In this video interview, Mark Freitas, managing director and life sciences practice lead at Alvarez & Marsal, makes the case for integrating reimbursement probability into the standard PTRs framework earlier in development, and what happens when sponsors reach approval without a viable value story.
Clinical trial delays often originate in early site selection decisions, where misalignment between protocol demands and site capabilities undermines startup, enrollment, and data quality despite later efforts to correct course.
FDA gives granted accelerated approval to lunsotogene parvec (Otarmeni) for OTOF-related sensorineural hearing loss based on early CHORD trial results in children.
Oral semaglutide lowered HbA1c vs placebo in a phase 3 trial in adolescents with type 2 diabetes, pending full data and review.
In this video interview, Mark Freitas, managing director and life sciences practice lead at Alvarez & Marsal, discusses how crowded pipelines, compressed IP timelines, and competitive differentiation pressures are forcing smaller sponsors to treat protocol design as a core element of business strategy.
In today's ACT Brief, we explore organizational factors behind CRL decisions, methodological concerns with pooling amyloid antibody trial data, and how compounding addresses care gaps when commercial options fall short.
In this video interview, Mark Freitas, managing director and life sciences practice lead at Alvarez & Marsal, explains why the most consequential shift in clinical trial design is not methodological but organizational, and what an analysis of 200 FDA complete response letters reveals about where decisions are going wrong.
A systematic review pooling data from 17 clinical trials—including 15 involving failed or withdrawn therapies—concludes that amyloid-targeting antibodies offer little clinically meaningful benefit, prompting widespread pushback from Alzheimer's researchers.
In today's ACT Brief, we explore practical community engagement strategies that drive representative enrollment, how sponsors should structure development plans around the plausible mechanism framework, and the organizational prerequisites for scaling AI effectively in drug development.
In this Q&A, Mwango Kashoki, MD, MPH, SVP and global head of regulatory strategy at Parexel, breaks down the FDA's plausible mechanism framework and what it means for sponsors developing individualized therapies in ultra-rare disease settings.
In this video interview, Del Smith, PhD, CEO and co-founder of Acclinate, outlines what a practical, sustained community engagement framework looks like in practice and how sponsors can translate that investment into faster, more efficient, and more representative enrollment.
In today's ACT Brief, we explore practical steps sponsors can take to prepare for the plausible mechanism framework, how behavioral science addresses root causes of recruitment and diversity challenges, and FDA acceleration of psychedelic therapy reviews through executive order.
Behavioral science reveals how recruitment failures, site disengagement, and underrepresentation in clinical trials are rooted in early design decisions, and what sponsors can do to address them before they become costly problems.
In this video interview, Mwango Kashoki, MD, MPH, senior vice president and global head of regulatory strategy at Parexel, outlines the steps sponsors can take today—from early FDA engagement and robust non-clinical programs to adaptive trial designs and confirmatory evidence packages—to avoid delays as the guidance moves toward finalization.
In today's ACT Brief, we explore how disease heterogeneity confounds natural history controls, the execution translation gap converting identified problems into action, and policy recommendations for strengthening FDA's accelerated approval pathway.
The execution translation gap—the failure to convert identified problems into coordinated, timely action—costs millions per trial through delayed amendments, persistent deviations, and slow site activation, yet remains addressable through aligned accountability and proactive execution management.
In this video interview, Mwango Kashoki, MD, MPH, senior vice president and global head of regulatory strategy at Parexel, discusses how heterogeneity in disease course and patient characteristics creates confounding risk when using natural history data as an external control, and what sponsors need to do to achieve meaningful patient matching.
In today's ACT Brief, we explore biological and dosing considerations for basket trial designs, how human-relevant data earlier in development reduces late-stage failures, and FDA's shift toward single-trial approvals supported by real-world evidence.
In this Q&A, Jenna DiRito, PhD, COO and co-founder of Revalia Bio, discusses how earlier, human-relevant data is reshaping go/no-go decision-making in drug development and what a truly front-loaded workflow looks like in practice.
In this video interview, Mwango Kashoki, MD, MPH, senior vice president and global head of regulatory strategy at Parexel, examines the biological, dosing, and population-level considerations that sponsors must get right from the start when using basket and other master protocol designs to evaluate individualized therapies across multiple conditions.
In today's ACT Brief, we explore how FDA evaluates effectiveness for ultra-rare individualized therapies, the agency's enforcement push for mandatory trial result reporting, and Obsidian's merger backed by $350 million to advance next-generation TIL cell therapy.
Agency outreach targets a compliance gap affecting thousands of registered trials, with nearly 30% of studies subject to mandatory reporting showing no results submitted to ClinicalTrials.gov.
In this video interview, Mwango Kashoki, MD, MPH, senior vice president and global head of regulatory strategy at Parexel, breaks down how the FDA evaluates substantial evidence of effectiveness for individualized therapies in ultra-rare conditions, and why that determination depends on the totality of mechanistic, biomarker, and clinical outcome data rather than trial numbers alone.
In today's ACT Brief, we examine how protocol interpretation and source document preparation delay study startup, how the plausible mechanism framework accelerates individualized therapy timelines, and how real-time tracking technologies improve supply chain and sample integrity in decentralized trials.
A collaborative study by the Tufts Center for the Study of Drug Development and CRIO identifies protocol interpretation and source document preparation as an understudied yet significant bottleneck in study start-up timelines that may hold key opportunities for efficiency gains.
In this video interview, Mwango Kashoki, MD, MPH, senior vice president and global head of regulatory strategy at Parexel, explains how the plausible mechanism framework reshapes the development timeline for individualized therapies by allowing first-in-human studies to serve as pivotal trials and giving sponsors earlier clarity on the evidence needed for approval.
In today's ACT Brief, we examine Thermo Fisher's expanded real-world data access through HealthVerity, how FHIR-based standards are finally enabling scalable eSource implementation, and FDA's second complete response letter for Replimune's oncolytic immunotherapy.
New agreement provides enterprise-level access to claims data covering more than 270 million de-identified patient lives, supporting trial feasibility, recruitment, and real-world evidence generation.
Sponsored Content
In this video interview, Nick Frenzer, general manager of site solutions at Veeva Systems, discusses why eSource has been difficult to implement at scale, how FHIR-based technology is changing that, and what Veeva eSource means for sites, sponsors, and the broader clinical trial ecosystem.
In today's ACT Brief, we explore industry perspectives on operational and methodological shifts defining 2026, how agentic AI will augment clinical trial roles over the next two years, and Roche's expanded partnership with C4 Therapeutics on next-generation antibody conjugates.
