How FDA Is Approving More Drugs To Treat Rare Diseases | RealClearPolitics

RealClearPolitics

by Marty Makary, WSJ

February 24, 2026

AI-Generated Deep Dive Summary

The FDA is significantly increasing its approvals of drugs to treat rare diseases, marking a notable shift in regulatory focus. This trend reflects growing efforts to address the unique challenges faced by patients with rare conditions, which often lack effective treatments. According to Marty Makary of the Wall Street Journal, this push aligns with broader healthcare policies aimed at accelerating access to innovative therapies while maintaining safety standards. The rise in approvals can be attributed to several factors, including regulatory reforms and incentives for drug developers. Programs like the orphan drug designation provide financial benefits to companies working on treatments for rare diseases, encouraging investment in this space. Additionally, the FDA’s Rare Disease Program has streamlined processes to evaluate these therapies more efficiently, reducing delays that once hindered progress. This shift is particularly significant for patients and advocacy groups who have long called for greater attention to rare diseases. While some criticize the potential for higher costs associated with orphan drugs, supporters argue that these treatments offer life-changing benefits for underserved patient populations. The political implications of this trend are also notable, as it underscores ongoing debates about healthcare access, drug pricing, and government’s role in regulating medical innovation. Ultimately, the FDA’s expanded focus on rare diseases highlights a broader cultural and policy shift toward valuing innovation and prioritizing patient needs. While questions remain about balancing speed with safety, these developments signal progress for those affected by rare conditions and set the stage for future advancements in this field.

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Originally published on RealClearPolitics on 2/24/2026