HHS makes history with $4.5M Hemophilia B treatment delivery
Outpatient pharmacy receives first-ever delivery of groundbreaking $4.5M Hemophilia B treatment
A Hamilton Health Sciences (HHS) outpatient pharmacy recently made history by becoming the first in Canada to receive a delivery of Hemgenix, a $4.5 million, one-time gene therapy for adults with Hemophilia B, a rare genetic bleeding disorder.
Hemgenix is one of the most expensive medications in the world.
The delivery to the outpatient pharmacy at HHS McMaster University Medical Centre (MUMC) involved following a wide range of protocols for receiving and storing this highly valuable drug, and underscores the HHS pharmacy team’s leading-edge capabilities in handling ultra-specialized medications.
“I’m proud of our pharmacy teams across HHS, whose specialized training makes life-changing, lifesaving treatments available to our patients.” — Wassim Houneini, HHS outpatient pharmacies manager
HHS outpatient pharmacies serve many specialized patient populations, including patients visiting clinics during the day for appointments rather than staying overnight, and patients being discharged from hospital. There are HHS outpatient pharmacy locations at MUMC/McMaster Children’s Hospital, Hamilton General Hospital and Juravinski Hospital and Cancer Centre.
Hemgenix can dramatically improve the quality of life for people with Hemophilia B. About 800 people in Canada have this disease, where their bodies can’t make enough of the Factor IX protein needed for blood to clot properly. Hemgenix’s effects are often long-lasting with many people having fewer bleeding episodes, or none at all, for years after just one treatment. While Hemgenix is approved for use in Canada, it’s not yet on the market. Ontario Health handles approvals for patients receiving this medication, and covers the cost.
HHS is among Canada’s largest and highest-ranked academic health sciences centers, and is a national leader in gene therapy through involvement in advanced treatments and pioneering research. Our pharmacies are among the few in Canada qualified to handle medications worth several million dollars, which require highly specialized delivery and storage protocols, and advanced inventory tracking.
A very special delivery
Given Hemgenix’s hefty price tag, there’s no room for error when it comes to receiving and storing it, says Wassim Houneini, HHS outpatient pharmacies manager.
Mock delivery runs are held to prepare for the arrival of ultra-specialized medications.
Houneini was on hand to personally receive Canada’s first-ever shipment of Hemgenix in April, which involved following standard operating procedures so detailed, the document containing them is almost 50 pages long. “Only three hospitals in Canada are currently receiving Hemgenix, and we were the first,” says Houneini.
The playbook
Standard operating procedures include a temperature-controlled supply chain so the drug stays at the correct temperature at every stage, including transportation, handling, delivery and storage.
Hemgenix is shipped from Europe to Toronto, and then transported by a courier to Hamilton. It was a fairly easy drug to transport and store because it didn’t require dry ice packaging or extra-cold storage, says Houneini.
Every handoff from the manufacturer, to the distributor, to the pharmacy was documented and tracked, and the drug could only be accepted and handled by qualified staff who checked to ensure it arrived in a sealed and undamaged tamper-proof container with a tracking number.
Pharmacy staff got advance notice of the delivery, so they would be ready. Upon approval it was immediately inspected, temperature logs were checked, and it was quickly transferred to a freezer for storage.
All steps along the way were logged, including delivery time, the medication’s condition and the staff members involved. Insurance, security and other risk protocols were also in place. And because it’s a personalized treatment, information about the patient receiving it is directly attached to the medication.
Built on know-how
This isn’t the first time that the HHS outpatient pharmacy has accepted delivery of a highly valuable medication involving a myriad of protocols, says Houneini, adding that mock delivery runs are held to prepare for the arrival of medications like these.
A few weeks before Hemgenix’s delivery, the lab held a mock delivery run using a placebo medication for a different expensive drug also used to treat Hemophilia B. This medication arrived in dry ice, with specialized instructions that included storage at -70° C. A standard lab freezer is -25° C.
“Our storage is able to accommodate this, since we have a -100° C freezer,” says Houneini. However, Hemgenix treats the same patient population with easier storage and compounding requirements. But that dry run helped the team prepare for Hemgenix’s arrival.
The HHS outpatient pharmacy was also the first in Canada to receive and store the gene therapy drug Zolgensma, a $3 million, one-time treatment for kids born with spinal muscular atrophy, a genetic disease that damages the muscles and impairs the child’s ability to move.
“I’m proud of our pharmacy teams across HHS, whose specialized training makes life-changing, lifesaving treatments available to our patients,” says Houneini.
