更快的概念验证路径：生物技术公司如何加速发展

Accelerating clinical development is a priority across the biopharma industry, but thepressure for speed is often most intense among small and mid-sized biotechs. Whiletheir leanness allows them to respond with agility to shifting landscapes, it also makesthem susceptible to significant financial risk. By reaching proof of concept as efficiently Recently, Parexel asked biotech leaders across the U.S. and Europe about strategiesto accelerate their development work. We surveyed 120 decision makers at smalland mid-sized companies with an annual R&D spend of up to $200 million and Through this research, we’ve identified the strategies biotechs believeare most important to their success as well as the challenges that most Research methodology When asked aboutthe circumstancesshaping biopharmadevelopment, the10 biotech leaderswith whom we Trends or challenges most impactingclinical development, accordingto 10 biotech leaders. In addition to anoverview of ourfindings, this reportoffers insights into Global expansion:Capitalizing on opportunities Why biotechs are expanding globally Increase accessto patients While the U.S. and Western Europe continue to be centers for biotech development,regulatory uncertainties and slower startup times are pushing sponsors to expand into increase their access to patients. select regions based on speed of regulatory processes and operational execution. reduce spending by working in lower-cost regions. secure access to sites with less competition. According to our survey, small and mid-sized biotechs are currently prioritizingAustraliafor early-phase trials, thanks to relatively short regulatory timelinesand ample access to key opinion leaders. Biotech leaders are also interested China, where a large general population often leads to faster patientrecruitment. The cost of running a trial in China is also significantly South Korea, a scientifically and technologically advanced countryin which regulators are often aligned with the FDA. Japan. Conducting trials in Japan allows biotechs to prepare forcommercial opportunities in the country and protect future value. Eastern Europe, as countries in this region are eager to As biotech leaders consider their options for expansion, they need greater understanding ofregion-specific operational and logistical challenges as well as broader insight into patientrecruitment strategies and enrollment rates. Many were also interested in working with CROs In addition, survey responses point to a clear knowledge gap around region-specific regulatoryand submission requirements. We found that mid-sized companies often need more 70% of biotech leaders say acceleratedregulatory pathways have high or Regulatory strategy:Pursuing accelerated pathwaysand engaging in scenario planning as early as possible In our research, 70 percent of survey respondents aligned on accelerated regulatorypathways as a major driver of faster development. Within the FDA in particular,these pathways shorten overall review and approval timelines, reduce the likelihood Most biotech leaders we spoke with pursue accelerated regulatory pathwayswhenever appropriate — most commonly through the FDA’s BreakthroughTherapy or Orphan Drug Product Designations. “Once we have clinical data [tosupport our position], we’ll certainly apply for Breakthrough [Therapy] Designation, Survey responses indicated that biotechs need to better understand howmuch time can be saved through specific regulatory programs as well asregulatory designations and programs for specific therapeutic areas or To ensure alignment on approval pathways, the leaders we surveyed areincreasing their proactive engagement with regulatory authorities —interactions that also help hold regulators to agreed-on timelines andstandards. Taking initiative with regulators will continue to be critically When asked about challenges regarding regulatory strategy,biotech leaders were most concerned about the variabilityand complexity of regulations across global regions as wellas the perceived difficulty of navigating evolving regulatory Operational innovation:Leveraging adaptiveand seamless study design, RWE and AI According to our research, biotechs areincreasingly exploring the use of innovativetrial designs and statistical approaches.Biotech leaders are particularly interested ininnovative methodologies that allow them to about synthetic or external control armsthat can reduce patient enrollment needs.Regulatory uncertainty and lack of clear The leaders we spoke with and surveyedalso cited real-world evidence (RWE) as oneof the factors most influencing innovative inform endpoint selection and trial When asked about using Bayesian or otherstatistical models to extract rich informationfrom small sample sizes, biotech leadersacknowledged that this approach can beespecially valuable for rare disease researchwith extremely small patient populations. replace control arms in studies with support and potenti