How FDA Is Approving More Drugs To Treat Rare Diseases | RealClearPolitics
RealClearPolitics
by Marty Makary, WSJFebruary 24, 2026
AI-Generated Deep Dive Summary
The U.S. Food and Drug Administration (FDA) is increasingly prioritizing approvals for drugs treating rare diseases, marking a significant shift in its focus. While the agency's processes were historically designed with common ailments in mind, it now recognizes the urgent need to address the unique challenges faced by those with uncommon conditions. With approximately 30 million Americans affected by rare diseases, this shift reflects a growing emphasis on improving access to treatments for these underserved populations.
The FDA has made notable strides in approving orphan drugs—medications developed specifically for rare diseases. The Orphan Drug Act of 1983 established incentives for pharmaceutical companies to develop such treatments, including tax breaks and market exclusivity. These policies have led to a surge in approvals, with hundreds of orphan drugs now available. This trend underscores the FDA's commitment to accelerating the development of therapies that might otherwise be overlooked due to limited patient populations.
From a political perspective, this shift highlights the intersection of public health and policy. Rare diseases often disproportionately affect vulnerable communities, making access to treatment a matter of social equity. Politicians and policymakers are increasingly pressured to support initiatives that prioritize rare diseases, as these efforts align with broader goals of improving healthcare outcomes for all Americans. This focus also signals a move toward more patient-centered policies, reflecting the growing influence of patient advocacy groups in shaping regulatory decisions.
While progress has been made, challenges remain. Many rare diseases lack effective treatments, and drug development continues to face significant hurdles, including high costs and limited research funding. However, the FDA's expanded role in this space offers hope for patients and their families, signaling a potential turning point in how rare diseases are addressed in the U.S. political and healthcare landscape.
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Originally published on RealClearPolitics on 2/24/2026