BOSTON, MA - Clearway Health, a specialty pharmacy accelerator partnering with hospitals and health systems, has pioneered a model to support hospitals in enabling access to novel gene therapy treatments. The model was used to execute and accelerate payor, legal and procurement processes for Children’s National Hospital in Washington, D.C., to treat their first pediatric patient with ZYNTEGLO®, a one-time, potentially curative breakthrough gene therapy treatment for transfusion-dependent beta-thalassemia, a rare blood disorder requiring regular red blood cell transfusions.
“Novel gene therapies represent the advancement of emerging therapies addressing the possibility of untethering patients from a lifetime of burdensome, costly, chronic care with a single treatment. While very promising, the full clinical impact of these treatments is not yet clear, driving questions on coverage, cost efficiency and delivery,” said Shawn Francis, PharmD, director of specialty pharmacy at Clearway Health.
By 2030, 54 approved gene and cell therapies are expected in the FDA pipeline, making it essential for healthcare institutions to adequately prepare to deliver gene therapy.
Navigating a potentially multimillion-dollar drug therapy involves a multidisciplinary approach that can be extremely time intensive and costly for health systems, involving an understanding of payor dynamics, prior authorizations, geographic complexities, patient financial access, assessment of proper staff training, legal and risk management, orchestration of stakeholders and determining a procurement pathway. Clearway Health’s innovative framework supports health systems in navigating these dynamics, as well as the various intricacies that make it possible to absorb the high costs of a one-time, lump sum payment while protecting the financial integrity of the institution.
“Many health systems are not set up to coordinate the procurement of a novel gene therapy. There are an incredible amount of steps and considerations involved in the process,” said Eric Manuel Balmir, BS, MS, PharmD, CIM, vice president of clinical ancillary services and chief pharmacy officer of Children’s National Hospital. “Clearway Health’s framework allowed us to quickly and efficiently address these concerns, positioning our hospital to provide the latest pharmaceutical advancement.”