The objective of this article is not to discuss whether the Norwegian Medicines Agency or Bristol-Myers Squibb have calculated correctly, the objective is to call into question this way of making political decisions.
A number of problems are associated with the sequence of reasoning from quality of life data collected during the trial of ipilimumab (4) to the calculated price per QALY. Small variations in the calculation methods (none of which can be seen as more correct than the others) may have a decisive impact on the conclusion, as we have seen.
The use of highly sophisticated mathematical models gives a false impression of exactitude, and conceals the fact that no estimates are better than the assumptions upon which they rest. Are psychometric data for EORTC QLQ-C30 collected from patients with myelomatosis relevant when the form is to be used to measure the benefits of treatment in patients with melanoma? Are relevant aspects of the patients’ experience of their health condition preserved throughout the almost impenetrably complex processes needed to convert the 3.5 · 1018 response alternatives in EORTC QLQ-C30 to the 81 920 response alternatives in EORTC-8D and from there to the 85 alternatives that were finally evaluated?
Can we accept that Norwegian political priorities are to be based on a Gallup survey of 350 randomly selected persons in Northern England (with approximately 60 % attrition) on how long they would be willing to live with various degrees of symptoms and reduced ability to function? Is the questionnaire technique involved, asking people to weigh years of life in full health against years of life with reduced health, at all acceptable as a method for arriving at a decimal fraction for the value of life? If so, can we estimate the value of those 81 835 health conditions that were not evaluated directly by using regression analyses based on those 85 that were actually assessed? When cost estimates differ by 24 %, is it OK to simply use the average?
The complexity of the methods partly serves to conceal plain errors, as seen in the study done by the Norwegian Medicines Agency (6). More seriously, value choices are here presented as if they were calculations with an exact answer.
It is fine to have an opinion regarding whether NOK 53 – 80 million per year is too much to pay to permit approximately 150 patients with advanced melanoma increase their chances of long-term survival from near zero to around 20 %. This question can be debated openly, counterarguments can be put forward, and the electorate can let their vote be swayed by the response from the politicians. However, when the Norwegian Medicines Agency calculates that ipilimumab costs NOK 990 739 per QALY, the question is transformed into technical sophistry, with which it is impossible to agree or disagree. In terms of democracy and ethics, this is reprehensible, and it means that the agency’s bureaucrats are acting as politicians, but masquerading as researchers.
In the ipilimumab case, it gradually became obvious that the pharmacoeconomic analyses returned answers that were at odds with what most people found reasonable. Therefore, Støre needed to find a way to annul all the calculations made by the civil service, but preferably without making this about-turn appear too obvious.
The pretext was to launch a government-funded research project on predictors of the effect of ipilimumab. Thereby, all relevant patients could receive this as an «investigational drug». The reality is that all patients for whom the medical professionals determine an indication are permanently assured treatment with ipilimumab.
However, the regulations on how pharmacoeconomic analyses are to be performed have not been amended (10). It’s about time they were. Those who have a political point of view should declare it, and not disguise it as quasi-scientific mumbo-jumbo.