Michael Schlander1, Charalabos-Markos Dintsios2, Afschin Gandjour3. 1. Institute for Innovation and Valuation in Health Care, Wiesbaden, Germany; German Cancer Research Center, University of Heidelberg, Heidelberg, Germany. Electronic address: m.schlander@dkfz.de. 2. Institute for Health Services Research and Health Economics, Medical Faculty Heinrich-Heine University Düsseldorf, Düsseldorf, Germany. 3. Frankfurt School of Finance and Management, Frankfurt, Germany.
Abstract
OBJECTIVES: To review recent studies reporting health care expenditures (budgetary impact) for orphan medicinal products (OMPs) in Europe and to contribute to our understanding of the cost drivers of nononcological OMPs by means of an empirical analysis in Germany. METHODS: A systematic search for relevant studies on rare diseases was conducted in PubMed and Embase (until December 2016). In addition, annual treatment costs of nononcological OMPs in Germany were analyzed with respect to five explanatory variables: total prevalence of disease, prevalence with added benefit, availability of alternative treatments for the same indication, extent/probability of treatment benefit, and evidence for a treatment effect on mortality. RESULTS: A total of nine studies with specific estimates of the budget impact of OMPs for a total of 11 countries were identified; one study addressed specifically ultrarare diseases. Annual per-capita spending for OMPs ranges from €1.32 in Latvia to €16 in France. Per-patient annual treatment costs vary between €27,811 and €1,647,627 in Germany. On the basis of the German data set, the regression analysis shows that log prevalence has a significant inverse relationship with log annual treatment cost. In this model, doubling the prevalence leads to a 43% decrease in annual treatment cost. CONCLUSIONS: Despite per-patient annual treatment costs ranging up to several hundreds of thousands of euros for some OMPs, per-capita spending for OMPs is relatively small. In this study an inverse relationship between prevalence and annual treatment costs was found.
OBJECTIVES: To review recent studies reporting health care expenditures (budgetary impact) for orphan medicinal products (OMPs) in Europe and to contribute to our understanding of the cost drivers of nononcological OMPs by means of an empirical analysis in Germany. METHODS: A systematic search for relevant studies on rare diseases was conducted in PubMed and Embase (until December 2016). In addition, annual treatment costs of nononcological OMPs in Germany were analyzed with respect to five explanatory variables: total prevalence of disease, prevalence with added benefit, availability of alternative treatments for the same indication, extent/probability of treatment benefit, and evidence for a treatment effect on mortality. RESULTS: A total of nine studies with specific estimates of the budget impact of OMPs for a total of 11 countries were identified; one study addressed specifically ultrarare diseases. Annual per-capita spending for OMPs ranges from €1.32 in Latvia to €16 in France. Per-patient annual treatment costs vary between €27,811 and €1,647,627 in Germany. On the basis of the German data set, the regression analysis shows that log prevalence has a significant inverse relationship with log annual treatment cost. In this model, doubling the prevalence leads to a 43% decrease in annual treatment cost. CONCLUSIONS: Despite per-patient annual treatment costs ranging up to several hundreds of thousands of euros for some OMPs, per-capita spending for OMPs is relatively small. In this study an inverse relationship between prevalence and annual treatment costs was found.
Authors: Carina Schey; Maarten Jacobus Postma; Paul F M Krabbe; Olekdandr Topachevskyi; Andrew Volovyk; Mark Connolly Journal: Front Public Health Date: 2020-05-08
Authors: Monika Wagner; Dima Samaha; Roman Casciano; Matthew Brougham; Payam Abrishami; Charles Petrie; Bernard Avouac; Lorenzo Mantovani; Antonio Sarría-Santamera; Paul Kind; Michael Schlander; Michele Tringali Journal: Int J Health Policy Manag Date: 2019-07-01
Authors: Jaime Espin; Michael Schlander; Brian Godman; Pippa Anderson; Jorge Mestre-Ferrandiz; Isabelle Borget; Adam Hutchings; Steven Flostrand; Adam Parnaby; Claudio Jommi Journal: Appl Health Econ Health Policy Date: 2018-12 Impact factor: 2.561