Paying For Gene Therapy: Biggest Cost Challenges Two To Three Years Away?
Executive Summary
Only about one-quarter of the 40 gene therapies projected to launch by the end of 2025 would have potential US patient populations of more than 50,000. But a handful of treatments could target millions of individuals.
Upcoming gene therapies for diabetic neuropathy, osteoarthritis and wet age-related macular degeneration are on track for launch in the US within the next three years and have expected patient populations in the multi-millions, which could present a significant challenge for payers, including Medicare and state Medicaid programs.
To date, the individual impact of gene therapies on the market, even with prices that approach and exceed $1m, may seem relatively manageable. Most gene therapies on the market and due to reach it in the next two years target patient populations that are well below the 200,000 statutory threshold for orphan drugs, according to a gene therapy pipeline assembled by CVS Health Corp..
Only about one-quarter of the 41 gene therapies listed have projected patient populations of more than 50,000. Among them, bluebird bio’s LentiGlobin for sickle cell disease could treat 58,000 adult and pediatric patients, the pharmacy benefit manager projects. But the collective cost of those treatments as they continue to proliferate, combined with a handful of upcoming treatments targeting much bigger audiences, will intensify pressure on payers and policymakers to develop innovative payment models that can accommodate such high-cost, potentially curative agents.
The CVS pipeline report projects the launch of 11 new treatments (including new indications for existing gene therapies) in 2022, 16 launches in 2023, eight launches in 2024 and four in 2025. (See chart at end of the story.)
South Korean biotech Helixmith Co., Ltd.’s gene therapy for diabetic neuropathy targets a very common condition with a lifetime prevalence of around 50% in people with diabetes. As a result, the cost of the treatment could have a major impact on both Medicaid and Medicare. Kolon TissueGene, Inc.’s gene therapy for osteoarthritis and Regenxbio Inc.’s treatment for wet age-related macular degeneration both target widespread conditions associated with aging, so could be especially challenging for Medicare.
But individuals below the age of 65 may also suffer from OA or AMD, so the impact will reach Medicaid as well. In addition, Medicaid agencies are legally obliged to pay Medicare cost sharing for individuals who are eligible for both programs and that expense could be significant for very high-cost treatments.
Stumbles In Diabetic Neuropathy, Osteoarthritis Development Programs
Helixmith began a new Phase III study of its gene therapy for diabetic neuropathy Engensis (donaperminogene seltoplasmid) in 2020 after an earlier Phase III study failed to show a benefit over placebo. (Also see "Helixmith Mods Engensis Trial Plans As Probe Unravels PK Anomalies" - Scrip, 17 Feb, 2020.) The treatment, which is administered via injection, could target 7.1 to 13.5 million patients and could launch during the second half of 2023, CVS notes.
Kolon’s gene therapy Invossa (tonogenchoncel-L) is in development as a one-time injectable treatment for OA of the knee. It could reach the market in the second quarter of 2025, with a projected 13 million individuals as candidates for treatment, according to CVS.
The company received US Food and Drug Administration approval to resume Phase III testing in the spring of 2020 one year after the agency issued a clinical hold due to product characterization concerns. The two pivotal trials will enroll close to 1,020 patients at over 50 clinical sites across the US. (Also see "A Glimpse Of Light For Kolon As US Invossa Trials To Resume" - Scrip, 14 Apr, 2020.) In addition to significant pain and function improvements, the company has designed the trials to achieve a disease modifying osteoarthritis drug designation.
The disease modifying arena for OA has been notoriously difficult for drug developers to navigate and none have been approved to date. (Also see "Osteoarthritis Drug Development: ‘Clinically Meaningful Endpoints’ Remain Elusive" - Pink Sheet, 16 Aug, 2021.)
AbbVie Joins Forces With Regenxbio On Gene Therapy For AMD
Regenxbio is developing RGX-314 as a one-time gene therapy for neovascular (wet) age-related macular degeneration. The company anticipates filing a biologics license application for RGX-314 in 2024 and the treatment is on track to reach the market in the second half of 2025, according to CVS. The pharmacy benefit manager estimates RGX-314 could target 2 million adults.
Regenxbio recently entered a development and commercialization deal with AbbVie for the gene therapy as it moves toward regulatory filings. Under the arrangement, Regenxbio will complete a Phase II/III study of 300 patients comparing RGX-314 against Roche Holding AG’s Lucentis (ranibizumab) and the two companies will collaborate on a second pivotal trial set to begin in the fourth quarter of 2021. AbbVie will become responsible for the majority of development costs starting in 2023. (Also see "AbbVie Extends Reach Of Regenxbio’s Wet AMD Program" - Scrip, 13 Sep, 2021.)