CGTs ‘a Big Deal for Pharmacy,’ So Getting Payment Right Is Crucial

By Bruce Buckley and Karen Blum

A growing number of cell and gene therapies offer significant symptom relief—and in some cases, even cures—for a wide range of often debilitating conditions. But at millions of dollars per dose, CGT treatments pose demanding procurement and reimbursement challenges for U.S. hospitals that are qualified to administer them.

Several health systems are rising to that challenge. Some are partnering with consultants with proven expertise in securing prior authorizations for CGTs. Others have developed in-house pharmacy and therapeutics (P&T) committees that specialize in managing CGTs. The common thread to these efforts is having clearly defined processes for clearing the considerable clinical, financial and operational hurdles posed by these groundbreaking therapies.

Betibeglogene autotemcel (beti-cel; Zynteglo), Bluebird Bio’s gene therapy for transfusion-dependent beta-thalassemia, was one of the proving grounds for the CGT strategy of Children’s National Hospital (CNH), in Washington, D.C. A single infusion of beti-cel 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, the treatment could be a budget buster if not handled properly.

“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.”

Duchenne muscular dystrophy offered a proving ground for the CGT programs of two hospitals. Children’s National Hospital (CNH), in Washington, D.C., was one of he nation’s first to administer delandistrogene moxeparvovec-rokl (Elevidys, Sarepta Therapeutics).

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.”

Since October 2022, Clearway Health has partnered with CNH to manage the hospital’s CGT practices. “Clearway Health serves as an educator and navigator to help insurance companies basically do a benefit–risk analysis for our patients,” said Eric Manuel Balmir, PharmD, CNH’s vice president of clinical ancillary services and chief pharmacy officer.

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 HBB gene that makes betaglobin 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. That’s based on data showing that 90.2% of patients (37/41) treated with beti-cel achieved transfusion independence, as reported in a study presented at the 2023 ASH (American Society of Hematology) Annual Meeting & Exposition (poster 1102), in San Diego.

Beta-thalassemia wasn’t CNH’s first CGT experience. They recently became one of the nation’s first hospitals to administer delandistrogene moxeparvovec-rokl (Elevidys, Sarepa Therapeutics) to a patient, a 5-year-old boy with Duchenne muscular dystrophy.

Above, a UC Davis team helps manage a Duchenne patient on the gene therapy.

Getting hospitals paid for CGTs is a critical element of success with these cutting-edge therapies, noted Shawn Francis, PharmD, Clearway Health’s director of specialty pharmacy. “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, CGT procurement often varies significantly by payor. “Some payors may have a quicker turnaround for submitting a response to a claim. 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, he noted, can lead to “barriers in product procurement and the prompt 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.”

UC Davis Health’s Approach

UC Davis Health took more of a do-it-yourself approach to its CGT strategy. Before pharmacists at the hospital implemented a streamlined process to approve and institute CGT treatments for patients, the procedure was somewhat disjointed due to product sourcing, contracting, prior authorization and the logistics of administration.

Traditionally, the P&T committee didn’t necessarily review therapies until the FDA approved them, communication among departments was limited, and the work involved “lots and lots of emails,” recalled Sarah Bajorek, PharmD, MBA, BCACP, DPLA, the Sacramento, Calif.–based health system’s assistant chief pharmacy officer of clinical ambulatory care. “This was an inefficient and unsustainable process that needed to be fixed, knowing that more of these agents were coming down the pipeline,” Dr. Bajorek said at AMCP 2024, in New Orleans. She noted that the onboarding process could take well over a year. “For most of these patients, time delays mean more disease progression and a loss of function that they won’t be able to regain.”

So, Dr. Bajorek and her colleagues formed an emerging therapy (ET) subcommittee under the P&T committee to prospectively review CGTs from financial and operational perspectives while coordinating the clinical reviews.

The ET committee established criteria to review all high-cost products, including CGTs with a cumulative annual cost for one patient of $300,000 or more. They also included any drugs with high operational or logistical needs such as special handling or monitoring. The committee’s objectives included establishing business support and a process for determining initiation, duration and setting of therapies; coordinating reimbursement; and proactively assessing the financial impact of acquiring CGTs.

Jenny Craven, PharmD, BCPS, was hired as a senior clinical pharmacist in emerging therapies to oversee the program. She facilitates all onboarding activities for CGTs, including organizing ET committee and workgroup meetings and discussions of potential concerns. She also assists with some clinical formulary reviews. When a patient is scheduled to receive a CGT, Dr. Craven coordinates the process with all stakeholders and tracks patients’ progress. She also ensures that all billing and reimbursements are completed, and arranges post-dose meetings to review any issues.”

It Takes a Team

The multidisciplinary ET committee includes an ethicist and representatives from pharmacy; executive leadership; and the finance, contracts, utilization management and information technology departments. Initially, the group met monthly to review logistics, finance and the clinical review, but the committee lacked stakeholders for robust clinical discussion, Dr. Craven said. They have evolved to a new model, in which:

• a P&T subcommittee conducts the monograph review;
• ET leaders meets weekly as needed when onboarding a medication to discuss operational logistics; and
• the ET committee (consisting of stakeholder leaders) meets monthly to review financial impact and feasibility.

Today, the group has a well-defined, eight-phase process for onboarding new patients, from determining patient eligibility through treatment administration to a post-dose debriefing. Everything is tracked using a shared computer program (Microsoft Planner) that spells out details for each step. For example, the dose administration planning section has a checklist that includes which staff are involved, if there is a preferred day of the week for administration, and if any new protocols are needed.

Improving Time to Therapy

Treatment delays are one of the main barriers to effective CGT treatment. That’s why changing the initial review process was such a critical component of UC Davis Health’s approach: The new steps they employed significantly improved time to therapy, Dr. Bajorek said. When a clinic identifies a patient eligible for a CGT, they enter the order, which kicks off a referral request. Then the authorization is started, the contracts team assesses whether a single case agreement is needed, patient financial services determines if there is a copay, etc. The process has streamlined communication, which has been simplified from 60 or more emails to just three: two for approvals and one for ordering.

So far, the committee and workgroup onboarded the gene therapy delandistrogene moxeparvovec-rokl (Elevidys, Sarepta) for Duchenne muscular dystrophy. UC Davis Health dosed the first patient in California within 43 days of FDA approval. The facility has administered gene therapy doses for Elevidys and onasemnogene abeparvovec-xioi (Zolgensma, Novartis) for 14 patients, with a turnaround time of two to four weeks per authorization.

All of these efforts “have worked really well for us,” Dr. Craven said. “They help ensure that with all of our CGTs, we have all the fine details ready to go so that we can have a successful patient and UC Davis experience overall.”

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