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

Open Access 01-12-2018 | Research

Determining the value contribution of selexipag for the treatment of pulmonary arterial hypertension (PAH) in Spain using reflective multi-criteria decision analysis (MCDA)

Authors: Alberto Jiménez, Arantza Ais, Amélie Beaudet, Alicia Gil

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

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Abstract

Background

Pulmonary Arterial Hypertension (PAH) is a chronic rare disease that can lead to serious cardiovascular problems and death. Additional treatments that increase effectiveness, that are safe and with a convenient administration that improve outcomes and quality of life for patients are needed. The aim of this study was to assess the value contribution of the new, oral prostacyclin receptor agonist, selexipag, for PAH treatment in Spain through reflective Multicriteria Decision Analysis (MCDA) methodology.

Methods

A comprehensive literature review was performed to develop an evidence matrix, composed of twelve quantitative criteria and four contextual criteria, based on an EVIDEM MCDA framework adapted to orphan drugs evaluation by the Spanish region of Catalonia. Quantitative performance scores, qualitative impact of contextual criteria and individual reflections from stakeholders were collected for each MCDA framework criteria. The value contribution of selexipag to PAH treatment compared to inhaled iloprost was calculated.

Results

Oral selexipag for PAH treatment was considered as a treatment which adds value, compared to iloprost, in the following MCDA quantitative criteria: comparative efficacy, patient reported outcomes, preventive benefit, therapeutic benefit, other medical costs and other non-medical costs, without significant differences in safety profile but with a higher acquisition cost than inhaled iloprost.

Conclusions

Selexipag was considered to provide value to PAH treatment. It was perceived as an intervention indicated for a severe rare disease with high unmet needs, supported by high quality clinical evidence. When compared to inhaled iloprost, oral selexipag has demonstrated improvements in efficacy and patient reported outcomes, with a similar safety profile and some additional costs.
Reflective MCDA provided a standardised, transparent approach to evaluate multiple criteria relating to the overall value contribution of selexipag to PAH treatment facilitating decision-making.
Literature
1.
Galiè N, Humbert M, Vachiery J-L, Gibbs S, Lang I, Torbicki A, et al. 2015 ESC/ERS guidelines for the diagnosis and treatment of pulmonary hypertension. Eur Heart J. 2016;37(1):67–119.CrossRefPubMed
2.
Simonneau G, Galiè N, Rubin LJ, Langleben D, Seeger W, Domenighetti G, et al. Clinical classification of pulmonary hypertension. J Am Coll Cardiol. 2004;43(12 SUPPL): 5S-12S.
3.
Peacock AJ, Murphy NF, McMurray JJV, Caballero L, Stewart S. An epidemiological study of pulmonary arterial hypertension. Eur Respir J. 2007;30(1):104–9.CrossRefPubMed
4.
5.
6.
Lang IM, Gaine SP. Recent advances in targeting the prostacyclin pathway in pulmonary arterial hypertension. Eur Respir Rev. 2015;24(138):630–41.CrossRefPubMed
7.
8.
Terap IDEP, Hap L, Ii-iv CF. Informe de Posicionamiento Terapéutico de selexipag ( Uptravi ® ) en hipertensión pulmonar. 2017;1–6.
9.
Sitbon O, Channick R, Chin KM, Frey A, Gaine S, Galiè N, et al. Selexipag for the treatment of pulmonary arterial hypertension. N Engl J Med. 2015;373(26):2522–33.CrossRefPubMed
10.
Bayram MM, Walters B. Assessment Report 2008;44(April):1–12.
11.
Annemans L, Aymé S, Le Cam Y, Facey K, Gunther P, Nicod E, et al. Recommendations from the European working Group for Value Assessment and Funding Processes in rare diseases (ORPH-VAL). Orphanet J Rare Dis. 2017;12(1):50.CrossRefPubMedPubMedCentral
12.
13.
Thokala 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 Heal. 2016;19(1):1-13.
14.
Marsh K, IJzerman M, Thokala P, Baltussen R, Boysen M, Kalo Z, et al. Multiple criteria decision analysis for health care decision making--emerging good practices: report 2 of the ISPOR MCDA emerging good practices task force. Value Heal J Int Soc Pharmacoeconomics Outcomes Res. 2016;19(2):125–37.CrossRef
15.
Gilabert-Perramon A, Torrent-Farnell J, Catalan A, Prat A, Fontanet M, Merino-Montero S, Goetghebeur MMBX. Drug evaluation and decision making in Catalonia: development and validation of a methodological framework based on multi-criteria decision analysis (MCDA) for orphan drugs. ISPOR 20th Annu Eur Congr 4-8 Novemb 2017. Glas Scotl. 2017;1:111–20.
16.
17.
Riva JJ, Malik KM, Burnie SJ, Endicott AR, Busse JW. What is your research question ? An introduction to the PICOT format for clinicians. J Chiropratic Assoc. 2012;56(3):167–71.
18.
19.
20.
21.
22.
23.
EVIDEM Collaboration. A non profit organisation to promote health through efficient decisionmaking. [Internet]. [cited 2017 Jul 18]. Available from: https://​evidem.​org.
24.
25.
European Medicines Agency. Summary of Product Characteristics. Uptravi (selexipag). 2016;279.
26.
European Medicines Agency. Summary of product characteristics. Ventavis (iloprost). 2000;14–6.
27.
McKenna SP, Doughty N, Meads DM, Doward LC, Pepke-Zaba J. The Cambridge pulmonary hypertension outcome review (CAMPHOR): a measure of health-related quality of life and quality of life for patients with pulmonary hypertension. Qual Life Res. 2006;15(1):103–15.CrossRefPubMed
28.
Rabin R, Oemar M, Oppe M, Janssen B, Herdman M. EQ-5D-5L user guide. Basic Inf how to use EQ-5D-5L Instrum. 2015;(April):28.
29.
Garau M, Marsden G, Devlin N, Amedeo N, Profico A. Applying a Multi Criteria Decision Analysis (MCDA) Approach to Elicit Stakeholders Preferences in Italy. The Case of Obinutuzumab for Rituximab Refractory Indolent Non Hodgkin Lymphoma (iNHL). PharmacoEconomics - Open. 2017;11.
30.
31.
32.
Gilabert-Perramon A, Betolaza J, March J, Espín J, Merino-Montero S, Gil A, et al. Multi-Criteria Decision Analysis (MCDA): common tools for different needs supporting healthcare decision making in Spain. ISPOR 19th Annu Eur Congr 29 October-2 Novemb 2016, Vienna; 2016.
33.
34.
Metadata
Title
Determining the value contribution of selexipag for the treatment of pulmonary arterial hypertension (PAH) in Spain using reflective multi-criteria decision analysis (MCDA)
Authors
Alberto Jiménez
Arantza Ais
Amélie Beaudet
Alicia Gil
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/s13023-018-0966-4

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