Shining a light in the black box of orphan drug pricing

Abstract

BackgroundThe pricing mechanism of orphan drugs appears arbitrary and has been referred to as a “black box”. Therefore, the aim of this study is to investigate how drug- and disease-specific variables relate to orphan drug prices. Additionally, we aim to explore if certain country-specific pricing and reimbursement policies affect the price level of orphan drugs.MethodsAnnual treatment costs per indication per patient were calculated for 59 orphan drugs with a publicly available price in Belgium, the Netherlands, Czech Republic, France, Italy and the United Kingdom. A multiple linear regression model was built with 14 drug- and disease-specific variables. A Mann-Whitney U test was used to explore whether there is a correlation between annual treatment costs of orphan drugs across countries with different pricing and reimbursement policies.ResultsRepurposed orphan drugs, orally administered orphan drugs or orphan drugs for which an alternative treatment is available are associated with lower annual treatment costs. Orphan drugs with multiple orphan indications, for chronic treatments or for which an improvement in overall survival or quality-of-life has been demonstrated, are associated with higher annual treatment costs. No association was found between annual treatments cost of orphan drugs across countries and the different pricing and reimbursement systems.ConclusionsThis study has shown that prices of orphan drugs are influenced by factors such as the availability of an alternative drug treatment, repurposing, etc. Current debate about the affordability of orphan drugs highlights the need for more transparency in orphan drug price setting.

Are new models needed to optimize the utilization of new medicines to sustain healthcare systems?

“…New orphan medicinal products (OMPs), some costing over €1 million per patient per year (20,23,100), are also putting pressure on healthcare budgets, especially with some OMPs achieving blockbuster status (9,100,101). This increases the debate whether pharmaceutical companies should also consider social good alongside financial returns (20), and whether health authorities should be more critical when assessing the value of new OMPs.…”

“…The Transparent Value Framework (TVF) was developed as part of the European project on coordinated access to orphan medicinal products (MoCA) under the platform Access to Medicines (101,159). Its development arose as a result of the ongoing challenges posed by pricing and funding of the new orphan medicinal products (OMPs) (108), conceived as an aid to the MoCA process by providing a framework for discussions on clinical effectiveness, recognizing that it would be more efficient to avoid duplication of effort for appraising OMPs among the member states.…”

Current Problems in Provision of Orphan Drugs and Ways to Solve Them

“…К про-чим особенностям орфанных лекарственных средств, благоприятным для производителей, можно отнести относительно низкие затраты на клинические испыта-ния, в т.ч. по причине небольших по объему выборки исследований, и невысокую конкуренцию на рынке пре-паратов этого класса [39].…”

Pharmaceutical Expenditure And Policies

“…According to a study on prices of 59 orphan drugs in Belgium, the Netherlands, Czech Republic, France, Italy and the United Kingdom, prices do not seem to be affected by national pricing and reimbursement policies. Instead, they are likely to be higher when orphan medicines have multiple indications, when no alternative treatment is available, when they target chronic disease and when an improvement in survival of quality of life has been demonstrated (Picavet et al 2014). …”