July 2014

This PiNSA statement supports international calls to ensure access to the best suited immunoglobulin (IG) replacement therapy, as prescribed by physicians for patients living with a primary immunodeficiency (PID)[1] that requires IG replacement therapy. It is estimated that there are between 20,000- 40,000 South Africans living with PID, of whom 99% remain undiagnosed.

Some PIDs are potentially life-threatening, chronic conditions which are the result of faulty inherited immune systems, with the World Health Organisation identifying more than 200 kinds of PID diseases[2].

Ongoing, regular, life-saving IG replacement therapy, either intravenously or subcutaneously, is critically important for some PID patients. As such, IG replacement therapies are listed on the World Health Organisation (WHO) list of essential medicines for the treatment of PIDs[3].

IG replacement therapy helps to protect patients who require it against a range of infections and reduces autoimmune symptoms, treating (amongst other conditions): Common Variable Immunodeficiency (CVID), X-Linked Agammaglobulinaemia (XLA), X-linked and non X-linked Hyper-immunoglobulin M (Hyper-IGM) Syndrome, Wiskott-Aldrich Syndrome (WAS), Severe Combined Immunodeficiency (SCID) and other combined immunodeficiencies.

There is no alternative treatment to IG therapy for qualifying patients and without treatment, the quality of life, life expectancy, as well as their productivity, is severely compromised. In addition, the effect of the disease burden on an already over-burdened health care services should not be underestimated, and IG replacement therapy has been proven as a pharmaco-economically sound protocol for management of patients with PIDs.

PiNSA recommends that measures are taken to ensure that PID patients who require IG replacement therapy have continuous and equal access to the widest range of safe and effective IG therapies. Given that many PIDs are recognised as conditions on the Medical Schemes Act Chronic Disease List, PiNSA which includes a medical panel of experts in the field of primary immunodeficiencies, and supported by the International Patient Organisation for Primary Immunodeficiencies (IPOPI), welcomes the opportunity to work with the Department of Health, Council for Medical Schemes and individual medical aids in ensuring IG replacement therapies are added to the SA Essential Drugs List and in standardising treatment protocols in this regard.