The health benefits, costs, and cost-effectiveness of ultra-orphan drugs.

To examine ultra-orphan drugs in terms of incremental health, costs, and cost-effectiveness compared to more prevalent disease drugs. We identified FDA drug approvals from 1999-2019. For drugs approved for multiple indications, we considered each drug-indication pair separately. Utilizing FDA's orphan drug designation and US disease prevalence, we categorized drug-indication pairs as: ultra-orphan (<10,000 patients), 'other' orphan (≥10,000 and <200,000), and non-orphan (≥200,000). We searched the PubMed database for cost-effectiveness and cost-utility studies. We excluded manufacturer-funded studies. We extracted estimates of incremental health gains in terms of quality-adjusted life-years (QALYs) and incremental costs associated with drug-indication pairs compared to the standard of care at the time of their approval. We compared QALY gains, added costs, and incremental cost-effectiveness ratios (ICERs) using the Kruskal-Wallis (KW), Mann-Whitney U (MWU), and Kolmogorov-Smirnov (KS) tests. Median incremental QALYs, costs, and ICERs differed across non-orphan, 'other' orphan, and ultra-orphan categories (KW p<0.01). Compared to non-orphan drugs, ultra-orphan drugs had larger QALY gains (0.700 vs. 0.050, MWU p<0.01, KS p<0.01), larger costs ($172,231 vs. $3,360, MWU p<0.01, KS p<0.01), and larger ICERs ($1,216,184/QALY vs. $114,061/QALY, MWU p<0.01, KS p<0.01). Compared to 'other' orphan drugs, ultra-orphan drugs had larger QALY gains (0.700 vs. 0.310, MWU p=0.65, KS p=0.32), larger costs ($172,231 vs. $69,308, MWU p=0.03, KS p=0.03), and larger ICERs ($1,216,184/QALY vs. $223,472/QALY, MWU p<0.01, KS p<0.01). Novel ultra-orphan drugs typically offer larger incremental health gains than drugs for more prevalent diseases, but due to their substantial added costs, are typically less cost-effective.

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