GVS assessment of tafamidis (Vyndaqel®) for the treatment of wild-type or hereditary transthyretin amyloidosis

Zorginstituut Nederland has completed its assessment whether tafamidis (Vyndaqel®) for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) can be included in the Medication Reimbursement System (GVS). The Zorginstituut's advice is not to include tafamidis 61 mg free acid (Vyndaqel®) in the GVS.

Registered indication

Tafamidis 61 mg free acid is indicated for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). It is available as a capsule and contains 61 mg of tafamidis (free acid). The recommended dosage is one capsule once a day, added to the standard treatment.

Interchangeability

To determine the placement of a medicinal product in the GVS, its interchangeability with medicinal products already included in the GVS must first be assessed.

The tafamidis capsule 61 mg (free acid) is registered under the same brand name as the tafamidis capsule 20 mg (Vyndaqel®). The tafamidis capsules 20 mg are already included in the GVS on List 1B. Vyndaqel® capsule 20 mg is registered for the treatment of transthyretin amyloidosis in adults with stage 1 symptomatic polyneuropathy, to delay peripheral neurologic impairment. The reimbursement of tafamidis capsule 20 mg is limited to the registered indication via List 2 of the Health Insurance Regulation.

Tafamidis 20 mg is not registered for the treatment of cardiomyopathy. For the tafamidis capsules 61 mg (free acid), reimbursement is now requested for the use in case of cardiomyopathy.

Therapeutic value

The Zorginstituut has concluded that tafamidis 61 mg free acid has a therapeutic added value compared to placebo in the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) and NYHA class I or II. The added value consists of a reduced risk of death and a better quality of life.

Cost-effectiveness

The cost-effectiveness analysis provided by the market authorisation holder is of insufficient quality, despite the fact that the market authorisation holder has been given and has made use of the opportunity to improve it. In particular, the Zorginstituut has observed uncertainties about the validity and transparency of the pharmaco-economic analysis.

The Zorginstituut cannot provide a realistic estimate of cost-effectiveness, nor can it give an indication of the price reduction required to get close to an acceptable cost-effectiveness.

Unfortunately, this means that the Zorginstituut cannot give advice on possible price negotiations. This is essential for the minister of Medical Care and for the Zorginstituut, because reimbursement of tafamidis 61 mg free acid at the current asking price by the market authorisation holder leads to a very high budget impact.

Zorginstituut's advice

The Zorginstituut advices the Minister of Medical Care not to include tafamidis 61 mg free acid (Vyndaqel®) in the GVS. The Zorginstituut remarks that it is aware that the outcome of the assessment is disappointing for both patients and treating physicians. The Zorginstituut therefore invites the market authorisation holder to better substantiate the cost-effectiveness.

This report is a summary of recommendations by Zorginstituut Nederland. The original text is in Dutch.