Rare diseases are highly complex and over 50% of rare diseases begin in childhood. 30% of children born with a rare disease are unlikely to see their 5th birthday [1,2]. Some examples of rare diseases are hemophilia, Gaucher disease, Hunter syndrome, Pompe disease, and so forth. Today, the majority of rare diseases are still unknown and yet to be fully understood.
Through its sharp focus on rare diseases and highly specialized conditions, Shire is delivering high impact for patient communities with rare and life-threatening diseases. Combined a year ago with Baxalta, Shire focuses on precision innovation and personalized treatment to make a huge impact on patients’ lives.
Shire has 17 manufacturing sites across seven different countries, and Singapore is part of this world-class network. In Asia Pacific, Shire is headquartered in Singapore, with a state-of-the-art manufacturing site for its hematological pipelines in Woodlands.
Mr. Peter Fang, Head of Asia Pacific, Shire, shared at a media roundtable that there are many good reasons for Shire to look at growing its global presence in the Asia Pacific region.
Shire aims to be more than a biotechnology company
Peter Fang, Head of Asia Pacific, Shire tells us what differentiates Shire’s vision from others in its class.
“We have a few differentiating factors when it comes to rare diseases. At present, we are the market leader for a rare bleeding disorder called hemophilia, and have over 60 years’ experience in treating it, longer than many more recent hemophilia entries. Hemophilia is a lifelong disease, and therapy lasts the entire lifetime of a patient. Therefore, it is vital we deliver innovation and go “beyond the vial” to find ways to improve the entire experience for the patient. This includes improving time to diagnosis, enabling broader access and reimbursement, and ensuring consistent supply.
When it comes to innovation, Shire has a broad portfolio and a strong pipeline of about 40 programs in clinical development, largely focused on rare diseases. Our goal is to accelerate the launches of such innovative therapies across APAC to address areas of high unmet medical need and improve treatment options for people with rare diseases.
Furthermore, Shire supports campaigns to improve diagnosis on rare diseases. For example, a lot of patients with rare diseases don’t know what is wrong with them, and even some doctors don’t know how to diagnose the disease. We develop disease awareness and diagnosis programmes with patient organizations, and offer new and easy diagnostic tools to increase diagnosis for people with rare diseases.
Shire also serves as a community partner with patient organizations to help advocate for improved standards of care and treatment guidelines. Most recently, we worked with Rainbow Across Borders and the Rare Disease Society (Singapore) to form the ASEAN+ Rare Disease Network, which will help the organization to mobilize support for rare diseases across the region.
Finally, Shire has a dedicated manufacturing platform and network strategy. Many companies do not have in-house manufacturing or only rely on a single site for manufacturing. For our hematology pipeline, we have 13 high tech manufacturing sites across six countries to ensure the adequacy of supply of our medicine to people living with hemophilia around the world. This is key to avoid supply shortages and to have better control of the quality of our product for the lifetime of the patients.”
Shire’s Asia Pacific focus
As a patient-centric company, Shire plans to have to have more than 10 new product launches this year, primarily in rare diseases across the Asia Pacific region, providing more access to new therapies. With global research expertise, Shire has a strong base in hematology. Hemophilia is a rare bleeding disorder for people who are missing a protein called clotting factor VIII, causing them spontaneous bleeding, such as bleeding in the joints. Genetic diseases such as Hereditary Angioedema (HAE) – spontaneous swelling of hands and body – is another core therapeutic area. HAE sometimes causes high mortality because of breathing difficulties due to the swelling of the throat. Hunter Syndrome and Gaucher Disease are rare genetic diseases that if left untreated, may likely cause death before the age of five. Fang said, “In Asia Pacific, the need to treat rare diseases is significant. For instance, there is an estimated 200K cases of hemophilia in Asia Pacific but only 26K are diagnosed, and less than 30% of them get treated.”
In terms of patients, Shire invests to improve the standard of care and patient programs. Today, only 5% of rare diseases have FDA approved treatment . Thus, the need to invest in rare diseases is significant and this is the key that motivates the research teams at Shire when developing their pipelines and moving them forward.
Shire’s pipeline and programs are pioneering and innovative in their approach across multiple therapeutic areas. Excitingly, under the 40 clinical development programs, most of those are actively developed in APAC.
“We make sure that we are taking a personalized approach with the biggest impact for patients,” added Fang. “Not only that, we are also optimizing the precision in terms of dosage for every single patient.”
Shire also develops immunotherapies for a disease called Primary Immunodeficiency (PI), a group of more than 300 genetic disorders in which part of the body’s immune system is missing or failing. The patients get infections easily or display severe reactions to the external environment. Moreover, Shire also has portfolios in neuroscience – ADHD drug development, internal medicine for chronic kidney disease and gastrointestinal disorder, oncology, and a new pipeline for ophthalmics.
Fang reflects on the areas for improvement in this region’s approach to rare disease. “In general, therapies for rare diseases are difficult to develop due to challenges such as limited public, patient and physician knowledge of the signs and symptoms to look out for, due to the vast number of rare diseases as a whole (approximately 7,000 identified), and the rarity of those diseases individually. In Asia, some countries do not have clear definitions of rare diseases, hence for regulatory purposes, rare diseases are treated similarly as any other medicines. Overall, in Asia Pacific, there is huge opportunity for improvement. We believe that we can expand our leadership and our presence with our patient-centric model in the rare diseases space.”
Shire constantly partners with governments, ministries of health, patient advocacy organizations and clinical organizations to enable more access of its therapies in various countries.
Fang elaborates that for this small community of rare diseases, only a handful of doctors have the specialization to understand and see the patients. “We must help to connect worldwide specialists with local physicians and local patients to improve local standards of care.”
“We seek to reinforce leadership. It is about being the champion for people with rare diseases, and a champion for patients. We tend to evaluate our success by measuring how much impact we have brought to every single patient in a single day, and we talk about – and share – patient stories,” said Fang.
With a strong patient-centric and entrepreneurial culture, Shire is the leading global biotechnological company focused on developing and delivering innovative medicines to under-served patients with rare diseases and highly specialized conditions.
1. EURORDIS 2005
2. Global Genes accessed October 2015
3. Miyamoto & Kakkis 2011