Cell and Gene Therapies

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.

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.

The US Food and Drug Administration’s simultaneous approval of two gene therapies for sickle cell disease from Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG and bluebird bio on 8 December provides the competitors an equal start out of the gate, and offers another test for the Rx policy concept that intra-class competition can drive down prices.

Based on the initial list prices, though, it seems like perhaps competition cannot do that, at least not in this case, or at least not yet. Bluebird bio’s Lyfgenia has a wholesale acquisition cost of $3.1m, while the WAC for Vertex and CRISPR’s Casgevy is $2.2m, which might be a significant handicap for bluebird in securing reimbursement.

The recent approvals of the first two gene therapies for sickle cell disease represent potential major breakthroughs for patients who have faced significant burdens associated with the condition. However, there are still questions about how and whether payers will cover the high-cost treatments and how many patients will be open to taking the new treatments.

The FDA on Dec. 8 approved Casgevy (exagamglogene autotemcel) from CRISPR Therapeutics and Vertex Pharmaceuticals Inc. and Lyfgenia (lovotibeglogene autotemcel) from bluebird, Inc. for patients who are 12 or older and have recurrent vaso-occlusive events (VOEs), the term for severe pain and organ damage. The agency noted about 100,000 people in the U.S. have sickle cell disease and that VOEs “can lead to life-threatening disabilities and/or early death.”

As the PBM and specialty pharmacy worlds have become more integrated, PBMs now are providing more specialty management services than they previously did. Only 15% of respondents to a recent survey said they carve out specialty drugs from the PBM that is providing their pharmacy benefit management, while 26% said they were considering taking this step, according to the 2023 Pharmacy Benefit Manager Customer Satisfaction Report from Pharmaceutical Strategies Group (PSG), an EPIC company.

The 85% of respondents whose PBMs provide both specialty and traditional pharmacy benefit management ranked formulary management of specialty drugs and financial reporting in the pharmacy benefit as the services they were most satisfied with, while they were least satisfied with management of specialty agents in the medical benefit and offering competitive discounts on specialty medications.

The specialty drug landscape continues to be a dynamic space, as new agents enter the market and existing ones gain FDA approval for additional indications. Global health care and specialty pharmacy solutions organization AscellaHealth recently released its quarterly breakdown of insights into treatments within the segment.

The Q3 2023 Specialty & Rare Pipeline Digest examines new approvals and launches of specialty drugs, including biosimilars, generics, and cell and gene therapies, as well as ones in the pipeline.

The specialty drug landscape continues to be a dynamic space, as new agents enter the market and existing ones gain FDA approval for additional indications. Global health care and specialty pharmacy solutions organization AscellaHealth recently released its quarterly breakdown of insights into treatments within the segment.

The Q3 2023 Specialty & Rare Pipeline Digest examines new approvals and launches of specialty drugs, including biosimilars, generics, and cell and gene therapies, as well as ones in the pipeline.

Among the highlighted agents in the recently released report were Alzheimer’s disease drug Leqembi (lecanemab-irmb) from Eisai Inc. and Biogen, myasthenia gravis therapy Rystiggo (rozanolixizumab-noli) from UCB, Inc. and Pfizer Inc.’s alopecia areata medication Litfulo (ritlecitinib).

As more and more high-cost therapies, including cell and gene therapies (CGTs), enter the U.S. market, commercial health plans have multiple tools at their disposal to manage these agents. Medicaid plans, however, are limited in what they can do. But a multistate value-based contracting (VBC) tool offered by Magellan Rx Management and its parent company, Prime Therapeutics LLC, is helping Medicaid programs access CGTs and ensuring that the agents’ costs are linked to patient outcomes.

A new Medicaid Pharmacy Insights report, titled The State of Value-Based Contracting: Reinventing the Current Drug Payment Model in Medicaid, notes that Medicaid is usually the largest expenditure in state budgets. States need to be able to offer costly CGTs while also managing their budgets. But various barriers to offering value-based contracts — including a lack of resources to negotiate them, as well as collect data and measure outcomes — have limited adoption of these agreements.

The FDA has approved multiple new agents for the treatment of acute lymphoblastic leukemia (ALL) — which is also known as acute lymphocytic leukemia — and recently converted an accelerated approval it had given one of them to full. However, even with all these options, respondents to a Zitter Insights survey said that unmet need exists in treating the disease.

On June 21, 2023, the FDA granted full approval to Amgen Inc.’s Blincyto (blinatumomab) for the treatment of adults and pediatric patients with CD19-positive B-cell precursor ALL first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%. The agency first approved the CD19-directed CD3 T-cell engager on Dec. 3, 2014; the accelerated approval for MRD-positive B-cell ALL was granted on March 29, 2018.

Specialty drug trend continues to be in consistent double digits, with increases due to both spend and trend. That’s one of the findings of the 2023 Artemetrx Specialty Spend & Trend Report from the Pharmaceutical Strategies Group (PSG), an EPIC company. And with the specialty drug pipeline continuing to pump out more and more expensive agents, opportunities for savings exist through the use of biosimilars and generic specialty drugs, among other strategies.

Published July 25, the report is sponsored by Walmart Specialty Pharmacy and includes data from 2022. Findings are based on an analysis of 347 million medical claims and 133 million pharmacy claims from PSG’s Artemetrx book of business. Artemetrx is a proprietary SaaS platform developed by PSG. This is the seventh annual version of the report, which started under the Pharmacy Benefit Management Institute (PBMI) moniker. In April 202, MJH Life Sciences acquired PBMI’s trademarks, conference, website, education and membership assets, while the current and future research and analytics projects remained with PSG.