Cell and Gene Therapies

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.

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.

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.

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.

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.

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.

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.

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.

March 29: The FDA approved Alexion, AstraZeneca Rare Disease’s, Voydeya (danicopan) as an add-on therapy to ravulizumab (currently available as the company’s Ultomiris) or eculizumab (currently available as the company’s Soliris) for the treatment of extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria. The agent is a first-in-class factor D inhibitor, and it has breakthrough therapy designation. Initial dosing for the tablet is 150 mg three times a day, which can be increased to 200 mg three times a day. Drugs.com lists the price of 180 50 mg-100 mg tablets as more than $4,359.

April 5: The FDA expanded the patient population of AstraZeneca’s Fasenra (benralizumab) to include the add-on maintenance treatment of people aged 6 to 11 with severe asthma with an eosinophilic phenotype. The agency first approved the interleukin-5 receptor alpha-directed cytolytic monoclonal antibody on Nov. 14, 2017. Dosing for the newest indication for pediatric patients weighing less than 35 kg is 10 mg via subcutaneous injection every four weeks for the first three doses and then once every eight weeks; for those at least 35 kg, dosing is 30 mg every four weeks, followed by once every eight weeks. The list price for one 30 mg/mL solution is $5,511.41.

Rebates continue to be a huge part of the specialty pharmacy space, with 93% of respondents to a recent survey receiving them for drugs in the pharmacy benefit. Meanwhile, 44% of respondents said their firm received rebates for medical benefit drugs, representing an increase from 39% in the prior year’s survey. Those are just some of the findings in the Trends in Specialty Drug Benefits Report from Pharmaceutical Strategies Group (PSG), an EPIC company.

The 11th annual survey, which reflects 2023 information, was fielded from Sept. 18 through Oct. 13, 2023. The primary source of respondents was PSG’s proprietary database of drug benefit decision makers, and they included people from employers, health plans (or third-party administrators or insurance companies) or union/Taft-Hartley plans. There were 185 benefits leaders from plan sponsors with an estimated 86.6 million lives.