The Coalition for Rare Diseases has gained access to an important paper prepared in collaboration with Sanofi on prioritization of rare diagnoses and socio-economic assessments. This paper sheds light on a crucial issue related to prioritization and access to treatment for patients with rare diagnoses in Norway.
What is the challenge? Rare diagnoses represent a significant burden, not only for the patients themselves, but also for their relatives and society as a whole. Menon has estimated the societal costs associated with rare diagnoses in 2022 to be as much as NOK 167 billion. This cost includes loss of quality of life, early death, limited participation in working life and increased burdens for relatives. At the same time, the lack of effective interventions limits patients' ability to receive necessary treatment and care in the specialist health service.
Medical advances and time constraints. With new medical advances, including gene therapy, it has become possible to offer life-saving treatments for many rare diagnoses. Unfortunately, it often takes a long time for these treatments to become available to patients. This leads to patients missing out on vital treatment, which is unacceptable. Decision-making systems are not sufficiently adapted to the rapid medical development associated with advanced therapies.
Prioritization criteria that do not reflect severity. This paper reveals a problematic reality: current prioritization criteria are not designed to prioritize rare diagnoses according to the severity they represent, neither for patients nor for society. This can lead to treatment for rare diagnoses often being given lower priority than should be the case. We also see that the cost-effectiveness of treating rare diagnoses is often underestimated, which further affects accessibility.
Ability to pay for rare event insurance. So what is the solution? The paper points to the need for public health services to reflect citizens' willingness to pay to reduce the risk of negative health outcomes, especially related to rare diagnoses. This means that the prioritization criteria in the health sector must take into account the actual willingness to pay to be insured against rare diagnoses. Socio-economic profitability must not overshadow fair distribution and equal access to health services for all citizens.
Lessons from other countries; Norway can look to other countries such as Sweden, the UK, Canada and the US for possible solutions to these challenges. These countries have developed systems that better ensure access and effective treatment for rare diagnoses, including increased willingness to pay and acceptance of risk associated with such diagnoses.
We are pleased to announce that Sanofi has commissioned this paper on the prioritization of rare diagnoses and the socio-economic assessments, and the Coalition has been granted permission to publish this paper. Together, we are working to raise awareness of the importance of equitable access to treatment for rare diagnoses and to promote a fairer and more efficient prioritization process.