GVS advice on the reassessment of tafamidis (Vyndaqel®)
Zorginstituut Nederland has completed its reassessment whether tafamidis (Vyndaqel®) can be included in the Medication Reimbursement System (GVS). Tafamidis (Vyndaqel®) is indicated for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). The Zorginstituut recommends not to include tafamidis in the GVS, unless a price reduction of at least 50% can be negotiated and agreements can be made regarding appropriate and efficient use of the treatment.
Registered indication
Tafamidis 61 mg free acid (Vyndaqel®) is indicated for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM), who are in NYHA class I or II. The reason for this advice is the manufacturer’s request for a reassessment of tafamidis 61 mg free acid.
Zorginstituut's advice
The Zorginstituut has concluded that tafamidis 61 mg free acid (Vyndaqel®) is not interchangeable with another product included in the GVS. This means that tafamidis is eligible for inclusion on List 1B. Tafamidis 61 mg free acid complies with the established medical science and medical practice for the treatment of patients with wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy and who are in NYHA class I or II.
However, the Zorginstituut also concludes that tafamidis 61 mg free acid cannot be provided at socially acceptable costs. The Zorginstituut therefore recommends the Minister not to include tafamidis 61 mg free acid on List 1B of the GVS, unless the following conditions are met:
- a substantial price reduction (of at least 50%) of tafamidis 61 mg free acid, given the very high budgetary impact and the unfavourable cost-effectiveness ratio;
- agreement about the appropriateness (in an orphan drug arrangement) for effective use of the treatment. The Zorginstituut is already involved in discussions with stakeholders responsible for providing treatment about how arrangements for appropriate use can be made.
Orphan drug arrangement
If it is included in the health care package, the Zorginstituut will set up an orphan drug arrangement with the stakeholders. This will contain agreements about the starting and stopping criteria, a committee for indications, and a register for collecting and evaluating data.
If the application of tafamidis 61 mg free acid is included in the health care package after a successful price negotiation, the Zorginstituut recommends the following reimbursement condition:
Condition for tafamidis 61 mg free acid (Vyndaqel®):
- Only for insured parties aged eighteen or older with wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy and who are in NYHA class I or II.