Advanced search
Publication Cover
Journal of Market Access & Health Policy Volume 4, 2016 - Issue 1
Journal homepage
1,497
Views
13
CrossRef citations to date
0
Altmetric
Position Papesr

Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement

Michael SchlanderInstitute of Public HealthMannheim Medical Faculty, University of Heidelberg, Mannheim, Germany;University of Applied Economic Sciences LudwigshafenLudwigshafen, Germany;Institute for Innovation and Valuation in Health Care, Wiesbaden, GermanyCorrespondence[email protected] [email protected]
,
Silvio GarattiniIRCCS – Istituto di Ricerche Farmacologiche Mario Negri, Milan, Italy
,
Peter Kolominsky-RabasInterdisziplinäres Zentrum für Public Health, Universitätsklinikum Erlangen, Erlangen, Germany
,
Erik NordSchool of PharmacyUniversity of Oslo, Oslo, Norway
,
Ulf PerssonThe Swedish Institute for Health EconomicsLund, Sweden
,
Maarten PostmaDepartment of PharmacyUniversity of Groningen, Groningen, Netherlands;Department of EpidemiologyUniversity Medical Center Groningen, University of Groningen, Groningen, Netherlands
,
Jeff RichardsonCentre for Health EconomicsMonash University, Clayton, Victoria, Australia
,
Steven SimoensDepartment of Pharmaceutical and Pharmacological SciencesKatholieke Universiteit (KU) Leuven, Leuven, Belgium
,
Oriol de Solà-MoralesHealth Innovation Technology Transfer (HITT)Barcelona, Spain
,
Keith TolleyTolley Health Economics LtdBuxton, Derbyshire, Great Britain
&
Mondher ToumiPublic Health Department – Research Unit EA 3279, Faculty of Medicine, Aix Marseille University, Marseille, France
 show all
Article: 33039 | Received 01 Aug 2016, Accepted 21 Sep 2016, Published online: 27 Oct 2016

Citations (13)

Keep up to date with the latest research on this topic with citation updates for this article.

Subscribe to citation updates

Read on this site (2)

Domenico Moro, Michael Schlander, Harry Telser, Oriol Sola-Morales, Michael David Clark, Andrew Olaye, Charlotte Camp, Mohit Jain, Thomas Butt & Sumeet Bakshi. (2022) Evaluating discrete choice experiment willingness to pay [DCE-WTP] analysis and relative social willingness to pay [RS-WTP] analysis in a health technology assessment of a treatment for an ultra-rare childhood disease [CLN2]. Expert Review of Pharmacoeconomics & Outcomes Research 22:4, pages 581-598.
Read now
Maria Kamusheva, Konstantin Tachkov, Guenka Petrova, Alexandra Savova & Manoela Manova. (2018) Orphan medicinal products’ access to the Bulgarian pharmaceutical market – challenges and obstacles. Expert Opinion on Orphan Drugs 6:2, pages 95-104.
Read now

Articles from other publishers (11)

F. Reed Johnson, Angelyn Fairchild, Dale Whittington, Amit K. Srivastava, Juan Marcos Gonzalez & Liping Huang. (2022) How Does the Public Evaluate Vaccines for Low-Incidence, Severe-Outcome Diseases? A General-Population Choice Experiment. The Patient - Patient-Centered Outcomes Research 16:2, pages 139-151.
Crossref
Néboa Zozaya, Teresa Caballero, Teresa González-Quevedo, Pedro Gamboa Setien, M. Ángeles González, Ramón Jódar, José Luis Poveda-Andrés, Encarna Guillén-Navarro, Agustín Rivero Cuadrado & Álvaro Hidalgo-Vega. (2022) A multicriteria decision analysis (MCDA) applied to three long-term prophylactic treatments for hereditary angioedema in Spain. Global & Regional Health Technology Assessment 9, pages 14-21.
Crossref
Alessandra Blonda, Teresa Barcina Lacosta, Mondher Toumi & Steven Simoens. (2022) Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries. Frontiers in Pharmacology 12.
Crossref
Michał Jakubczyk, Maciej Niewada, Robert Plisko, Magdalena Władysiuk, Michał Jachimowicz, Cezary Pruszko, Katarzyna Wepsięć & Joanna Lis. (2021) What matters in treating non‐oncological rare diseases?—Eliciting experts' preferences in Poland with PAPRIKA . Journal of Multi-Criteria Decision Analysis 29:1-2, pages 110-121.
Crossref
Michael Schlander, Karla Hernandez-Villafuerte, Chih-Yuan Cheng, Jorge Mestre-Ferrandiz & Michael Baumann. (2021) How Much Does It Cost to Research and Develop a New Drug? A Systematic Review and Assessment. PharmacoEconomics 39:11, pages 1243-1269.
Crossref
Bettina M. Zimmermann, Johanna Eichinger & Matthias R. Baumgartner. (2021) A systematic review of moral reasons on orphan drug reimbursement. Orphanet Journal of Rare Diseases 16:1.
Crossref
Alessandra Blonda, Yvonne Denier, Isabelle Huys & Steven Simoens. (2021) How to Value Orphan Drugs? A Review of European Value Assessment Frameworks. Frontiers in Pharmacology 12.
Crossref
Cornelis Boersma & Maarten J. Postma. (2021) Health Economics of Vaccines: From Current Practice to Future Perspectives. Value in Health 24:1, pages 1-2.
Crossref
A. van Egmond-Fröhlich & K. Schmitt. (2018) Öffentliche Lenkung und Preisbegrenzung für Orphan-DrugsPublic guidance and price limitation for orphan drugs. Monatsschrift Kinderheilkunde 166:9, pages 785-797.
Crossref
Kati Sarnola, Riitta Ahonen, Jaana E. Martikainen & Johanna Timonen. (2018) Policies and availability of orphan medicines in outpatient care in 24 European countries. European Journal of Clinical Pharmacology 74:7, pages 895-902.
Crossref
Oriol de Solà-Morales, David Cunningham, Mathias Flume, Paul M. Overton, Natalie Shalet & Stefano Capri. (2018) DEFINING INNOVATION WITH RESPECT TO NEW MEDICINES: A SYSTEMATIC REVIEW FROM A PAYER PERSPECTIVE. International Journal of Technology Assessment in Health Care 34:3, pages 224-240.
Crossref

Related research

People also read lists articles that other readers of this article have read.

Recommended articles lists articles that we recommend and is powered by our AI driven recommendation engine.

Cited by lists all citing articles based on Crossref citations.
Articles with the Crossref icon will open in a new tab.