At Hansa Biopharma, we are committed to advance the science of rare immunologic diseases to help develop new treatments that can address these complex, progressive, and often fatal conditions. To achieve this, we work with key stakeholders – including patient advocacy groups, clinicians, payers, other industry players, and policy makers – to increase the understanding of rare diseases and advance the science around these conditions, to ultimately bring innovative treatment solutions to all patients who need them.
The urgent need in rare diseases
Rare diseases place a significant burden on patients and those who live with them, as well as on the entire healthcare system. There is a need for better and dedicated treatment options for rare diseases that can improve the quality of patients’ lives and reduce the impact on healthcare systems. Between 260 to 450 million people globally live with a rare disease.3 Approximately 50% of rare diseases affect children, with one in three dying before five years of age.4 The higher medical needs of people living with a rare disease means that they often miss work or are forced to early retirement, and may require the assistance of a caregiver.5
People living with rare diseases face unique challenges due to the rare nature of the disease which can result in incorrect or missed diagnoses, inadequate clinical management, and limited access to effective treatment.6,7
Delays and misdiagnosis can cause the disease to progress unchecked and worsen patients’ quality of life. This is particularly dangerous in rare acute diseases, including autoimmune conditions, where acting quickly can be lifesaving.
Guillain-Barré Syndrome (GBS), for example, is a severe acute autoimmune disease targeting peripheral nerves and their spinal roots that can lead to severe paralysis of the arms and legs with approximately 25% of patients requiring mechanical ventilation for days to months and 20% unable to walk after six months.8,9 The rare nature of the disease causes delays in diagnosis and in the subsequent start of immunosuppressive treatments that can help limit the long-term damage to the nerves.
Guided by patients’ voice
People living with rare diseases are keen to play an active role in the clinical development of innovative treatments, with the large majority open to consider using an investigational treatment.10 At Hansa, we collaborate with patients to ensure patients’ view and preferences are embedded in our strategy. Understanding and incorporating patients’ voice throughout the development of a treatment involves a decision-making process informed by patients’ views and insights. From early research and clinical trial design to regulatory evaluation and reimbursement, patients’ voices and their insights can help build a more comprehensive understanding of the value they see in accessing an innovative therapy. This understanding, combined with clinical input from treating physicians, can help assess the real impact an innovative therapy has on a patient’s life.11,12
"At Hansa, we work to ensure that the patient voice is heard and understood during clinical decision-making regarding their priorities and needs and in reimbursement access decisions" said Gina Ewy VP of Global Market Access at Hansa Biopharma. "As an example, we are working closely with the American Association of Kidney Patients (AAKP) to conduct a discrete choice study investigating the factors highly sensitized kidney transplant patients most value when making a therapeutic choice to remain on dialysis or proceed with an HLA-incompatible kidney transplant surgery."
