Cell and Gene Therapies

Report Reveals Plan Sponsor Uncertainty Around CGTs’ Impact

With more than 4,000 cell and gene therapies (CGTs) in development, plan sponsors should not be ignoring or underestimating the potential impact of these agents, not only on their beneficiaries’ health but also on their own bottom lines. A recent report by Pharmaceutical Strategies Group (PSG), an EPIC company, found that some improvement is needed in this area.

Sponsored by Prescryptive Health and published June 11, “2024 Trends in Drug Benefit Design Report” examines trends among traditional — or nonspecialty — drug benefits, as well as gene therapies.

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News Briefs: BCS Financial Unveils Gene Therapy Rider

BCS Financial Corp. unveiled the Gene Therapy Rider on its EssentialCare Critical Illness insurance policy, the company said on June 18. The offering by the insurance and financial services company, which is owned by all 34 Blues plans, can pay the entire policy face value amount when a beneficiary is diagnosed with a covered disease and chooses to undergo treatment with an FDA-approved gene therapy. “The payments, which are based on the policy's face value, are made directly to the insured to use how they see fit,” says the company.

Prime Therapeutics LLC and Magellan Rx Management launched the technology-enabled specialty pharmacy solution Pharmacy Match, the companies said June 19. The offering, which is powered by Free Market Health’s cloud-based technology platform, is designed to drive competition by comparing prices across the market for specialty drugs and identifying the specialty pharmacy “best suited to meet their needs.” It also tracks each claim through fulfillment.

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Midsize Plans Struggle to Pay for Cell, Gene Therapies, Execs Say

It's no secret that health plans are concerned that they may not be able to afford gene and cell therapies with new or anticipated approvals. With expensive launches on the horizon, plans are searching for payment models that can grant patients access to lifesaving or life-changing therapies without blowing budgets.

Those concerns are particularly acute for smaller, regional health insurers and employer plan sponsors. Those payers may have tight balance sheets and lack the pricing advantages that national insurers can leverage in negotiations with manufacturers. Midsize plan executives say their organizations are figuring out what to do as they go along.

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New FDA Approvals: FDA Expanded Mircera’s Patient Population

April 30: The FDA expanded the patient population of CSL Vifor’s Mircera (methoxy polyethylene glycol-epoetin beta) to include the treatment of anemia associated with chronic kidney disease in pediatric patients 3 months old to 17 years old on dialysis and not on dialysis who are converting from another erythropoiesis-stimulating agent after their hemoglobin level was stabilized with an ESA. The agency also approved a subcutaneous route of administration for pediatric patients. The FDA first approved the long-acting ESA on Nov. 14, 2007. Dosing for the newest use is once every four weeks based on total weekly epoetin alfa or darbepoetin alfa dose at the time of conversion. The agent is available in both intravenous and subcutaneous formulations, and patients younger than 6 years old should maintain the same route of administration as the previous ESA. Drugs.com lists the price of one 75 mcg/0.3 mL injectable solution as more than $237.

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How Will Beqvez Fare in Challenging Market for Cell and Gene Therapies?

The FDA recently approved the second gene therapy for hemophilia B, Pfizer Inc.’s Beqvez (fidanacogene elaparvovec-dzkt). While the agent offers an additional treatment option with the potential for freedom from regular infusions of factor therapy, its price — which is equal to that of its competitor — may be too high for many payers, according to a Zitter Insights survey. Industry experts say that it may suffer from some of the challenges other cell and gene therapies have faced in gaining a foothold in the U.S. market.

On April 25, the FDA approved Beqvez for the treatment of adults with moderate to severe hemophilia B who use factor IX (FIX) prophylaxis therapy; have current or historical life-threatening hemorrhage; or have repeated, serious spontaneous bleeding episodes and do not have neutralizing antibodies to adeno-associated virus (AAV) serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. The manufacturer launched a warranty program for the intravenous infusion based on durability of patient response to treatment.

