Advanced Technologies
AI
The risk-based framework described in a new draft guidance starts with defining the question of interest and context of use and includes development and execution of a credibility assessment plan.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.
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.”
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.
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
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.
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.
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.
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
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.
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.
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 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
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.
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.
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.”
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.