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Clinical Trials/R&D

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Preparing For The Next Pandemic: Experts Talk AI Vaccines, Regulatory Acceptance & Earlier Human Trials

AI-generated vaccines, fast-moving regulatory systems and scalable manufacturing platforms will be key in preparing for future pandemics, according to vaccine and immunology experts including the CEOs of CEPI and Gavi.

BioPharmaceutical International

US FDA’s Cell-Gene Therapy Office Head Wants Sponsors To Seek Out Meetings

Companies should talk to FDA staff in between formal meetings if they need clarity on agency advice that is several years old or development programs hit a snag, Office of Therapeutic Products Director Nicole Verdun says.

Gene Therapy Clinical Trials

US FDA Commissioner Robert Califf Says No Formulas For Trial Diversity

The FDA does not appear likely to provide a calculation for sponsors to show clinical trial diversity, but agency officials seem split on how tough to enforce the new regulations intended to ensure all groups are adequately represented.

Diversity & Inclusion BIO

ICH Cuts Burden Of Assessing Drug-Drug Interactions; Argentina Gains Membership

New guidance from the International Council for Harmonisation is designed to tackle problems companies face by having to meet different requirements around the world when evaluating the potential of DDIs for their investigational products.

International Drug Safety

Switzerland Adopts Changes To Human Research & Clinical Trial Regulations

Updated clinical trial reporting rules and stronger participant protections are among the several changes to research in Switzerland that will apply from November, while new regulations on clinical trial transparency will apply from March 2025.

Europe Switzerland

EU Launches New Mechanisms To Boost Quality Of Clinical Trial And Marketing Applications

As of today, drug developers can apply to take part in two pilot programs that will involve regulators across the EU strengthening their coordination when it comes to providing companies with advice on their applications for clinical trials and marketing authorization.

Europe Clinical Trials

AI Assurance Lab Concept Leaves Potential Regulatory Gap

In an interview with the Pink Sheet, Troy Tazbaz, director of the US FDA's Digital Health Center of Excellence, says the agency lacks authority to regulate assurance labs, which would be used to help AI developers ensure their models are working correctly before submitting them for approval.

Artificial Intelligence Research & Development

EMA Stresses Importance Of Scientific Advice After Requests Drop 17% In 2023

The European Medicines Agency has encouraged companies to seek “guidance and direction” early in the development process after it reported a significant fall in the number of companies that sought scientific advice and protocol assistance compared with the three previous years.

Europe EU

Immuno-Oncology Add-On Trials: More May Not Be Better, US FDA Cautions

Oncology developers should ensure clinical development plans involving early-stage trials adding on to perioperative treatment regimens will show that new therapies are adding benefit in each phase, Oncology Center of Excellence Director Richard Pazdur said.

Cancer Clinical Trials

England’s Genomics Service Working With Industry On Precision Medicine & Personalized Vaccines

England’s chief scientific officer explains how the NHS Genomic Medicine Service has led to faster access to targeted cancer drugs and outlines the organization’s ongoing work in pharmacogenomics, liquid biopsies and personalized RNA vaccines.

Europe United Kingdom

‘Threading The Needle:’ Finding A Regulatory Foundation For Ultra Rare Disease Development

Sometimes even the US FDA’s regulatory flexibility may not be enough to approve rare disease treatments in very small populations, but agency officials worry that creating a new pathway could be problematic.

Rare Diseases Drug Approval Standards

Gene Editing And Cell Therapy: US FDA Still Wants Careful Characterization

The US FDA does not seem ready to depart from its safety-focused emphasis on product characterization as cell therapies become more complex, despite calls for approaches to allow human studies that let different edits “compete” and “find the winners inside the human tumor.”

Clinical Trials Gene Therapy

C3TI's Mission: Leverage FDA Trial Innovation Expertise, Educate Inspectors

The Center for Clinical Trial Innovation was created to be CDER’s hub for expertise and information on innovative clinical trial approaches. C3TI lead Kevin Bugin talks to the Pink Sheet about the program's genesis and what it hopes to accomplish.

Clinical Trials Real-World Evidence

How Pharma Can Use England’s Genomics Data To Improve Regulatory Filings & Trial Design

A real-world data research partnership involving Genomics England is helping pharma companies to continuously draw “more insights” from genomic information to improve their regulatory submissions and optimize trials.

Europe BioPharmaceutical

US FDA Drug Center Tries New Approach To Old Consistency Problems

Drug developers should not expect the US FDA's new Quantitative Medicine Center of Excellence to dramatically impact the agency's long-standing problem of ensuring more consistent use of innovative regulatory approaches in application reviews.

Clinical Trials Review Pathway

NGGT Progresses First US Gene Therapy Trials For Rare Conditions

Next Generation Gene Therapeutics is fast-tracking clinical trials in the US for its gene therapy candidates for Bietti's crystalline dystrophy and classic phenylketonuria, supported by China data, the Chinese venture's CEO says in an interview.

China Clinical Trials
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