全球视野下：ADC的trial策略

Shuhua Zhou（周淑华）PhDCitelineMar. 9th2024 Outline 1.ADC领域的市场空间和临床试验启动趋势 2.临床可行性评估（患者数量，临床时长，Patient journey）3.ADC临床开发的适应症和患者、主要终点选择4.ADC临床试验的Combo趋势 ADC Drugs with High Likelihood of Approval ADC Drugs WW sales forecast ADC Drugs Trials by Start Date Outline 1.ADC领域的市场空间和临床试验启动趋势 2.临床可行性评估（Patient journey，患者数量，临床时长与成本） 3.ADC临床开发的适应症和患者、主要终点选择 4.ADC临床试验的Combo趋势 Patient Demographics and Patient Journey •Better understanding of patient dynamics to assist with pharma company strategic planning – improve clinical trialdesign for pipeline drugs and patient targeting for launched drugs Description Examples •Reimbursement claims data analysis enables theinvestigation of patient demographics for moreaccurateestimation of the patient populationwitha particular disease,as well as patientdistribution by state or other geographic region •Such analysis is vital when planning clinical trialsand assessing the potential future value of aproduct,particularly when other sources ofepidemiology data are unavailable or unreliable,as is often the case with rare diseases •Reimbursement claims data analysis can also beused toinform pharma companies about thepatientjourney,e.g.,whether patients mostlyseek care at hospitals or clinics, referral patterns,treating physician specialties, etc. Turn Real World Data (RWD) into Insights +2.1M HCPs 98% of Payers +300M Patients Patient Volumes Payer Detail Available Granularity Granularity Specifics by benefit planSubmissions with PriorAuthorization (PA), Quantity Limit(QL), and Step Therapy (ST)specificity Affiliation HierarchyDifferentiation of treatment settings-(inpatient / outpatient)Line-item charge detailsBehaviors & decisions of physicians,assistants, nurse practitioners,registered nurses, and other healthcarestaff 300M+ total patients from 2017forward200M+ patients annually +7K Hospitals+5,600 Health Systems+1,500 ACOs+6.5M AffiliationsGranularity Remittances with allowed amountsDenials with rationale specificityDenial reasonsRebates, coupons, deductibles, co-insurance and full paymentamounts Patient Geography at Zip levelSite-of-care addressDemographics (age, race, gender)Out-of-pocket costsWeekly data refreshes Coverage by Payer Type~80% of commercial lives NPI, First/Last NameIDN Affiliations ~60% of Medicare lives~40% of Medicaid lives Benchmark to ADC Phase I & Phase III Trials in Last 10 Years ADC Trial Enrollment Efficiency in Top 10 Country Last 10Years Trial Cost Mean Benchmark Outline 1.ADC领域的市场空间和临床试验启动趋势2.临床可行性评估（患者数量，临床时长，Patient journey） 3.ADC临床开发的适应症和患者、主要终点选择 4.ADC临床试验的Combo趋势 Patient Segment for ADC Trials Patient Segment for ADC Trials Primary Endpoints in Registration and Expanded Indication Trialswith Positive Outcome Outline 1.ADC领域的市场空间和临床试验启动趋势2.临床可行性评估（患者数量，临床时长，Patient journey）3.ADC临床开发的适应症和患者、主要终点选择 4.ADC临床试验的Combo趋势 ADC Combination Trials in Phase II and Phase III Forecasting with Evolving Assumptions at Various Drug Life Cycle