At a time when health policy and medical care are constantly evolving, the issue of prioritizing treatments for rare diagnoses has become an important area of focus. Recently, an important discussion was held that brought together representatives from patient organizations, industry and other stakeholders involved in health policy. The aim of this discussion was to highlight the challenges and opportunities inherent in designing a holistic approach to prioritizing treatments for rare diagnoses. Here we share some of the most salient perspectives that were shared during this discussion.
Unique challenges require specific solutions
A crucial insight highlighted is that rare diagnoses cannot be treated with a one-size-fits-all approach. The varied and complex nature of such diagnoses requires tailored assessment criteria. This perspective emphasizes the importance of avoiding a "one-size-fits-all" approach to prioritization, and instead focusing on adapting guidelines to each individual situation.
Stronger together: collaboration and unification
The discussion highlighted the need for increased collaboration between patient organizations as a way to gain greater attention and resources. Identifying common goals and causes was seen as a key to increasing impact. This perspective recognizes that when patient organizations work together and coordinate their efforts, they can achieve more significant change.
Those of us who fight on behalf of rare diseases need to organize ourselves smarter. I have asked relatives what their plan is to win in the prioritization debate. The feedback is that they don't have the time, finances and energy required. They have more than enough to deal with their own challenges.
Fredrik Arneberg, Coalition for Rare Diseases (UCB)
Increased emphasis on rare diagnoses in the prioritization report
It was clearly expressed that rare diagnoses should receive special attention in the upcoming prioritization announcement. This includes lowering the documentation requirements for treatments for rare diagnoses and considering higher willingness to pay to address the unique challenges these diagnoses present. This perspective emphasizes the importance of developing a tailored approach that addresses the specific needs of rare diagnoses.
Strengthening user representation
A significant observation was the lack of strong user representatives in the expert groups. Participants highlighted the importance of giving people suffering from rare diagnoses a powerful voice in decision-making processes on treatment priorities. This perspective emphasizes that those most affected by these decisions must be actively included to ensure a more fair and inclusive prioritization process.
Learning from Nordic cooperation and international best practices
A discussion around Nordic collaboration in the field of rare diagnoses revealed both benefits and challenges. Sharing best practices from other countries and regions was seen as a valuable resource to improve access and prioritization of treatments. This perspective emphasizes the importance of looking beyond national borders to find solutions that work best for rare diagnoses.
Holistic assessment: long-term perspective and quality of life
The discussion also addressed the need for a holistic assessment of treatments for rare diagnoses. This includes considering the long-term consequences of treatments on patients' quality of life, as well as societal and economic implications. This perspective recognizes that successful treatment prioritization must take into account more than just medical factors.
Patients with rare diseases face unique challenges that require specialized care and access to innovative treatments. We need to work together to ensure that these patients are not overlooked and that they receive the support and treatment they deserve.
Bjørn-Kristian Svendsrud, Coalition for Rare Diseases (Sanofi)
The role of communication in raising awareness
Despite increased knowledge about rare diagnoses, the task of raising awareness among decision-makers remains. Participants emphasized the importance of education and communication to ensure that the priority announcement reflects the needs of rare diagnoses and their affected communities.
Patient rights and equal treatment
The discussion emphasized the importance of safeguarding patient rights and ensuring equal access to treatment. Every patient, regardless of the rarity of the diagnosis, has the right to good health care. This perspective highlights the importance of including a rights perspective in the prioritization report.
This discussion shed light on the complex landscape of prioritizing treatments for rare diagnoses. It reveals the need for a tailored approach, increased user representation, collaboration across organizations and borders, as well as a deeper understanding of both medical and non-medical factors. As we work towards the development of the upcoming prioritization announcement, it is crucial to include these perspectives to ensure that rare diagnoses are treated with the fairness and care they deserve.