治疗保证对商业付款人的影响

© Oliver WymanEXECUTIVESUMMARYMuch attention has been focused on the impact of gene and cell-based therapies (GCTs) ontotal US healthcare expenditures. Far less emphasis has been directed toward analyzinghow these exposures can vary widely across unique payer types — the employers, insurers,and government agencies that substantially pay for these treatments. The magnitudeof that GCT financial burden is significant, however, with increasing potential impactand variability on smaller payers, according to an analysis by Octaviant Financial andOliver WymanActuarial.Given the likely cost variability experienced by these constituencies, studies that providegreater insight into the anticipated range of expectations across payers are crucialto facilitate informed payer risk management. In this report, we focus specificallyon commercial payers and their risks, characterized as both overall expense outlaysfor GCTs and the economic loss associated with GCTs that fail to result in a clinicallymeaningfuloutcome.We use a simplified statistical model to estimate the probabilistic distribution of theexpense burden for GCTs, as well as the distribution of economic “waste” associated withunsuccessful treatment across several illustrative commercial payer types covering differentsized populations and risk financing structures. A novel risk mitigation tool, the therapeuticwarranty, is also modeled to determine its value in reducing the economic uncertainty andthe total costs of GCTs for different payertypes.In summary, our modeling confirms that the magnitude of the GCT financial burden issignificant, with increasing potential impact and variability on smaller payers. We alsovalidate that the wasted expense of GCT failure could be significantly reduced when aninstrument like a therapeutic warranty, which addresses a specified clinical outcome andprovides remuneration to payers when that outcome is not met, is available. This initialanalysis supports ongoing payer risk analysis and GCT planning and serves as a clarioncall for novel solutions that can help contain a payer’s net expenses, reduce overallfinancial risk by minimizing wasted expenditure and potentially enable greater coverageand authorization of GCTs for patients. This work also brings to the forefront the needfor additional studies, and we highlight important additional research opportunities inourconclusions. © Oliver WymanUS POPULATION GCT COST ESTIMATES AND PAYERMIXWhile GCTs in theory hold the promise of durable remission for many debilitating diseasessuch as sickle cell anemia, hemophilia, and Duchenne muscular dystrophy, the therapeuticsalone are typically priced in the range of $1 million to $4 million, exclusive of associatedhospital and professional fees. This is significantly more than the approximate $100,000cost of newly FDA-approved specialty therapies and the $1,432 per capita pharmaceuticalspending in 2022.1,2To date, 14 in-vivo gene therapies have been FDA-approved with anaverage price of $2.3 million while 12 cellular-based, non-cord, ex-vivo therapies have beenFDA-approved approved with an average price of $350,000.3Hundreds of additional GCTsare currently being developed and are advancing through clinicaltrials.Exhibit 1:FDA approved gene therapies (as of8/2/2024)GenetherapyDateWAC1($million)CelltherapyDateWAC1($million)BEQVEZ2024$3.5TECELRA2024$0.73LENMELDY2024$4.25AMTAGVI2024$0.52CASGEVY2023$2.2OMISIRGE2023$0.34ELEVIDYS2023$3.2LANTIDRA2023$0.30/yearLYFGENIA2023$3.1BREYANZI2022$0.41ROCTAVIAN2023$2.9CARVYKTI2022$0.47VYJUVEK2023$0.63/yearABECMA2021$0.42ADSTILADRIN2022$0.06/doseTECARTUS2021$0.37HEMGENIX2022$3.5KYMRIAH2017$0.48SKYSONA2022$3.0YESCARTA2017$0.40ZYNTEGLO2022$2.8LAVIV2011$0.01ZOLGENSMA2020$2.1PROVENGE2010$0.07LUXTURNA2017$0.85IMLYGIC2015$0.061. WAC: Wholesale AcquisitionCostSource:Oliver Wymanand OctaviantAnalysisVarious academic analyses have attempted to quantify the projected total financial impact ofGCTs on the US healthcare system. In 2021, Wong et al. estimated that a total of 1.09 millionpatients would be treated with gene therapies in the US between 2020 and 2034, with totalspending of $306 billion. In 2022, Young et al. of the NEWDIGS consortium predicted amore modest range of 54 to 74 GCT approvals by 2030, impacting over 600,000 patientsand costing a cumulative total of $201 billion.4While these estimates suggest that the expense of gene therapies will likely only increasethe overall $4.5 trillion US healthcare annual expenditure by 0.5% to 1.0%, they do notexplore the variability of impacts on specific payer segments, including the stop loss and © Oliver Wymanreinsurance markets.5Because the distribution of GCT-eligible patients across payers islargely random, the economic impact can be quite disparate across the range of payer typesrefenced in Exhibit2. These analyses also overlook payer-specific impacts associated withtherapeutic “failure,” or the waste associated with the inability of a GCT to achieve the clinicalobjectiv