A single infusion of betibeglogene autotemcel (beti-cel; Zynteglo), Bluebird Bio’s gene therapy for transfusion-dependent beta-thalassemia, frees most people with the severe inherited blood disorder from the need for regular long-term blood transfusions. At $2.8 million per dose, however, beti-cel—similarly to a growing number of high-cost cell and gene therapies—poses a demanding procurement challenge for U.S. hospitals that are qualified to administer CGTs.
“It’s not your typical prior authorization process,” said Nicole Faucher, MS, the president of Clearway Health, a specialty pharmacy accelerator that partners with health systems to build and strengthen specialty pharmacy programs. “Very few, if any, payors have policies or procedures specifically outlining coverage for cell and gene therapy.”
This means, Ms. Faucher said, that in almost every instance, a single-case agreement is required “that really talks about efficacy and durability of the treatment.” The agreement also needs to outline what the approved payment covers, including both the therapy and hospital stay because, she added, “there’s a lot of follow-up required after this therapy is administered.”
Children’s National Hospital Partnership
Since October 2022, Clearway Health has partnered with Children’s National Hospital (CNH), in Washington, D.C., to manage the specialty pharmacy space, including the hospital’s CGT practices. “We needed an expert who knew how to navigate the specialty pharmacy payment process,” said Eric Manuel Balmir, PharmD, CNH’s vice president of clinical ancillary services and chief pharmacy officer. “Clearway Health serves as an educator and navigator to help insurance companies basically do a benefit–risk analysis for our patients.”
Children’s National Hospital, in Washington, D.C., was the nation’s first hospital to administer Elevidys to a 5-year-old boy with Duchenne muscular dystrophy.
In January, CNH joined the group of qualified U.S. treatment centers that had already administered beti-cel for transfusion-dependent beta-thalassemia. The patient was a 6-year-old girl who lacked the functional beta-globin gene needed to produce red blood cells and avoid life-threatening anemia.
The successful infusion of beti-cel, manufactured using the girl’s own blood stem cells, greatly increased her odds for lifelong freedom from blood transfusions and frequent chelation therapy to reduce iron buildup resulting from the transfusions. (A Bluebird Bio graphic traces the lengthy beti-cel treatment journey from pretreatment consultation with a hematologist to the actual infusion of altered blood cells to the 15-plus years of post-procedure monitoring; bit.ly/3ViOYZX.) (For efficacy data, see box.)
Help With Duchenne CGT
The treatment of a transfusion-dependent beta-thalassemia patient wasn’t CNH’s first incursion into the CGT space. CNH recently became the nation’s first hospital to administer delandistrogene moxeparvovec-rokl (Elevidys, Sarepta Therapeutics) to a patient, a 5-year-old boy with Duchenne muscular dystrophy (bit.ly/3TAnTQE).
Dr. Balmir told Specialty Pharmacy Continuum that CNH also was set to receive one of the newly approved gene therapies for sickle cell disease: exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals/CRISPR Therapeutics) or lovotibeglogene autotemcel (Lyfgenia, Bluebird Bio).
Key Beti-cel Efficacy Data
- 90.2% of patients (37/41) achieved transfusion independence (TI).a
- TI was maintained through last follow-up (up to six years) across ages and genotypes.
- 68.2% of patients (15/22)b achieved TI, with 14 sustaining TI through the last follow-up (up to nine years).
- Clinically meaningful improvements in quality-of-life assessments up to 36 months occurred in both adults and children.c
a Phase 3 studies.
b Phase 1/2 studies.
c Phase 1/2 and 3 studies.
Source: 2023 ASH (American Society of Hematology) Annual Meeting & Exposition; poster 1102 (bit.ly/3TAnTQE)
Cell and gene therapies aren’t the only operational areas for which CNH seeks guidance. Shawn Francis, PharmD, Clearway Health’s director of specialty pharmacy, said five Clearway Health liaisons are embedded in some of the hospital’s top specialty clinics—including gastroenterology, rheumatology and endocrinology/diabetes—with a focus on outpatient services. Clearway Health also is building a specialty pharmacy in Maryland for CNH, which is expected to open in September.
‘A Longitudinal Undertaking’
But it is the CGT arena where some of the largest challenges lie, Dr. Francis stressed. “This is not just a one-time process where we pick the drug off the shelf and then we bill for it,” he said. “It’s a longitudinal undertaking that involves inpatient stays, outpatient billing, charges and coding.” And from a revenue cycle standpoint, he added, the complexities “can prolong the entire process.”
Moreover, Dr. Francis said, the whole procurement process varies by payor. “Some may have a quicker turnaround for submitting a response. They may implement certain prior authorization criteria because they are incentivized to control costs. They may require specific laboratory monitoring and genetic testing for diagnosing the disease.” All these requirements, Dr. Francis noted, can lead to “barriers in product procurement and the delivery of care to patients.”
With dozens of CGTs in the pipeline, the need for specialty pharmacy experts is likely to increase exponentially. “This market has exploded,” Dr. Balmir said. “We’ve gone from treating symptoms to actually prolonging life. Now we’re at this curative phase, which is a big deal for pharmacy.”
