The Coalition for Rare Diseases (the Coalition) refers to the Storting's website and the invitation to participate in the consultation on Prop 1S (2022-2023) for the budget year 2023 under the Ministry of Health and Care Services. In this consultation response, the Coalition requests that the Health and Care Committee:
- Ensuring new national funding models that address known challenges for people living with a rare diagnosis
- Ensures that rare diagnoses are taken into account in the further development of New Approaches and in the work on a new Prioritisation Communication
- Ensure good infrastructure to enable further participation in European Reference Networks
- Changes in funding schemes for specialist care must result in better funding models for rare diagnoses
From 2023 onwards, the basic allocations will account for 60% of the allocations to the somatic specialist health services. The change is intended to give the regional health authorities greater strategic room for manoeuvre. It is important that this room for manoeuvre is used to meet the need for new funding models in the rare disease area.
One reason why rare diagnoses are not approved in the New Approaches system is that there are few patients and great variation between patients. This makes it particularly challenging to obtain reliable knowledge about the effect of these treatments. Rather than pointing out that the knowledge is uncertain, it would be appropriate to put in place temporary financing arrangements, preferably in the form of a national fund for financing orphan drugs. This would allow treatment to be funded at the same time as new evidence is being gathered.
The coalition therefore wants the Health and Care Committee to ask the Government to clarify how to develop new funding models for financing innovative treatments in the upcoming National Health and Care Coordination Plan. Central to this should be a national fund for temporary approval/financing of treatment for rare diagnoses.
Further development of New Approaches must take into account rare diagnoses
In 2019, the majority of the Storting called for the system for new methods to be evaluated on the basis of "the challenge seen in connection with the introduction of new methods for rare diseases and other small patient groups, and believes that the decision-making processes must take these into account to a greater extent".
The final evaluation was submitted to the government in 2021, pointing out that "challenges related to personalised medicine, advanced therapies and treatments for rare conditions will be the main ones" in the future.
It is gratifying to see that fresh funds have been allocated to New Methods in this year's state budget and that the regional health authorities are working to develop New Methods within the framework of the current prioritisation report. The Government has also announced a new priorities report.
At the same time, few of the Storting's repeated instructions to take care of rare diseases are reflected in the ongoing work in New Methods or in the signals given for what to investigate in a new prioritisation report. This is made concrete by the fact that rare diseases are mentioned in the RHAs' update to the Ministry on the further development of New Methods, or in the Ministry's update to the Storting in this proposition (Chapter 11, Further development of New Methods). The Coalition believes that the current challenges with the introduction of treatments for rare diseases are a consequence of the guidelines provided by the Storting in the previous prioritisation report (Meld. St. 34 (2015-2016) - Values in the patient's health service)
These challenges must therefore be addressed in a new Priorities Communication. The Coalition therefore calls on the Health and Care Committee to ensure that rarity is adequately discussed in the upcoming Priorities Communication.
International cooperation is essential for early diagnosis and good treatment of rare diagnoses
The National Strategy for Rare Diagnoses highlights the importance of participation in European Reference Networks (ERNs). The aim of the networks is to deal with rare diseases and conditions that require highly specialised treatment and a concentration of knowledge and resources. The strategy specifies that the regional health authorities should facilitate increased Norwegian participation in all the European reference networks. This work has been given high priority, and Norway is currently a member of 17 out of 24 networks. This gives Norwegian patients with rare diagnoses the opportunity for faster diagnostics and better treatment.
A requirement for participation in an ERN is the ability to communicate through the Clinical Patient Management System (CPMS). This is a digital platform for communication that safeguards patient privacy. Norwegian clinicians do not currently have access to CPMS, and clear signals have been given by the EU that Norway risks being excluded from the ERN unless access to CPMS is put in place.
The coalition therefore wants the Health and Care Committee to ask the government to ensure that the measures in the national strategy for rare diagnoses are followed up and that access to critical infrastructure for further participation in the ERN is arranged.
The Coalition thanks you for the opportunity to provide input to this year's state budget and wishes the committee well in its further work. The Coalition is available to elaborate on our points and please do not hesitate to contact us if more information is required.