A new year brings new opportunities, and that includes for innovative medical treatments. The U.S. Food and Drug Administration (FDA) will be busy reviewing a number of novel therapies this year. First up could be a treatment for Alzheimer’s disease from Eli Lilly that promises to be the most effective yet in reducing the buildup of disease-contributing amyloid plaques in the brain. If approved, it would give patients another powerful option for managing the memory disorder. The FDA will also consider expanding approval of the first gene editing treatment, using CRISPR, which it approved to treat sickle cell anemia in 2023. This year, the agency will decide whether people with another genetic blood disorder, beta thalassemia, could also benefit from the technology, which gives people more blood cells that can carry oxygen. In the field of mental health, people with schizophrenia could see the first new treatment in decades if the FDA greenlights a medication that targets a different brain chemical than existing ones to reduce the condition’s peaks and valleys of positive and negative thoughts and mood. Health experts and patient advocates say that ensuring affordable access will be just as important as securing approval for these exciting new treatments. Many of the therapies are designed as one-time medications, rather than chronic interventions, and come with high upfront costs. That’s led some insurers to not commit to reimbursing for them. But many experts believe these novel medicines could end up saving the health care system money—and lives—in the long run. READ THE REST |