Why is access to all brands of plasma protein therapies important?

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One-size-fits-all policies are unsuitable for plasma protein therapies and endanger patient health. Each therapy is unique due to the pharmacologic and manufacturing differences that exist across different brands and patients’ unique responses to the treatments. Plasma protein therapies are non-interchangeable, sole-source biologics, therefore it is essential that patients have access to their medically justifiable therapy.

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

37%: 1971
78%: 1993
90%: 2008

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

1900: 13 years
1960: 20 years
2017: 77 years

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

"Given the variable nature of these diseases, individualized treatments depending on patient need and physician judgment are important."

American Academy of Neurology

"It is unacceptable to limit availability of augmentation therapy in any way and especially to a single product."

Alpha-1 Foundation

"Because not all patients respond the same to each medication, it is the responsibility of the coordinating expert physician to work with each patient to define the optimal medication(s) for that particular patient."

haea

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.

The rare and chronic diseases treated by plasma protein therapies are often difficult to diagnose, treat, and manage. Accordingly, to ensure the best possible health outcomes for patients, the individuals who rely on plasma protein therapies as part of their lifesaving treatment require medically appropriate access to the specialists who best understand the complexities of their diseases. A relatively select few specialists across the country have developed the enhanced knowledge and experience necessary to effectively treat the rare diseases treated by plasma protein therapies. Therefore, we recommend payer policies that allow individuals with plasma protein deficiencies access to those physicians who have experience treating these rare conditions.

Immune Deficiency Foundation. (2009).