While both sides of the U.S. political aisle are focused on bringing down drug prices, they’re missing a critical lever for long-term change: raising the evidentiary bar for what qualifies as clinical value. In late 2025 the FDA signaled a major policy shift in removing barriers to the use of real-world evidence in regulatory submissions, heralding an opportunity for innovative approaches to researching the impact of new drugs on patients. This is especially important for patients suffering from rare diseases where traditional research methodologies make it difficult to recruit small samples and raise ethical concerns for patients entered into traditional RCTs.

Lasting affordability in the face of rapid advances in technology depends on distinguishing between statistical difference and real clinical improvement. Patients—and U.S. taxpayers—deserve evidence that every dollar spent delivers measurable value. Consideration of study design, relevance of clinical endpoints, patient reported outcomes and other parameters are essential to determination of meaningful clinical value.