National approval of an orphan product in Sweden

Wickstrøm & Langkilde ApS supported Akcea Therapeutics by preparing documentation requested by the NT Council for Tegsedi (inotersen) in Sweden. Based on the clinical and economic evidence, Tegsedi was recommended to be used in Sweden by the NT Council in October 2019.

Tegsedi (inotersen) is indicated for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). hATTR is a rare and devastating disease where patients experience a rapid decline in functional status, mobility, and independence. hATTR is associated with premature death.

Tegsedi was selected for managed introduction in Sweden. Under the managed introduction program, TLV are responsible for assessment of clinical and health economic evidence on behalf of the NT Council. Companies are invited to submit documentation. A key part of the managed introduction program is the possibility of entering into a common agreement with all of the Swedish health care regions.

Akcea Therapeutics engaged Wickstrøm & Langkilde to prepare and submit a dossier for Tegsedi in line with the requirements defined by TLV. This included adaptation of a global model to reflect the country specific requirements. Furthermore, Wickstrøm & Langkilde participated in meetings and negotiations. 

The project was undertaken in close collaboration with the European organization within Akcea Therapeutics, key input was retrieved from leading Swedish specialists and a close dialogue with TLV and the NT Council was ensured throughout the process.

When working in small organizations in the Nordics, the in-depth knowledge of country specific demands when it comes to preparing Health Economic Dossiers to national reimbursement agencies is often not sufficient. Therefore, there is a crucial need for an experienced local contractor to lead the work, preparing the Health Economic Dossier in order to be in line with national demands. With their expertise and knowledge, Wickstrøm & Langkilde ApS, delivered a high-quality submission to TLV and NT Council in Sweden, which lead to the best outcome we could get.

Suzanna Theut Stehr-Nielsen, Head of Nordics and Peter Rydqvist, Field Medical Director Nordics, Akcea Therapeutics