The Dutch HTA system for high-cost drugs in hospitals

The Netherlands has a statutory social health insurance system with a relatively generous basic benefit package. This open system allows more than 90 percent of services and technologies to be included in the benefit package without the need for a formal health technology assessment (HTA) or a national reimbursement decision.

If doctors consider that the effectiveness of a service or technology is sufficiently proven and the benefit-risk ratio is positive, they can offer it. Negotiations with individual health insurance companies about reimbursement are then necessary. Only if a service or technology requires a significant additional budget will it be necessary to submit a request for a new payment code. In such cases, the decision can be based on a formal HTA carried out by the Dutch HTA body, the Zorginstituut Netherlands (ZIN).

The open cumulation process provides for three exceptions that require mandatory health technology assessment (HTA): certain drugs (see Figure 1), the national vaccination programme and the national screening programme. Reimbursement of expensive drugs for hospital treatment, especially oncology drugs, represents a major challenge as they take up an increasing share of the hospital budget. To address this problem, the Dutch government has introduced the so-called “blocking procedure” to manage market access. Drugs are included in the “ban” if they meet certain criteria:¹

• If the total costs of a new indication or several new indications together are expected to exceed EUR 20 million per year, all new and future indications will be included in the block (before 1 July 2023 they were more than EUR 40 million);
• If the total costs for a new indication are ≥ 50,000 euros per patient per year AND the total costs are > 10 million euros per year, the indication will be included in the blocking process.

As part of the blocking procedure, developers must submit a comprehensive pharmacoeconomic dossier in addition to the clinical dossier. ZIN evaluates this dossier based on four main criteria:

• Necessity, determined primarily by the severity of the disease, requiring that treatment be reimbursed from public funds;
• Relative efficacy, measured by the additional therapeutic benefit compared to existing standard treatment;
• cost-effectiveness, measured by the cost per quality-adjusted life year (QALY) gained;
• Viability, which is defined primarily in financial terms to ensure that the budgetary impact is affordable.

The “cost per QALY” of an intervention is compared to a threshold that increases as disease severity increases (see Figure 2)^two. If the cost per QALY falls below the threshold, the drug is considered cost-effective.

Depending on the assessment, ZIN recommends negotiating a discount on the drug to increase cost-effectiveness and reduce the impact on the budget. Price negotiations are carried out by a team from the Ministry of Health. In 2021, the team managed to negotiate an average discount of 46 percent, resulting in a cost reduction of 745 million euros. Even if this seems to be a success from the public’s point of view, it is not known to what extent the promoters expected this profit margin. Just like the decision of the Minister of Health in 2023, Troveldy^® (sacituzumab govitecan) and Libmeldy^® (Atidarsagen autotemcel) not refunding shows that negotiations are not always successful.

The “lock-in procedure” emerged as a replacement for a relatively unsuccessful experiment with conditional reimbursement arrangements that required reassessment after four years of additional data collection.³ In many cases, the data collected have not significantly reduced uncertainty around effectiveness and health economic evaluations. Although conditional reimbursement remains an option in the Netherlands, it is only used to a limited extent. Furthermore, outcomes-based arrangements for controlled access to medicines are being discouraged due to concerns about social risk tolerance, feasibility, administrative burden, costs associated with measuring outcomes, discussions about compensation in case of inadequate treatment effectiveness, and lack of support facilities for physicians to monitor outcomes have not been widely adopted. Consequently, in the Netherlands, controlled access arrangements predominantly take the form of financial arrangements.

It is also interesting that ZIN has introduced a new assessment framework adapted to tumor diagnosis and drugs recognized by the European Medicines Agency (EMA) based on single-arm studies.⁴ Depending on the context, ZIN may take into account:

• Evidence from single-arm studies, even if they do not allow assessment of efficacy by tumor location;
• Recognition of potential data gaps in the natural history or standard treatment of patients with the same mutations targeted for tumor diagnosis, since these patients have not been studied in the past;
• Acceptance of interim results such as objective response rate (ORR), duration of response (DoR), disease-free survival (DFS) and progression-free survival (PFS), provided that the choice of outcome and the minimal important difference (MID) in PICOs are defined during the scoping phase and these results can predict overall survival (OS);
• Recognition of the need to build a synthetic control arm in health economic models.

These considerations depend largely on the plausibility of the assumption that without treatment there will be no tumor response.

ZIN is gradually migrating to a cyclical, risk-based ATS system⁵starting with reliable strategic foresight methods (so-called horizon scanning) to identify potentially transformative health technologies at an early stage and quantify the associated risks. A rapid risk assessment is then performed, including uncertainties regarding therapeutic value, financial considerations and the potential for inappropriate use. These risks are addressed through prudent risk management strategies that include HTA, conditional reimbursement, controlled approval programs, price negotiations and agreements to ensure appropriate use in practice.

Dr. Maureen Rutten-van Molken is Professor of Economic Evaluation of Health Innovations at the Erasmus School of Health Policy & Management, Erasmus University Rotterdam, and Scientific Director of the Institute for Medical Technology Assessment (IMTA).

She obtained a Master’s degree in Health Policy and Management and a PhD in Health Economics from Maastricht University (1994). In 1996 she joined IMTA as Scientific Director and was Assistant Professor of Health Economics and Medical Technology Assessment at Maastricht University.

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