Literature DB >> 24453281

Limits on use of health economic assessments for rare diseases.

H I Hyry1, A D Stern, T M Cox, J C P Roos.   

Abstract

Funding of expensive treatments for rare (orphan) diseases is contentious. These agents fare poorly on 'efficiency' or health economic measures, such as the quality-adjusted life years, because of high cost and frequently poor gains in quality of life and survival. We show that cost-effectiveness assessments are flawed, and have only a limited role to play in reimbursement decisions for orphan drugs and beyond.

Mesh：

Year:  2014        PMID： 24453281     DOI： 10.1093/qjmed/hcu016

Source DB:  PubMed          Journal:  QJM        ISSN： 1460-2393


  8 in total

1.  Orphan drugs: expensive yet necessary.

Authors:  H I Hyry; J C Roos; T M Cox
Journal:  QJM       Date:  2015-09-03

2.  Cost-Effectiveness Analysis of Maintenance Olaparib in Patients with Metastatic Pancreatic Cancer and a Germline BRCA1/2 Mutation Based on the POLO Trial.

Authors:  Mei Zhan; Hanrui Zheng; Yu Yang; Zhiyao He; Ting Xu; Qiu Li
Journal:  Cancer Manag Res       Date:  2020-12-16       Impact factor: 3.989

3.  Evaluation of orphan diseases global burden.

Authors:  Ekaterina V Makarova; Ivan S Krysanov; Tatiana P Valilyeva; Mikhail D Vasiliev; Rena A Zinchenko
Journal:  Eur J Transl Myol       Date:  2021-05-14

Review 4.  A systematic review of moral reasons on orphan drug reimbursement.

Authors:  Bettina M Zimmermann; Johanna Eichinger; Matthias R Baumgartner
Journal:  Orphanet J Rare Dis       Date:  2021-06-30       Impact factor: 4.123

5.  Compassionate use of orphan drugs.

Authors:  Hanna I Hyry; Jeremy Manuel; Timothy M Cox; Jonathan C P Roos
Journal:  Orphanet J Rare Dis       Date:  2015-08-21       Impact factor: 4.123

6.  Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs.

Authors:  Katarzyna Kolasa; Krzysztof M Zwolinski; Zoltan Kalo; Tomasz Hermanowski
Journal:  Orphanet J Rare Dis       Date:  2016-03-10       Impact factor: 4.123

7.  The economics of moonshots: Value in rare disease drug development.

Authors:  Nathan Yates; Jennifer Hinkel
Journal:  Clin Transl Sci       Date:  2022-03-25       Impact factor: 4.438

Review 8.  Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries.

Authors:  Tamás Zelei; Mária J Molnár; Márta Szegedi; Zoltán Kaló
Journal:  Orphanet J Rare Dis       Date:  2016-06-04       Impact factor: 4.123
  8 in total

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