Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs
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BACKGROUND: Orphan drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than nonorphan drugs, leading to questions about their costeffectiveness. OBJECTIVE: To compare the value of orphan and nonorphan drugs approved by the FDA from 1999 through 2015. DESIGN: We searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a drug was approved for multiple indications, we considered each drug-indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions). RESULTS: We identified estimates for 49 orphan drugindication pairs, and for 169 non-orphan drug-indication pairs. We found that orphan drug-indication pairs offered larger median incremental health gains than non-orphan drug-indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p < 0.001, KS p < 0.001) and less favorable cost-effectiveness ($276,288 vs. $100,360 per QALY gained; MWU p = 0.0068, KS p = 0.009). CONCLUSIONS: Our study suggests that orphan drugs often offer larger health gains than non-orphan drugs, but due to their substantially higher costs they tend to be less cost-effective than non-orphan drugs. Our findings highlight the challenge faced by health care payers to provide patients appropriate access to orphan drugs while achieving value from drug spending. KEY WORDS: cost-effectiveness; orphan drugs; rare disease; access; managed care.
J Gen Intern Med DOI: 10.1007/s11606-020-05805-2 © Society of General Internal Medicine 2020
INTRODUCTION
Since 2015, almost half of the drugs approved by the FDA have been orphan drugs, defined as drugs indicated for diseases affecting fewer than 200,000 patients in the USA, and this proportion has increased over each of the past three decades.1, 2 Rare diseases are often inherited disorders and severe in nature, and affect very young, vulnerable populations. Examples of inherited rare diseases include cystic fibrosis, spinal muscular atrophy, Duchenne muscular dystrophy, and Fabry disease. Orphan drugs have provided important therapeutic options for many of these conditions, for which few, if any, prior treatments were available. For instance, in 2019, the FDA approved pexidartinib for symptomatic tenosynovial giant cell tumor, and caplacizumab-yhdp for acquired thrombotic thrombocytopenic purpura (a lifethreatening disorder that causes blood clotting); these drugs were the first therapies approved for these conditions.3, 4
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