Specialty Drugs

Biosimilars Are Picking Up Market Share, but Some Uncertainties Still Exist

Since the FDA’s approval of the first biosimilar — Zarxio (filgrastim-sndz) from Sandoz, a division of Novartis Pharmaceuticals Corp. — on March 6, 2015, the agency has approved almost 40 more agents via the 351(k) pathway established under the Biologics Price Competition and Innovation Act (BPCIA), itself part of the Affordable Care Act (ACA). Although not all of those agents have launched yet, and almost all of the ones that have are all professionally administered, industry experts say they expect to see more competition in the space, depending on interchangeability status, provider uptake and the impact of the Inflation Reduction Act.

Cell and Gene Therapies Hold Promise, but Stakeholders Must Overcome Challenges to Meet Their Full Potential

Researchers continue to make progress in developing cell and gene therapies that offer the promise of slowing a disease’s progression and even offering a potential cure to patients. And while these agents may offer hope to patients, some challenges exist, including access to the treatments. In order for these products to reach their full potential, stakeholders must work together to overcome these potential barriers.

With its Feb. 28 FDA approval, the Janssen Pharmaceutical Companies of Johnson & Johnson and Legend Biotech USA, Inc.’s Carvykti (ciltacabtagene autoleucel or cilta-cel) became the sixth chimeric antigen receptor T-cell (CAR-T) therapy approved in the U.S. In addition, the existing CAR-Ts continue to get additional FDA-approved indications added to their labels, including for use in earlier line settings. And in August and September alone, the FDA approved two bluebird bio, Inc. gene therapies: Zynteglo (betibeglogene autotemcel or beti-cel) and Skysona (elivaldogene autotemcel or eli-cel).

Biosimilars Are Making Inroads Into U.S. Market, but Challenges Remain

Since the FDA’s approval of the first biosimilar — Zarxio (filgrastim-sndz) from Sandoz, a division of Novartis Pharmaceuticals Corp. — on March 6, 2015, the agency has approved almost 40 more agents via the 351(k) pathway established under the Biologics Price Competition and Innovation Act (BPCIA), itself part of the Affordable Care Act (ACA). Although not all of those agents have launched yet, and almost all of the ones that have are all professionally administered, industry experts say they expect to see more competition in the space, depending on interchangeability status, provider uptake and the impact of the Inflation Reduction Act.

Prescription Drug Spending Increases 16% Over Five Years, Driven by Rising Drug Prices

Total inflation-adjusted spending on prescription drugs grew from $520 billion in 2016 to $603 billion in 2021, before accounting for rebates, according to a report published by HHS’s Assistant Secretary for Planning and Evaluation (ASPE). Retail drug expenditures represented about 70% of prescription drug spending in 2021. For retail prescription drugs, there was a 13% increase in drug spending over the five years studied, yet only a 5.7% increase in the number of prescriptions.

Meanwhile, more and more Americans received their drugs from mail order pharmacies, clinics and home health care organizations between 2016 and 2021.

Prescription Drug Spending Increases 16% Over Five Years, Driven by Rising Drug Prices

Total inflation-adjusted spending on prescription drugs grew from $520 billion in 2016 to $603 billion in 2021, before accounting for rebates, according to a report published by HHS’s Assistant Secretary for Planning and Evaluation (ASPE). Retail drug expenditures represented about 70% of prescription drug spending in 2021. For retail prescription drugs, there was a 13% increase in drug spending over the five years studied, yet only a 5.7% increase in the number of prescriptions.

Meanwhile, more and more Americans received their drugs from mail order pharmacies, clinics and home health care organizations between 2016 and 2021.

Novel Therapies Take Center Stage in Optum Rx Drug Pipeline Report

In its latest report highlighting notable drugs nearing FDA approval, UnitedHealth Group’s Optum Rx PBM singles out two therapies designated as orphan drugs and one that has been granted priority review. Experts tell AIS Health that payers deciding how to cover these drugs will undoubtedly have to weigh factors such as a high unmet need for treatment, the durability of patients’ response to the therapy, and potentially high price tags.

