Profitability and Market Value of Orphan Drug Companies:A Retrospective, Propensity-Matched Case-Control Study

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.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.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%).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

Challenges for economic evaluation of strategies to contain antimicrobial resistance

The threat of antimicrobial resistance has global health and economic consequences. Medical strategies to reduce unnecessary antibiotic prescribing, to conserve the effectiveness of current antimicrobials in the long term, inevitably result in short-term costs to health care providers. Economic evaluations of health care interventions therefore need to consider the short-term costs of interventions, to gain future benefits. This represents a challenge for health economists, not only in terms of the most appropriate methods for evaluation, but also in attributing the potential budget impact over time and considering health impacts on future populations. This commentary discusses the challenge of accurately capturing the cost-effectiveness of health care interventions aimed at tackling antimicrobial resistance. We reflect on methods to capture and incorporate the costs and health outcomes associated with antimicrobial resistance, the appropriateness of the quality-adjusted-life year (QALY), individual time preferences, and perspectives in economic evaluation

What should patients do if they miss a dose? A systematic review of patient information leaflets and summaries of product characteristics

PurposeMedicines regulatory authorities advise that patient information leaflets (PILs) should provide specific advice on what actions to take if one or more doses are missed. We aimed to assess the content in this regard, of PILs and Summaries of Product Characteristics (SmPCs) of prescription only medicines (POMs) marketed in the UK.MethodsPILs and SmPCs were accessed via the electronic Medicines Compendium. The following terms were used in the advanced search facility: miss(ed), omit(ted), adhere(d), delay(ed), forgot, forget, lapse. Identified documents were screened for instructions on missed doses which were categorised according to level of specificity, and cross-referenced to the National Patient Safety Agency (NPSA) grading of risk of harm from omitted and delayed medicines. Any supporting clinical or pharmacological evidence was identified from SmPCs.ResultsTwo thousand two hundred eighty-four documents were identified from 7248 PILs and SmPCs relating to 1501 POMs. Seven hundred eighty-three (52%) POMs had SmPCs or PILs with no instructions on missed doses; 487 POMs (32%) included non-specific advice (e.g. "take as soon as possible"); 138 (9%) provided specific instructions; and 93 (6%) referred patients to seek medical advice. SmPCs for only 13/138 (9%) of those which included specific instructions provided any supporting clinical or pharmacological evidence. Instructions were absent for several medicines where the NPSA assessed that dose omissions may result in significant risk of harm.ConclusionsAdvice on missed doses is generally inadequate. Pharmaceutical companies and regulatory authorities should produce clear and concise instructions on what patients should do if they miss doses, with supporting evidence where necessary