Position
statement on managing immunoglobulin replacement therapy for primary
immunodeficiency (PID) patients during stock shortage
By the
Primary Immune Deficiency Working Group (PIDDSA) of the Allergy Society of
South Africa (ALLSA)
Summary
statement
Immunoglobulin
replacement therapy is a life-saving treatment for patients suffering from
antibody deficiency secondary to PID. Most these conditions are prescribed
minimum benefits (ICD10 coding D80.1-84.9).
Stock
shortage of registered immunoglobulin products occurs in South Africa, leaving
PID patients vulnerable. Immunoglobulin therapy cannot be discontinued by PID
patients during stock shortages.
We
strongly call on:
i) Doctors, patients and patient support organisations to assist patients with
the burdens of frequent, unanticipated product changes
ii) Medical Funders to be responsive to request for short-term use of
alternative, usually more expensive products during stock-outs
iii) Suppliers of immunoglobulin to improve the supply chain process and limit
stock shortages
Immunglobulin
therapy cannot be discontinued when stock shortages occur
Replacement
immunoglobulin therapy (IRT) is life-saving and cost-effective for patients
with hypogammaglobulinemia or functional antibody deficiencies owing to primary
genetic immunodeficiencies. Most patients require 0.4-0.6g/kg/month to provide
protective antibody levels and prevent serious bacterial infections. PID
patients are completely reliant on this expensive and logistically challenging
therapy. Failure of regular replacement therapy will lead to a dangerous
decline in protective antibodies within 1-3 months, given a half-life of
approximately 21 days for IgG. A single serious bacterial infection can lead to
death or serious morbidity, such as the neurological impairment that may result
from bacterial meningitis. PID patients cannot discontinue life-saving IRT during
stock shortages
The
route of administration can be changed to ensure Immunoglobulin treatment
continues
PID
patients can receive IRT via either an intravenous or subcutaneous routes, with
largely equal efficacy but distinct side-effect profiles, pharmacokinetics and
administration challenges. Thus, it is reasonable in times of shortage to
consider changing the route of administration to ensure that IRT treatment
interruptions are limited. However, changing the route of administration may
NOT be appropriate if the reason for an original change from the intravenous to
subcutaneous route was serious side-effects. Patients with medical reasons for
using the subcutaneous route of administration for IRT must be fully funded and
supported to use an alternative available subcutaneous product as this is a PMB
condition.
The
additional expense of short-term product change must be paid by medical funders
when change is forced by stock shortage in PMB conditions
Product
change for PID patients during times of stock shortage in many instances may
mean using a more expensive alternative therapy to avoid treatment interruption.
It is mandatory that medical funders support the additional expense of
short-term product change. It is unacceptable that patients be expected to
provide large co-payments to support the use of more expensive alternative
products.
Patients
should call on treating physicians, patient organisations and medical funders
to help ensure treatment interruptions are avoided
Stock
shortages and the resultant need for product change is a challenging situation
for patients and their treating clinicians. We need to act fast to ensure that
antibody levels do not drop dangerously low. We would encourage individual
patients to contact treating physicians and vice versa to notify them of
available stock and make an immediate plan. Treating clinicians then need to
liaise with the patient and funder to ensure continuity of IRT. Patients can
also reach out to their patient organisation PINSA to assist them in ensuring
continued therapy through direct support and more widespread advocacy.
Available
products need to be increased and existing supply chains strengthened
Immunoglobulin
therapy is derived from donor plasma through a process of fractionation. It is
complex to manufacture and make safe for patient administration; it is
expensive and dependent on donor availability. SA has only one fractionator,
that provides an intravenous and intramuscular immunoglobulin product. There
are two other registered, imported products. Several patients access imported
products via section 21 MCC approvals. This is a very limited number of products
and suppliers by international standards. The consequence of this is inevitable
stock shortages, which is a catastrophe for vulnerable patients’ dependent on
these products. All suppliers in SA have experienced stock shortages at some
time point. Thus, we call on our local manufacturers to make renewed efforts to
ensure that the current problem is resolved and that future problems are
avoided. We also call on government to facilitate the registration of new
products in the SA market to allow for the unavoidable fluctuations in plasma
donation. The current situation is untenable and may cost lives.
Compiled
by:
Prof
Jonny Peter
Prof Monika Esser
Prof André van Niekerk
Members of PIDDSA. Adopted by the Executive Members of ALLSA during an
executive meeting on 13 May 2017.
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