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.