Today is Rare Disease Day — a day to acknowledge the people living with rare conditions and their need for better access to life-changing treatments.
There have been several noteworthy updates to the Nordic market access landscape with regards to rare diseases in the past few months:
In January, the TLV released a report as the outcome of a government assignment aimed at improving access to medicines for severe diseases. From now on, TLV will increasingly consider the number of patients and sales value in decisions on pricing and reimbursement with the aim to enhance access to medicines for patients with severe and rare health conditions
This Wednesday, the DMC approved the gene therapy Casgevy (exagamglogene autotemcel) for patients with severe sickle cell disease and transfusion dependent β-thalassemia. The treatment is the first CRISPR-Cas9-based product to be reimbursed in the Nordics, a promising sign for continued increased access to advanced treatments for patients with rare diseases
The Macanda team is dedicated to supporting clients in bringing innovative orphan therapies to market.
If you are interested in discussing these recent developments or the landscape for rare diseases in general, please reach out to us here or via contact@macanda.se.
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