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Cell and Gene Therapies Pose Multiple Challenges but No Easy Solutions

While health care payers are facing a variety of issues, paying for multimillion-dollar cell and gene therapies (CGTs) is one of the most pressing, as evidenced by sessions at two recent AHIP conferences. Multiple speakers discussed various approaches to the agents, as well as challenges payers need to tackle, but all acknowledged that a truly successful model has yet to be implemented.

Many CGTs are in the pipeline, impacting potentially millions of patients and prompting many questions around affordability and accessibility, stated Sean Dickson, senior vice president of pharmaceutical policy at AHIP, during a session in Baltimore titled “Cell and Gene Therapies: Regulatory Updates and Coverage Policies.” “Oncology is where it will get really interesting,” and these agents will have the greatest impact on Medicare payers.

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FDA Approves Another Hemophilia Gene Therapy, but Will Price Prove to Be Too High?

The FDA recently approved the second gene therapy for hemophilia B, Pfizer Inc.’s Beqvez (fidanacogene elaparvovec-dzkt). But while the manufacturer priced the agent at parity to the other treatment, that price may still be too high for many payers, according to a Zitter Insights survey.

On April 25, the FDA approved Beqvez for the treatment of adults with moderate to severe hemophilia B who use factor IX prophylaxis therapy; have current or historical life-threatening hemorrhage; or have repeated, serious spontaneous bleeding episodes and do not have neutralizing antibodies to adeno-associated virus (AAV) serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. The manufacturer launched a warranty program for the intravenous infusion based on durability of patient response to treatment.

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Walgreens Will Launch New Specialty Pharmacy With ‘Significant’ Cell and Gene Therapy Offering

On April 24, Walgreens Boots Alliance Inc.’s Walgreens revealed that it will launch Walgreens Specialty Pharmacy on Aug. 1. The company is touting the new division, which will include AllianceRx Walgreens Pharmacy and four central specialty pharmacies, as being able to swiftly serve patients without being invested in a PBM. And that could mean enhanced opportunities for contracting with payers and manufacturers, according to one expert.

The new unit will also feature nearly 300 community-based specialty pharmacies across the U.S. and a new 18,000-foot Gene & Cell Services Pharmacy and Innovation Center in Pittsburgh. The division will have more than 1,500 specialty-trained pharmacists, 5,000 patient advocacy support staffers and 10 Specialty360 teams. It also has access to more than 240 limited distribution drugs, including 40 narrow networks and 12 exclusive limited distribution agents.

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Specialty Drug Benefits Survey Spotlights Gene Therapy, Biosimilar Strategies

While over 90% of health plans receive specialty medication rebates under the pharmacy benefit, the receipt of medical-benefit rebates has risen in the past one to two years, according to the 2024 Trends in Specialty Drug Benefits Report, published by Pharmaceutical Strategies Group, an EPIC company. The report also covered topics like the management strategies of Humira biosimilars and the financial risk associated with cell and gene therapy.

The report is based on responses from 185 benefits leaders from employers, unions/Taft-Hartley plans and health plans representing plan sponsors of approximately 86.6 million covered lives, conducted from Sept. 18, 2023, through Oct. 13, 2023.

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Specialty Drug Benefits Survey Spotlights Gene Therapy, Biosimilar Strategies

While over 90% of health plans receive specialty medication rebates under the pharmacy benefit, the receipt of medical-benefit rebates has risen in the past one to two years, according to the 2024 Trends in Specialty Drug Benefits Report, published by Pharmaceutical Strategies Group, an EPIC company. The report also covered topics like the management strategies of Humira biosimilars and the financial risk associated with cell and gene therapy.

The report is based on responses from 185 benefits leaders from employers, unions/Taft-Hartley plans and health plans representing plan sponsors of approximately 86.6 million covered lives, conducted from Sept. 18, 2023, through Oct. 13, 2023.

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