Clinical Trials/R&D

Securing a place on the European Medicines Agency’s priority medicines scheme is not easy, but some companies have managed to make the grade more than once.

FDA’s Center for Biologics Evaluation and Research saw IND clinical holds for gene and cell therapies dropped to 70 in calendar year 2022, a 52% decline from 2018. Center for Drug Evaluation and Research’s total clinical holds in fiscal year 2022 were 380, the highest number in the past 12 years.

However, FDA officials appear skeptical that imaging-based and circulating biomarkers are ready for prime time when it comes to supporting accelerated approval of drugs for noncirrhotic nonalcoholic steatohepatitis.

Proposals on regulatory data protection and transferable exclusivity vouchers to encourage antimicrobial R&D have received mixed reactions from the parliament’s rapporteurs for the draft legislation.

In a Pink Sheet interview Chief Scientist Bumpus discusses need to move diversity conversation up further in the pipeline. Better ‘organs on a chip’ should help.

US FDA’s biologics center intends to ask for volunteers to help demonstrate the concept, modeled on the COVID vaccine effort, which includes speedier communication with sponsors to expedite development of products for rare diseases.

The new designation could streamline development of multiple products that use the same backbone, said Nicole Verdun, director of the US FDA’s Office of Therapeutic Products.

Governments should increase financial resources for novel antimicrobial drug candidates in the later stages of development and tackle shortages of existing, underused antibiotics to combat antimicrobial resistance, says a new report from the OECD.

Pink Sheet reporter and editors discuss the somewhat novel pricing language in Regeneron’s deal with the US government to develop a COVID-19 monoclonal antibody, US FDA Commissioner Robert Califf hiring a new senior advisor, and optimizing dosing in cancer combination therapies.

The evidence is growing that comparative efficacy trials may not be necessary for all biosimilars, but regulators said they need a standard for sponsors to meet in order to waive them.

‘We’ve got these enormous markets,’ Rob Califf says. ‘Should 65% of the US population be treated with obesity drugs?’

Narrow, restrictive, unclear, ambiguous, challenging to achieve, and likely to hamper innovation and deter R&D investment. Just some of the comments that have been leveled at EU proposals for a formal definition of unmet medical need.

FDA initiative aimed at optimizing cancer drug dosage earlier in development started with single-agent therapies, but moving into combinations is ‘a natural progression,’ Oncology Center of Excellence’s Richard Pazdur says. At a recent workshop, experts discuss considerations for combination product dosage optimization and review case studies that reflect Optimus principles.

An expert panel formed by the Council of Canadian Academies says drug developers that provide “novel, valuable antibiotic solutions” should be rewarded with a “fair and reasonable guaranteed revenue” for their drugs.

As FDA clears new COVID vaccines, the Regeneron deal is a reminder of the politics of drug pricing. Sen. Sanders touts the agreement, which attaches product price caps to funding for clinical development, and he allows the confirmation effort for the NIH director nominee to proceed.

Pink Sheet reporter and editors discuss the US Federal Trade Commission ending its attempt to block the Amgen acquisition of Horizon Therapeutics, a proposed public-private partnership to develop ultra-rare cancer drugs, and a slow-down in ANDA submissions to the US Food and Drug Administration.