OBJECTIVES: High prices of orphan drugs are often linked to the necessity to recover the development costs from sales to small patient populations. If this is true, drugs with smaller target population sizes should be more costly. We sought to compare the prices of orphan drugs in oncology with their respective patient population sizes. METHODS: A list of orphan drugs designated by the FDA between June 2011 and June 2016 was retrieved from the FDA website. We include: a) drugs approved by the FDA; b) treatments in oncology. We exclude: a) products not specifically indicated to treat cancer (e.g.,imaging, palliative care or treatment of a cancer-associated condition). Diseases prevalence data was obtained from the FDA orphan drug approval reviews. Average Wholesale Prices per unit were obtained from the Micromedex database. Prices per
year of treatment were calculated based on the drugs’ dosage from the FDA labels. We used descriptive statistics to compare drug prices per year of treatment to the target populations of patients for each indication. RESULTS: Out of 187 orphan designated indications, 70 led to drug approvals by the FDA. 37 approvals were in oncology. Eight regimens had two approved indications and one had five indications. There were 25 unique regimens of which two were for drug combinations.
There was no clear association between the drug prices and the sizes of their respective patient populations.
CONCLUSIONS: The current orphan drug policies have encouraged the development of novel treatments, but have also led to extremely high prices of these drugs. Whereas drug prices may depend on factors other than population size alone, our findings suggest that there is no apparent link between the prices and target population sizes. This should help policy makers formulate future orphan drug policies that encourage innovation, but that are based on drivers other than potential market size alone.