Voretigene neparvovec (Luxturna®) for the treatment of vision loss due to inherited retinal dystrophy with bi-allelic RPE65 mutations

Zorginstituut Nederland has completed its assessment whether voretigene neparvovec (Luxturna®) for the treatment of vision loss due to inherited retinal dystrophy with bi-allelic RPE65 mutations can be included in the insured package. Due to its expected high costs the Minister of Medical Care and Sports has placed voretigene neparvovec in the so-called ‘package lock’ or ‘sluice’ for expensive drugs. These products can only be accepted into the insured package after the Zorginstituut has advised on their inclusion in the insured package and, where applicable, subject to Ministry negotiations to arrive at a financial arrangement with the supplier. The Zorginstituut advises the Minister to only admit the treatment to the package if the price can be significantly reduced.

Criteria for including treatments in the insured package

The Zorginstituut assessed the above-mentioned combination treatment based on the four package criteria effectiveness, cost-effectiveness, necessity and feasibility. Assessing from the perspective of the basic package which is paid from collective premiums, the Zorginstituut looks at whether new care is better than what is currently available. In doing this we look not only at the degree of certainty that has been achieved, from a scientific perspective and from the perspective of societal support, but also at efficiency aspects. The Zorginstituut was advised by two independent committees: the Scientific Advisory Board (WAR) which examines data on established medical science and medical practice and determines the cost-effectiveness, and the Insured Package Advisory Committee (ACP) which considers the societal assessment. Stakeholders are also consulted during the process.

Zorginstuut's advice

The treatment meets the ‘current state of science and practice’ criterion, but looking at the great uncertainties about the long-term effects of this gene therapy and about the cost-effectiveness, the Zorginstituut advises the Minister to only admit the treatment to the package if the price can be significantly reduced. In the price negotiations, we ask to particularly take into account the above-mentioned uncertainty of this gene therapy. A pay for performance agreement, whereby agreements are made about step-by-step compensation linked to the degree of effectiveness and the available burden of proof, would be a very relevant option. Novartis has indicated that they are open to such an agreement.

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