Specialty Drugs

The Institute for Clinical and Economic Review’s (ICER’s) new president and CEO Sarah Emond took over the role from the organization’s longtime leader and founder Steve Pearson on Jan. 1, and in an interview at the J.P. Morgan Healthcare Conference in San Francisco, she discussed some of the pressing drug pricing issues on ICER’s radar for the year ahead.

Emond has had a lengthy career at ICER, having worked for the organization for 14 years — most recently as vice president and chief operating officer, helping to lead strategic operations. She said the leadership change was part of a long and planned transition and Pearson continues to work as an advisor.

Tremendous strides have been made in personalized medicine over the past several years — not only in drugs but in the various biomarkers used to identify the right drug for the right patient at the right time. While many people may think of this approach as one mainly solely for cancer, the potential for personalized medicine to have a broader impact on health outcomes when it is used proactively has not fully been realized.

In September, InformedDNA — which provides genomic solutions to an array of health care stakeholders to improve outcomes — acquired gWell Health, a digital health, genomics and wellness company. As part of the deal, gWell founder and CEO Surya Singh, M.D., transitioned to CEO of InformedDNA. Singh has worked in various health care organizations in more than 20 years, including serving as corporate vice president and chief medical officer at CVS Health Specialty.

This past year, the FDA continued to rebound from a drop in approvals, marking the highest number in years. The agency’s Center for Drug Evaluation and Research (CDER) approved 55 novel drugs last year, and its Center for Biologics Evaluation and Research (CBER) approved 17 agents. That’s up from 37 CDER-approved therapies in 2022 and 51 in 2011. In 2022, CBER OK’d 13 agents, up from 10 in 2021. Specialty agents, such as cell and gene therapies, continued to make up a large portion of those new approvals, while the FDA approved several biosimilars, including a handful that were the first versions of their reference drugs. AIS Health, a division of MMIT, spoke with industry experts about what they view as the most notable FDA approvals of 2023.

Dec. 8: The FDA expanded the patient population of ADMA Biologics, Inc.’s Bivigam (immune globulin intravenous, human) to include the treatment of people at least 2 years old with primary humoral immunodeficiency. The agency initially approved the therapy on Dec. 21, 2012. The initial intravenous infusion rate is 0.5 mg/kg/minute for the first 10 minutes for a dose of 300-800 mg/kg every three to four weeks. The maintenance infusion rate may be increased every 20 minutes by 0.8 mg/kg/minute up to 6 mg/kg/minute. Drugs.com lists the price of a 50 mL intravenous solution as more than $756.

Dec. 13: The FDA approved US WorldMeds’ Iwilfin (eflornithine) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-disialoganglioside (anti-GD2) immunotherapy. Between 700 and 800 cases of neuroblastoma are diagnosed annually in the U.S., with 90% of those before age 5. About half of those cases in children are HRNB. The agency used the Real-Time Oncology Review pilot program and the Assessment Aid. It also granted the application priority review and breakthrough drug and orphan drug designations. The recommended dosing of the tablet is based on body surface area.

A district court judge wasted no time in responding to the latest motion in an ongoing lawsuit filed against HHS by patient advocacy groups over the use of copay accumulators. In his decision, the judge reiterated his September decision vacating one rule and reinstating another that prohibits the use of accumulators for drugs that do not have a medically appropriate generic equivalent. It’s not entirely clear what next steps will be, but one patient advocacy group says it hopes to see HHS enforce the policy limiting plans’ use of accumulator programs.

While pharma manufacturers began offering the programs to help patients stay on costly therapies, payers have pushed back, saying they lead to higher-cost agents to be used over lower-cost ones. At the same time, similar programs, such as copay maximizers and alternate funding programs, have increased in use, to payers’ dismay.

Elevance Health, Inc. has agreed to acquire infusion services provider Paragon Healthcare, Inc., the health insurer said Jan. 4. The companies did not disclose financial details of the deal, which is expected to close in the first half of 2024. Paragon Healthcare serves more than 35,000 people at more than 40 ambulatory infusion centers in eight states, as well as in the home setting, and it treats more than 300 conditions. Once the deal is finalized, Paragon Healthcare will operate as part of CarelonRx, which is the pharmacy segment within Elevance Health’s Carelon health services division. The deal follows Elevance Health’s acquisition of BioPlus, a specialty pharmacy subsidiary of CarepathRx, a portfolio company of Nautic Partners, which it closed in February 2023.

