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doi:10.1111/jpc.12531
EDITORIAL
Ethical dilemmas about orphan drugs for orphan diseases
Our hospital Drug Committee recently received a request from the renal team to prescribe eculizumab for a young child with refractory atypical haemolytic uraemic syndrome (aHUS). Eculizumab, which inhibits a terminal component of complement, is relatively safe and effective in aHUS but not curative, so currently, it is usually continued indefinitely.1 The main barrier is cost. The pharmaceutical company agreed to a modest price reduction for our patient, but the annual drug cost was still over $160 000 and is twice as much for older children and adults. One or two Australian children with aHUS receive eculizumab free from the company (‘compassionate use’), but as evidence of efficacy accumulated, the company’s compassion dissipated, and now, hospitals have to pay for new patients. The company has applied to the Pharmaceutical Benefits Advisory Committee for Australian Government funding of eculizumab for aHUS, but similar applications elsewhere have been unsuccessful because of the high cost. Our hospital Drug Committee and senior clinical administrators felt as if we were being held to ransom: The patient and her family were the hostages who would suffer if we refused. After careful consideration, we agreed to pay for eculizumab, but other hospitals might have reached a different decision, scarcely an equitable situation. Orphan diseases are rare diseases (defined as a prevalence ≤50 per 100 000 in the European Union or
doi:10.1111/jpc.12531
EDITORIAL
Ethical dilemmas about orphan drugs for orphan diseases
Our hospital Drug Committee recently received a request from the renal team to prescribe eculizumab for a young child with refractory atypical haemolytic uraemic syndrome (aHUS). Eculizumab, which inhibits a terminal component of complement, is relatively safe and effective in aHUS but not curative, so currently, it is usually continued indefinitely.1 The main barrier is cost. The pharmaceutical company agreed to a modest price reduction for our patient, but the annual drug cost was still over $160 000 and is twice as much for older children and adults. One or two Australian children with aHUS receive eculizumab free from the company (‘compassionate use’), but as evidence of efficacy accumulated, the company’s compassion dissipated, and now, hospitals have to pay for new patients. The company has applied to the Pharmaceutical Benefits Advisory Committee for Australian Government funding of eculizumab for aHUS, but similar applications elsewhere have been unsuccessful because of the high cost. Our hospital Drug Committee and senior clinical administrators felt as if we were being held to ransom: The patient and her family were the hostages who would suffer if we refused. After careful consideration, we agreed to pay for eculizumab, but other hospitals might have reached a different decision, scarcely an equitable situation. Orphan diseases are rare diseases (defined as a prevalence ≤50 per 100 000 in the European Union or
