Seven panelists are regular members of the Cellular, Tissue and Gene Therapies advisory committee, while many of the 13 temporary voting members bring neurology and neuroscience expertise. In a conflict-of-interest waiver, the agency noted a struggle to find experts in clinical neurology and neurodegenerative diseases who lack disqualifying financial interests.

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.

Pink Sheet’s Drug Review Profile looks at the timeline behind the development and FDA review of CellTrans’ treatment for type 1 diabetes.

A report comparing the health technology assessment methods used in nine European countries, Australia and Canada found that England has the most favorable reimbursement landscape for gene therapies – but outlined several areas for improvement across the board.

The median approval time for new drugs in Switzerland lengthened in 2022, mainly because of applications filed during the peak of the COVID-19 pandemic, says the country’s regulator.

A newly-translated guidance document from Japan’s drug and device regulator outlines the points that clinical trial sponsors should consider when using electronic methods to collect informed consent from participants.

Rhythm is working with country-level authorities and centers of excellence in Europe to gain reimbursement and access for its obesity treatment Imcivree.

Nonclinical testing questions should be answered in a pre-IND meeting, rather than the new Type D session and indicated support for alternatives to animal testing when possible, a US FDA Center for Biologics Evaluation and Research official suggests.

Drug manufacturing in the EU would grind to a halt in under three years if a proposal to restrict per- and polyfluoroalkyl substances in the European Economic Area is implemented in its current form, says industry group EFPIA.

Users of the new catalogs will benefit from a modern technology with enhanced view, search, export and data submission functionalities, says the European Commission.

Convincing payers to fund a $3m therapy is no mean feat, even for innovations with curative potential. Orchard Therapeutics, a company with much experience in this field, tells the Pink Sheet why it values open dialogues, and outlines how health technology assessment methods could be improved.

The other seven drugs on the initial list for negotiation are already heavily rebated and CMS could choose not to go much lower than current net prices, SSR Health report points out.

Chamber files petition to have FTC amend its disqualification rules to have commissioners explain their rejection of recusal requests. FTC is seeking public comments on the petition.

Givinostat, Italfarmaco’s investigational treatment for Duchenne muscular dystrophy, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

The World Health Organization has shortlisted over 30 quality assurance and good manufacturing practice guidelines that are over seven years old or need a major review.

A move to cut senior FDA positions including CDER Director through the appropriations process is rare and almost certain to fail, but experts say the attempt is indicative of an environment that seems to be encouraging these personal amendments.

BIO objects to guidelines seeking to block mergers that would eliminate a potential entrant in a concentrated market and give greater scrutiny to transactions that are part of a series of company deals. APhA and NCPA call for FTC to consider more factors in PBM mergers.

Seven panelists are regular members of the Cellular, Tissue and Gene Therapies advisory committee, while many of the 13 temporary voting members bring neurology and neuroscience expertise. In a conflict-of-interest waiver, the agency noted a struggle to find experts in clinical neurology and neurodegenerative diseases who lack disqualifying financial interests.

New tendering rules in Belgium aim to improve competition between biosimilars and originators, for example by setting contract durations for tendering contracts and limiting the criteria for such contracts.