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 efficientlyas possible, biotechs can make the best use of their resources, putting expertise andfunding behind the products most likely to succeed in the market. 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$1.5 billion, respectively. We also held in-depth conversations with vice presidentsand C-level executives at 10 U.S. and European biotechs. Through this research, we’ve identified the strategies biotechs believeare most important to their success as well as the challenges that mostsignificantly impact them. Research methodology decision makers surveyed120 small and mid-sized biotechs(R&D spend up to $1.5B) in-depth executive interviews U.S. and European companies When asked aboutthe circumstancesshaping biopharmadevelopment, the10 biotech leaderswith whom wespoke at lengthnamed the sametrends andchallenges identifiedin our larger survey. Figure 1 Trends or challenges most impactingclinical development, accordingto 10 biotech leaders. In addition to anoverview of ourfindings, this reportoffers insights intothe strategies onthe rise amongbiotech innovators. Global expansion:Capitalizing on opportunitiesoutside of the U.S. and Western Europe 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 intoother global regions. This allows biotechs to: Select regions based onregulatory speed 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. Reduce spending inlower-cost regions 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 interestedin locating studies in: Secure access to lesscompetitive sites China, where a large general population often leads to faster patientrecruitment. The cost of running a trial in China is also significantlylower than in the U.S. 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 tosupport biopharma development. Survey respondents indicate regions of interest for clinical development expansion. 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 CROsthat have staff in these regions as they were hesitant to execute trials in a country in whichthey had no local presence. 70% In addition, survey responses point to a clear knowledge gap around region-specific regulatoryand submission requirements. We found that mid-sized companies often need moreinformation on which regions are best suited to specific therapeutic areas or indications. of biotech leaders say acceleratedregulatory pathways have high orsignificant impact on clinicaldevelopment timelines 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 likelihoodof trial design modifications and allow biotechs to prioritize studies with the fastestpotential path to market. 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,which is important,” one U.S. C-level leader told us. “And we’re evaluating all these[additional regulatory] approaches to see if we can really utilize all [of them].”Like this executive, many biotech decision makers seek to combine acceleratedpathways when possible. Survey responses indica