Managed Healthcare Executive: Pharmacy expert discusses disease impacts of new pipeline gene therapies

April 27, 2018

The complexities of gene therapies, current and future indications and budget impacts as outlined by UMass Medical School's Nicole Trask, PharmD, are featured in a Managed Healthcare Executive article. Trask presented on the topic April 24 at the Academy of Managed Care Pharmacy’s AMCP Managed Care and Specialty Pharmacy Annual Meeting in Boston.

Trask, a clinical consultant pharmacist in Clinical Pharmacy Services, presented “A Whole New World: Navigating the Gene Therapy Pipeline.” She provided a summary of what gene therapies are able to accomplish and acknowledged safety concerns around their use, including patient death.

The majority of Trask’s presentation detailed the current and future focus of gene therapy. Investigational indications for gene therapy include conditions such as ALS, HIV/AIDS, Sickle cell disease and Tay-Sachs disease. The FDA approved a treatment for biallelic RPE-65-mediated retinal dystrophy in 2017 at a cost of $425,000 per eye.

She closed the presentation with an overview of gene therapies in late-stage development including,  

  • Valoctocogene roxaparvovec for hemophilia A

  • EB-101 for recessive dystrophic epidermolysis bullosa

  • GS010 for Leber hereditary optic neuropathy

  • Elivaldogene tavalentivec for childhood cerebral adrenoleukodystrophy

  • RT-100 for heart failure

These groundbreaking new treatments come not only with a high risk, but at a high cost - some running up to $1 million per patient, which can be a challenge for payers.

Trask is a vital part of the Clinical Pharmacy Services team, which provides comprehensive prescription drug management support and consulting services that help our clients build clinically effective and cost-efficient pharmacy programs.

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