AI


Artificial Intelligence: US FDA Outlines 7 Steps To Establishing Model Credibility

 
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The risk-based framework described in a new draft guidance starts with defining the question of interest and context of use and includes development and execution of a credibility assessment plan. The guidance is limited to AI models used to support regulatory decisions about drug safety, effectiveness or quality.

Boosting Reliability: EMA Backs AI Tool For MASH Trials

 

An artificial intelligence-based pathology tool for metabolic dysfunction-associated steatohepatitis shows promise for a drug development landscape that is said to be “fraught with trials that have shown borderline results or outright failures based on liver histology.”

Real-World Evidence Improving, Not Replacing, Traditional Studies

 

As RWE advances, a multiplicity of roles are emerging, but case studies show how it is more effective filling evidence gaps during drug development and improving surveillance than creating a randomized controlled trial alternative.

AstraZeneca: AI Regulations ‘Tricky’ To Interpret & Navigate

 

The pharma industry wants regulators around the world to engage with companies and “articulate the value added” when introducing new regulations and guidance around the use of AI in drug development, AstraZeneca’s director for data and AI policy says.

Digital Technologies


‘Ambitious Platform’ Among EU Proposals For Increasing mHealth Data Use In Regulatory Decision-Making

 

mHealth data generated by smartphones and wearables show potential for enhancing the clinical evidence used in regulatory decision-making, but there are “notable challenges” that may hinder the use of such data, EU regulators say.

Decentralized Trials: FDA Final Guidance Eliminates Need For Local Provider Activity Tracking

 
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Draft recommendation for investigators to maintain a task log of activities that local health care providers perform was removed from final guidance on decentralized clinical trials. Other changes include more detail on US FDA oversight and what HCPs can and cannot do in such studies.

Pharma Warming Up To Benefits Of Australia’s Teletrials Initiative

 
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The Australian teletrials program has surpassed expectations at its mid-way point and is being used by drug and device sponsors looking to enhance trial participant recruitment and retention by enabling access to rural, regional and remote areas.

EFPIA Calls For Active Inclusion Of Pharma Industry In Roll Out Of European Health Data Space

 

The implementation phase of the European Health Data Space is an opportunity to address “critical aspects” of the new regulation that require clarification, says pharmaceutical industry federation EFPIA.

Cell & Gene Therapies


Innovation Drives Divergence: US FDA Drugs and Biologics Centers Follow Own Trajectories

 

Regenerative medicines using expedited review pathways dominate novel approvals at the Center for Biologics Evaluation and Research, while the Center for Drug Evaluation’s higher volume comes with lower first-cycle approval rates and more standard reviews.

Why 2025 Could See A Surge In EMA Approval Opinions For ATMPs

 

Marketing authorization applications for five new advanced therapy medicinal products were filed with the European Medicines Agency in 2024, the highest number in any year since 2020. The Pink Sheet looks at these and two other ATMPs that might get the thumbs up from the European Medicines Agency in 2025.

Shorter Gene Therapy Postmarket Studies ‘On The Table’

 

Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.

China Greenlights First Stem Cell Therapy On Mixed Clinical Findings

 

China has granted its first approval for a stem cell therapy, to domestic firm Platinum Life Biotech’s umbilical cord-derived mesenchymal stem cell product for corticosteroid-failed acute graft-versus-host disease with gut involvement.

Platform Technologies


‘Ambitious Platform’ Among EU Proposals For Increasing mHealth Data Use In Regulatory Decision-Making

 

mHealth data generated by smartphones and wearables show potential for enhancing the clinical evidence used in regulatory decision-making, but there are “notable challenges” that may hinder the use of such data, EU regulators say.

US FDA Not Ready For Sponsors Citing Others’ Platforms Yet, Marks Says

 

Many platform designation requests have been from sponsors eager to cite other sponsors’ products, but CBER Director Peter Marks said in an interview with the Pink Sheet his office likely is years away from accepting those applications.

Genome Editing: US FDA Eyes Platform-Based Approach To Development And Approval

 
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CBER Director Peter Marks outlines a streamlined process to approval for treating different mutations of the same gene. NCATS’ Philip Brooks tells the Pink Sheet the approach avoids the need to “start from scratch for every new mutation.”

Platform Tech Designation: US FDA Emphasizes “Significant Efficiencies” Criteria In New Guidance

 

New draft guidance defines the necessary components of a platform technology designation request, including that sponsors demonstrate how it will save the FDA review time.