No correlation between the prices of oncology orphan drugs in the US and their patient population sizes

• Published in: Journal of cancer policy 2017-12, Vol.14, p.1-4
• Main Authors: Jarosławski, Szymon, Auquier, Pascal, Toumi, Mondher
• Format: Article
• Language: English
• Subjects: Drug pricing; Orphan drugs; Public policy; Rare diseases
• ISSN: 2213-5383; 2213-5383
• DOI: 10.1016/j.jcpo.2017.09.005

•The prices of orphan drugs were previously correlated to the target population sizes in Europe where prices are regulated by the government.•However, there is no linear or exponential relationship between the drug price and the size of their patient population in the US.•Current drug pricing policy in the US may have contributed to orphan drug price escalation. -Further studies are needed to elucidate other factors that can explain the differences in prices.•Greater transparency of pricing could potentially align the prices of orphan drugs with target population size. We sought to assess if orphan oncology drugs that target smaller populations are costlier than those that target larger ones. A list of orphan drugs designated by the FDA and diseases prevalence was retrieved from the FDA. Average Wholesale Prices per unit were obtained from the Red book database. Between June 2011 and June 2016, the FDA approved 31 unique regimens. Their annual treatment costs ranged from $9474 to $230,400, with a median of $140,893. There was no linear or exponential relationship between drug price and the size of their patient population. Further research is needed to bring more transparency of drug pricing in the US and align the prices of orphan drugs with the target population size and other relevant factors.