Advanced Technologies

Clinical trial sponsors in the rare disease space face “big safety demands” from regulators, which can be challenging for those with limited resources. A CRO founder makes the case for “embedding automation” into the clinical trial process to keep pace with regulatory requirements.

A UK research team has used artificial intelligence to find new treatments for cancer using existing US Food and Drug Administration-approved medicines that are not normally not used for the disease.

Silicon Valley investor Jim O’Neill’s Senate confirmation hearings showcased the unique background of someone with government and tech experience and avoided discussions of his past statements about lowering the bar for regulatory approval.

Recognizing that the evidence it receives in applications for health technology assessments will increasingly be informed by artificial intelligence, the CDA-AMC has clarified its expectations for companies that use AI methods in the generation and/or reporting of evidence.

Despite following the path of the popular Project Orbis, the cell and gene therapy international collaborative review pilot is being reconsidered by the FDA's new management.

Highlights from day one of the BIO convention include advice for firms hoping to go public, a call for companies to push the US Congress on rare disease priority review vouchers, and updates on next-generation gene therapies.

Madrigal Pharmaceuticals’ resmetirom could become the first approved treatment for non-cirrhotic metabolic dysfunction-associated steatohepatitis in the EU, if the European Medicines Agency issues a positive opinion for the drug later this week.

The Pediatric Advisory Committee will review postmarketing safety for three vaccines and the oncologic drugs panel will consider the return of GSK’s belantamab mafodotin. An FDA notice about a 30 July meeting on Capricor’s deramiocel for Duchenne muscular dystrophy was withdrawn.

Sponsors can expect faster evaluation processes for key medicines and greater support in mitigating medicines supply shortages this year as part of the European Medicines Agency’s digital transformation overhaul.

The European Health Data Space framework will allow companies to accelerate R&D processes and identify new molecular targets faster by facilitating centralized access to certain types of high-quality data, Finland’s Orion Pharma says.

The European Health Data Space Regulation is deliberately “vague” when it comes to defining trade secrets because the EU wants the pharma industry to make recommendations on safeguarding intellectual property, a policy officer for the European Commission says.

Legal heavyweights from Nishith Desai Associates, Anand and Anand, Corporate Law Group and EY partner and national cyber leader talk about compliance requirements that pharma and global capability centers may need to consider as India strengthens the framework for the protection of digital personal data.

Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.

Cellares’ fully automated cell therapy manufacturing platform is the first system to receive an Advanced Manufacturing Technology designation from US FDA

The UK’s drug regulator is developing a “clear and streamlined” regulatory pathway for individualized cancer mRNA immunotherapies.

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.