title: Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.
abstract: BACKGROUND
  Concerns about the high cost of orphan drugs has led to questions being asked about the generosity of the incentives for development, and associated company profits.
  
  
  METHODS
  We conducted a retrospective, propensity score matched study of publicly-listed orphan companies. Cases were defined as holders of orphan drug market authorisation in Europe or the USA between 2000-12. Control companies were selected based on their propensity for being orphan drug market authorisation holders. We applied system General Method of Moments to test whether companies with orphan drug market authorization are valued higher, as measured by the Tobin's Q and market to book value ratios, and are more profitable based on return on assets, than non-orphan drug companies.
  
  
  RESULTS
  86 companies with orphan drug approvals in European (4), USA (61) or both (21) markets were matched with 258 controls. Following adjustment, orphan drug market authorization holders have a 9.6% (95% confidence interval, 0.6% to 18.7%) higher return on assets than non-orphan drug companies; Tobin's Q was higher by 9.9% (1.0% to 19.7%); market to book value by 15.7% (3.1% to 30.0%) and operating profit by 516% (CI 19.8% to 1011%). For each additional orphan drug sold, return on assets increased by 11.1% (0.6% to 21.3%), Tobin's Q by 2.7% (0.2% to 5.2%), and market to book value ratio by 5.8% (0.7% to 10.9%).
  
  
  CONCLUSIONS
  Publicly listed pharmaceutical companies that are orphan drug market authorization holders are associated with higher market value and greater profits than companies not producing treatments for rare diseases.
authors: Centre for Health Economics and Medicines Evaluation, Bangor University, Ardudwy, Holyhead Road, Bangor, LL57 2PZ, United Kingdom.
authors: Dyfrig A
authors: DA
authors: Hughes
authors: University of Liverpool Management School, University of Liverpool, Chatham Street, Liverpool, L69 7ZH, United Kingdom.
authors: Jannine
authors: J
authors: Poletti-Hughes
mesh_terms: D004345:Drug Industry; D006801:Humans; D007182:Income; D009965:Orphan Drug Production / Q000191:economics*; D012189:Retrospective Studies