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The Dutch Orphan Drug Access Protocol

Earlier in 2022, in the Netherlands, the Dutch National Health Care Institute (Zorginstituut Nederland) and the platform Medicijn voor de Maatschappij (Medicine for Society, specialising in the availability of drugs for rare diseases) started a pilot of the Orphan Drug Access Protocol (ODAP for short). The ODAP pilot is a significant attempt at mitigating pricing and access problems in the rare disease space, for it seeks to make available promising-but-expensive medicines with highly uncertain effectiveness sooner while collecting more data. According to the ODAP, medicines should serve patients with a defined unmet need without alternatives.

This pilot has outlined that it will experiment with a newly proposed working method for three to five medicines. Every selected medicine is to have a committee with doctors, patient representatives, insurers, pharmacists, and representatives of the National Health Care Institute.

The relevant hospital-based expertise centre for this particular rare disease will set up the registry, collect the data, and report back regularly (Medicijn voor de Maatschappij, 2022). Zorgverzekeraars Nederland (the umbrella organisation for all Dutch health insurers) finances the pilot and will come to an agreement with all concerned parties based on the protocol which will outline the payers for each phase of the protocol.

The phases are:

  1. First, the manufacturer will make the medicines available free of charge pending further data collection;

  2. Then the insurers will pay for the medicines on an individual basis;

  3. Finally, the medicine will be reimbursed ‘at group level’.

The agreements also contain information on the disease and the place of the treatment vis-à-vis other treatments, and specify what data should be collected, potential start and stop criteria for treatment, and when the treatment should be considered effective.

Importantly, these agreements between the different parties should be settled (or be very close to settling) when the promising drug enters the European market. This last point shows that ODAP will likely significantly shorten the usual timeline: in the Netherlands, such drugs for rare diseases tend to go through a lengthy cycle. A medicines’ reimbursement tends to be paused pending a decision by the Minister of Health because of their high price per patient per year or total budget impact.

The Dutch National Health Care Institute then examines the cost-effectiveness and nearly always advises the Minister that because of the significant uncertainty in effectiveness, reimbursement through the Dutch collective basic benefits package is only advisable provided substantial cost reductions. The ‘price negotiation bureau’ at the Ministry then negotiates a secret price reduction with the manufacturer, and the medicine becomes available to patients without further data collection.

The Medicine for Society platform highlights that we need “new routes to access. Routes that ensure that urgently needed medicines become available quickly but that also immediately realise a reasonable price and appropriate use. A new working method is tested with the ODAP pilot” (Medicijn voor de Maatschappij, 2022).

The ODAP, and the pilot, in particular, is thus clearly recognised as an experiment. One definition of an experiment is “an act or operation for the purpose of discovering something unknown or of testing a supposition”. Experiments have positive connotations: they carry ideas of unbiased data collection and careful re-examining and, if necessary, altering one's suppositions once the data is in. Experiments draw stakeholders together, allow for institutional and practical learning, and act as a ‘niche’ in which innovation may develop. As such, experiments are also a crucial mode of social pharmaceutical innovation.

Health care sociologists, however, tell us that policy experimentation is complex, rarely as effective as advertised, and notoriously tricky to ‘scale up’ or implement more permanently. In fact, many policy experiments, especially in health care, have adverse and unexpected consequences for actors involved at a lesser and greater distance from the experiment (Felder, 2020).

Accordingly, those involved in SPIN should remain attentive to any negative consequences, especially affecting stakeholders not directly involved in the experiment, and to the broader system and how it allows or impedes scaling up.



Medicijn voor de Maatschappij, 2022. Orphan Drug Access Protocol. Accessed on 22 July 2022 via:

Felder, M. (2020). Together Alone: An analysis of policy experimentation in Dutch healthcare governance.


Written by: Tineke Kleinhout-Vliek, PhD

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