美国新兴生物制药的选择拓展：变革的十年

Introduction Emerging biopharma companies — those with R&D spending less than $200Mnand revenues less than $500Mn a year — have become central to biomedicalinnovation over the past decade, driving the majority of novel drug launches,notably in specialty, orphan, and first-in-class categories. Their agility, resilience, This report examines the evolving landscape ofemerging biopharma (EBP) companies in the U.S. overthe past 10 years. Emerging biopharma companiesare those with R&D spend less than $200Mn per yearand revenue below $500Mn globally, and includes The study was produced independently by the IQVIAInstitute for Human Data Science as a public service,without industry or government funding. Find Out More If you wish to receive future reports from the IQVIAInstitute for Human Data Science or join our mailing list, MURRAY AITKENExecutive Director REFERENCING THIS REPORTPlease use this format when referencing content from this report: ©2025 IQVIA and its affiliates. All reproduction rights, quotations, broadcasting, publications reserved. No part of this publication may be reproduced ortransmitted in any form or by any means, electronic or mechanical, including photocopy, recording, or any information storage and retrieval system, without Expanding Options for Emerging Biopharma in the U.S.: A Decade of Change OverviewEBPs definition and classificationFunding and strategic transactionsClinical trial activityClinical development productivity Overview DEFINING EMERGING BIOPHARMA (EBP) AND THEIR FUNDING AND STRATEGIC TRANSACTIONS Biopharma funding levels have been especially dynamicsince the beginning of the pandemic, rebounding in2024 to reaching $100Bn, but IPO activity has remainedhistorically low, with only $1.4Bn raised throughJuly 2025. Venture funding has declined in 2025 to date,but mergers and acquisitions (M&A) and licensing deals EBPs are defined as companies with estimated R&Dspend below $200Mn or prescription sales up to$500Mn, including both pre–commercial and commercialentities. They represent a major segment of thebiomedical innovation ecosystem, responsible for CLINICAL TRIAL ACTIVITY STRATEGIC ROLES AND COMPANY SEGMENTATION EBPs pursue varied strategies: some develop assetsfor acquisition by larger companies, others licenseout assets to reinvest in research, and some remainindependent, commercializing drugs themselvesor partnering to maximize returns. This diversity EBPs have become the engine of the research–basedindustry, sponsoring the majority of trial starts in recentyears. Pre–commercial EBPs initiated more trials thanlarge companies across all phases, accounting for 66% oftrial starts in 2024, up from 42% in 2015. Trial starts have EBPs have increased their share of trial starts in keytherapy areas, notably diabetes, cardiovascular,neurology, ophthalmology, oncology, infectious Biopharma funding levels havebeen especially dynamic sincethe beginning of the pandemic, China and the U.S. are the most utilized countriesfor EBP trials, with Australia also prominent forPhase I studies. Country selection reflects patient In 2024, EBPs launched 38 drugs in the U.S., eight ofwhich took less than 10 years from first patent filing.The median time across the full cohort of EBP–originatedNAS was 14.3 years, almost 10 months faster than thenon–EBP originated median of 15.1 years. Over the past CLINICAL DEVELOPMENT PRODUCTIVITY EBPs demonstrate higher productivity than largercompanies, driven by less complex and faster trialsdespite lower success rates. Complexity indices for EBPshave been consistently below the industry average, The Clinical Program Productivity Index (CPPI) forEBPs reached 10.6 in 2024, compared to 10.2 for largercompanies. While EBPs have lower success rates, theirlower complexity and shorter durations offset this EBPs are increasingly responsiblefor discovering and patentingspecialty, orphan, oncology, and INNOVATION AND LAUNCHES EBPs originated 570 of the 1,005 global NAS launchesover the past 20 years, with 38 EBP–originated launchesof innovative medicines globally in 2024. EBPs areincreasingly responsible for discovering and patentingspecialty, orphan, oncology, and first–in–class drugs, Notable aspects of trial and program design — suchas compressed trial sequences, single–arm trials,and predictive biomarkers — contribute to shorter First–in–class molecules have accelerated, with108 launched in the past five years, up from 66 in theprior five years, and increasing especially in oncology improvements over the standard of care, though thisis a lower percentage than larger companies. MostEBP–launched drugs are similar to or only slightly COMMERCIALIZATION AND MARKET IMPACT EBPs commercialized 197 of 497 U.S. NAS launchesin the past decade, including 205 drugs through theabbreviated 505(b)(2) pathway. Their share of launcheshas grown, reaching 46% of NAS and 56% of 505(b)(2) SALES UPTAKE AND PROFITABILITY EBP–launched NAS have varied sales upt