切割时间，而非角落：通过监管效率加快临床准入

Accelerating clinical entry through regulatory efficiency JEAN DEHDASHTI,MS, RAC, Manager, Regulatory Science and Strategy, Research & Development Solutions, IQVIAREID D’AMICO,PhD, Sr. Principal, Regulatory Science and Strategy, Research & Development Solutions, IQVIAJENNY FAM,Sr. Director, Drug Development and Regulatory Strategy, IQVIA Table of contents Executive summary3Introduction: The case for leaner, smarter evidence3A framework for regulatory efficiency4Key principles4Area of focus5Phase-appropriate, risk-based examples6Operational readiness: making ‘lean’ work6Faster, more efficient clinical entry6Conclusion6References7 Executive summary Introduction The case for leaner, smarter evidence Traditional development programs often accumulatestudies out of caution or convention, ratherthan demonstrable regulatory need. In today’s Leaner evidence plans can help de-risk timelines by: 1.Avoiding duplicative non-clinical investigationswhen well-characterized platforms or prior- 2.Replacing or re-sizing studieswith validatedmodeling, simulation, or in vitro/in silico methods Sponsors face increasing pressure to reach the clinicfaster, but on restrictive budgets. By prioritizingmore informative studies, cost savings are maximizedwithout compromising patient safety or regulatory 3.Designing clinical programs that maximizelearning per patientthrough adaptive features, Increasingly, regulatory agencies expect sponsorsto justify not only what studies are conducted, butwhy they are needed at a given point in development.IQVIA supports sponsors by reframing development The approach leverages prior knowledge and historicaldata, robust scientific rationale, and innovativemethodologies (e.g., modeling and simulation, The result: earlier first-in-human (FIH) trials, streamlinedprogression across clinical phases, and lower totaldevelopment costs through transparent engagement Sponsors face increasing pressureto reach the clinic faster, but onrestrictive budgets. By prioritizingmore informative studies, cost A structured, question-based assessment of everyproposed study is central to this approach. For eachnon-clinical or clinical activity, sponsors shouldevaluate whether the study materially contributes to Key principles A framework for regulatory •Regulatory intelligence (know what’s necessaryand what’s not):Monitor global precedents,guidance documents, and reviews to identify where Sponsors often submit regulatory packages to FDA withextensive testing and study designs based on historicalprecedent and perceived agency expectations forspecific modalities and patient populations. Drawing on •Regulatory strategy (align business and benefit–risk): Translate intelligence into a plan thatevaluates planned studies, explicitly tying eachproposed study to the regulatory question it isintended to answer, patient safety considerations,and program objectives. Application of a consistent, structured decisionframework to evaluate each proposed study againstregulatory expectations and overall developmentobjectives supports fit-for-purpose evidence •SME-based pre-clinical and clinical expertise: Apply the expertise of former FDA/NIH professionalsskilled at strategizing minimum phase-appropriate •Evidence planning (fit-for-purpose, phase-appropriate): Build an integrated evidenceplan that right-sizes non-clinical and clinical activitiesby phase,ensuring that each study has a clear role The key principles outlined below clarify how thisframework is applied in practice. These define theregulatory intelligence, strategic planning, expert •Regulator interaction (engage early and often,document transparently): Secure alignmentvia early scientific advice meetings with briefing We recognize that many pre-clinicalstudies, clinical assays, and trialdesigns might be redundant, provide Similarly, the focus areas below translate theseprinciples into the specific domains where disciplined,phase-appropriate decision-making is most critical Faster, more efficientclinical entry Phase-appropriate,risk-based examples The examples illustrate how applying explicitregulatory and development questions can enable Time to FIH: Months saved by deferring orsubstituting low-value non-clinical work. Protocol optimization: Streamlining designs,reducing patient burden, minimizing Non-clinical study minimization: evidence that canbe applied across a platform and exposure-drivenbridging supported by Physiologically Based Evidence economics: Cost avoided by leveraging Clinical acceleration: Seamless Single AscendingDose (SAD)/Multiple Ascending Dose (MAD) designs,adaptive Phase 1b/2 protocols, master protocols, and Regulatory clarity: Number of key uncertainties Patient protection metrics: Event-driven Sponsors can responsibly minimizeregulatory study requirements andaccelerate time to clinic by applying Conclusion Sponsors can responsibly minimize regulatory studyrequirements and accelerate time to clinic by applyingdiscip