Radar on Specialty Pharmacy

More than 10 years after the passage of the Biologics Price Competition and Innovation Act of 2009 (BPCIA) as part of the Affordable Care Act, the FDA finally has approved the first interchangeable biosimilar. But while many in the industry are hailing the move, at least one expert wonders whether interchangeables will hamstring use of biosimilars that do not have that designation.

On July 28, the FDA approved Viatris Inc. and Biocon Ltd. subsidiary Biocon Biologics Ltd.’s Semglee (insulin glargine-yfgn) to improve glycemic control in adults and pediatric patients with Type 1 diabetes mellitus and in adults with Type 2 diabetes mellitus. It is a biosimilar to and interchangeable with its reference product, Sanofi Aventis’ Lantus (insulin glargine), a long-acting insulin analog. The product will be available in 10 mL vials and 3 mL prefilled pens and is administered subcutaneously once daily. It also has 12 months of exclusivity under section 351(k)(6) of the Public Health Service (PHS) Act during which the FDA cannot approve another biosimilar that is interchangeable with Lantus.

The FDA recently approved a third agent to treat paroxysmal nocturnal hemoglobinuria (PNH). With some conditions, that number of treatments may prompt payer preferencing, but that is unlikely to happen with this ultra-rare, potentially fatal disease, observe industry experts.

On May 14, the FDA approved Apellis Pharmaceuticals, Inc.’s Empaveli (pegcetacoplan) as the first and only targeted C3 therapy for the treatment of adults with PNH. It is approved for both treatment-naïve people, as well as ones switching from any C5 inhibitor. The FDA has approved two of those drugs: Soliris (eculizumab) and Ultomiris (ravulizumab-cwvz), both from Alexion Pharmaceuticals, Inc., which was just acquired by AstraZeneca in July.

✦ Merck & Co., Inc. will voluntarily withdraw the accelerated approval indication for Keytruda (pembrolizumab) for the treatment of people with recurrent locally advanced or metastatic gastric or gastroesophageal junction adenocarcinoma whose tumors express programmed death-ligand 1 (PD-L1), as determined by an FDA-approved test, with disease progression on or after at least two lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and, if appropriate, human epidermal growth factor receptor 2 (HER2)/neu-targeted therapy. The move follows the April 29 Oncologic Drugs Advisory Committee meeting in which members voted against keeping the indication after late-stage confirmatory trials did not show clinical benefit. Merck says it will begin the withdrawal in six months.

✦ The expected average cost of new FDA-approved specialty drugs in 2020 was just under $350,000 per year. That was one of the findings of analytics company AMS’s 2020 Specialty Drug Trends Report, based on drugs added to the firm’s PredictRx module, which incorporates pertinent clinical information and AMS Cost Projection (ACP) pricing. It also found that the average yearly cost for newly approved orphan drugs was $449,507, compared with $144,130 for traditional drugs.

✦ June 7: The FDA granted another indication to Alexion Pharmaceuticals, Inc.’s Ultomiris (ravulizumab-cwvz) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in children at least 1 month old and adolescents. It is the only PNH therapy approved for use in these age groups. The agency initially approved the C5 inhibitor on Dec. 21, 2019, for PNH in adults. Dosing is weight-based; administration intervals also are weight-based and are either every four weeks or every eight weeks. The estimated average cost per year in adults is $458,000.

✦ June 9: The FDA gave another indication to Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis who are 6 to 11 years old and have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data. The agency also approved a new dosage strength of the tablet: elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg. The FDA first approved the drug on Oct. 21, 2019. Dosing in this new age group for people less than 30 kg is two elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg tablets in the morning and one ivacaftor 75 mg tablet in the evening. For those weighing at least 30 kg, dosing is two elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg tablets in the morning and one ivacaftor 150 mg tablet in the evening. Website Drugs.com lists the price of 84 tablets of the latter dose as more than $24,900.

As more high-cost drugs, including one-time gene therapies, come onto the market, employers are considering implementing a variety of contracting models to make sure their employees have access to these agents. However, even as innovative new approaches are being explored, employers have experienced challenges in executing them. But with the pharma pipeline full of specialty products, employers should explore which approaches may work best for their company.

“The emerging financial models for employers stemming from these new ultra–high-cost gene therapies include various reinsurance products from organizations like Cigna, CVS/Aetna, Prime Therapeutics and others,” explains Jorge Font, MPH, senior vice president of the access experience team at PRECISIONvalue. “These products involve a regular per-member-per-month premium paid to cover the risk of these low-frequency, high-cost claims.”