Gene therapy, which got its start in the 1960s, is finally starting to take hold.
Nicole Trask, PharmD, clinical consultant pharmacist at University of Massachusetts Pharmacy Services in Shrewsbury, MA, opened her session at the Academy of Managed Care Pharmacy Managed Care and Specialty Pharmacy (AMCP) Annual Meeting, in Boston April 24, by defining gene therapy as the administration of genetic material to modify or manipulate the expression of genes or alter the biological properties of living cells. She added that gene therapy can be used to accomplish one of the following actions:
The first clinical study on gene therapy, which included five participants, was published in 1990. In that study, a retroviral vector was used to transfer a neomycin resistance marker gene into tumor-infiltrating lymphocytes for the treatment of metastatic melanoma. In the study, lymphocytes were removed and expanded, she said.
Trask attributed gene therapy’s slow clinical progress largely to safety concerns and vector inefficiencies.
One of the challenges with gene therapy is the need for precision in delivery to the appropriate cells and/or tissues. Further, the gene must be either activated or remain inactivated. Delivery to the wrong cells and/or tissues could be catastrophic.
That’s why focusing on safety is wise. In 1999, one patient died after experiencing an adenovirus gene transfer in ornithine transcarbamylase deficiency, a rare genetic disorder characterized by a complete or partial lack of the enzyme ornithine. And in 2000, two patients with SCID-XI, an inherited disease impacting the immune system, were successfully treated using the MoMLV vector; these were two of 11 patients involved in the clinical trial, where three patients developed leukemia as a result of the gene transfer procedure, according to Trask.
Gene therapy current focus
Investigational indications for gene therapy include conditions such as:
FDA approved in 2017 Luxturna (voretigene neparvovec-rzyl) for the treatment of confirmed biallelic RPE65-mediated retinal dystrophy. The treatment, which is associated with significant improvements in functional vision, costs $425,000 per eye, or $850,000 for most patients, she added.
Trask also highlighted gene therapies that are in late-stage development, including:
While expressing enthusiasm for these therapies, Trask also noted their challenges for payers, including their extremely high costs-as much as $700,000 to $1,000,000 per patient-and the reality that some gene therapies may be associated with safety risks.
David Calabrese of OptumRx Talks New Role, Market Insulin Prices and Other Topics 'On His Mind'
April 13th 2023In this month’s episode of the "What's On Your Mind podcast," Peter Wehrwein, managing editor of MHE connects with the now Chief Clinical Officer of OptumRx Integrated Pharmacies, David Calabrese. In this conversation, David touches on his transition in January as OptumRx’s former chief pharmacy officer and market president of health plans and PBMs to his new role as Chief Clinical Officer where he now focuses more on things such as specialty pharmacy to home delivery — with an overall goal of creating whole-patient care. Throughout the conversation, Calabrese also touched on the market’s hot topic of insulin prices and behavioral health services within the OptumRx community, among other topics.
Listen
Upended: Can PBM Transparency Succeed?
March 6th 2024Simmering tensions in the pharmacy benefit management (PBM) industry have turned into fault lines. The PBMs challenging the "big three" have formed a trade association. Purchaser coalitions want change. The head of the industry's trade group says inherent marketplace friction has spilled over into political friction.
Read More
Briana Contreras, editor of Managed Healthcare Executive, spoke with Nancy Lurker, CEO and president of EyePoint Pharmaceuticals. Nancy shared a bit about EyePoint and how the organization’s innovative therapies are addressing patient needs through eye care, and most importantly, she addressed C-Suite positions like the CEO role. Nancy shared advice for those seeking to reach the CEO level, especially toward women in healthcare and other roles, and what it takes to run a biopharma company.
Listen
The deliberate disconnection of Change Healthcare to ring fence a cyberattack entered its seventh day today. Prescribers are finding ways to get pharmacy claims processed, and UnitedHealth Group says disruption to the dispensing of prescriptions has been minimal. But independent pharmacies want more information and protection from financial consequences from pharmacy benefit managers.
Read More