In year two, Humira biosimilar uptake – or the lack thereof – continues to build the case for policy reform to incentivize use of cheaper biologics in the outpatient setting.

The streamlined process is expected to shorten the turn-around for the CAR-T therapy to about the same time as from an office visit to front-line cancer treatment, Kite Pharma said.

While at least 25 accelerated approval cancer indications or drugs have been pulled or requested withdrawn since 2020, there has not yet been a similar concerted regulatory effort in the non-oncology space despite a large number of products with overdue postmarketing requirements.

Of 11 NMEs and novel biologics, only one – a vaccine for a tropical disease – did not have confirmatory studies underway or completed at the time of accelerated approval. Statutory changes are forcing companies to prioritize confirmatory trials earlier than in the past.

International interest in fast-track regulatory reliance and collaboration pathways such as Project Orbis and the ACCESS Consortium has grown in recent years, a trend that looks set to continue in 2024. But some policy experts have warned that faster approvals are not always better, with one academic claiming the bar for marketing authorizations has dropped “far too low.”

Sixteen cancer therapies have been granted marketing authorizations or label variations in the US this year via the Project Orbis scheme. However, the majority of these are still under review by Orbis partners, which have also been outpaced by the non-Orbis EU.

European Union reform proposals could reduce ‘clock stop’ time in drug reviews, which a new study identified as the primary driver of longer review times at the EMA compared with the US FDA.

Regulators from Canada, the UK and Australia discuss their experiences of working together under both the ACCESS Consortium, an international regulatory worksharing coalition, and Project Orbis, a parallel review process designed to speed up access to oncology medicines.

Some states are adding more ‘muscular attributes’ to their Rx boards, such as setting upper limits based on the pending Medicare prices, but federal ERISA legislation still limits the boards’ impact.

Commissioner Califf says the technology exists and the idea would allow the agency to be more nimble, but the Paperwork Reduction Act makes the process difficult.

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered. 

An updated priority pathogen list, a review of the clinical pipeline for antibacterials, and high-level meetings with other global regulators are among the steps that will be taken by the World Health Organization in the year ahead to combat antimicrobial resistance.

The WHO sees the HTAP as an important step in securing more equitable access to a wide range of health products by sharing intellectual property and scientific knowledge. But the pharmaceutical industry wants to see more detail and has warned against a global “one-size-fits-all approach” to the licensing of medicines and vaccines.

Biogen’s decision to discontinue is Alzheimer’s treatment Aduhelm cements its status as one of the most spectacular commercial failures in biopharma history. But its impact on biopharma policy is still extraordinary.

FDA Commissioner is ‘pretty excited’ about obtaining high quality evidence in the postmarket space, noting agency's collaboration with NIH. He cites need to address the low cost of generic drugs as a factor in drug shortages.

Separate, and higher, payments outside the bundle means manufacturers will owe Medicaid rebates, and that is OK, Alliance for Regenerative Medicine tells Sen. Cassidy. But manufacturers want assurances they won't be liable for rebates on treatments included in bundled payments.

Revised document on best surveillance practices for US FDA staff deletes some product categories subject to more extensive monitoring. It also no longer recommends focusing on adverse events reported in a specific population.

Patients in England with relapsed or refractory diffuse large B-cell lymphoma will have another treatment option after the health technology appraisal institute NICE recommended AbbVie’s bispecific antibody Tepkinly for funding on the National Health Service . The product is also undergoing a benefit assessment in Germany.

Stimulating the development of novel antibiotic drugs and diagnostics will require new funding models as part of the broader “push and pull” incentives strategy, says the director of health & life sciences at Innovate UK.

Outcomes-based contract that federal government will negotiate with manufacturers on behalf of Medicaid programs might not be appealing to large states. Bluebird and Vertex can apply to the demonstration project this spring and negotiations would follow between May and November.