年度临床试验综述：2025年版：创新、监管与传承

2025 Edition: Innovation,Regulation, and Preservation Heidi Chen, PhDAssociate Director, Research& Commercial Services C I T E L I N E . C O M Agenda Total trial initiations and Y-O-Y trendsClinical trials by therapeutic areasTop 10 diseasesRare disease trialsTop industry sponsorsGeographic distributionsConcluding thoughts 2024 Clinical Trial Initiations by the numbers Scope of trial analysisPhase I-III, disclosed initiation date in 2024, Trialtrove curated trials Growth in industry-sponsored (67%) trials significantly declined•Tightening capital markets and focus shift •Increased trial costs – complex protocols•Evolving diversity requirements•Global harmonization challenges•Political conflicts – supply chain disruption; patient/site displacement Phase I-III trials by therapeutic area Oncology (37%) remains a core area of research•Immunotherapy, T-cell therapies, antibody-drugconjugates (ADCs) & bispecifics•Radiopharmaceuticals CNS grew by 14.7%•Pain, depression, Alzheimer’s disease Autoimmune/Inflammation (A/I)•Impressive growth of 14.6%•Cell therapy, i.e. CAR-T expansion beyondoncology Industry-sponsored Phase I-III trials by TA Industry sponsored trials by therapeutic area Oncology (30% share) hadthe most trials but declinedby 5% in 2024 Autoimmune/Inflammationgrew by 17%, second largestTA for pharma industry; CNSsustains its growingpresence at 15.5% Infectious disease grew by22%. Cardiovascular andmetabolic experiencedmodest decline but remain astrong focus area for theindustry Trial distribution by phase and TA •Phase II growth by 14.3% •Phase III share grew from2.7% to 15.4% •Even TA distributions inPhase I and III •Oncology is the clearwinner in Phase IIdevelopment Top 10 diseases in 2024 trial initiations Top 10 diseases and their rankings •Diseases with most Phase I trials: T2D, hypertension,respiratory infection•Esophageal cancer entered top 10 – rare disease development•Obesity made the list, riding on the success of GLP-1 boom•Phase III blue ribbons: NSCLC, respiratory infections, breastcancer, pain (nociceptive). •New comers: pancreatic cancer, dyslipidemia•Biggest leap: obesity: from #21 in 2023 to #6 Rare Disease Trials •52% novel drugs approved by theFDA are for rare diseases•Rare disease drugs: 7,721 in2025, 32.3% share of pipeline•Trial sponsorship split betweenindustry (48%) and academic(52%) Top rare diseases •ALS had more than double the trialinitiations•Scleroderma gained momentum•ITP slipped through the rank with adecrease of 27%•New comers: myasthenia gravis andprurigo nodularis •Majority of development in oncology•NHL had the most trial initiations Where is rare disease research happening? •China leads with 1,476trials (47%)•US follows with 1,063trials (34%)•Japan with easedregulatory requirementfor rare disease joinedthe rank Top sponsors in rare disease trials •Close competitions intop sponsor leaderboard•AstraZeneca leads inrare disease trials,replacing Roche’s #1spot in 2023•Commitments fromChinese companiesrepresent 40% of thislist Key industry sponsors in trial initiations Disease priorities of top 10 sponsors •20 Unique diseases in 2024 trialinitiations•Consistency in 6 out of 10 companies•Commitment to oncology: NSCLC,breast cancer•Rise in obesity focus 2024 Trial initiations by region FDAAA 801 relaxed Ph 1 reporting in the USled to smaller share of Phase I trial counts 5EU and Japan had majority of developmentsin Phase III China leads trial initiations with >80% share in AsiaDriven by big increase in Ph I trials Clinical trials affected by geopolitical conflicts •Recovery in mostcountries exceptRussia•Poland has the largestrebound with 442 trials•Continued tensions riskfurther disruptions toclinical research Top locations by therapeutic areas