For example, regarding the only non-orphan drug included in the report, Apellis Pharmaceuticals, Inc.’s pegcetacoplan, there are currently no other approved treatments for the condition it’s targeting, “so that will have an impact on how payers approach coverage,” says Bill Dreitlein, senior director of Pipeline & Drug Surveillance at Optum Rx.

Oncologists May Prescribe New Neutropenia Agent Over Others in Class

The FDA recently approved the first novel long-acting granulocyte colony-stimulating factor (G-CSF) in more than 20 years. Payers say they are likely to manage the new agent similar to existing ones, but oncologists have indicated that they are willing to prescribe it in place of other neutropenia agents, according to Zitter Insights.

On Sept. 9, the FDA approved Spectrum Pharmaceuticals, Inc.’s Rolvedon (eflapegrastim-xnst) to decrease the incidence of infection, as manifested by febrile neutropenia, in adults with nonmyeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. The company developed the drug with South Korea’s Hanmi Pharmaceutical Co. The recommended dose is 13.2 mg administered subcutaneously once per chemotherapy cycle. The company says it expects the product to be available in fourth-quarter 2022. It has not revealed the agent’s price yet.

FDA Grants Tentative Approval to Extended-Release Narcolepsy Agent

A new formulation of a narcolepsy drug may get uptake among providers, according to Zitter Insights research. But payers indicate that they are likely to require patients to fail at least one generic drug before getting access to the new agent and other branded medications. Still, the agent’s less onerous dosing may allow it to pull market share from similar medications within the class.

On July 19, Avadel Pharmaceuticals plc said that the FDA had granted tentative approval to its Lumryz (sodium oxybate) for the treatment of excessive daytime sleepiness or cataplexy — an abrupt loss of muscle tone that can be triggered by strong emotion — in adults with narcolepsy. The drug — which is also known as FT218 — is a once-at-bedtime extended-release version of Jazz Pharmaceuticals plc’s Xyrem, a drug that requires one dose at bedtime and then another dose between two-and-a-half to four hours later.

Specialty Pharmacy’s Dose Optimization Program Saved More Than $6 Million Last Year

As spending on specialty drugs continues to rise, payers are implementing various utilization management strategies in an effort to rein in their costs. While prior authorization and step therapy are perhaps the most common tactics, other approaches, such as quantity limits, site-of-care optimization and dose optimization also can be successful tools, as evidenced by one specialty pharmacy’s recent announcement.

In September, AllianceRx Walgreens Pharmacy revealed that its use of dose optimization resulted in $6.2 million in savings in 2021. The strategy reduces the number of dispensed units of an oral, injectable or infusible agent while delivering the same appropriate dose. Some manufacturers charge the same or similar price for different doses of a drug, so if a patient is given a prescription for two 10 mg tablets per day, and a 20 mg tablet is available, it can be more efficient and economical for the patient to take the single 20 mg tablet.

New FDA Approvals: FDA Approves Sotyktu

Sept. 9: The FDA approved Bristol Myers Squibb’s Sotyktu (deucravacitinib) for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. The agent is a first-in-class, oral, selective, allosteric tyrosine kinase 2 (TYK2) inhibitor. The recommended dosage of the tablet is 6 mg once daily. The list price for a 30-day supply is $6,164.

Sept. 9: The FDA approved Spectrum Pharmaceuticals, Inc.’s Rolvedon (eflapegrastim-xnst) to decrease the incidence of infection, as manifested by febrile neutropenia, in adults with nonmyeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. The manufacturer says it is the first novel long-acting granulocyte colony-stimulating factor (G-CSF) approved in more than 20 years. The recommended dose is 13.2 mg administered subcutaneously once per chemotherapy cycle. The company says it expects the product to be available in fourth-quarter 2022.