Regulatory

A quick look at FDA’s 2025 transparency moves, what CRLs are (and aren’t), how FAERS works and its limits, who can report, and where the agency is applying AI.

The designation supports expedited development of SAR402663, Sanofi’s one-time gene therapy designed to reduce treatment burden and slow vision loss in patients with wet age-related macular degeneration.

New guidance and technology advances are reshaping eClinical systems, positioning them as tools to accelerate enrollment, improve data quality, and reduce site and patient burden.

FDA approves COVID-19 vaccines from Moderna, Pfizer-BioNTech, and Novavax targeting the LP.8.1 sublineage of SARS-CoV-2, with eligibility limited to adults aged 65 years and older and those with underlying medical conditions that place them with a high risk for severe disease.

The FDA will now publish adverse event data from FAERS on a daily basis, marking a major step toward greater transparency in drug development.

The Supreme Court has ruled 5-4 to allow the Trump administration’s NIH funding cuts to continue, impacting more than 1,700 medical research grants in areas including heart disease, HIV/AIDS, Alzheimer’s disease, and mental health.

In this episode of the Applied Clinical Trials Brief, we spotlight a recent video interview in which Jon Walsh, founder and chief scientific officer of Unlearn.AI, shared how digital twins can improve trial efficiency, enhance patient-centric designs, align with regulatory expectations, and accelerate access to new therapies.

In this episode of the Applied Clinical Trials Brief, we spotlight a recent video interview in which Jon Walsh, founder and chief scientific officer of Unlearn.AI, shared how digital twins can improve trial efficiency, enhance patient-centric designs, align with regulatory expectations, and accelerate access to new therapies.

With approval to proceed, the Phase II trial (NCT06092034) of RP-A501 for Danon disease restarts under an optimized dosing and immunomodulatory strategy.

An overview of how recent funding reductions are reshaping clinical trial operations, from site sustainability and patient recruitment to trial design and long-term strategy.

Breakthrough Therapy Designation was based on results from the Phase II IDeate-Lung01 trial (NCT05280470), which demonstrated clinically meaningful benefits in patients with extensive-stage small cell lung cancer treated with ifinatamab deruxtecan.

Ron Lanton, partner, Lanton Law, explains how shifts in policy and government guidance could reduce public participation and complicate the design and recruitment of future vaccine trials.

In this video interview, Meri Beckwith, Co-CEO of Lindus Health, shares practical ways clinical teams can strengthen trial protocols for expedited programs—such as incorporating control groups where possible, leveraging synthetic and real-world data, and adopting adaptive trial designs to reduce regulatory risk.

In this Q&A, Rohit Nambisan, CEO of Lokavant, and Jonathan Crowther, head of predictive analytics, Pfizer, explore how AI is transforming study feasibility, regulatory review, and trial execution.

In this video interview, Meri Beckwith, Co-CEO of Lindus Health, discusses whether recent trial design rejections signal a broader shift in FDA expectations or simply reflect changes in individual reviewers, emphasizing that current unpredictability may be more situational than systemic.

In this video interview, Meri Beckwith, Co-CEO of Lindus Health, explains why sponsors pursuing breakthrough or priority review designations must maintain rigorous trial design while building organizational agility.

In this video interview, Meri Beckwith, Co-CEO of Lindus Health, discusses recent Complete Response Letters issued to Replimune and Capricor, explaining how shifts in FDA reviewers can affect single-arm trial acceptance, especially in oncology settings where control groups may be deemed unethical.

Jon Walsh, founder, chief scientific officer, Unlearn, explains how regulators are clarifying best practices for integrating AI and digital twins into clinical research.

In this episode of the Applied Clinical Trials Brief, we recap our three most-viewed stories of the previous week with a look into the FDA’s heightened scrutiny of trial design, the wind-down of federal mRNA vaccine programs, and how digital innovation is reshaping the clinical research landscape.

In this video interview, Meri Beckwith, Co-CEO of Lindus Health, examines how recent developments with Complete Response Letters have shifted industry perceptions of FDA breakthrough designation—highlighting that it offers faster review timelines and investor visibility, but not a higher likelihood of final approval.

A unified technology approach improves study efficiency and data collection.

In this video interview, Luke Wilson, senior director, biotech, pharma services at Thermo Fisher Scientific, discusses how integrated systems and data transparency enable sponsors to meet evolving regulatory expectations and streamline FDA inspections through enhanced operational readiness.

As clinical trials become more complex, success depends on early collaboration, thoughtful protocol design, and practical strategies to manage risk, reduce delays, and support patients.

In this video interview, Michael Miller, chief operating officer at Quanterix, discusses how biopharma companies—especially smaller biotechs—can leverage platform trials and biomarker-driven accelerated approvals to remain efficient and competitive amid shifting public-private funding dynamics.

In this pilot episode of the Applied Clinical Trials Brief, we examine the renewed push for placebo-controlled vaccine trials in the US, why experts warn it violates core ethical standards, and how these proposals could jeopardize both participant safety and future innovation.