Cell & Gene Therapies
Cell and gene therapy manufacturers must consider the practicalities of their product within the context of a health care system before it comes onto the market to be successful, experts from Novartis, AstraZeneca and England’s National Health Service say.
A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.
Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.
Not all companies will be able to access joint scientific consultations under the EU Health Technology Assessment Regulation, but success is still possible for those that engage with national agencies early on, says EUCOPE’s Alexander Natz.
Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.
Pink Sheet reporter and editors discuss the early retirement and buyout offers to FDA employees and significant agency departures in the Center for Biologics Evaluation and Research and chief counsel’s office as a new commissioner moved closer to confirmation.
Center for Biologics Evaluation and Research Director Peter Marks expects no fundamental changes in support for FDA initiatives and said the Rare Disease Innovation Hub, which promotes cross-center collaboration, is “consistent with what we're hearing in the current environment.”
Among those now leaving the US FDA’s biologics center is Lola Fashoyin-Aje, who was considered a rising star at the agency when she became director of the Office of Therapeutic Products’ Office of Clinical Evaluation in 2024.
US FDA Commissioner nominee Martin Makary’s 6 March Senate confirmation hearing should offer insight on his alignment with HHS Secretary Robert F. Kennedy Jr.'s views and willingness to insulate the FDA from an unusual level of micromanagement by the department and White House.
The FY 2024 fourth quarter had the highest number of formal meetings cancelled because written responses were sufficient since the beginning of FY 2018.
The European Medicines Agency has recommended in favor of approving four new products, and has also reaffirmed its positive opinion for the Alzheimer’s disease drug, Leqembi.
The US FDA is expected to refine warnings recently added to CAR-T cell therapies about the risk of secondary malignancies after reassuring new data, which is a positive sign for the future of the therapies in autoimmune disorders.
Health technology assessment agencies are willing to pay more than usual for advanced therapies that treat rare or severe diseases, but an advanced therapy for a common condition would not qualify for such a modifier, a former senior figure at England’s NICE says.
The European Medicines Agency is set to issue opinions this week on whether pan-EU marketing approval should be granted to a number of new products.
While CSL Behring has successfully negotiated reimbursement contracts for its one-time gene therapy Hemgenix in several European countries, it says there are still “barriers to innovative contracting solutions” in some member states that need to be addressed.
CSL Behring explains how it worked with authorities in Denmark, Austria, England, Scotland, Spain and Switzerland to secure innovative access arrangements suitable to each nation’s “unique needs” for its one-time gene therapy, Hemgenix.
The "interwoven" nature of the offices within the agency likely means Medicare and Medicaid benefits may be disrupted by staff cuts despite assurances from the administration, Brooks-LaSure warned.
The US FDA is receiving more requests for regenerative medicine advanced therapy designation and granting more of them, according to recently reported agency data.
Brazil’s drug regulator ANVISA has also published its first post-marketing authorization monitoring reports on advanced therapies Yescarta, Zolgenma, Kymriah, Carvykti and Luxturna.
The world’s first CRISPR gene editing therapy, Casgevy, has been made available to patients with sickle cell disease in England, adding to access arrangements in the US, Austria, Bahrain, Germany, Luxembourg, Italy and Saudi Arabia.