![]() | Audun Brendbekken PhD Candidate - University of Bergen |
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04.08.2025-30.09.2025
High-cost therapies and priority setting: following orphan drugs from public deliberation to clinical rationing
Objectives of the study
The aim is to study public deliberation and preferences concerning reimbursement of high-cost orphan drugs and how clinicians ration such drugs in patient treatment.
Sub-objective 1: To describe and assess the development of the public debate on Norwegian healthcare rationing through three cases in print media.
Sub-objective 2: To explore public preferences for start-stop criteria and alternative pricing agreements regulating access to orphan drugs.
Sub-objective 3: To explore clinicians’ and hospital leaders’ experiences with clinical implementation and rationing of orphan drug CFTR-modulators against cystic fibrosis in Norway.Research questions and study hypotheses
Article 1: “Public participation: Healthcare rationing in the newspaper media.”
Results: We found that New Methods depoliticized the Norwegian healthcare rationing debate. Our findings were published in BMC Health Services Research in April 2022.Article 2: “Public preferences for orphan drug start-stop criteria and price agreements: a discrete choice experiment”
Hypothesis: We expect to find more knowledge on public preferences for start-stop criteria and alternative pricing agreements.Article 3 “Clinical rationing of orphan drugs: decentralized implementation of CFTR- modulators against cystic fibrosis in Norway”
Initial results: Our initial findings relate to clinicians molding stop criteria in order to avoid patients losing access to treatment and to clinicians describing financial incentivization mechanisms changing according to treatment cost allocation levels in hospital budgets.
My PhD project concerns high-cost therapies and priority setting within a Norwegian context, with a focus on high-cost drugs against rare diseases. The project applies both qualitative and quantitative methods in following such drugs from public deliberation to clinical rationing. Priority-setting is challenged by the high cost and long-term evidence uncertainty of these drugs. The aim is to gain knowledge on public preferences for rare disease therapeutics and on the mechanisms for their clinical rationing. Findings will add to literature gaps and be relevant to clinicians, decision-makers, and patients.