HTA Quarterly | Fall 2019

By Xcenda

In this issue, our editors examine cost-effectiveness assessments for innovative but high-cost treatments, market access for cell and gene therapies in key European countries, and the newest joint HTA assessment agency, FINOSE.


The Cost of Cures: What Does Zolgensma Teach Us About the Use of Cost-Effectiveness Assessments?

On May 24, 2019, the United States (US) Food and Drug Administration (FDA) approved ZOLGENSMA (onasemnogene abeparvovec), a 1-time treatment for spinal muscular atrophy (SMA) and the first gene therapy approved to treat the most severe form of the disease. Although it was hailed as a medical breakthrough, the media attention was primarily on the US$2.125 million price tag, which currently represents the world’s most expensive medicine. In this article, we explore how and when CE assessments are utilized for making reimbursement and pricing decisions across several major markets.


Market Access for Cell and Gene Therapies in Key European Countries

Cell and gene therapies are classified as advanced therapy medicinal products (ATMPs) by the European Medicines Agency (EMA), which offer groundbreaking new opportunities for the treatment of disease and injury. Several ATMPs have recently received marketing authorization in Europe (Table 1), a trend that is likely to continue and accelerate. Although the precise technology used in each therapy and the disease mechanism targeted for correction vary widely, overall, these therapies attempt to replace or regenerate human cells, tissues, or organs, either to restore or establish normal function.
DNA double helix


FINOSE Finishes 2 Joint HTAs in the Nordics

Since 2016, 4 joint assessment agencies have been created across Europe: BeNeLuxAIr (Belgium, Netherlands, Luxembourg, Austria, and Ireland), La Valetta (Italy, Spain, Greece, Portugal, Slovenia, Cyprus, Malta, and Croatia with France as an observer), Visegrad (Czech, Hungary, Poland, Slovakia, Croatia, and Lithuania), and most recently, FINOSE (Finland, Norway, and Sweden). We take a closer look at FINOSE.
Flags of Finland, Norway, Sweden

Heard on the Street

“Understand the ideas, concerns, and expectations of whoever you are talking to. You have to recognize that around the world, there are some really good people who want to make a difference.”

– Sir David Haslam’s advice to the new Chair at NICE as he prepares to step down after 6 years in the role.

HTA by the Numbers

3 or 3:1 or 3 in 1

The number of agencies concurrently approving combination Keytruda plus Lenvima for the treatment of patients with advanced endometrial carcinoma that is not microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR) and who have disease progression following prior systemic therapy but are not candidates for curative surgery or radiation. The approvals from the US FDA, the Australian Therapeutic Goods Administration, and Health Canada mark the first collaboration between the agencies under Project Orbis. The collaboration aims to reduce the burden and delays associated with regulatory reviews of cancer therapies and identify regulatory divergence across review teams.

Source: Simultaneous review decisions for pembrolizumab plus lenvatinib in Australia, Canada and US