Ernesto Aycardi
Global Head of Development, Kyowa Kirin
Angela Williams
VP, Global HEOR Head, Kyowa Kirin
To make a real difference in the rare disease space, pharma companies are driving innovation via partnerships with each other — and with the patients they are aiming to help.
Pharma companies working in the area of rare diseases will, by definition, only impact a smaller percentage of the population. Yet, the size of a patient group isn’t the point, insists Dr Ernesto Aycardi, Global Head of Development at Kyowa Kirin, a Japan-based global specialty pharma company. It’s about helping those struggling with unmet health needs and who often feel unheard and overlooked.
Targeting rare disease treatments
“Our mission is to develop innovative products that deliver life-changing value to patients and their families,” Dr Aycardi explains. “We want to centre our activities on meeting patients’ needs and representing those who too often feel they have no voice. For those reasons, the rare disease space is exactly where we want to be.”
It’s a stance that has delivered notable successes for Kyowa Kirin, a company with expertise in biologics and stem cell gene therapies and a focus on developing therapeutics for bone and mineral, haemato-oncology, and rare diseases. X-linked hypophosphatemia (XLH), a rare, lifelong, progressive musculoskeletal disease that affects around one in 20,000 people worldwide, is one key area of specialty for the company.
“For people living with XLH, the impact can be debilitating,” says Dr Aycardi. “With limited options available, we worked tirelessly to bring a new therapy for these patients, and we remain committed to addressing the unmet needs of patients and their families.”
We always engage with patients on
the design of our studies to find out
what we should be measuring.
Partnership accelerates access to life-changing treatment
Naturally, none of this is straightforward because rare disease drug development is fraught with challenges. By being open to partnering with other companies, Kyowa Kirin has been able to speed up patient access to potentially life-changing medicines. “Most recently, we partnered with Ultragenyx to hasten both late-stage clinical studies and delivery of therapy to our patients around the world,” said Dr Aycardi.
It’s not just good collaboration between pharma companies that can facilitate the discovery and delivery of rare disease medicines. Dr Aycardi stresses the importance of pharma firms working with academia, healthcare providers, regulatory agencies, advocacy groups, and patients themselves.
It’s a point that Angela Williams, VP, Global HEOR Head, reiterates. She notes that Health Economics and Outcomes Research (HEOR) is a vital piece in the drug development jigsaw. “It involves ensuring we understand and measure what is important to patients.
“We always engage with patients on the design of our studies to find out what we should be measuring,” explains Dr Williams. “For example, a drug in development may measure a health issue well known to the clinicians, such as itching; but the clinicians may be less aware of other problems patients have living with a disease, such as fatigue, which is severely affecting the patient’s quality of life. The best way for us to find out about the full spectrum of unmet needs is from the person who is actually living with the disease.”
Importance of a deep and lasting relationship with patients
HEOR teams must go beyond simply ‘talking’ to patients about their experience to inform a clinical trial. Fostering lasting, trust-based relationships will gather the most valuable insights. “It’s why we engage with patients and patient groups at the very start of the research and development process and continue to work through the whole development process together,” says Dr Williams. “We’ve worked this way, not only in XLH but also in cutaneous T-cell lymphoma (CTCL), partnering and collaborating with patient groups all around the world.”
That includes continuing studies after approval to understand the benefit and safety of a new drug in the broader population of people with the disease.
“I believe stem cell gene therapy is going to be revolutionary with regards to rare diseases — and other diseases,” says Dr Aycardi. “Patients with a rare disease are looking for hope. Their voices are becoming louder and it is incumbent on all of us to listen to them.”
