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Methods and criteria for the assessment of orphan drugs: a scoping review

Published online by Cambridge University Press:  22 June 2022

Marita Mohammadshahi Open the ORCID record for Marita Mohammadshahi [Opens in a new window] ,
Alireza Olyaeemanesh Open the ORCID record for Alireza Olyaeemanesh [Opens in a new window] ,
Elham Ehsani-Chimeh Open the ORCID record for Elham Ehsani-Chimeh [Opens in a new window] ,
Mohammadreza Mobinizadeh Open the ORCID record for Mohammadreza Mobinizadeh [Opens in a new window] ,
Zeinab Fakoorfard Open the ORCID record for Zeinab Fakoorfard [Opens in a new window] ,
Ali Akbari Sari  and
Mohammad Aghighi
Marita Mohammadshahi
Affiliation:
Health Economics, National Institute for Health Research, Tehran University of Medical Sciences, Tehran, Iran
Alireza Olyaeemanesh
Affiliation:
Health Policy, National Institute for Health Research and Health Equity Research Center (HERC), Tehran University of Medical Sciences, Tehran, Iran
Elham Ehsani-Chimeh
Affiliation:
Health Services Management, National Institute for Health Research, Tehran University of Medical Sciences, Tehran, Iran
Mohammadreza Mobinizadeh*
Affiliation:
Health Services Management, National Institute for Health Research, Tehran University of Medical Sciences, Tehran, Iran
Zeinab Fakoorfard*
Affiliation:
Health Economics, National Institute for Health Research, Tehran University of Medical Sciences, Tehran, Iran
Ali Akbari Sari
Affiliation:
Health Policy, Department of Health Management and Economics, School of Public Health, Tehran University of Medical Sciences, Tehran, Iran
Mohammad Aghighi
Affiliation:
Head of Special Diseases Office, Vice Chancellery for Treatment, Iran Ministry of Health and Medical Education, Tehran, Iran
*
* Authors for correspondence: Mohammadreza Mobinizadeh and Zeinab Fakoorfard, E-mail: mr.mobini1986@gmail.com; z-fakoorfard@farabi.tums.ac.ir
* Authors for correspondence: Mohammadreza Mobinizadeh and Zeinab Fakoorfard, E-mail: mr.mobini1986@gmail.com; z-fakoorfard@farabi.tums.ac.ir
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Abstract

Objectives

This study aimed to identify different criteria for priority setting of rare diseases to help policy makers in making evidence-informed decisions.

Methods

A scoping review was conducted to comprehensively examine the existing various methods and criteria for prioritizing orphan drugs and rare diseases. We performed searching in Scopus, PubMed, Embase, and websites of health technology assessment (HTA) agencies, 2000–21, and data were extracted.

Results

From the 1,580 identified publications, eleven articles were included. Multicriteria decision analysis was the most frequent method (seven out of eleven studies) used for priority setting. The extracted criteria for priority setting of orphan products were analyzed based on six main categories as follows: health outcomes and clinical implications (six subsets which showed clinical implications), economic aspects (four subsets that indicated the economic effects of orphan drugs and rare diseases), disease and population characteristics (six subsets that included the characteristics of the rare diseases), therapeutic alternatives and uniqueness of orphan technologies (two subsets which discussed the alternatives and uniqueness of orphan technologies), evidence (three subsets which regarded the quality and availability of evidence), and other criteria (three subsets dealing with social and organizational criteria). Cost-effectiveness, budget impact, and disease severity were the most frequent criteria in the studies.

Conclusions

Because of the high price of orphan drugs and limitations of using HTA for reimbursement of them, it is critical to explore them by precise technical methods like multiple criteria decision making in priority setting.

Keywords

Orphan drugsRare diseasesPriority settingCriteria
Type
Policy
Information
International Journal of Technology Assessment in Health Care , Volume 38 , Issue 1 , 2022 , e59
Copyright
© The Author(s), 2022. Published by Cambridge University Press

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