- Global Pharma News & Resources

Alexion Launches Video for Rare Disease Day 2022 Highlighting Disparity in Rare Disease Journey and Care

Alexion Launches Video for Rare Disease Day 2022 Highlighting Disparity in Rare Disease Journey and Care

Based on a survey of 1,020 people affected by or caring for someone with a rare disease in the UK, the video reiterates the urgent need to reduce the element of chance around rare disease diagnosis and care

Uxbridge, UK, February 16, 2022 – Alexion Pharma UK today launches a new video, “Reforming Rare Diseases: Making the Rare Path the Right One”, for Rare Disease Day 2022. The video brings to life the theme of this year’s Rare Disease Day focused on championing equity for people living with a rare disease by highlighting the urgent reforms needed to improve patient care for the rare disease community. While 3.5 million people in the UK are affected by a rare condition,1 there are still major unmet needs in the standard of care for these patients. The video shows the long and frustrating journey that so many people with a rare disease experience to reach a diagnosis and receive the care they need. Created in consultation with Genetic Alliance UK, Beacon, Medics 4 Rare Diseases and Rare Revolution, the video is based on the findings of a recent patient/caregiver survey on the standard of care for people with a rare disease in the UK, led by Genetic Alliance UK and supported by Alexion.

“Rare conditions are often severe, chronic and progressive and can have a devastating impact on both individuals and their families,” said Lauren Roberts, Joint Interim Chief Executive and Director of Engagement and Support, Genetic Alliance UK. “We urgently need to address the unmet needs of this population and resolve the issues that those living with a rare condition experience across the entire patient pathway. By raising awareness of these challenges, we hope to continue to find ways to improve the standard of care for this community.”

The video focuses on the current realities of care outlined in the findings of the Rare Experience 2020 survey, whereby over one third (37%) of people living with a rare condition rated their overall experience of care as poor or very poor, 2 41% waited more than five years for an accurate diagnosis,2 and 71% manage their own care plans.3 Stark contrasts in the patient journey are presented throughout the video, which follows one individual on two very different pathways. This includes the all-too-common journey of chance which plays out in parallel to the ideal journey that all those living with a rare condition deserve.

Rare Disease Day is a global awareness initiative that takes place on the last day of February each year. The day was launched by EURORDIS-Rare Disease Europe and its Council of National Alliances in 2008 to raise awareness amongst policymakers and the public about the impact of rare diseases on patients with the ultimate goal of improving lives.

“We need to urgently address the current shortcomings in care for the rare disease community, reducing the element of chance in their patient journey, so the right path is no longer the rare path,” said Sean Richardson, General Manager, UK and Ireland, Alexion, AstraZeneca Rare Disease. “We hope this video is another reminder of the importance of implementing the new Rare Disease Framework so that faster diagnosis and better coordination of treatment and care are available to all those affected by rare diseases.”

The “Reforming Rare Diseases: Making the Rare Path the Right One” video is accessible here: For more information on the Rare Experience 2020 Survey, please visit:

About Alexion

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology, and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. Please visit

About AstraZeneca

AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide.

AstraZeneca is based in five different locations across the UK, with its global headquarters in Cambridge. In the UK, around 8,300 employees work in research and development, manufacturing, supply, sales and marketing. We supply 40 different medicines to the NHS.

For more information, please visit and follow us on Twitter @AstraZenecaUK.

Media Inquiries

Lilianna Husseini

+33 (0) 647641720


1 UK Department of Health and Social Care. Policy Paper: The UK Rare Diseases Framework. 9th January 2021. Available at:

2 Alexion, Reforming Rare Diseases Report, December 2020. Available at: Accessed December 2021

3 Genetic Alliance, Rare Experience 2020 Report. Available at: https://rareexperience2020.geneticalliance.

Editor Details

Last Updated: 22-Feb-2022