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细胞和基因疗法的商业前景

医药生物2024-05-17奥纬咨询邓***

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Cell and gene therapies (CGTs) have significant potential to revolutionize treatment but face challenges in commercial viability due to high costs, small patient populations, and reimbursement issues. The global market is projected to grow at a 46% compound annual growth rate, reaching nearly $37 billion by 2028, with an increasing number of approvals and technological advancements like CRISPR gene editing. However, most CGTs have underperformed revenue forecasts due to approval delays, supply constraints, and unfavorable reimbursement, exemplified by Roctavian's low sales. Prices of CGTs are rising, with eight out of ten of the most expensive drugs in the U.S. being CGTs. Outcome-based agreements (OBAs) are being explored to secure reimbursement, but adoption varies by country, with Europe being more advanced. Improving operating models is critical, particularly through cost optimization in manufacturing. For cell therapies, reducing costs by at least 50% is necessary for commercial viability, while gene therapies face challenges in recouping R&D investments due to small patient populations and complex manufacturing. Advancing manufacturing technologies at a pace equal to scientific progress is essential to avoid falling short of commercial success.

© Oliver WymanINTRODUCTIONCell and gene therapies (CGTs) have the potential to revolutionize the treatmentlandscape and even cure previously incurable diseases. Optimism surrounding theseinnovative therapies is propelling a 46% compound annual growth rate over the nextfive years, bringing total the global market size to nearly $37 billion.Yet given the high cost of the drugs, combined with smaller population sizes, earlyinnovators still face significant issues securing reimbursement from payers which limitstheir ability to recoup investments. Ongoing funding challenges for health systems aroundthe globe could further tighten reimbursement and add pressure to lowerprices.At the same time, manufacturing and quality processes remain immature and non-standardized with low automation and high material cost, resulting in margin levels thattest the commercial viability of the new product classes — both in cell and genetherapies.Closing the commercial viability gap will be a key priority to successfully give large patientpopulations access to the great potential these therapies can bring. This report detailssteps organizations can take to make greater progress on pricing and bring the cost ofgoods down to viablelevels.FROM SCIENCE FICTION TO BLOCKBUSTER DRUGSCell and gene therapy refers to the introduction, removal, or change in the content of aperson’s genetic code to treat or cure a disease. This is achieved by either directly injectinggenetic material into their bodies or using modified cells and (re)inserting those as thetherapy (Exhibit1). What was once an unimaginable leap in science has now become areality for manypatients.Exhibit 1: Cell and Gene therapy taxonomyGenetic interventionoccursinside the bodyIn vivoIn vivoGene therapiesSource: Oliver Wyman analysis1EvaluatePharma 1Usecells as startingmaterial, geneticmodification madeoutside the body andcorrected cells infusedback in the patientUsespatient’s cellsas starting material,genetic modificationmadeoutside thebodyand engineered cellsinfused back in the patientUsesnon-selfalternative sourcesasstarting material, offthe shelf geneticallymodified productTypes of Cell and Gene therapiesGene modified cell therapiesGenetic intervention occursoutside the bodyEx vivoAutologousAutologousAllogeneic © Oliver WymanCell and gene therapies started getting regulatory approval in the mid-2010s. Since then,the number of annual approvals has been mounting, reaching 21 total approvals in theUS and Europe as of early April 2024. Not only has the volume of therapies increased,but there have been many technological advancements within the therapies such asthe approval of Casgevy in December 2023 which is the first therapy approved usingCRISPR gene editing (Exhibit2).Exhibit 2: Count of CGT approvals in Europe or the US per year and recentapproval highlightsApproval DateTherapyManufacturerRecent approval breakthroughsDecemberJuneMayCasgevyElevidysVyjuvekVertexFirst everCRISPR/Cas9 basedgene editingtherapy approved indicated for sickle cell diseaseand transfusion-dependent beta thalassemiaFirst adenoviral vector-based gene therapyapprovedfor Duchenne muscular dystrophyFirstMultidose & topical gene therapy— delivers COL7A1 gene via modified HSV totreat dystrophic epidermolysis bullosaSareptaKrystal202320222022120162017201820192020202120232122364DecemberEbvalloAdstiladrinFirstallogenic T-cell immunotherapyapproved— indicated for Epstein‑Barr virus positivepost‑transplant lymphoproliferative diseaseFirst adenoviral vector-based gene therapyapproved forsolid tumours-indicated for BCGunresponsive non-muscle invasive bladder cancerAtara BioFerringpharmaceuticalsGene therapiesGene modified cell therapies+22%Source: Oliver Wyman analysis © Oliver WymanTo date, two CGTs which have reached blockbuster status, Zolgensma and Yescarta.This will likely increase to six by 2028. Taking late-stage pipeline candidates into account,the potential addressable patient population magnitude is forecasted to increase by morethan 207% by 2028. This is driven by both a larger volume of CGTs entering the market andexpansion into such larger indications as systemic lupus erythematosus (Exhibit3). As aresult, CGTs are becoming a common therapeutic class with applications across millionsof patients globally, expanding beyond the traditional utilization for ultra rarediseases.Exhibit 3: Cumulative total addressable patient population based on approved/pipeline therapies in the US and EU. Magnitude of total addressable patient populationcategorized by therapy indication incidence rane67,883Historical launches(pre-2022)Recent launches(2022-2023)Future launces(2024-2028)78,914169,70784,854118,795402,206509,122169,707458,20984,8541,235,4688,48525,4563,39413,5771 in 1,000,000 – 1 in 100,0001 in 100,000 – 1 in 50,0001 in 50,000 – 1 in 10,0001 in 10,000 – 1 in 5,0001 in 5,000 – 1 in 1,000+207%Source: Evaluate Pharma, Oliver Wyman Analysis © Oliver WymanStill, the commercial viability of CGTs remains uncertain. Se

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