Orphan drug arrangement givosiran (Givlaari®) for the treatment of acute hepatic porphyria
Agreements have been made to promote appropriate use of givosiran (Givlaari®) as treatment of acute hepatic porphyria, a rare metabolic disease. The agreements were made following the National Health Care Institute's 2021 advisory report, in which we advised to only reimburse givosiran after price reduction and with agreements on appropriate use. The agreements are laid down in the orphan drug arrangement.
Acute hepatic porphyria is a rare metabolic disease
Acute hepatic porphyria is relatively most common in women of childbearing age and has a major impact on a person's ability to function and their quality of life. A small group of patients, estimated to be 15 to 21 persons in the Netherlands, suffer from regular porphyria attacks: 2 or more every 6 months. People with acute porphyria may experience abdominal pain attacks. These may be associated with pain in the back and upper legs. Nausea, vomiting and constipation are common. Patients may also become confused and paralysis symptoms may occur. Severe attacks can lead to epilepsy and coma.
Package management orphan drugs
At the end of 2015, the National Health Care Institute published the ‘Packet management orphan drugs’ report. It states that it is important to allow orphan drugs to be admitted into the basic health care package in a responsible way, if the National Health Care Institute, after weighing the package principles, concludes that it is desirable to include the product in the basic health care package. This responsible approach is often linked to high costs or unfavourable cost-effectiveness. In this way, we will ensure the accessibility of orphan drugs, promote effective use and control the risks for the basic health care insurance.
Orphan drug arrangement
In the orphan drug arrangements on givosiran, agreements have been made on:
- Which patients are eligible for treatment with givosiran, in other words the so-called starting criteria.
- How often the treating physician should evaluate the treatment and under what circumstances it is more appropriate to discontinue givosiran treatment, in other words the so-called stop criteria.
- Setting up an indication committee. This committee decides per case whether to start givosiran and when it is more appropriate to stop the treatment, based on the start and stop criteria.
- The most optimal dose and dose reduction.
- Preventing waste of givosiran.
- Data collection and reporting with the aim of monitoring the implementation of the start and stop criteria in practice.