This article has been developed and funded by Chiesi Group Rare Diseases.
Enrico Piccinini
Head of Europe and International Rare Diseases, Chiesi Global Rare Diseases
Inflation and rising public debt are creating barriers for the rare disease community to access the latest therapies.
Public healthcare budgets are being reduced, making access to new treatments difficult while stricter evidence requirements further limit availability across Europe. The lack of treatment availability, combined with the difficulties and delays in diagnosis of rare diseases, poses a societal challenge.1
Burden of rare diseases is unacceptably high
Chiesi Global Rare Diseases (GRD) is a business unit of the Chiesi Group established to deliver solutions for people living with rare diseases.
Last year, Chiesi GRD published the report ‘Rare disease burden of care and the economic impact on citizens in Germany, France and Italy’ as part of the PENDULUM EU project, aimed to trigger action in Europe by encouraging a more refined approach to understanding rare diseases.
The report found that the cost burden for rare diseases is significantly higher than for high-prevalence diseases, with rare diseases averaging €107,000 per patient per year compared to €7,000 for high-prevalence diseases. Indirect costs make up a substantial portion of this burden, averaging 29% when treatment is available, which rises to 45% when no treatment is available. These indirect costs are primarily shouldered by families and caregivers, creating broader economic impact.1
Living with a rare disease comes with challenges for families and caregivers, including delays to diagnosis, managing the physical and emotional burden, finding adequate support and loss of income and expenses associated with the condition.1.2
Complementing the economic focus of the PENDULUM EU project, these challenges are highlighted through Chiesi GRD’s It’s Rare for Me special edition, launched in the UK, which offers a platform for people with rare diseases to share their stories.
Accelerating treatment availability to alleviate burdens
To positively impact the future of the rare disease community, long-term changes need to be implemented at both the national and European levels. Technological advancements are enhancing the understanding and diagnosis of rare diseases as well as generating an increase in research funding.
To support this, European pharmaceutical legislation should be revised to boost competitiveness and innovation in Europe. On a national level, payers need to adopt flexible payment models to ensure timely access to therapies.1
“We urge all stakeholders to collaborate in finding solutions that enhance patient quality of life and outcomes. By improving funding, access and innovation for rare diseases, we can create positive value and reduce the significant economic burden.”
This article has been written and funded by Chiesi Global Rare Diseases.
UK-CHI-2400700 | September 2024
References
[1] Chiesi. Global rare diseases report: Rare disease burden of care and the economic impact on citizens in Germany, France and Italy. https://chiesirarediseases.com/assets/pdf/rare-disease-burden-of-care-and-the-economic-impact-on-citizens.pdf [Accessed September 2024]
[2] Mcmullan J, Lohfeld L, McKnight AJ. Needs of informal caregivers of people with a rare disease: a rapid review of the literature. BMJ Open. 2022;12(12):e063263
