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Genetics and genomics are starting to have an impact on cardiovascular care. Here’s what payers should consider when deciding whether to cover a gene expression test.

Five questions for payers to ask when deciding coverage of genomic tests

Like many novel therapies, gene therapies are not without challenges that can be solved through a combination of policy and market changes. In order to improve patient access to these life-saving therapies new payment solutions, education efforts around the availability of these treatments and their benefits and risks, and the use of appropriate value assessment methods are all needed, according to a white paper from the National Pharmaceutical Council (NPC).

Journal of Comparative Effectiveness Research

There are a total of 14 gene therapies are now available for 

 and some cancers, and these potentially curative therapies often only require a single treatment, which can be complicated to deliver. The finances of paying for costly gene therapies is a challenge in the U.S., as well. Lenmeldy (atidarsagene autotemcel) is the 

, with a price tag of $4.25 million. The therapy is approved to treat metachromatic leukodystrophy, a rare disorder that damages the nervous system. The 

 starts around 2 years of age or younger and is the most common form. It progresses fast and the mortality rate is high.

“The unique characteristics of gene therapy treatments, in conjunction with uncertainties about long-term durability and future market competition, necessitate the development of innovative models to assess the value of these treatments, promote appropriate payment and advance meaningful patient access while maintaining incentives for future research and development,” the authors wrote.

In addition to the high cost of these therapies, they are faced with challenges related to production and distribution, as well as the logistical and treatment administration obstacles that can create barriers to accessing gene therapies. A growing number of gene therapies require extracting cells from the patient, isolating and genetically modifying the cells and then re-administering them to the patient. This is a complex and time-intensive processes, which limits the ability to scale production of these therapies.

For patients and caregivers, there is the added burden of special diagnostic testing and treatment administration, which can require that they travel long distances from home, sometimes necessitating staying in another location for a significant amount of time. Only specific providers and locations that are qualified for handling gene therapies can administer them.

Coverage by health plans varies greatly for gene therapies, which “holds important implications for equitable patient access to life-changing treatments,” the NPC authors noted. Medicaid’s coverage is more restrictive than the FDA approved label more than two-thirds (68.3%) of the time, and commercial plans have more restrictive policies more than half (53.5%) of the time.

“As payers often cite uncertainty about long-term durability and patient churn to justify coverage exclusions and strict utilization management, policy and market solutions aimed at improving patient access should consider opportunities to address these concerns,” they wrote.

Overall, health policy proposals and market-based solutions to improve access to gene therapies need to consider all of these challenges that affect patients, manufacturers, providers, payers and more.

Innovative payment models, such as outcome-based payments and risk pooling can improve patient access to treatments while alleviating payer concerns about the efficacy of an expensive, one-time treatment and patient churn in plans. In addition, these value-based payment arrangements should not be applied to all treatments because they may not be the right fit for all gene therapies, the authors noted.

“To ensure that their potential is realized, we must incentivize the development of gene therapies for those most in need,” they concluded. “Several opportunities exist to improve patient access to gene therapy treatments, including relying on appropriate value assessment methods; advancing innovative payment solutions; removing inappropriate access barriers imposed by health; and improving education and awareness of treatment availability, benefits and risks.”

Gene therapies have a lot of potential to improve health outcomes for patients with life-threatening diseases but face a number of barriers that restrict access.

Clinical

Potential Solutions to Remove Barriers to Gene Therapies

Jazz Pharmaceuticals announced the recent FDA accelerated approval of Ziihera (zanidatamab-hrii) for adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer. This approval makes Ziihera the first and only dual HER2-targeted bispecific antibody treatment for this indication. An investor webcast will be held on Dec. 11 at 4:30 p.m. EST to discuss further details, according to a 

Potential Ziihera patients may have their cancer identified using the PATHWAY HER2 (4B5) test, 

 under an FDA label expansion. This test, approved on Nov. 25, is the first and only FDA-approved companion diagnostic for HER2-positive biliary tract cancer. 

Ziihera is produced in Chinese hamster ovary cells via recombinant DNA technology, official prescribing information 

. These cells are chosen because they can produce complex proteins similar to human cells. More than 70% of all recombinant antibodies have been produced from Chinese hamster ovaries since 2016, a 

Biliary tract cancer affects bile ducts within the gallbladder, liver and small intestine. The cancer becomes HER2-positive when the tumor cells overexpress the HER2 protein, which causes the tumors to grow rapidly. When it becomes metastatic, the five-year survival rate is less than 5%.

