Journal Article

Pricing Zolgensma - the world's most expensive drug

Nuijten, Mark

2022

Overview

A heated discussion has recently broken out in Europe about the price of Zolgensma, ‘the most expensive drug ever’. The National Institute for Health and Care Excellence (NICE) approved Zolgensma in March this year, which is set to become the most expensive treatment ever approved by NICE. Zolgensma is a gene therapy medicine for treating spinal muscular atrophy (SMA), a serious and rare condition of the nerves that causes muscle wasting and weakness [1]. It is estimated that the drug will cost approximately €1.9 million per course of treatment [2]. Patients with SMA have a defect in a gene known as SMN1, which the body needs to make a protein essential for the normal functioning of nerves that control muscle movements. Zolgensma is a gene therapy containing a functional copy of this gene which, after injection, passes into the nerves from where it provides the correct gene to make enough of the protein and, thereby, restore nerve function [1]. At first impression, the price level of Zolgensma raises many understandable questions, because €1.9 million sounds exorbitantly high in the public domain (often driven by emotions and lack of specialised knowledge of the costs and risk of the development of a new pharmaceutical). However, there are many factors that may justify NICE’s decision to approve the intervention for use. In the Netherlands, since the debate in 2013 about the high price of medicines for Fabry and Pompe diseases, ‘expensive’ medicines are increasingly only reimbursed after tough price negotiations with the Ministry of Health [3]. This usually concerns medicines for the treatment of rare diseases, the so-called ‘orphan drugs’ such as Zolgensma. For example, it is estimated that only one in 11,000 children is born with SMA [4]. These price negotiations have since become a permanent and important part of the market access process for new ‘expensive’ orphan drugs, where expenditure weighed against patient suffering, a difficult and ethically difficult task for all parties [3].

• Publisher: Routledge,Taylor & Francis Ltd,MDPI AG
• Subject: Biotechnology industry / Discounted cash flow / Disease / Drugs / Emotions / Ethics / Gene therapy / Health care / Health services / Intervention / Medical treatment / Medicine / Muscles / Negotiation / Nerves / Neuromuscular diseases / orphan disease / Patients / Pharmaceuticals / Price levels / Price valuation reimbursement / Prices / Production costs / Public domain / R&D / Rare diseases / Reimbursement / Research & development / Short Communication / Strength / Therapy / Valuation