Is it ethical to reimburse “SMA Innovative Treatments” from government resources?

Abstract

Abstract Background Spinal Muscular Atrophy (SMA) is a rare genetic disorder that disturbs the quality of life. This paper aims to gather current understanding of safety, efficacy and economic aspects of common SMA treatments to help Iranian policy-makers to adopt such novel treatments for this vulnerable population. Methods This is a rapid health technology assessment (HTA) research that was conducted in four steps: database review, screening, data extraction, and thematic analysis. Inclusion criteria consisted of studies focused on assessing medical interventions’ safety, efficacy and economic aspects in SMA patients compared to those who did not receive such interventions. Results Based on current data Nusinersen was found to be most effective (increased mobility achievements in SMA type 1 and 2) with the least side effects for SMA types 1 and 2. In terms of economic evaluations, all treatments were found to be not cost effective. Conclusions The decision regarding the reimbursement for such medical drugs must not focus on their cost-effectiveness but rather on creating access to such essential care and meeting patient needs and also national budget limitations. Managed Entry Agreements (MEAs) are flexible tools that can be used by service providers or payers to negotiate and reduce the financial burden for patients and payers.