Can the decentralized global healthcare ecosystem to turn real-world evidence from a buzzword to a useful approach to fit-for-purpose evidence generation?

Agency offers three informal ‘buckets’ organized around data, study design and tools.

The first BCMA-targeting agent to gain approval in myeloma never became the blockbuster for which GSK had hoped, but Blenrep appears to have set a new standard for speed of voluntary withdrawal of an accelerated approval drug after a confirmatory trial failure.

Revised labeling requirements for unauthorized investigational and auxiliary medicinal products under the EU Clinical Trial Regulation have been published in the Official Journal.

Access to expedited regulatory pathways, marketing exclusivity and tax credits in exchange for enrolling and retaining diverse clinical trial populations could provide a ‘carrot’ to the ‘stick’ in US FDA guidance and legislative proposals; attorneys Sarah Thompson Schick and Winston Kirton also call for a re-examination of concerns about remuneration for trial participants.

CBER Director Peter Marks said more sophisticated manufacturing expected in the next decade could lead to questions about whether gene therapies should be regulated by his center or CDRH.

ANVISA, Brazil’s medicines regulator, is asking for feedback on the regulatory pathway for biosimilars.

Key Republican committee chairs on health issues are familiar faces, largely supportive of the US FDA’s approach to drug regulation. Implementation of the new pricing law, however, will get a lot more scrutiny.

Research institutions, CROs, biopharma industry and other stakeholders are asked to comment on potential governance structure that would determine when trials are needed to address disease outbreaks and other public health emergencies.

US FDA’s Theresa Mullin envisions having a national catalog of standard core clinical outcomes assessment measures that could apply to every disease area.

The European Medicines Agency’s newly established Quality Innovation Expert Group will focus on innovative pharmaceutical manufacturing approaches, starting with continuous manufacturing for biologicals and end-to-end processes, and decentralized manufacturing.

OCE Director Richard Pazdur says idea for the latest initiative stemmed from a clinical trial in lung cancer that he thought only needed to measure the impact on overall survival.

David Ricks says the challenging political environment is empowering regulators. The Lilly leader also talked Twitter and drug prices, in a STAT Summit interview.

Pharma CEOs must balance optimizing access and profitability, former Merck CEO and soon-to-be ex executive chairman of the company said. Frazier says health care industry must come to table and admit the “system ain’t working,” talks about the need to protect democracy, in a sweeping interview at the STAT Summit.

Harm Reduction Therapeutics submits NDA for OTC approval of a 3-mg naloxone nasal spray branded RiVive. CEO Michael Hufford says the NDA touches all the bases FDA detailed in notice it published on what’s needed in naloxone OTC switch proposals.

CBER Director Peter Marks said more sophisticated manufacturing expected in the next decade could lead to questions about whether gene therapies should be regulated by his center or CDRH.

The European Medicines Agency’s newly established Quality Innovation Expert Group will focus on innovative pharmaceutical manufacturing approaches, starting with continuous manufacturing for biologicals and end-to-end processes, and decentralized manufacturing.

The European Medicines Agency and the international association of pharmaceutical inspectorates, PIC/S, say they need to make extensive changes to their guide on using computerized systems when manufacturing medicines.