Budgetary Impact and Cost Drivers of Drugs for Rare and Ultrarare Diseases

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
Michael SchlanderAfschin Gandjour

Abstract

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. 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. 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 prevalen...Continue Reading

Citations

Jan 7, 2020·PharmacoEconomics·Franziska Worm, Charalabos-Markos Dintsios
Mar 29, 2019·The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care·Oriol de Sola-Morales
Jun 15, 2020·International Journal of Cancer. Journal International Du Cancer·Tao RanMichael Schlander
Aug 9, 2018·Applied Health Economics and Health Policy·Jaime EspinClaudio Jommi
Dec 14, 2019·Health Economics Review·Ana BabacJ-Matthias Graf von der Schulenburg
Dec 13, 2019·Orphanet Journal of Rare Diseases·Jorge Mestre-FerrandizAdam Parnaby
May 27, 2020·The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care·Afschin GandjourCharalabos-Markos Dintsios
Nov 11, 2018·PharmacoEconomics·J Jaime CaroMichael Schlander
Feb 4, 2021·International Journal of Environmental Research and Public Health·Patrizio ArmeniYllka Kodra
Apr 15, 2021·European Journal of Human Genetics : EJHG·Tessel RigterMartina C Cornel
May 11, 2021·Frontiers in Pharmacology·Khadidja AbdallahSteven Simoens

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