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Input to expert groups - the prioritization report

    The Coalition for Rare Diseases consists of five biotech companies working to improve the quality of life and health of patients with rare diseases. We work for good and predictable framework conditions for medical treatment, when such treatment is available.

    With the current prioritization criteria, people with rare diseases experience major challenges in gaining access to effective treatments for their disease. The prioritization criteria and the system for assessing and funding new therapies are not suitable for small patient groups with serious diseases.

    According to the latest W.A.I.T report from IQVIA (1), only 34% of the 61 drugs for rare diseases that received European marketing authorization in the period 2018-2021 are available to Norwegian patients. The report also states that for the few medicines that make it through the eye of the needle in Norway, it takes an average of 627 days from marketing authorization in Europe to availability for Norwegian patients.

    The Coalition for Rare Diseases has noted that the Minister of Health and Care Services, in her answer to a question from representative Tone W. Trøen (Doc. no. 15:1190), rejects the idea that a public committee will be established in connection with the Government's announced white paper on prioritization in the specialist health service. In her answer, the Minister of Health and Care Services emphasizes that expert groups will instead be set up in selected areas.

    The coalition believes that this will mean that the announced prioritization report will have a weaker foundation than desired. We are also concerned that it will not have the necessary legitimacy in the health service and in society at large. This is further exacerbated by the speed of medical developments that have taken place since the Norheim Committee delivered its report in 2014. We assume that this development will continue in the years to come, which in our opinion indicates a need for a full review of the prioritization area.

    This will particularly apply to people with rare diseases, who feel that the overall structural changes in the field have made everyday life much more difficult for these people.

    A dedicated expert group for rare diseases

    There are up to 300,000 patients living with a rare disease in Norway today who need to be taken seriously. Treatment of rare diseases is at the forefront of precision medicine, and has already helped to solve several medical puzzles, as well as developing new technology that is transferable to other areas.
    The Coalition for Rare Diseases therefore asks the Minister of Health and Care Services to consider the need to appoint a separate expert group to investigate the conditions for prioritization for people with rare diseases. This could provide important proposals for necessary changes for a large and marginalized patient group who feel that the current system does not work.
    The experience of patients, relatives, doctors and the pharmaceutical industry is that it has become more difficult for an already vulnerable group to gain access to new effective treatment that comes on the market. The prioritization announcement and the associated framework are therefore best suited to prioritizing major diseases such as diabetes, cardiovascular disease, etc. but have an unfortunate impact on small patient groups such as rare diseases.

    Below are some specific suggestions for the areas that the various expert groups should look at more closely in order to address the specific challenges faced in the field of rare diseases.

    Personalized medicine and efficacy documentation (overall input)

    Today, the effect of good years of life is discounted for future benefits. Several treatments are given as a one-off treatment, such as cell and gene therapies where the effect is expected to last a lifetime. For younger patients, this will be unfortunate as the effect can last up to 70 years (and often longer) but the cost is immediate. When the health benefits are reduced due to discounting, the cost-effectiveness is reduced and the treatment becomes less attractive to finance.
    Health service perspective versus a societal perspective (group 1)

    A report from Menon (2) finds that the annual societal costs of rare diseases are approximately NOK 167 billion (36-350 billion*), the majority of which are related to disease burden. The total health service costs, including hospitalizations, consultations, pharmaceuticals, assistance and informal care, vary between 12 and 90 billion in 2022, with an expected value of about 46 billion annually. Hospitalizations, consultations and the need for medical treatment account for 55 percent of total healthcare costs, while costs related to municipal healthcare and informal care account for 25 and 20 percent of total healthcare costs, respectively. The social costs of Duchenne muscular atrophy alone are estimated at NOK 700 million (3).

    It is a paradox that drugs are considered safe and effective enough to be used by humans, but are repeatedly judged too unsafe to fund. Like the Health Personnel Commission, the Coalition is of the opinion that humans are one of our most limited resources (4). This should logically lead to the introduction of measures to improve the quality of life of people with illnesses in order to increase societal efficiency by reducing the burden of illness for patients and their families or correcting the illness and allowing people to contribute to working life.

