An analysis of orphan medicine expenditure in Europe: is it sustainable?

Patient access to orphan drugs in France

(2019) Marion Bourdoncle et al.   Orphanet Journal of Rare Diseases

Outrageous prices of orphan drugs: a call for collaboration

(2018) Lucio Luzzatto et al.   LANCET

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

(2018) Michael Schlander et al.   VALUE IN HEALTH

Projecting Pharmaceutical Expenditure in EU5 to 2021: Adjusting for the Impact of Discounts and Rebates

(2018) Jaime Espin et al.   Applied Health Economics and Health Policy

Gene therapies: the challenge of super-high-cost treatments and how to pay for them

(2016) David R Carr et al.   Regenerative Medicine

Is the Orphanage Filling Up? Projecting the Growth and Budget Impact of Orphan Drugs in Europe

(2016) S Flostrand et al.   VALUE IN HEALTH

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

(2016) Dyfrig A. Hughes et al.   PLoS One

GENERATING HEALTH TECHNOLOGY ASSESSMENT EVIDENCE FOR RARE DISEASES

(2014) Karen Facey et al.   INTERNATIONAL JOURNAL OF TECHNOLOGY ASSESSMENT IN HEALTH CARE

Orphan drugs expenditure in the Netherlands in the period 2006–2012

(2014) Tim A Kanters et al.   Orphanet Journal of Rare Diseases

Estimating the budget impact of orphan medicines in Europe: 2010 - 2020

(2011) Carina Schey et al.   Orphanet Journal of Rare Diseases

Drugs for Rare Diseases: Influence of Orphan Designation Status on Price

(2011) Eline Picavet et al.   Applied Health Economics and Health Policy

Global Spending on Orphan Drugs in France, Germany, the UK, Italy and Spain during 2007

(2010) Javier Orofino et al.   Applied Health Economics and Health Policy