Problem A call was received out of hours by the specialist PICU pharmacist (SP). A five month old baby with rapidly spreading paralysis of unknown cause had been admitted to the unit. A toxin had been extracted from the stool culture and tested on mice. Within hours all mice had died, confirming a positive result for Botulism toxin. The SP was asked to obtain an urgent supply of Human Botulism Anti-Toxin however the only worldwide manufacturer/supplier, the Infant Botulism Treatment and Prevention Program (IBTPP), is based in California.¹ BabyBIG, Botulism Immune Globulin Intravenous (Human) (BIG-IV), is an orphan drug that consists of human-derived anti-botulism toxin antibodies that is approved by the U.S. Food and Drug Administration for the treatment of infant botulism types A and B in patients <1 year old.

Pharmacist contributions Day 1: After confirmation with PHE (Public Health England) that the use of their heptavalent horse botulisum anti-toxin would be inappropriate, it was suggested that a supply should be obtained from the USA. SP phoned the IBTPP on call consultant and discussed need for an urgent supply.

Consequently SP ensured the appropriate provision and recording of required information and the seamless transition of relevant paperwork.

Day 2: SP liaised with the Clinical Director for Children’s Services, the PHE consultant on call and the Trust Silver on call manager to authorise large out of hours drug expenditure. Prompt authorised signature of contract between the above parties was arranged by SP via email. SP contacted the MHRA duty officer on call to obtain an import permit authorisation letter (Notification of Intent to Import an Unlicensed Medicinal Product) to allow for this unlicensed import of a human medicinal product from outside the EEA, re-affirming this was of urgent clinical need.

Trust Chief Pharmacist was alerted to the situation by SP, and access to the to the Trust import/specials licence required by the MHRA was granted to the SP to finalise the MHRA import licence. A courier from California was organised by SP liaising with the on call IBTPP consultant, ensuring all paperwork was accurately completed. Dosing, administration and reconstitution advice was given by SP to PICU medical and nursing staff via email. SP immediately confirmed receipt via phone and provided clarification of this when required. SP remained contactable throughout the weekend to resolve any queries the staff had with regards to BabyBIG.

Day 3: The SP attempted contact with border control at Heathrow airport to ensure a timely transition through customs and liaised with the courier in the UK to ensure rapid delivery once BabyBIG had been cleared. Allowing sufficient transit time from Heathrow, the SP then called to confirm receipt of BabyBIG on PICU.

Outcome and lessons learned BabyBIG obtained and patient treated successfully, avoiding potential for serious complications and dramatically reducing PICU and overall inpatient stay. A cost analysis done by SP confirmed treatment with BabyBIG reduced overall Trust spend on this admission by half; accounting for average expected PICU stay for infant botulism cases(∼6 weeks) versus this patient’s stay (∼1.5 weeks).

References

1. Division of Communicable Disease Control, California Department of Public Health. Infant Botulism Treatment and Prevention Program [online] California Department of Public Health, 2010. (accessed 02 Aug 2018) Available from: http://www.infantbotulism.org/general/babybig.phpSave