    Replacing the current health service perspective with a societal perspective will help create a more sustainable health service and societal model.

    Group level and access for individual patients (group 2)

    The coalition's view of the current system is that it is too rigid and makes too many decisions at group level, depriving doctors of the necessary autonomy to provide patients with the optimal treatment. The National Insurance scheme was originally designed with an understanding of the differences between patients and that some diseases were less prevalent than others, with the result that there was little value in requiring full documentation. This was particularly true for rare diseases that are more costly to treat, which led the Norwegian Parliament to introduce a safety valve with individual reimbursement.

    The current system has removed this safety valve for patients with rare diseases and patients who deviate from the normal patient population. Even people with rare diseases can be characterized by heterogeneity within a condition. The Coalition therefore believes that it should be investigated whether a system such as individual reimbursement should be introduced in the specialist health service to ensure that patients who deviate from the normal patient population or who are more severely affected than the normal patient population receive treatment.

    Transparency and verifiability (group 3)

    For the Coalition, it is somewhat unclear why transparency and verifiability should be addressed in a prioritization report rather than a legislative proposal.

    It is a recurring challenge that the processes in New Methods appear as a black box where parts of the input values are known and results are known through the method report and the decision in New Methods. It is unclear how input to the HTA and disagreements are handled, and what input is received.

    The coalition believes that the Norwegian authorities should take inspiration from NICE in England to ensure more transparency and broader involvement in the case processing5. A protocol should be drawn up that includes discussions with questions, any disagreements and how these have been handled, which questions have been asked and how they have been answered. Furthermore, input, especially from clinicians, should be included in the methodology report as a standard.

    Denmark's approach through the Medicines Council is another model that can be considered, where the pharmaceutical industry and others are also represented (6).

    The coalition believes that this can contribute to greater transparency and involvement of the affected parties in a way that the current system is unable to provide.

    General input for assessment across the expert groups

    The way in which study data are assessed in the HTAs has an unfortunate impact on people with rare diseases. The prioritization report assumes that uncertainty about the effect, interpreted through study data, should count negatively in the decision-making process. Because there are few people in the world with a specific rare disease and it would often be unethical to put people in a control group without treatment, there is natural uncertainty in the data. This often results in Norwegian patients being "punished" due to circumstances beyond their control.

    The Storting's safety mechanism for small patient groups, operationalized through the Norwegian Medicines Agency's memorandum "Scheme for rapid health technology assessment of medicines for particularly small patient groups with very serious conditions" is perceived to be weakly founded and sets too high thresholds to benefit most rare diagnoses. This is despite the fact that most diagnostic groups within rare diseases are generally not large enough to conduct large studies that provide sufficient documentation for New Methods. It is also questioned whether uncertainty is weighted twice by counting it negatively both in the HTA and in the Decision Forum (7).

    Furthermore, measuring the treatment's effect on quality of life is a tool that is poorly suited for some diseases, yet better for "traditional and common" diseases, but is limited in its ability to measure changes in quality of life in people with rare diseases. This means that the effect of the drug is often underestimated in relation to the actual perceived benefit for patients and may present the cost-effectiveness less accurately than if several dimensions of quality of life were mapped.

    The current prioritization report is based on the Magnussen Group's payment ladder, which operationalizes the severity criterion and standardizes how much the Norwegian healthcare system is willing to pay for a good year of life after the loss of prognosis (how many good years of life the patient loses due to the disease).

    This payment ladder stops after 20 lost years of life and thus neglects the serious rare diseases where patients can lose up to 70 good years of life. There is therefore a need for further development to reflect a life course with associated willingness to pay for the most serious diseases.

    If the Ministry of Health and Care Services would like to meet with us for further clarification, we would be happy to accommodate this.

    Kind regards
    Bjørn-Kristian Svendsrud
    Sofia Nordin
    Patrick Svarvar
    Mattias Jánzen
    Fredrik Arneberg
    Amicus Therapeutics