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The term “rare disease” can be misleading. Despite the individual rarity of each condition, there are over 7,000 rare diseases collectively affecting 4% of the global population. This underscores the critical need for increased research into rare diseases and their symptoms, and improved access to quality treatment for people living with these conditions.

February 28 marks Rare Disease Day, a day dedicated to raising awareness of rare diseases and the unique challenges faced by people living with them. The “More than You Can Imagine” 2025 campaign emphasises the resilience and power of this community as they navigate life with rare diseases. CSL Behring is honoured to join the rare disease community in advocating for better diagnosis and treatment, and recognition for the 300 million people living with a rare disease worldwide

Written by CSL Behring

It is important to recognise that rare disease patients continue to encounter challenges, and global commitments to raising awareness must remain steadfast throughout the year to drive meaningful actions in policy and practice.

A key area for improvement is the pathway to optimal diagnosis and treatment.

For people living with rare diseases, the path to optimal diagnosis and treatment is fraught with obstacles, taking a considerable emotional, social, and financial toll. An accurate diagnosis often opens the door to targeted therapy, yet obtaining this diagnosis can often take about five years and varies significantly by country, disease, and the level of medical awareness. Access to optimal care and innovative treatments remains fragmented, with barriers such as limited funding and awareness among healthcare providers.

One rare disease that exemplifies these challenges is hereditary angioedema (HAE), a debilitating and potentially life-threatening genetic disorder. HAE affects approximately 1 in 50,000 individuals and is characterised by episodic and unpredictable swelling (HAE attacks) in various parts of the body, including the hands, feet, face, gastrointestinal tract, and airway.

Diagnosing HAE, like many rare diseases, remains a significant challenge due to a lack of awareness, leading to late or incorrect diagnoses and taking a heavy psychological toll. While an accurate diagnosis and access to effective preventative treatments that significantly reduce the frequency of HAE attacks can bring relief, the burden remains high. HAE attacks are highly debilitating, disrupting both professional and personal life for those affected and their caregivers. The unpredictability of these attacks places a significant psychological strain on everyone involved, serving as a painful reminder of past struggles or family history. As we continue to celebrate groundbreaking scientific strides and the resilience of those living with HAE, we call on governments to acknowledge the immense physical, emotional, financial and social toll of living with a rare disease like HAE. Urgent action is needed to fast-track diagnosis and ensure access to personalised treatments that match individuals’ lifestyles and needs. Through greater education and multi-stakeholder collaboration, we can turn awareness into action and ensure no one living with a rare disease is left behind.

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CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients’ needs by using the latest technologies, we discover, develop and deliver innovative therapies for people living with conditions in the immunology, hematology, cardiovascular and metabolic, respiratory, and transplant therapeutic areas.

We use three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full lives.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. Our parent company, CSL, headquartered in Melbourne, Australia, employs 32,000 people, and delivers its lifesaving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit CSL.com/Vita and follow us on Twitter.com/CSLBehring

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