Sponsored Insight
How can innovation address unmet needs? What is the value of improving a person’s quality of life? Who benefits when health outcomes improve?
Stakeholders across the healthcare system are tackling these big questions as research into new treatment options for hemophilia B builds on the immense success of the past 70 years.
Painting the story of progress.
For people with hemophilia B, innovation has come a long way. Today’s gene therapy research stands in stark contrast to the 1950s when fresh frozen plasma was the only treatment, and the average life expectancy was about 20 years. In the 1990s, recombinant factor products revolutionized treatment and allowed people with hemophilia B to have a longer life. In 2016 recombinant products further evolved treatment standards with the first replacement product that offered sustained, high factor levels for up to 14 days.
Grandparents whose childhood memories involve long stays at the hospital due to a lack of outpatient treatment options for hemophilia B and sometimes unbearable pain from bleeds are now watching their grandchildren pursue active and fulfilling lives thanks to the progress in care and treatment options.
Innovations in care undoubtedly benefit a person living with hemophilia B, but the positive impacts can go even further. Understanding how hemophilia B affects family, friends, caregivers, and the larger healthcare system is critical to appreciating the value that improvements in care may make for society as a whole.
Managing the life-long impacts of hemophilia B.
Despite the enormous progress, people with hemophilia B can still face challenges.
Current standard of care for people with moderate to severe hemophilia B involves life-long infusions. They may also experience spontaneous bleeding episodes, which can lead to joint damage and difficulty carrying out everyday tasks, long-term pain, reduced quality of life and for some, surgical replacement of the damaged joints.
The life-long effects of living with and managing hemophilia B aren’t just physical. More than 40% of people living with hemophilia B experience depression, anxiety, or other psychological disorders.
Moreover, recent estimates show that people living with hemophilia B miss an additional 3.5 days from work per year and have higher rates of early retirement, which accounts for approximately $6,000 in annual indirect costs per person in the United States. In fact, overall healthcare costs can be 25 times higher for people living with hemophilia B compared to individuals who do not have a bleeding disorder.
Understanding the potential of innovation.
Gene therapy research continues to build on the revolutionary progress that has already been made in hemophilia B treatment. Gene therapy is currently being explored as a one-time treatment option that aims to address the underlying genetic cause of the condition.
By generating stable and durable FIX levels, reducing annual bleed rates, and reducing or eliminating prophylactic therapy, gene therapy has the potential to lower the physical, mental and economic impacts of hemophilia B.
If we are able to realize the potential of gene therapy, it could make significant strides toward reducing the overall burden of hemophilia B — to the patient and their family, caregivers and the healthcare system.
Learn more about innovation in bleeding disorders here.