The Coalition for Rare Diseases thanks the Progress Party for choosing to focus on rare diseases with this input meeting. My name is Fredrik Arneberg and I am here with Bjørn-Kristian Svendsrud on behalf of the Coalition for Rare Diseases. "There is something beautiful about a whole society caring about a small group of people". These were the words of Torstein Lerhol, an SMA patient who has waited far too long for effective treatment, on yesterday's Dagsrevyen.
The struggle of Torstein and SMA patients reinforces the Coalition's view that the current funding system is unsustainable and that it primarily affects patients with rare diseases.
On behalf of the Coalition, Menon Economics produced a report last year which found that the cost to society of rare diagnoses is 167 billion a year, compared to 210 billion for cancer. Rare diagnoses consume large amounts of public and family resources and prevent people with rare diagnoses from participating in working life.
In connection with the Medicines White Paper and the Prioritisation White Paper, the funding of medicines for rare diseases was transferred from the National Insurance Scheme to the regional health authorities back in 2019. At the same time, it was decided that all new treatments must undergo a health technology assessment and meet the prioritization criteria. This has proved to have unintended consequences, despite the fact that the Parliament and the Committee had other, better intentions.
The Prioritisation Report assumes that uncertainty about efficacy should count negatively in the decision-making process. Less uncertainty can be achieved by recruiting more patients to the clinical trials. By definition, this cannot be done, because there are few patients to recruit. The committee noted that other requirements should be set for study data for rare diseases. Nevertheless, the Norwegian Medicines Agency's guidelines contain the same documentation requirements for rare diseases as for other diseases. Most decisions in the Decision Forum end with a decision not to introduce the drug on the grounds that "the documentation for the effect is too uncertain in relation to the price".
This often means that Norwegian patients have to wait for new data to be generated in countries capable of adopting new treatments and are 'penalized' for reasons beyond their control.
The Priority Announcement, New Methods and HTA have had a particularly negative effect on the uptake of new, innovative medicines for the treatment of rare diseases. Three main challenges with the rare disease system are:
- It uses a quality of life measurement tool that is not suitable for rare diseases. For example, it does not measure that you only need half as much help breathing as you did before you took the drug.
- Willingness to pay is increasing until patients have "gained" 20 good years of life, but patients who "gain" 60 or 70 good years of life instead should not count more positively than more common diseases.
- Finally, the effect is reduced because the Parliament has agreed that a good year you get 40 years in the future is worth less than the year you get today.
These are examples of the system gearing up for the major but "less" serious diseases, which unfortunately means that people with rare diseases often end up with a "double penalty".
The evaluation of New Methods carried out by Proba Analyse points out, like the experiences of patients, doctors and industry, that New Methods are not adapted to treat methods for rare diseases. The coalition believes that it is unlikely that vague management signals will succeed and that the Storting must therefore give clear management signals in order to have an impact.
There are several serious rare diseases where the course of the disease can progress rapidly to irreversible conditions that can require lifelong treatment and care, and ultimately death. These people are currently being harmed because the system is not adapted to minor diseases. Many people with rare diseases are currently waiting for treatments that are in development or have marketing authorization but are not funded.
I would also like to highlight the importance of the pharmaceutical industry's need for predictable framework conditions if the Norwegian Parliament wants Norwegian patients to have access to innovative medicines.
The Coalition's primary position is that the system must be changed and we believe that the measures implemented in the field of rare diseases have hit people with rare diseases undesirably hard. The financing system should therefore be changed, either through individual reimbursement as before, through a rare disease prescription or a rare disease fund. The Coalition believes that the representative's proposal is good and can help to correct unintended consequences for a vulnerable group.