Access to pharmaceuticals for very small patient groups with extremely severe conditions
The Norwegian Medicines Agency has prepared a memorandum that describes arrangements for the single technology assessment (STA) of pharmaceuticals for such patient groups.
In connection with the STA of such pharmaceuticals, the Priority-setting White Paper (Prioriteringsmeldingen) states that the following may be accepted when assessing the documentation:
- a lower level of documentation
- a higher level of resource use for specific interventions compared to other interventions
There are three guiding criteria regarding the assessment of whether or not a pharmaceutical is intended for the treatment of a very small patient group with extremely severe conditions. These are as follows:
- Very small patient group:
a) Fewer than approx. 1 patient per 100,000 inhabitants on a global basis per pharmaceutical
b) Fewer than approx. 50 patients in Norway per pharmaceutical
- Extremely severe condition: Level of severity measured as absolute shortfall corresponding to at least 30 good life years.
- Considerable expected benefit from the pharmaceutical: The expected benefit from the specific treatment is considerable and a minimum of two gained good life years compared to standard treatment.
All three of these indicative criteria should be fulfilled in order for a pharmaceutical to be considered under Section 14-5, third paragraph, of the Norwegian Regulation on Medicinal Products.