Hospital Supply of Off-label Immunomodulatory Drugs

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There has been an increase in the demand for new immunomodulatory drugs for the treatment of immunemediated inflammatory diseases. Many of these indications represent the off-label use of immunomodulatory drugs.


To evaluate the spectrum of requests, clinical outcomes and costs for the hospital-approved, off-label use of immunomodulatory drugs.


A list of all new individual patient use approvals at a Sydney teaching hospital (February 2005 to June 2006) was obtained and the approved immunomodulatory drugs were selected for audit. A literature review was conducted to determine the level of evidence available for the approved immunomodulatory drugs.


61 individual patient use approvals for off-label immunomodulatory drugs were analysed. The most commonly requested drugs were infliximab (n = 22), mycophenolate (n = 13), rituximab (n = 10) and cyclosporin (n = 7). These drugs were initiated for treatment of a heterogeneous group of serious medical disorders. 10 hospital departments initiated the requests. The cost of new approvals was $458 301, accounting for 61% of the total cost of $749 236 for off-label immunomodulatory drugs within the study period. The most expensive drugs were rituximab, infliximab and mycophenolate. Efficacy reports were available for 64% of approvals and a favourable therapeutic response was observed in 74% of cases. 4 deaths occurred and significant treatment-related adverse events were noted in 24% of cases. High-level evidence supported the use of immunomodulatory drugs for 41 individual patient use approvals.


Off-label use of immunomodulatory drugs was diverse and supported by good evidence in over 50% of cases. Methods used to assess the response to immunomodulatory drugs were suboptimal and non-standardised. Formalising the supply of immunomodulatory drugs may enable aggregation of data to inform future decisions and resource allocation.

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