Easing Hemophilia Burden: From Disease to Treatment
By Karin Knobe, MD, PhD, Head Rare Diseases Clinical Development
There is more than meets the eye when Sanofi is chasing the miracles of science. What may be a clinically meaningful benefit from a medical standpoint may create a new burden for a patient. That is what leads Sanofi to chase solutions that treat the 'whole person' so that all aspects of a person's health can be improved.
This is exemplified in the work we do for people living with hemophilia, a rare lifelong condition in which a person’s blood does not clot properly due to a lack of clotting proteins, insufficient thrombin generation, and ineffective clot formation. While we have come a long way in the more than 25 years since I first practiced in pediatric hematology, there is more to go for this community that deserves to live their lives free from the burdens and limitations of this disorder.
Bridging Science and Patient Care
I began my career at the prestigious Malmö Center for Thrombosis and Haemostasis in Sweden immersed in both clinical practice and laboratory research, focusing on coagulation disorders – which are conditions where the blood is able or unable to clot properly. When I started in 1997, the hemophilia community was starting to see new breakthroughs that helped improve care. Even with those advancements, a team of us were hard are work in labs discovering some of the coagulation proteins and molecules that we use to treat patients today.
Beyond the lab, I treasured my work as a practicing pediatric hematologist. My clinical practice allowed me to connect with these children and their parents on a personal level, witnessing firsthand the challenges they faced on a day-to-day basis beyond what you could read on a medical chart. Even outside of office visits, I would join the community at summer camps and education sessions to support and empower them as they worked to live their lives while managing their hemophilia.
These experiences reinforced my commitment to advancing meaningful care in hemophilia that helps this community live as ‘normal’ a life as possible.
Dr. Karin Knobe continues to dedicate her professional life to advancing care in rare diseases, including rare blood disorders
Reducing the Burden of Disease and Treatment
What I find unique about the advancements in hemophilia care is that while we helped solve the burden of disease, the community found themselves with a new burden – their treatment. For some, the burden of treatment can be enormous.
While advancements in hemophilia care have led to people living longer, it first meant patients had to make frequent visits to a hospital for transfusions. Future innovations came in the form of at-home treatments; however, people living with hemophilia were required to carry bulky supplies and perform time-consuming infusions – sometimes daily – making it difficult for some to live their daily lives.
Understanding how close the hemophilia community is, researchers and physicians – including those at Sanofi – have closely collaborated with them to make sure future advancements are directly informed by their needs. In doing so, this dynamic partnership is enabling us to develop the next line of treatments that could help this community lead fuller, healthier lives.
The Path Ahead in Hemophilia
Together with the community, Sanofi is elevating understanding of what it means to live with hemophilia - the impact on physical and mental health, lifestyle, relationships, career, family and more - through the eyes of people living it, so that we can meaningfully advance the care paradigm.
Even now, leading Global Development for Sanofi’s Rare Diseases – including the Rare Blood Disorders franchise – I carry the faces of patients I treated years ago. Their stories fuel my drive to build on today's progress and envision a future with even greater breakthroughs.
The burden of treatment is one of a list of pressing issues we at Sanofi need to address, and we recognize there is more to hemophilia than just preventing or treating bleeding. Working as a committed partner with the hemophilia community, we’re going Beyond the Bleed.