PORTAL generates evidence to advance safe, effective, and affordable medications.

The US Orphan Drug Act and EU Orphan Regulation both offer incentives to develop rare disease treatments, but of 344 rare disease drug indications approved from 2011 to 2020, 97.7% received FDA Orphan Drug designation versus only 40.4% from the EMA, with the agencies aligning most on advanced therapies and genetic diseases but diverging on cancer subsets and pediatric indications of common diseases like HIV. These differences reflect the broader US indication-based framework versus the EU’s narrower focus on distinct conditions, suggesting policymakers should target incentives toward truly rare diseases rather than subsets of common conditions like cancer.

Brand-name drug manufacturers accumulate thickets of patents on their drugs that they then use to delay generic and biosimilar competition. Studies show many of these patents are obvious variations of earlier patents, granted only after applicants file “terminal disclaimers.” The ETHIC Act allows one patent per group of obvious variation patents to be asserted against generic or biosimilar competitors in litigation, while maintaining brand-name manufacturers’ ability to enforce any patents that fall outside these patent groups. The ETHIC Act substantially reduces the number of patents that could be enforced against generic or biosimilar competitors in litigation, making market entry of generics and biosimilars faster and more efficient, leading to greater health care savings and broader patient access.

Standard cost-effectiveness thresholds used in the US and other high-income countries already exceed the marginal cost of a unit of health, building in an “innovation premium” that allows new drugs to be deemed cost-effective even when adopting them produces a net population health loss. Rather than abandoning thresholds, policymakers should fund research to identify the true opportunity-cost threshold, use it as a primary result in analyses, and acknowledge this premium for “newness” as a deliberate policy choice rather than a hidden default.