SUPPLY AND LOGISTICS
often the best choice, Engel says. For example, Origin Biosciences recently signed an exclusive distribution deal with specialty pharmacy Biologics by McKesson. Origin’s Nulibry (fosdenopterin) treats molybdenum cofactor disorder with around 150 patients worldwide.
Specialty pharmacies offer expertise If one pharmacy is the sole provider for a disease with a population in the thousands, insurers are motivated to work with that one pharmacy, Engel adds. In addition, the small specialty pharmacy could provide expertise key clinicians, and knowledge of negotiating coverage with insurers, she says. The National Institutes of Health (NIH) estimates there are around 7,000 rare diseases in the US, many with very small, niche pools of clinical experts. manufacturer helps cut down vendors and decreases their costs,” says Dr Brandon Salke, of the specialty pharmacy Optime Care. In general, drugs for rare diseases with fewer from an exclusive agreement with a specialty pharmacy, he notes. Exclusive contracts allow manufacturers to
control the list price of their drug, contractually limiting the specialty pharmacy to a set manufacturer will usually pay the specialty pharmacy fees for services like ensuring rare disease therapy, she adds. This grants manufacturers more knowledge and oversight over which patients receive their drug and the reimbursement obstacles they face, she says. In contrast, with other contracts, a manufacturer sells the product to the pharmacy
Risk-sharing contracts for gene therapies sharing strategies in contracts, Vogenberg says. based contracts should have as few parties as possible, making it easier to assign risk and structure outcomes that trigger payments.
36 | Clinical Trial Supply Handbook
“Exclusive contracts allow drug manufacturers to control the list price of their drug, contractually limiting the specialty pharmacy to a set percentage mark-up”
In these cases, there would be an incentive
to remove PBMs from the supply chain and reduce or eliminate the number of involved specialty pharmacies where possible, Vogenberg notes. Manufacturers could seek more direct relationships with hospitals for gene therapies administered in the inpatient setting, he adds. Hospital systems are also joining the trend
toward consolidation, with many operating or acquiring their own specialty pharmacies. “Pharmacies owned by hospital systems are now very aggressive in growth in the marketplace and are very much a force to be reckoned with by manufacturers,” says reimbursement consultant Doug Paul of Medical Marketing Economics. However, displace other specialty pharmacies, he notes. The most prevalent rare diseases likely to soon see gene therapy approvals are haemophilia, Pompe disease, and Duchenne muscular dystrophy (DMD), the drug distribution expert says. Spinal muscular the approval of Novartis’s Zolgensma, with a list price of over $2m. Still, if all these gene therapies gain approval, there would likely only be around 30,000 eligible patients by 2025 or 2026, he adds. the public may have mixed feelings, Salke says. Gene and cell therapies will be seen as breakthrough treatments, but people will likely question their exceedingly high costs, he explains. “Payers will seek out innovative payment structures to help curb the cost and provide a safety net,” he predicts.
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