Cell and Gene Therapies

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.

The pharmaceutical industry continues to bring innovative therapies to market, but payers are continuing to grapple with how they can manage their financial impact. And while the Inflation Reduction Act (IRA) may bring some relief to Medicare beneficiaries in the form of lower drug prices and out-of-pocket costs, uncertainties remain about how that legislation could impact commercial payers, according to speakers at AHIP’s 2024 Medicare, Medicaid, Duals & Commercial Markets Forum, held March 12 through 14 in Baltimore.

During a March 13 session, titled “Trends in Prescription Drug Affordability, Innovation and Access,” a large part of the panel discussion focused on the IRA, which put “a lot of pressure on all parts of our health care system, but pharma got the brunt of it,” observed Rena Conti, Ph.D., associate professor at Boston University’s Questrom School of Business. While much of the focus from pharma and politicians has been on the drug price negotiation process, the Medicare Part D redesign “is actually a really big thing, because that’s what’s going to lower prices for seniors,” she asserted. “And it also will put a fair amount of pressure back on plans to manage this benefit.”

One week before the Institute for Clinical and Economic Review released a white paper laying out ways to manage ultra-high-cost gene therapies, ICER President and CEO Sarah Emond shared frank views about the consequences that result when medications are priced far higher than they need to be.

“We’re making tradeoff decisions all the time,” Emond said during an April 16 presentation at the Academy of Managed Care Pharmacy annual conference in New Orleans. “Anyone who thinks that we are not rationing care in the United States is not paying attention. It has a lot to do with how good your health insurance is and how much money you have.”

March 13: The FDA approved BeiGene, Ltd.’s Tevimbra (tislelizumab-jsgr) for the treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma after prior systemic chemotherapy that did not include a programmed death receptor-1 (PD-1)/programmed death-ligand 1 (PD-L1) inhibitor. Recommended dosing for the PD-1 inhibitor is 200 mg via intravenous infusion once every three weeks. The initial dose is administered over 60 minutes; if it is tolerated, subsequent doses can be administered over 30 minutes. The drug will be available in the second half of 2024.

March 13: The FDA gave an additional indication to Mirum Pharmaceuticals, Inc.’s Livmarli (maralixibat) for the treatment of cholestatic pruritus in people at least 5 years old with progressive familial intrahepatic cholestasis (PFIC). The agency first approved the ileal bile acid transporter inhibitor on Sept. 29, 2021. The new indication has orphan drug designation, as well as breakthrough therapy designation for PFIC type 2. The starting dose for the newest use of the oral solution is 285 mcg/kg once daily in the morning, increasing to 285 mcg/kg twice daily, then 428 mcg/kg twice daily and then 570 mcg/kg twice daily. Drugs.com lists the price of 9.5 mg/mL for 30 milliliters as more than $56,239.

The FDA recently approved a first-in-class agent to treat a particularly deadly cancer. The therapy, Iovance Biotherapeutics, Inc.’s Amtagvi (lifileucel), signifies a promising new development in the treatment of solid tumors, which represent about 90% of all cancers in the U.S. While the drug comes with a price tag of more than $500,000, as well as additional costs, payers’ experience with chimeric antigen receptor T cells (CAR-T) therapies should be helpful in their coverage of the new agent, say industry sources.

On Feb. 16, the FDA gave accelerated approval to Amtagvi for the treatment of adults with unresectable or metastatic melanoma previously treated with a programmed death receptor-1 (PD-1) inhibitor and, if BRAF V600 positive, a BRAF inhibitor with or without a MEK inhibitor. The agency gave the therapy orphan drug, regenerative medicine advanced therapy, fast track and priority review designations.

Artificial intelligence (AI), drug shortages and cell and gene therapies are just a few of the areas on which the FDA is focusing during a major reorganization. Those were some of the topics that FDA Commissioner Robert Califf, M.D., discussed with Mike Tuffin, president and CEO of AHIP, at the insurer trade group’s 2024 Medicare, Medicaid, Duals & Commercial Markets Forum, held March 12 through 14 in Baltimore.

When asked his vision for the FDA in 2024 and beyond, Califf responded that 2024 priorities were different because it’s an election year, and the agency’s priorities are “tied to the administration that’s in place.”

Amazon.com Inc’s pharmacy division will be the exclusive home delivery partner for Eli Lilly & Co.’s direct-to-consumer business, Lilly Direct, which will distribute GLP-1s, among other drugs. In addition, the e-commerce giant now offers same-day delivery of many medications in New York and Los Angeles; it already offered same-day delivery in Austin, Indianapolis, Miami, Phoenix and Seattle. Analysts were positive about the LillyDirect deal: GlobalData’s Costanza Alciati wrote on March 14 that “Surely, by facilitating its medicines’ access in the world’s biggest obesity market, Eli Lilly made a great move to promote [GLP-1] utilization over competitor Novo Nordisk [A/S].” Bank of America analyst Allen Lutz wrote on March 13 that while “Amazon’s entrance into the pharmacy space has been underwhelming,” the LillyDirect deal “reflect[s] a shift in consumer preferences” that Amazon is wise to capitalize on. Lutz added that “patients taking GLP-1 drugs for the first time in 2024 could be introduced to Amazon’s mail pharmacy for the first time, which could potentially create greater awareness of the platform.”

Among the issues facing health care payers, paying for multimillion-dollar cell and gene therapies (CGTs) is one of the most pressing, as evidenced during AHIP’s 2024 Medicare, Medicaid, Duals & Commercial Markets Forum, held March 12 through 14 in Baltimore. While they were mentioned by multiple speakers throughout the three-day conference, speakers at one session focused on the topic said that while approaches such as short-term milestone-based contracts and risk pools are being used, no perfect solution has emerged yet.

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 the March 12 session, 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.