The recommended dose of Ziihera is 20 mg/kg given as an intravenous infusion every two weeks. It’s also recommended that patients be given acetaminophen, an antihistamine and a corticosteroid 30 to 60 minutes before treatment to avoid infusion-related reactions. Ziihera comes as a powder that must be reconstituted and diluted with sterile water before use.

This approval is based on the results of the phase 2b HERIZON-BTC-01 trial in which 52% of the 87 patients given Ziihera saw improvements. The median duration of response was 14.9 months, determined by independent central review. Results were published in 

Ziihera safety was demonstrated in the HERIZON-BTC-01 and ZWI-ZW25-101 trials, which enrolled 109 patients with biliary tract cancer and 124 patients with other cancers. The most common adverse reactions in HERIZON-BTC-01 were diarrhea (50%), abdominal pain (29%) and fatigue (24%).

"As a clinical investigator and medical oncologist focused on advancing the care of patients with biliary tract and liver cancers, I have experienced firsthand the significant unmet need for effective therapies for patients with these diseases," James Harding, M.D., associate attending, Gastrointestinal Oncology and Early Drug Development Services, at Memorial Sloan Kettering Cancer Center said in the news release. "[Ziihera] has demonstrated antitumor activity and is now a new option for patients with HER2-positive biliary tract cancer.”

A phase 3 HERIZON-BTC-302 trial investigating the effectiveness of Ziihera in combination with standard-of-care therapy is ongoing. Ziihera effectiveness is also being testing on metastatic breast cancer tumors and gastroesophageal adenocarcinomas.

Produced in Chinese hamster ovary cells, Ziihera (zanidatamab-hrii) is the first HER2-targeted bispecific antibody treatment for patients with previously treated, unresectable or metastatic biliary tract cancer.

Formulary Watch

FDA Approves Biliary Tract Cancer Antibody Treatment 

In a recent conversation with Greg Baker, CEO of AffirmedRx, the PBM expert outlined the company’s distinct approach in the PBM industry with MHE editors.

Baker, a member of Managed Healthcare Executive's Editorial Advisory Board, expressed that AffirmedRx prioritizes the public good over profits, aiming to eliminate conflicts of interest and enhance transparency.

The company charges a flat per-prescription administrative fee, which ensures financial decisions are patient-focused rather than driven by drug costs. AffirmedRx also provides clients with full visibility into claims and manufacturer revenues, fostering trust and accountability.

“We're to the point where we could credential our clients directly into the adjudication system... allow them to see all the claims, what's getting paid, then they know exactly what they should be paying,” he said. “Everything is in one place, so it makes it very transparent.”

By aligning its goals with clients and patients, Baker shared that AffirmedRx differentiates itself from traditional PBMs and those backed by private equity.

For example, regulatory changes, such as those under the Consolidated Appropriations Act, reinforce the company’s mission of ethical and transparent practices, setting a standard for the evolving PBM landscape.

In a recent conversation with Greg Baker, CEO of AffirmedRx, the PBM professional outlined the company’s distinct approach in the PBM industry with MHE editors.

A Transparent, Patient-First Approach Amid Ongoing PBM Industry Challenges

Binghampton University in New York state will join Mayo Clinic and at least 15 other investigators in developing an implant aimed to treat inflammatory diseases. The project, called Engage Assess SecretE (EASE): A Platform for Treating Chronic Inflammation, is meant to act as a “living pharmacy” triggering cells to produce antibodies that could target inflammatory conditions.

, Ph.D., biochemical engineer at Mayo Clinic, is funded by a $42.8 million grant from the 

Advanced Research Projects Agency for Health (ARPA-H)

, an agency nestled under the Department of Health and Human Services and the National Institutes of Health. ARPA-H funds and supports biomedical and health research with the potential for developing high-impact and transformative treatments.

Mayo Clinic is the primary research site for the EASE project with collaborators from several other sites, including Binghampton University, the University of Minnesota, Case Western Reserve University, the University of Texas, the University of California Davis,and biotechnology companies EnLiSense and Sersense Inc.

“I am very excited that this project has high societal impact and patient relevance. It represents a prime example of convergence science, where clinicians, biologists and engineers come together to produce a sophisticated solution to improve patient care,” Revzin said in a 

, Ph.D., will create electrodes to generate oxygen that will feed the cells that produce the pharmaceutical catalysts.

“Our research group aims to electrochemically generate oxygen to ensure the cells survive during implantation," said Koh. "I'm responsible for developing the 'cafeteria' in the 'factory,' ensuring that they 'eat' properly,” she continued.

Currently, the EASE platform is being developed for the treatment of inflammatory bowel disease, including Crohn’s disease and ulcerative colitis. The researchers hope to eventually use the device to treat autoimmune disorders, such as rheumatoid arthritis and psoriasis.

