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Can Sma Innovative Treatments Be Reimbursed? a Rapid Review Publisher



Yaftian F1 ; Mobinizadeh M2 ; Olyaeemanesh A3 ; Mohamadi E3 ; Habibi F4
Authors
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Authors Affiliations
  1. 1. National Center for Health Insurance Research, Tehran, Iran
  2. 2. National Institute for Health Research, Tehran University of Medical Sciences (TUMS), Tehran, Iran
  3. 3. Health Equity Research Center (HERC), Tehran University of Medical Sciences (TUMS), Tehran, Iran
  4. 4. Department of Health Economics and Management, School of Public Health, Tehran University of Medical Sciences, Tehran, Iran

Source: Iranian Journal of Pediatrics Published:2024


Abstract

Context: Spinal muscular atrophy (SMA) is a rare genetic disorder that significantly impacts the quality of life. This paper aims to gather current understanding of the safety, efficacy, and economic aspects of common SMA treatments to assist Iranian policymakers in adopting these novel treatments for this vulnerable population. Methods: This rapid health technology assessment (HTA) research was conducted in four steps: Database review, screening, data extraction, and thematic analysis. Inclusion criteria consisted of studies focused on assessing the safety, efficacy, and economic aspects of medical interventions in SMA patients compared to those who did not receive such interventions. Results: Based on current data, nusinersen was found to be the most effective treatment (increasing mobility achievements in SMA types 1 and 2) with the least side effects for SMA types 1 and 2. In terms of economic evaluations, none of the treatments were found to be cost-effective. Conclusions: The decision regarding reimbursement for such medical drugs should not focus solely on their cost-effectiveness but rather on creating access to essential care, meeting patient needs, and considering national budget limitations. Managed entry agreements (MEAs) are flexible tools that service providers or payers can use to negotiate and reduce the financial burden for both patients and payers. © 2024, Yaftian et al.