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Journal of Market Access & Health Policy (JMAHP) is published by MDPI from Volume 12 Issue 1 (2024). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with Taylor & Francis.
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Article

Determining the Value of Medical Technologies to Treat Ultra-Rare Disorders: A Consensus Statement

by
Michael Schlander
1,2,3,*,
Silvio Garattini
4,
Peter Kolominsky-Rabas
5,
Erik Nord
6,
Ulf Persson
7,
Maarten Postma
8,9,
Jeff Richardson
10,
Steven Simoens
11,
Oriol de Solà-Morales
12,
Keith Tolley
13 and
Mondher Toumi
14
1
Institute of Public Health, Mannheim Medical Faculty, University of Heidelberg, Mannheim, Germany
2
University of Applied Economic Sciences Ludwigshafen, Ludwigshafen, Germany
3
Institute for Innovation and Valuation in Health Care, An der Ringkirche 4, DE-65197 Wiesbaden, Germany
4
IRCCS—Istituto di Ricerche Farmacologiche Mario Negri, Milan, Italy
5
Interdisziplinäres Zentrum für Public Health, Universitätsklinikum Erlangen, Erlangen, Germany
6
School of Pharmacy, University of Oslo, Oslo, Norway
7
The Swedish Institute for Health Economics, Lund, Sweden
8
Department of Pharmacy, University of Groningen, Groningen, The Netherlands
9
Department of Epidemiology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands
10
Centre for Health Economics, Monash University, Clayton, VIC, Australia
11
Department of Pharmaceutical and Pharmacological Sciences, Katholieke Universiteit (KU) Leuven, Leuven, Belgium
12
Health Innovation Technology Transfer (HITT), Barcelona, Spain
13
Tolley Health Economics Ltd., Buxton, Derbyshire, Great Britain
14
Public Health Department—Research Unit EA 3279, Faculty of Medicine, Aix Marseille University, Marseille, France
 
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Author to whom correspondence should be addressed.
J. Mark. Access Health Policy 2016, 4(1), 33039; https://doi.org/10.3402/jmahp.v4.33039
Submission received: 1 August 2016 / Revised: 20 September 2016 / Accepted: 21 September 2016 / Published: 27 October 2016

Abstract

Background: In most jurisdictions, policies have been adopted to encourage the development of treatments for rare or orphan diseases. While successful as assessed against their primary objective, these policies have prompted concerns among payers about the economic burden that might be caused by an annual cost per patient in some cases exceeding 100,000 Euro. At the same time, many drugs for rare disorders do not meet conventional standards for cost-effectiveness or ‘value for money’. Owing to the fixed (volume-independent) cost of research and development, this issue is becoming increasingly serious with decreasing prevalence of a given disorder. Methods: In order to critically appraise the problems posed by the systematic valuation of interventions for ultra-rare disorders (URDs), an international group of clinical and health economic experts was convened in conjunction with the Annual European ISPOR Congress in Berlin, Germany, in November 2012. Following this meeting and during subsequent deliberations, the group achieved a consensus on the specific challenges and potential ways forward. Results: The group concluded that the complexities of research and development for new treatments for URDs may require conditional approval and reimbursement policies, such as managed entry schemes and coverage with evidence development agreements, but should not use as justification surrogate end point improvement only. As a prerequisite for value assessment, the demonstration of a minimum significant clinical benefit should be expected within a reasonable time frame. As to the health economic evaluation of interventions for URDs, the currently prevailing logic of cost-effectiveness (using benchmarks for the maximum allowable incremental cost per quality-adjusted life year gained) was considered deficient as it does not capture well-established social preferences regarding health care resource allocation. Conclusion: Modified approaches or alternative paradigms to establish the ‘value for money’ conferred by interventions for URDs should be developed with high priority.
Keywords: orphan drugs; health technology assessment; economic evaluation; cost-effectiveness; social cost value analysis; multicriteria decision making orphan drugs; health technology assessment; economic evaluation; cost-effectiveness; social cost value analysis; multicriteria decision making

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MDPI and ACS Style

Schlander, M.; Garattini, S.; Kolominsky-Rabas, P.; Nord, E.; Persson, U.; Postma, M.; Richardson, J.; Simoens, S.; de Solà-Morales, O.; Tolley, K.; et al. Determining the Value of Medical Technologies to Treat Ultra-Rare Disorders: A Consensus Statement. J. Mark. Access Health Policy 2016, 4, 33039. https://doi.org/10.3402/jmahp.v4.33039

AMA Style

Schlander M, Garattini S, Kolominsky-Rabas P, Nord E, Persson U, Postma M, Richardson J, Simoens S, de Solà-Morales O, Tolley K, et al. Determining the Value of Medical Technologies to Treat Ultra-Rare Disorders: A Consensus Statement. Journal of Market Access & Health Policy. 2016; 4(1):33039. https://doi.org/10.3402/jmahp.v4.33039

Chicago/Turabian Style

Schlander, Michael, Silvio Garattini, Peter Kolominsky-Rabas, Erik Nord, Ulf Persson, Maarten Postma, Jeff Richardson, Steven Simoens, Oriol de Solà-Morales, Keith Tolley, and et al. 2016. "Determining the Value of Medical Technologies to Treat Ultra-Rare Disorders: A Consensus Statement" Journal of Market Access & Health Policy 4, no. 1: 33039. https://doi.org/10.3402/jmahp.v4.33039

APA Style

Schlander, M., Garattini, S., Kolominsky-Rabas, P., Nord, E., Persson, U., Postma, M., Richardson, J., Simoens, S., de Solà-Morales, O., Tolley, K., & Toumi, M. (2016). Determining the Value of Medical Technologies to Treat Ultra-Rare Disorders: A Consensus Statement. Journal of Market Access & Health Policy, 4(1), 33039. https://doi.org/10.3402/jmahp.v4.33039

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