{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,8]],"date-time":"2026-05-08T08:10:20Z","timestamp":1778227820585,"version":"3.51.4"},"reference-count":24,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2026,5,5]],"date-time":"2026-05-05T00:00:00Z","timestamp":1777939200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Pharmacy Practice"],"abstract":"<jats:p>Between 2022 and 2025, several ultra\u2013high-cost, one-time therapies were approved in the United States, offering potentially curative options for conditions such as spinal muscular atrophy, hemophilia, and sickle cell disease. Despite their clinical value, upfront costs often exceeding $2-4 million create major challenges for payers, especially Medicaid programs with fixed budgets. Traditional reimbursement models, designed for chronic therapies, may not align with the long-term benefits and financial risks of these treatments. This has led to growing interest in alternative payment models, including outcomes-based agreements, installment payments, and risk-sharing contracts. However, adoption remains inconsistent due to regulatory barriers, administrative complexity, and limited long-term data. A narrative literature review was conducted to evaluate U.S. pricing strategies and manufacturer\u2013payer payment models for ultra\u2013high-cost, one-time therapies. Peer-reviewed and grey literature were identified through searches of PubMed, Embase, and Google Scholar, along with policy and regulatory sources. Publications from 2010 to 2025 were included and synthesized to categorize payment models and identify implementation challenges. Manufacturers used diverse pricing approaches rather than a single model. Outcomes-based agreements were more common when clinical endpoints were measurable, while other therapies relied on lump-sum payments with support programs. Adoption varied based on therapy characteristics and payer preferences, with limited standardization across stakeholders. These therapies are financed through a mix of traditional and alternative models, but regulatory and operational barriers continue to limit widespread adoption.<\/jats:p>","DOI":"10.1177\/08971900261450720","type":"journal-article","created":{"date-parts":[[2026,5,5]],"date-time":"2026-05-05T08:22:32Z","timestamp":1777969352000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Payer and Manufacturer Agreements for High-Cost Therapy: A Narrative Review"],"prefix":"10.1177","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-7352-2514","authenticated-orcid":false,"given":"Vishwanauth","family":"Persaud","sequence":"first","affiliation":[{"name":"Touro College of Pharmacy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Albert","family":"Wertheimer","sequence":"additional","affiliation":[{"name":"Touro College of Pharmacy"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2026,5,5]]},"reference":[{"key":"e_1_3_3_2_2","article-title":"The most expensive drugs in the US","author":"Maragkou I","year":"2024","unstructured":"Maragkou I. 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DIA Global Forum. 2025. https:\/\/globalforum.diaglobal.org\/issue\/may-2025\/accelerated-approval-as-the-new-norm-in-gene-therapy-for-rare-diseases\/"},{"key":"e_1_3_3_7_2","volume-title":"Changes in Medicaid\u2019s Best Price Rule Likely to Boost Value-based Purchasing Agreements","author":"Lyfegen","year":"2025","unstructured":"Lyfegen. Changes in Medicaid\u2019s Best Price Rule Likely to Boost Value-based Purchasing Agreements. Lyfegen HealthTech AG. https:\/\/www.lyfegen.com\/post\/changes-in-medicaids-best-price-rule-likely-to-boost-value-based-purchasing-agreements 2025."},{"key":"e_1_3_3_8_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.healthpol.2010.02.005"},{"key":"e_1_3_3_9_2","volume-title":"New Gene Therapy for Rare MDL to Cost $4.25 Million","author":"Myshko D","unstructured":"Myshko D. New Gene Therapy for Rare MDL to Cost $4.25 Million. Managed Healthcare Executive. https:\/\/www.managedhealthcareexecutive.com\/view\/new-gene-for-rare-mdl-to-cost-4-25-million"},{"key":"e_1_3_3_10_2","volume-title":"FDA Approves $3.5 Million Treatment for Hemophilia, now the Most Expensive Drug in the World","author":"McPhillips D","year":"2022","unstructured":"McPhillips D. FDA Approves $3.5 Million Treatment for Hemophilia, now the Most Expensive Drug in the World. CNN. https:\/\/www.cnn.com\/2022\/11\/23\/health\/hemophilia-drug-hemgenix 2022."},{"key":"e_1_3_3_11_2","unstructured":"Bratulic A. Insurer deal helps quell commercial concerns for bluebird bio's Lyfgenia. FirstWord Pharma. https:\/\/firstwordpharma.com\/story\/5810363. (2023 December 14)."},{"key":"e_1_3_3_12_2","volume-title":"At $2.1 Million, New Gene Therapy is the Most Expensive Drug Ever","author":"Stein R","year":"2019","unstructured":"Stein R. At $2.1 Million, New Gene Therapy is the Most Expensive Drug Ever. NPR. https:\/\/www.npr.org\/sections\/health-shots\/2019\/05\/24\/725404168\/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever 2019."},{"key":"e_1_3_3_13_2","doi-asserted-by":"publisher","DOI":"10.1089\/crispr.2024.0042"},{"key":"e_1_3_3_14_2","volume-title":"Launch Plans for Lenmeldytm (Atidarsagene Autotemcel), the Only Approved Therapy for Children with early-onset Metachromatic Leukodystrophy","author":"Orchard Therapeutics Outlines US","unstructured":"Orchard Therapeutics Outlines US. Launch Plans for Lenmeldytm (Atidarsagene Autotemcel), the Only Approved Therapy for Children with early-onset Metachromatic Leukodystrophy. Orchard Therapeutics. https:\/\/ir.orchard-tx.com\/news-releases\/news-release-details\/orchard-therapeutics-outlines-us-launch-plans-lenmeldytm"},{"key":"e_1_3_3_15_2","unstructured":"CSL\u2019s HEMGENIX Shows Durable Benefit. 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