The uncertainty about the added value of orphan medicinal products (OMPs) is often substantially high because the available clinical studies contain limited information about clinical outcomes and long-term effectiveness. Despite the uncertainties, there is social pressure to make these highly prized OMPs quickly available for a small group of patients with life-threatening conditions. The question is whether a cyclic Health Technology Assessment (HTA) approach could contribute to more controlled and affordable access to these medicines.

Your job
You will be on the frontline of developing a cyclic HTA approach to OMPs. Your work will focus on:

• mapping current international activities on cyclic HTA for OMPs. The emphasis will be on which elements of cyclic HTA approach, such as horizon scanning, scientific advice, assessment of efficacy and cost-effectiveness, and appropriate use, should have an initial priority in establishing cyclic HTA of OMPs;
• assessing whether and how the available national and international horizon scanning programmes maybe used to facilitate cyclic HTA of OMPs;
• evaluating current scientific methods for determining the value of OMPs (relative effectiveness and cost-effectiveness) and considering whether new, more experimental methods might be used in the future;
• determining the role of international patient registries for orphan diseases in monitoring appropriate care and using managed entry arrangements for OMPs.

The methods you will apply to study these questions include comparative document analyses, quantitative descriptive and association studies, and complex systems analyses.

This PhD position is part of the Academic Research Network HTA, a collaboration between the National Health Care Institute (Zorginstituut Nederland (ZIN)), Erasmus University Rotterdam, and Utrecht University. The network aims to closely bridge HTA research and HTA policy by supporting specific HTA research projects closely linked to policy questions. That means you will work closely with other PhD candidates within this network and structurally interact with the HTA policy officers at ZIN. Moreover, your work will entail close collaboration with stakeholders such as patients, clinicians and health technology developers on a national and international level.

We are looking for a candidate who meets the following criteria:

• an MSc in pharmaceutical or (bio)medical sciences, health economics and/or health technology assessment, epidemiology, or another relevant field;
• the ability and enthusiasm to learn quantitative and qualitative methods within Health Technology Assessment;
• an interest in cross-disciplinary work within Health Technology Assessment;
• the ability to work independently, be self-reflective, and take initiative and responsibility for your projects.
• good oral and written communication skills in English and Dutch are essential to ensure effective collaboration and communication with the Dutch health technology assessment stakeholders’ community.

We consider it a plus if you have experience with:

• complex health systems (i.e. international health policies);
• data analyses; qualitative (e.g. NVivo) and/or quantitative (e.g. R, SAS, or SPSS);
• working with different types of stakeholders, such as HTDs, clinicians, and healthcare decision-makers from public institutes or governments;
• working in a dynamic environment on multiple projects simultaneously;
• teaching graduate students and supervising bachelor and master research projects within the pharmaceutical policy domain.

• a position (1.0 FTE) for one year, with an extension to a total of four years upon successful assessment;
• a full-time gross salary between €2,770 in the first year and €3,539 in the fourth year of employment in scale P of the Collective Labour Agreement Dutch Universities (CAO NU);
• 8% holiday bonus and 8.3% end-of-year bonus;
• a pension scheme, partially paid parental leave, and flexible employment conditions based on the Collective Labour Agreement Dutch Universities.

In addition to the employment conditions from the CAO for Dutch Universities, Utrecht University has a number of its own arrangements. These include agreements on professional development, leave arrangements, sports and cultural schemes and you get discounts on software and other IT products. We also give you the opportunity to expand your terms of employment through the Employment Conditions Selection Model. This is how we encourage you to grow. For more information, please visit working at Utrecht University.

Universiteit Utrecht

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You will be employed within the Division of Pharmacoepidemiology and Clinical Pharmacology, one of the five divisions of the Utrecht Institute for Pharmaceutical Sciences (UIPS). This is the research institute of the Department of Pharmaceutical Sciences of Utrecht University.

The Division of Pharmacoepidemiology and Clinical Pharmacology comprises a multidisciplinary team of young, internationally oriented-researchers. The research programme is directed at several epidemiological, therapeutic, and policy aspects of chronic drug use, focusing on advanced, affordable biomolecular and cellular therapies. The PhD position falls under the Centre for Pharmaceutical Policy & Regulation (one of the three Centres of the Division). The Centre aims to develop, assess, and apply innovative methods in pharmaceutical policy, drug regulatory science, and health technology assessment (HTA). For this, an integrated approach to the drug life cycle and the context in which medicines are developed, assessed, and used is taken. Necessary research spearheaded by the Centre includes questions on authorisation, access to, and affordability of medicines. One of the unique features of the Centre is that it works closely with experts from the Dutch Medicines Evaluation Board (MEB), the Dutch National Health Care Institute (ZIN), and the International Pharmaceutical Federation (FIP). The WHO Collaborating Centre for Pharmaceutical Policy and Regulation is embedded in the Centre, which focuses on global access to medicines in pharmaceutical systems—the centre partners with the Regulatory Science Network Netherlands (RSNN) and the Academic Research Network HTA.