Clinical and economic assessment of nusinersen: the Bulgarian perspective

ABSTRACT

Introduction

Spinal muscular atrophy (SMA) is a very serious debilitating rare condition mainly affecting newborns and infants.

Areas covered

The aim of current chapter is to present the standard of care and treatment available in Bulgaria from both clinical and economic point of view. The authors are presenting the latest clinical studies in the area of this rare neuromuscular disorder as well as describing a very detailed economic evaluation from the perspective of Bulgarian healthcare insurance fund regarding Nusinersen. A systematic literature review of the published clinical studies of nusinersen for the period March 2015 – March 2019 was performed following predefined criteria.

Expert opinion

Nusinersen is a significant therapeutic advancement, and is the first option to delay the progression of the disease. A number of clinical trials have demonstrated the efficacy and tolerability of nusinersen and achieving better clinical outcomes after its use compared to placebo. Despite the expected significant increase of the budget for SMA, nusinersen provides new possibilities of treatment of children with SMA in Bulgaria and innovative disease-modifying approach to the unmet medical needs of the patients and their families.

KEYWORDS:

• Spinal muscular atrophy
• therapeutic options
• safety
• efficacy
• economic evaluation

Article Highlights

• Nusinersen and a new gene therapy approved in 2019 could be defined as a “therapeutic hope” for patients suffering from spinal muscular atrophy (SMA).

• A number of studies demonstrate nusinersen efficacy and safety as more real-world evidences are needed.

• Still scarce information regarding the effect of nusinersen on the SMA clinical progress is available.

• New alternative therapies such as gene replacement therapy are becoming available on the market or are still under investigation.

• Reimbursement of nusinersen for Bulgarian patients under 18 years of age is provided despite the high related costs. So, access of Bulgarian patients with 5qSMA to this innovative medicine is assured.

Acknowledgments

We would like to express our very deep gratitude to Professor Guenka Petrova for her guidance, encouragement, constructive suggestions, valuable support and reliable critiques during planning, conducting, and developing the content of current article.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Supplementary material

Supplemental data for this article can be accessed here.

Additional information

Funding

This paper was not funded.