Value to Patients

As different policies to slow health spending are debated, it is critical to maintain access to lifesaving treatments for rare disease patients. Although some value-based frameworks work for generic, interchangeable pharmaceuticals—a one-size-fits-all policy does not work for plasma protein therapies as these biologics are not interchangeable. Plasma protein therapies are high-impact pharmaceuticals because they increase life expectancy, improve quality of life, and reduce life-threatening complications for individuals with plasma protein deficiencies.

Plasma protein therapies provide immeasurable, lifelong benefits to the patients who use them.

10-year survival rate of patients with COMMON VARIABLE IMMUNE DEFICIENCY, by year

Source: Chapel H, Lucas M, Lee M, et al. Common variable immunodeficiency disorders: division into distinct clinical phenotypes. Blood. 2008; 112(2):277–286.

Life expectancy of a patient born with HEMOPHILIA, by year

Source: Aledort, L. The evolution of comprehensive haemophilia care in the United States: perspectives from the frontline. Haemophilia. 2016; 22(2):676-683.

Terry

individual with a Primary Immunodeficiency Disease

“To think about having to go back long term without my IVIG infusions, I would rather not be alive. I started to receive the plasma therapy and within a couple of months from being near death...I became very vital. These are lifesaving therapies for which there is no alternative for many patients. To take away the plasma therapy from a PI patient such as myself— what you’re doing is condemning those people to a life of sickness and possibly death.”

Kerry

mother of sons with hemophilia

“Plasma-derived therapies saved my children’s lives, literally. The first thing that would happen if we didn’t have access to them would be that we would not be able to stop bleeding inside their bodies, they would first be in a lot of pain, then they would become crippled and eventually they would die.”

VALUE TO THE SYSTEM The economic impact of diagnosing a Primary Immunodeficiency Disease and treating an individual with immunoglobulin therapy represents an average savings of $55,882 per year.

Source: Modell, V., Quinn, J., Ginsberg, G., Gladue, R., Orange, J., & Modell, F. (2017). Modeling strategy to identify patients with primary immunodeficiency utilizing risk management and outcome measurement. Immunologic Research.

Immunoglobulin replacement therapy (lgGRT) in Common Variable Immunodeficiency Disorders yields Incremental Cost Effectiveness Ratio-estimates that are of the same magnitude as those for the-by far-more common influenza vaccination program for adults.

Source: van Wilder P, Odnoletkova I. Mouline M. de Vries E (2021) lmmunoglobulin Replacement Therapy is critical and cost -effective in increasing life expectancy and quality of life in patients suffering from Common Variable Immunodeficiency Disorders (CVID): A health-economic assessment.