“This method is intended to treat bowel disease, but the whole platform is structured so that if the antibodies produced by the cells are altered, this might be used as a transformative treatment for other targeted diseases,” Koh commented.

Binghamton University, Mayo Clinic and Others Snare NIH Funding for IBD Research 

Seven years ago, when the FDA approved the first-ever chimeric antigen receptor (CAR)-T cell therapy, Novartis’ then-CEO Joseph Jimenez, MBA, said Kymriah (tisagenlecleucel) represented a “paradigm-changing” therapy.

In the years since then, the FDA has approved five more CAR-T therapies, and it is hard to disagree with Jiminez’s assessment. Yet the mainstreaming of CAR-T therapies has changed much more than solely which therapy a physician prescribes. It has also created a unique patient journey, set off a wave of new scientific questions and heightened concerns about the rising cost of care.

Umar Farooq, M.D., a hematologist-oncologist at the University of Iowa (UI) Health Care, has been treating patients with CAR-T cells since 2015, when he began collaborating on a CAR-T clinical trial. His health system now treats between 70 and 80 patients each year with CAR-T cell therapies. “The number of CAR-T cells we’ve been doing every year just continues to increase,” he says.

That’s great news in Farooq’s view because, he says, the therapy has led to dramatic improvements in many of his patients. “With what I’ve seen in the past 10 years, and how drastically it has improved patient outcomes, I’m … very optimistic for the next 10 years.”

As of September 2024, six CAR-T cell therapies covering 16 indications have been approved in the United States, according to the Leukemia & Lymphoma Society. All the therapies have been approved for the treatment of blood cancers, including non-Hodgkin lymphoma, acute lymphoblastic leukemia, multiple myeloma and, as of March 2024, chronic lymphocytic leukemia and small lymphocytic leukemia.

Bethany Lilly, J.D., executive director of public policy at the Leukemia & Lymphoma Society, says the expanding number of indications approved for CAR-T therapy has also increased interest.

“Awareness is growing, so more patients call knowing that they are eligible for the treatment, but [they] often still have questions,” she says.

Farooq says he recommends patients go to an academic medical center once their disease has progressed past one line of treatment.

“If we see you too late and you’re already too frail and the disease has affected your organ function, it would be harder to do,” he says.

Referral speed is important because CAR-T therapy is not a fast process. Farooq says by the time he typically sees a patient, the patient already has insurance authorization for the initial consultation. He and his colleagues will then run tests to ensure the patient’s heart, lungs and other major organs are sufficiently healthy for the therapy. If so, Farooq and colleagues will submit insurance paperwork to get approval for the procedure. Next, they reach out to the cell therapy lab to ensure they are ready to collect the patient’s white blood cells and to the drug manufacturer’s lab to ensure they are ready to receive the cells. It takes a minimum of two weeks from the first patient visit to the time the patient gets the go-ahead, he says.

Then comes the manufacturing itself, which generally takes at least three to four weeks. “Once we know for sure that the cells have been manufactured, meet the quality standards and have arrived at our hospital, it’s only then that we admit the patient to start the low-dose chemotherapy to suppress the immune system,” he says.

In the best-case scenarios, the entire process can take about six weeks in an optimal case, he says, and then the hospitalization is not over. Patients require daily monitoring for adverse events for two to three weeks, usually as an inpatient.

The frequent checkups are necessary because of the significant risk of toxicity from CAR-T therapy, including cytokine release syndrome (CRS) and neurotoxicity.

“And if the patient is … much farther away from the treating center, it would be a very complicated case for a local health facility to deal with,” he says.

Farooq says both CRS and confusion are common, though most of the time the adverse events are not severe.

He sees patients from as far away as Des Moines, which is a little less than two hours from Iowa City, where UI Health Care is based. He says he typically only sees out-of-state patients if it is for a clinical trial.

Still, all that time in the hospital means patients who live far away from their treatment center must plan for things like travel expenses or hotel rooms to undergo the necessary treatment and monitoring. That can create a barrier for many patients, Lilly says.

“It can be thousands of dollars, and not every patient can afford it,” she says. “This is especially the case for low-income patients, who also disproportionately live in rural areas and/or are patients of color.”

Of course, those travel costs are typically just the beginning of the extensive fees associated with CAR-T therapy. Most CAR-T treatments run between $300,000 and $400,000, and sometimes upwards of a half-million dollars, Farooq says. That is why one of the first steps his health system takes is to communicate with a patient’s insurer to verify the procedure will be fully paid for and that the patient will not face financial ruin.

But there are some concerns: high costs, extended hospitalizations 
and secondary cancers.

David Levison

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