Make More Pending Facility Approval, March 3, 2023
Survey Reveals Changing Genetic Testing Patterns, Persistent Disparities in Precision Oncology, Feb. 1, 2023