As gene therapies continue to grow in popularity for the treatment of rare diseases, stakeholders are navigating the benefits and challenges associated with these expensive treatments.
In a recent presentation at AMCP Nexus 2020, John Petrich, RPh, MS, manager, Investigational Drug Service, Cleveland Clinic, Edmund Pezalla, MD, MPH, CEO, Enlightenment Bioconsult, LLC, and Mari-Pat Pusey, MBA, senior product director, OptumRx, discussed the value of gene therapies regarding clinical end points, comparative effectiveness, financial implications to payers, and utilization rates.
“Gene therapy aims to restore healthy physiologic function or suppress aberrant activity via gene augmentation, gene suppression, or genome editing,” explained Mr Petrich during the first portion of the session. He continued to outline the principles of gene therapy, including the measurement of clinical outcomes for which he used chimeric antigen receptor T-cell therapy (CAR-T) and factor production therapies for hemophilia as examples.
“Cancer represents a key area in gene therapy, with CAR-T therapies approved and in development,” said Mr Petrich. CAR-T remains one of the most common gene therapies but also one of the most expensive. “Rare, monogenic diseases are another notable area for gene therapy, with approved treatments for LCA and SMA and agents in late-stage development for blood disorders.”
Dr Pezalla continued the presentation, focusing on utilizing the potentially curative benefits of gene therapies while attempting to remain cost-conscious.
He explained that specialty growth continues to outpace traditional pharmaceuticals and in turn, poses a significant impact on costs. Not only do these treatments address small patient populations, but there are regulatory and developmental challenges which create lengthy and high-cost approval processes.
Dr Pezalla emphasized the value of innovation but noted that in the end, “Value in health care innovation lies in the result of the innovation rather than the innovation itself.” Value is created through:
- Better patient outcomes;
- Health care system efficiencies;
- Improved societal outcomes; and
- Patients living longer and better lives.
Another challenge is assessing value. Dr Pezalla asked and polled how value should be measured with the majority of the attendees reporting that qualify adjusted life-years, or QALYs, are the most relevant measure—above treatment costs, disability adjusted life-years, and overall improvements measured by patient-reported outcomes.
The MIT FoCUS project is an example of a program dedicated to addressing the need for new, innovative financing and reimbursement models for specialty treatments, like gene therapy. Dr Pezalla explained that its focus is on “creating precision financing solutions for durable/potentially curative therapies with large, upfront costs whose benefits accrue over time” and not “assessing or setting value, or negotiating specific prices for specific products.” However, the program helps ease the minds of stakeholders with financial, effectiveness, regulatory, operational, and access concerns.
Ms Pusey delved into the intricate web of challenges and benefits of gene therapies from a payer perspective, noting that they “vary dependent upon size, financial strength, and ability to absorb risk at multiple levels.”
Examples of strategies being tested in the marketplace today are alternative payment models (risk pools) and outcomes-based agreements. Ms Pusey explained that new types of administrator entities are likely to emerge, that could aid in negotiating pricing on behalf of payer coalitions or provide
data and analytics infrastructure to measure outcomes. These specialized positions would allow for more effective and efficient care, explained
“The eventual choice of innovative access scheme will ultimately depend on individual health plan environment and characteristics,” said Ms Pusey.
Dr Pezalla concluded: “The juxtaposed needs and concerns of payers, providers, and patients must all be carefully weighed when evaluating the role and value of gene therapy in future care interventions.” —Edan Stanley