Raising Awareness in the Fight to Cure Rare Diseases

Vineet Singhal,
Country Head, Baxalta Bioscience India Pvt Ltd (Now part of Shire).
"Our purpose is to enable people with lifealtering conditions to lead better lives," Says Vineet Singhal, Country Head, Baxalta Bioscience India Pvt Ltd (Now part of Shire). In an exclusive interview with Mahesh Kallayil, he further talks about potential opportunities in rare disease market, ensuring affordability of specialty medicines, business strategies and much more.

Can you briefly explain about Shire's vision and its focus areas in India?

Shire is a leading global biotechnology company committed to serving people affected with rare diseases and other highly specialized conditions. These diseases (many of which are genetic) are often misunderstood, undiagnosed, and potentially life-threatening.

Shire keeps patients at the centre of everything we do. In India, we are focused on two therapeutic categories, one being Hematology with our portfolio covering antihemophilia factors. Hemophilia is an X-linked congenital bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII) Hemophilia A or factor IX (FIX) Hemophilia B. The deficiency is the result of mutations of the respective clotting factor genes.

The second broad franchise in India is Immunology where we sell high-quality albumin and immunoglobulins, which are used under critical care by leading hospitals across India. We have widened our reach across the country and are present in every state.

How do you see rare disease market in India?

The biggest challenge that we see is that awareness and understanding of most rare diseases remains limited in India.

Therefore our primary focus will be on three areas: the first is to increase awareness of these conditions amongst the medical community. Our second focus will be to improve the means of diagnosis for rare diseases. Thirdly and most importantly we want to make therapies accessible to patients.

We are making a concerted effort to nurture an ecosystem that supports patients in rare diseases and ensure these specialized conditions can be treated. The good news is that there is a fair level of awareness about some rare conditions like Hemophilia across the country. Various efforts by the Government, patient bodies and healthcare companies have been instrumental in driving this awareness. It is thanks to these collective efforts by various stakeholders that the disease is addressed in a holistic manner. There has been an increase in funding for Hemophilia around the country from three states 10 years ago to 21 states as of today.

The Government has played a pivotal role in this journey by ensuring that patients do not suffer from a lack of access clotting factors. Going forward , Shire is committed to supporting the Government in its effort to increase awareness of rare diseases, LSD (Lysosomal Storage Disorder) and specialized conditions.

What are the potential opportunities you see for Shire in India ?

Given the current scenario of rising incidences of disease - burden in Hemophilia and other rare diseases it is an imperative and a responsibility for Shire to be able to bring therapies to India that can help patients lead a better quality of life. From our experience of having played the role of enablers providing access to the diagnosis and treatment of Hemophilia, we plan to play a similar role in the treatment of LSD.

Shire's extensive portfolio means we have the potential to help thousands of patients who need treatments , not yet available within the country. We want to make therapies accessible to patients so that they can start leading a near normal life.

We also plan to bring our Oncology portfolio into the country, including a first in line treatment for Acute Lymphoblastic Leukemia.

For Shire, it's critical we are a catalyst in creating an environment that supports Rare Disease patients with a comprehensive program that will encompass awareness, education, diagnosis, and infrastructure to provide the right treatment options. We believe the only way to make this possible is through meaningful partnerships that bring together policy makers, patient bodies, patient communities and other stakeholders around a common agenda, promoting awareness and accessibility to therapies.

At Shire, we ensure we build a sustainable platform for future innovation and growth. Keeping in mind the current scenario, the company has a significant role to play in building awareness among all these stakeholders .

Can you give us a brief overview of the Indian pharmaceutical sector and growth potential of biotechnology?

The pharmaceutical market in India is growing at a double-digit rate. Having said that, it is difficult to benchmark any specific data for the biotechnology market, although it has the potential to grow at a much faster rate than the mass pharma market. With rising in awareness levels and Government initiatives, we feel that the biotechnology industry will continue to grow, comprising bio-pharmaceuticals which will can contribute to the overall growth of the economy.

What steps have been taken by Shire to address the affordability of medicines of these highly specialized conditions?

Shire is dedicated to developing innovative treatments for rare diseases and specialized conditions, which make a difference to the lives of patients and their caregivers. Developing innovative medicines, particularly for rare diseases, is not easy. For every successful therapy that is brought to the market, there are many failures.

We are committed to pricing our treatments in a way that is responsible and sustainable for healthcare systems around the world and also ensures that our company makes a return on investment enabling us to develop the next generation of treatment for patients with high unmet needs.

To support decision makers, Shire generates evidence, both before and after our therapies are approved, to demonstrate the impact our treatments make to the lives of patients, their caregivers and to the healthcare system. We are solution-focused, actively seeking ways to engage constructively with healthcare providers, payers and policy makers to achieve together the best possible outcomes for patients from the resources available.

At Shire, we never lose sight of our focus on patients and our drive for high-impact. Our goal is to ensure that all patients prescribed a Shire therapy have access to it and that they have the support they need to achieve the best possible results for them. Shire has programs to support patients along their treatment journey offering early, supported or charitable access.

We believe our commitment to advancing innovation for rare disease and specialized conditions is how we are making a significant contribution to society through the impact we have on the lives of patients, their families and caregivers.

Shire has a very strong focus towards both global and domestic partnership. What are the basic criteria for partnership and how do you ensure success?

Shire's journey over the past few years has seen the company strengthened its position through partnerships and mergers, which in turn have helped us to achieve our mission. We are constantly seeking out new opportunities through strategic partnerships and our approach to business .

We have a good track record of partnerships with other organisations to develop innovative therapies which build on our existing rare disease portfolio. These partnerships help to combine the best of both companies and contribute to making Shire, a nimble, performance-driven organization through a collaborative approach with the patient at the centre of it all .

How important has the Baxalta acquisition been for Shire?

Our combination with Baxalta has been an important step to help us achieve our goal of being the leading global biotech for rare diseases, bringing us an expanded portfolio and increased capabilities that enhance the impact we can have on patients around the world with significant unmet needs. Shire and Baxalta's complementary portfolios mean that our global therapeutic areas now include Hematology, Immunology, Neuroscience, LSD, Gastrointestinal/Internal/Endocrine, HAE, and a small but growing franchise in Oncology. Globally, our two biggest franchises are Hemophilia (a Baxalta legacy) and LSD (a Shire legacy).

Following our combination , we have an expanded 37 programs in clinical development, our most robust pipeline in history. The combined company has nearly 24,000 employees and expanded geographic reach across more than 100 countries.

What are the major challenges you are facing and how are you gearing up?

From an India standpoint, our biggest challenge is the lack of awareness of rare diseases and specialized conditions. To overcome this, it is important to create more conversations at various levels, including patients, doctors, the medical community and policy makers. We have been working continuously with all stakeholders through patient outreach, awareness programs for the medical community and engagement with doctors and policy makers to increase awareness of these life affecting conditions.

What would be your business strategy for the current financial year ?

Shire strives to support patients with rare diseases and other specialized conditions, and our strategy is to create and grow longterm value based on improved diagnosis and providing high impact medicines and therapies. We believe our commitment to advancing innovation means we are making a significant contribution to society through the positive impact we have on the lives of patients, their families and caregivers.

We continue to focus on commercial execution and new product launches, including geographical expansions to extend access to our portfolio. Our intent is to expand leadership therapeutic are as by enhancing our commercial capabilities, increasing our global footprint and broadening our portfolio of best-inclass products. Also, we want to focus on the effective execution of our late stage clinical development pipeline to support future growth.