High-cost rare disease drugs challenge Norwegian health priorities

New medical developments make it possible to treat an increasing number of severe and rare diseases with novel, high-cost pharmaceuticals. At the same time, there are limited resources. The aim of this thesis is to examine how the treatment of rare diseases challenges the priority-setting system in the Norwegian public health service. The findings can help inform Norwegian health policy in this field by contributing to the further development of national priority-setting processes and tools for allocating healthcare resources in clinical practice.

The thesis explores this aim both at the national level and in the encounters between physicians and patients in the clinic.

The first sub-study analyses how Norwegian health priorities were discussed in the media before and after the establishment of the Decision Forum and the National System for Managed Introduction of New Health Technologies (Nye Metoder), based on the debate surrounding three high-cost treatments for cancer and rare diseases between 2013 and 2019. The study shows that these cases engage a wide range of commentators, and that discussions about individual treatments increasingly moved out of party politics after the establishment of Nye Metoder.

The second sub-study investigates which criteria the public emphasises when making priority decisions between hypothetical patient groups. The findings show that patients who are expected to experience reduced informal caregiving needs or improved ability to work - societal criteria - are most likely to be prioritised. Patients who can gain more quality-adjusted life years or who have more severe conditions - health-related criteria - are also prioritised more highly. Responses related to work ability depend on whether the respondents themselves are employed.

The third sub-study is based on interviews with 18 physicians regarding the introduction of a new, high-cost treatment for cystic fibrosis in 2022. Two main findings are described: Physicians negotiate the content of clinical guidelines in their encounters with patients, and they interpret the treatment cost in ways that enable them to continue offering the therapy - even when the effect is uncertain - rather than discontinuing it.

The thesis increases understanding of the priority-setting challenges associated with expensive pharmaceuticals for rare diseases. The findings show that physicians often face difficult situations when translating overarching priority-setting principles into clinical practice, particularly when both the costs and the consequences for other patient groups are unclear. These insights may therefore support the further development of priority-setting tools in the healthcare system. Greater transparency in price agreements and clearer, evidence-based criteria in clinical guidelines for high-cost medicines can provide better support to clinicians when navigating uncertain treatment effects, while also contributing to fairer and more transparent priority-setting in the health service.