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Becoming a Champion for Rare Disease Patients

September 18, 2017
At Shire, our mission is to be a champion for the 350 million patients worldwide living with a rare disease. About half of all rare diseases begin in childhood, and many patients endure lifelong suffering. Sadly, rare diseases often result in a shorter lifespan, and around one in three children with a rare disease will not live beyond their fifth birthday. What these figures do not reflect, however, are the untold number of mothers, fathers, friends and family who painstakingly support a loved one living with a rare disease.

We believe that people with rare diseases, along with their families, need a champion. As the global leader in rare diseases, Shire has long believed that we have an opportunity to transform rare disease treatment. It is a responsibility we take seriously. And so we work tirelessly to seek innovative therapies, explore new approaches, and support patients with rare diseases every step of the way.

Our engagement with patient organizations is a key component of our continued efforts to support patients. Patients, caregivers and patient advocacy groups play a crucial role in our efforts to tackle the many challenges these communities face every day.

Every rare disease community is different and each faces its own challenges, that’s why our patient advocacy group has partnered with over 100 advocacy organizations around the world to create disease awareness and education programs, raise awareness of clinical trials and support patient conferences and engagements, such as sponsoring the 2017 BIO Patient and Health Advocacy Summit. These partnerships have been designed to progress opportunities for better care and to make a difference in the lives of patients and caregivers. As Greg Robertson of Shire’s UK patient advocacy team, said, “Whether working to shorten the time to diagnosis for patients or supporting education efforts to empower patients in managing their disease, our work with advocacy organizations helps address the needs of people living with rare diseases. Together, we make sure the patient voice is at the heart of everything Shire does.”

To maintain our patient-centric focus, Shire has developed an internal initiative which allows patients and caregivers to share their experience of living with a rare disease at the beginning of department meetings or employee town halls. By helping colleagues reach an understanding of disease burdens on a personal level, we feel it enables them to stay connected to the patients we serve and maintain motivation to deliver on our mission. For instance, Jeff Hackman, head of US oncology franchise at Shire said, “My impact on rare diseases is my inspiration to make a difference in patients’ lives because the most important patient that we treat is the next one.”

Further, as part of Shire’s dedication to bettering the lives of rare disease patients, we are committed to increasing worldwide access to healthcare. We support numerous patient programs designed to help those in need receive free or discounted products or services such as home healthcare and nursing.

Given the healthcare landscape is rapidly changing, our partnerships with patient advocacy organizations have proven to be vital to ensure our programs and efforts are addressing the evolving needs of our patients, as best they can. These connections continue to impact our development of new ideas, new innovations and new ways of thinking to enhance our capabilities.  As Kaaren Ruth, US patient advocacy lead, stated, “Patient advocacy organizations are change agents. They empower a connected community voice to raise awareness, educate, forge new policies, raise funds for research and provide hope for a better life living with a rare disease. They change lives.”
Next month, BIO will host the Patient and Health Advocacy Summit, an annual event that brings together stakeholders for timely policy discussions and best practice seminars. This is the third in a series of guest blogs highlighting efforts by BIO members to bring the patient voice into the discovery,  development and delivery of life-saving medicines

[caption id="attachment_26503" align="alignleft" width="120"] Marjorie Stewart-Hart[/caption]


About the author:
Marjorie Stewart-Hart is the Head of U.S. and R&D Patient Advocacy at Shire. She has worked on the Shire patient advocacy team since July 2016.