OBJECTIVES: To assess the trends and factors associated with the price at market entry of new molecular entities (NMEs) with oral formulations and chronic use approved by the FDA between 1980 and 2021.

METHODS: Data on regulatory information, defined daily dosages, and wholesale acquisition cost (WAC) at market entry were extracted from the FDA, World Health Organization, and IBM Micromedex, respectively. WAC was used as a proxy for private sector prices, as both Medicaid and the Federal Supply Schedule use WAC to estimate rebates and reimbursement for new drugs at market entry. Prices were converted to average 2021 US dollars using the consumer price index. Descriptive statistics and regression analysis were conducted.

RESULTS: The FDA approved 436 new drugs with oral formulations for chronic use from 1980 to 2021. Among these, 121 (27.1%) had orphan designation, 210 (47.1%) were approved using the FDA priority review designation, and 210 (47.1%) had generic competition as of December 2021. The median inflation-adjusted price per year at market entry was $923 (IQR=$872) in the 1980s (n=66, 15.1%), $1,467 (IQR=$3,134) in the 1990s (n=130, 29.8%), $2,434 (IQR=$9,924) in the 2000s (n=75, 17.2%), $76,390 (IQR=$138,847) in the 2010s (n=128, 29.4%), and $219,219 (IQR=$319,039) in 2020-2021 (n=37, 8.5%). The price at market entry per year was positively associated with orphan drug designation (2.64, p<0.001), priority review designation (0.63, p<0.001), approval date (0.1, p<0.001), antineoplastic and immunomodulating agents (4.71, p<0.001), anti-infectives for systemic use (2.54, p<0.001), nervous system (0.35, p=0.04), and alimentary tract and metabolism (0.58, p=0.01).

CONCLUSIONS: This study observed a significant upward trend in the prices at market entry of new molecular entities approved by the FDA between 1980 and 2021. The approval date, orphan drug and priority review designations, and drugs for oncology and viral infections were associated with a higher price at market entry.