Skip to main content
Key Specialties
Cardiology Diabetology Endocrinology Gastroenterology Geriatrics and Gerontology Gynecology and Obstetrics Hematology Infectious Disease Internal Medicine Nephrology Neurology Oncology Pediatrics Respiratory Medicine Rheumatology Surgery
Congress Coverage
2024 ACC Congress 2023 ESMO Congress 2023 EASD Congress 2023 ERS Congress 2023 ESC Congress
Clinical Collections
Advances in Alzheimer’s

At a glance: The ONWARDS insulin icodec trials

A round-up of the ONWARDS phase 3 clinical trials evaluating the novel weekly insulin icodec in people with diabetes.
ADVANCED SEARCH
Log in
Top
Orphanet Journal of Rare Diseases
Published in: Orphanet Journal of Rare Diseases 1/2018

Open Access 01-12-2018 | Research

Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis

Authors: Katarzyna Kolasa, Krzysztof Miroslaw Zwolinski, Vladimir Zah, Zoltán Kaló, Tadeusz Lewandowski

Published in: Orphanet Journal of Rare Diseases | Issue 1/2018

Login to get access

Abstract

Background

A Multi Criteria Decision Analysis (MCDA) technique was adopted to reveal the preferences of the Appraisal Body of the Polish HTA agency towards orphan drugs (OMPs).

Results

There were 34 positive and 23 negative HTA recommendations out of 54 distinctive drug-indication pairs. The MCDA matrix consisted of 13 criteria, seven of which made the most impact on the HTA process. Appraisal of clinical evidence, cost of therapy, and safety considerations were the main contributors to the HTA guidance, whilst advancement of technology and manufacturing costs made the least impact.