Hizentra (immune globulin subcutaneous [human] 20% liquid) is now available in a 10 g prefilled syringe, manufacturer CSL Behring disclosed Jan. 3. The agent is the first and only subcutaneous immune globulin treatment approved for the maintenance of chronic inflammatory demyelinating polyneuropathy in the U.S. It also is approved for primary immunodeficiency in people at least 2 years old. In addition to the new size, the drug also is available in 1 g, 2 g and 4 g prefilled syringes.

In December, the FDA approved the two newest cell and gene therapies, which were the first such agents approved for the treatment of sickle cell disease. As more of these products launch onto the U.S. market — the agency previously predicted that it would be approving 10 to 20 of the treatments by 2025 — payers are taking a variety of approaches to managing the therapies. Cost remains the main obstacle to their use, but their long-term durability also remains a question to some extent.

Dec. 8 saw the newest approvals, both for the treatment of sickle cell disease in people at least 12 years old: bluebird bio, Inc’s Lyfgenia (lovotibeglogene autotemcel; lovo-cel) and Vertex Pharmaceuticals Inc. and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel; exa-cel). The latter agent is the first CRISPR/Cas9 genome-edited cell therapy that the FDA has approved. The FDA gave both applications priority review, orphan drug, fast track and regenerative medicine advanced therapy designations. It also gave Lyfgenia rare pediatric disease designation.

Elevance Health, Inc. has agreed to acquire Paragon Healthcare, a company that provides infusion services to patients. Elevance announced the pending transaction on Jan. 4 and said it expects the deal to close in the first half of this year. The companies did not disclose financial details, but Axios reported Elevance will pay more than $1 billion for Paragon, which operates in Alabama, Colorado, Florida, Georgia, Missouri, Oklahoma, Tennessee and Texas. If the deal closes, Paragon will become part of CarelonRx, Elevance’s pharmacy services segment.

The Cigna Group is in “advanced talks” to sell its Medicare Advantage business to Health Care Service Corp. (HCSC) for between $3 billion and $4 billion, according to a Jan. 3 Wall Street Journal article. The report comes shortly after Bloomberg cited anonymous sources saying HCSC and Elevance Health were interested in acquiring Cigna’s Medicare business, a segment the company entered in 2012 through its acquisition of HealthSpring. Cigna has struggled to compete in Medicare with market leaders such as UnitedHealth Group and Humana Inc., and it has just 580,000 MA lives, according to AIS’s Directory of Health Plans. Earlier last month, the WSJ reported that discussions about a potential transaction between Cigna and Humana had fizzled.

While the U.S. finally had biosimilar competition for AbbVie Inc.’s Humira (adalimumab) in 2023, it may take longer than anticipated for the agents to truly have an impact. That’s one of the trends moving into 2024 that Adam J. Fein, Ph.D., CEO of Drug Channels Institute, discussed during a recent webinar. While the impact of the glucagon-like peptide 1s (GLP1s) and patient-paid prescriptions will persist, the uptake of copay offset tools is subsiding, he said during the Dec. 15 webinar, titled Drug Channels Outlook 2024. In this first of a two-part series, AIS Health highlights the first half of the trends projected by the longtime industry expert.

The FDA recently approved the two newest cell and gene therapies, with one of them earning the distinction of being the first of its kind approved by the FDA. As more of these products launch onto the U.S. market — the agency previously predicted that it would be approving 10 to 20 of the treatments by 2025 — payers are taking a variety of approaches to managing the therapies.

Dec. 8 saw the newest approvals, both for the treatment of sickle cell disease in people at least 12 years old: bluebird bio, Inc’s Lyfgenia (lovotibeglogene autotemcel; lovo-cel) and Vertex Pharmaceuticals Inc. and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel; exa-cel). The latter agent is the first CRISPR/Cas9 genome-edited cell therapy that the FDA has approved. The one-time treatments come with hefty price tags: Lyfgenia is priced at $3.1 million and Casgevy at $2.2 million.