Uncertain Policy Environment
May Shift Forecasts

The highly uncertain US policy environment – around IRA implementation, FDA approval rates and more – makes forecasting even trickier than usual. The Trump administration has begun to fire FDA staff as part of its wider efficiency drive, depressing staff morale and perhaps prompting further, voluntary departures among hard-to-replace experts. Until now, FDA has supported cell and gene therapies, and pushed for accelerated access to novel rare disease therapies, for instance with a new rare diseases hub coordinating action across its CDER and CBER approval divisions. These initiatives are now under threat. The staff cuts may slow product approvals, and lead to a more risk-averse approach to assessing medicines’ value. Meanwhile, moving IRA goalposts also make the backend of products’ revenue cycles harder to assess. The possibility of unwelcome broader health policies, such as a vaccine rollback, plus attempts by the Trump government to cut National Institutes of Health (NIH) funding, are stoking a general malaise.Since orphan drugs now occupy a significant, if steadying, share of overall drug sales and pipeline value, the category may be just as heavily impacted by these shifting policy and regulatory sands as mainstream prescription pharmaceuticals.

Moving IRA goalposts also make the backend of products’ revenue cycles harder to assess.