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Despite the clear order from the Parliament, the evaluation of New Approaches does not address the consequences for patients with rare diseases. It is now urgent to put in place a national funding model that ensures that these patients receive quality care regardless of their place of residence.

The article was originally published in Dagens Medisin:

Article: The Coalition for Rare Diseases;
Kristin Mesteig, Director of Government Affairs, Sanofi
act at Sanofi
Mattias Bankel, Nordic Director at Amicus Therapeuics
Mattias Janzen, Nordic Director at BioMarin Pharmaceutical
Patrick Svarvar, Nordic Director for Rare Diseases at Chiesi Group
Lidziya Ulvenes, Head of Market Access for Rare Diagnoses at Takeda

WHEN HOSPITALS decide on the treatment of a patient, they must comply with two overarching guidelines - the principles of prioritisation and the right to appropriate health care. The Norwegian Parliament has determined some national prioritisation principles that are operationalised through the New Methods system. At the same time, each patient has a statutory right to responsible health care.

Recently, there have been several cases in the media that show the problems that arise when these guidelines collide: Patients are not offered appropriate health care because of the priorities set by the New Approaches system. This is particularly true for patients with rare diseases.

GUIDELINES. The issue is not new. As early as two years ago, when the Storting enshrined the system in law, it was decided that the system should be evaluated in the light of the criticism it had received. The majority of the Storting, including the then opposition politician Ingvild Kjerkhol (Ap), requested that the evaluation should emphasise the input from the consultation round concerning patients with rare diseases.

Kjerkol, now Minister of Health and Care Services, received the evaluation of New Methods on 18 November. Despite clear guidance from the Norwegian Parliament on the importance of evaluating the framework for patients with rare diseases, this is almost absent from the report.

A national funding model for orphan drugs will lift the financial burden from individual hospitals - and ensure quality care for patients with rare diseases

OMITTED. There are critical challenges to the current system that have not been evaluated. The first challenge is that the prioritisation system is based on group assessments, while patient rights are individual. This was also an issue in the Storting's consideration of the legislation.

The chair of the committee, Geir Jørgen Bekkevold (KrF), pointed out at the time that individual assessments should be based on the individual's needs and actual ability to treat illness. If this is required to provide justifiable health care, the patient must be offered something that the system for new methods may have refused.

'INCORRECT UNDERSTANDING'. Since 2019, the State Administrator has, on several occasions, pointed out that patients' right to proper health care has not been met, and has concluded that the health authorities' assessment "... is based on an incorrect understanding of the Patients' and Users' Rights Act".

For Helse Førde, it is not possible to comply with the State Administrator's decision on the basis of the budgetary consequences of granting the patient's rights: "The amount is far above the cost that Helse Førde is able to approve for individual patients (...) In the overall assessment, both the severity criterion and the benefit criterion weigh in the patient's favour.It is only the resource criterion that weighs against".

WARNINGS. The second challenge is the introduction of a new model for financing medicines: The prescriber is now responsible for funding. This means that the hospital that determines that a patient needs treatment must cover the treatment through its own operating budget.

In 2019, Norwegian hospitals became responsible for funding a number of drugs for rare diagnoses that were previously covered by the National Insurance Scheme. In practice, the change meant that the clinic at the patient's local hospital has to pay for the treatment, and the treatment costs for individual patients increased significantly.

The consequences of this were already warned against in 2018.

RIGHTS VIOLATIONS. Rare diseases are often congenital, they are very serious, and often require close follow-up from both specialist and primary care services. For individual hospitals and municipalities that have a patient with a rare diagnosis, it is very costly to provide good treatment and follow-up. In its consideration of the current national budget, the majority of the Storting was clear that "... no patients with rare diseases should have their patient rights impaired as a result of the transfer of funding responsibility for pharmaceutical treatment to the regional health authorities".

Since 2019, these patient rights for patients with a rare disease have been violated on several occasions.

THE SOLUTION. The government is signalling that the further development of New Approaches needs to proceed on a number of levels. While it wants to implement some measures immediately, it wants to discuss other more fundamental issues more broadly before proposing changes.

One of the measures urgently needed is a national funding model for orphan drugs that lifts the financial burden from individual hospitals. This will ensure quality care for patients with rare diseases. Equal access to healthcare regardless of place of residence and the right to individual assessment must also apply to patients with a rare diagnosis.

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