Conclusions

MCDA can be regarded as a valuable tool for revealing decision makers’ preferences in the healthcare sector. Given that only roughly half of all criteria included in the MCDA matrix were deemed to make an impact on the HTA process, there is certainly some room for improvement with respect to the adaptation of a new approach towards the value assessment of OMPs in Poland.
Appendix
Available only for authorised users
Literature
1.
2.
3.
Gutierrez L, Patris J, Hutchings A, Cowell W. Principles for consistent value assessment and sustainable funding of orphan drugs in Europe. Orphanet J Rare Dis. 2015;10:53.CrossRefPubMedPubMedCentral
4.
5.
6.
Skoupa J, Annemans L, Hájek P. Health economic data requirements and availability in the European Union: results of a survey among 10 European countries. Value In Health Regional Issues. 2014;4(C):53–7.CrossRef
7.
8.
Iskrov G, Miteva-Katrandzhieva T, Stefanov R. Challenges to orphan drugs access in Eastern Europe: the case of Bulgaria. Health Policy. 2012;108(1):10–8.CrossRefPubMed
9.
10.
11.
12.
Diaby V, Goeree R. How to use multi-criteria decision analysis methods for reimbursement decision-making in healthcare: a step-by-step guide. Expert Rev Pharmacoecon Outcomes Res. 2014;14(1):81–99.CrossRefPubMed
13.
Zah V, Thompson S, Berjan M, Goeree R. Zrx Mcdm: a fully flexible tool to support the local adaptation of multiple-criteria decision criteria in health care. Value Health. 2015;18(7):A703.CrossRefPubMed
14.
Kolasa K, Zwolinski KM, Kalo Z, Hermanowski T. Potential impact of the implementation of multiple-criteria decision analysis (MCDA) into the polish pricing and reimbursement process of orphan drugs. Orphanet J Rare Dis. 2016;11:23.CrossRefPubMedPubMedCentral
15.
Marsh K, IJzerman M, Thokala P, Baltussen R, Boysen M, Kaló Z, Lönngren T, Mussen F, Peacock S, Watkins J, Devlin N. Multiple criteria decision analysis for health care decision making—emerging good practices: report 2 of the ISPOR MCDA emerging good practices task force. Value Health. 2016;19(2):125–37.CrossRefPubMed
16.
Kolasa K, Wasiak R. Health technology assessment in Poland and Scotland: comparison of process and decisions. Int J Technol Assess Health Care. 2012;28(1):70–6.CrossRefPubMed
17.
Thokaka P, Devlin N, Marsh K, Baltussen R, Boysen M, Kalo Z, et al. Multiple criteria decision analysis for health care decision making—an introduction: report 1 of the ISPOR MCDA emerging good practices task force. Value Health. 2016;19:1–13.CrossRef
18.
19.
Lootsma FA. Theory and methodology conflict resolution via pairwise comparison of concessions. Eur J Oper Res. 1989;40:109–16.CrossRef
20.
Salo A, Hamalainen R. On the measurement of preference in the analytic hierarchy process. J Multicrit Decis Anal. 1997;6:309–19.CrossRef
21.
Harker PT, Vargas LG. The theory of ratio scale estimation: Saaty’s analytic hierarchy process. Manag Sci. 1987;33(11):1383–403.CrossRef
22.
Ishizaka A, Balkenborg D, Kaplan T. Influence of aggregation and measurement scale on ranking a compromise alternative in AHP. J Oper Res Soc. 2011;62(4):700–10.CrossRef
23.
Schey C, Krabbe PFM, Postma MJ, Connolly MP. Multi-criteria decision analysis (MCDA): testing a proposed MCDA framework for orphan drugs. Orphanet J Rare Dis. 2017;12(1):10.CrossRefPubMedPubMedCentral
24.
Iskrov G, Miteva-Katrandzhieva T, Stefanov R. Multi-criteria decision analysis for assessment and appraisal of orphan drugs. Front Public Health. 2016;4:214.CrossRefPubMedPubMedCentral
25.
Sussex J, Rollet P, Garau M, Schmitt C, Kent A, Hutchings A. A pilot study of multicriteria decision analysis for valuing orphan medicines. Value Health. 2013;16(8):1163–9.CrossRefPubMed
26.
Kanters TA, de Sonneville-Koedoot C, Redekop WK, Hakkaart L. Systematic review of available evidence on 11 high-priced inpatient orphan drugs. Orphanet J Rare Dis. 2013;8:124.CrossRefPubMedPubMedCentral
27.
Joppi R, Bertele’ V, Garattini S. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur J Clin Pharmacol. 2013;69(4):1009–24.CrossRefPubMed
28.
29.
Onakpoya IJ, Spencer EA, Thompson MJ, Heneghan CJ. Effectiveness, safety and costs of orphan drugs: an evidence-based review. BMJ Open. 2015;5:e007199.CrossRefPubMedPubMedCentral
30.
Aballéa S, Toumi M, Vataire AL, Millier A, Lamure M. PHP6 quantitative analysis of the influence of disease and product characteristics on orphan drug prices in Europe. Value Health. 2010;13(3):A82.CrossRef
31.
Ogbah R. Orphan medicinal products – a European process overview. Regulatory Rapporteur. 2015;12(2):5–11.
32.
Kolasa K, Kalo Z, Hornby E. Pricing and reimbursement frameworks in Central Eastern Europe: decision tool to support choices. Expert Rev Pharmacoecon Outcomes Res. 2015;15(1):145–55.CrossRefPubMed
Metadata
Title
Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis
Authors
Katarzyna Kolasa
Krzysztof Miroslaw Zwolinski
Vladimir Zah
Zoltán Kaló
Tadeusz Lewandowski
Publication date
01-12-2018
Publisher
BioMed Central
Published in
Orphanet Journal of Rare Diseases / Issue 1/2018
Electronic ISSN: 1750-1172
DOI
https://doi.org/10.1186/s13023-018-0803-9

Other articles of this Issue 1/2018

Orphanet Journal of Rare Diseases 1/2018 Go to the issue

Letter to the Editor

Efficacy of sirolimus for the prevention of recurrent pneumothorax in patients with lymphangioleiomyomatosis: a case series

Letter to the Editor

Frequency of hereditary neuropathy with liability to pressure palsies (HNPP) due to 17p11.2 deletion in a Korean newborn population

Research

Missense mutation in SLIT2 associated with congenital myopia, anisometropia, connective tissue abnormalities, and obesity

Research

Recessive mutations in ATP8A2 cause severe hypotonia, cognitive impairment, hyperkinetic movement disorders and progressive optic atrophy

Research

Molecular diagnosis of anti-laminin 332 (epiligrin) mucous membrane pemphigoid

Research

Retinal and optic nerve degeneration in α-mannosidosis