Approval Standards
The former CDER Director spoke to the Pink Sheet about why a new statutory standard is needed for certain rare disease drug approvals. We lay out some of her thinking in part one of a multi-part series on the topic.
Regulators do not have the resources to “double check” that companies are using AI appropriately, meaning that manufacturers must ensure the AI tools they use meet relevant standards, says an EU regulatory expert.
Despite the recent anti-vaccine rhetoric in the final weeks of the Trump campaign, pulling an established safe and effective product off market would be difficult. But there’s little to stop political interference in approvals.
Japan is cautiously easing Japanese clinical data requirements for rare disease drugs to allow faster and more flexible approvals, including on a conditional basis supported by postmarketing studies.
Woodcock and the Haystack Project want to modify a foundational concept of modern FDA drug efficacy assessments, which may be seen as an attempt to more formally codify and define regulatory flexibility.
Lyfgenia and Zynteglo comprise complex mixtures of transduced cells that represent different active ingredients, the company said in administrative appeals to the FDA. Two lawmakers also raised bipartisan concerns about the agency’s decision on the voucher request for the sickle cell disease treatment.
Remarks at the NORD Breakthrough Summit from the FDA’s Lola Fashoyin-Aje may suggest the agency is becoming more comfortable with single-arm trials.
Oncology staff also said more work is needed before patient registries can be used as an external control in clinical trials, but they strongly encouraged continued investment.
A US FDA Advisory Committee responded enthusiastically to the potential use of challenge models to enable pivotal trials of novel pertussis vaccines, but most members suggested more refinements are needed before they are ready to use.
The FDA’s vaccine advisory committee endorsed a new plan to update pandemic influenza vaccines “inter-pandemic,” but encouraged the agency to consider how to make the approach feasible for novel vaccine technologies.
Brazil has also published its recent regulatory reliance regulations in English, a move designed to increase confidence in its decisions as it presses on with aligning its national practices with global best practices.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
The program for collaborative gene therapy reviews with the European Medicines Agency is starting with simple applications, but after launch could be expanded to earlier discussions of CMC and pharm/tox data requirements, the FDA’s Nicole Verdun said.
As need for new antibiotics keeps increasing, a recent US FDA advisory panel offers a case study of why industry isn’t keen on the space.
The company will file a new drug application with the US FDA by the end of the year for tradipitant in a different indication, motion sickness.
The European Medicines Agency has highlighted issues for drug developers to consider when seeking to submit clinical data from SATs as the pivotal evidence in their marketing authorization applications instead of randomized controlled trial data.
Studies should enroll a representative subgroup of US patients based on the incidence or prevalence of cancer in the US, and the comparator arm should use the US standard of care when possible, the FDA says in draft guidance.
The European Commission has registered a European citizens' initiative that explains how the EU can foster equitable, timely, affordable, safe and legal access to innovative psychedelic-assisted treatments. Work towards securing the one million signatures needed for the next step is starting soon.
Disputes over orphan and new clinical investigation exclusivity are among the early drug and biologic cases where legal filings cite the Loper Bright decision, which overturned the Chevron doctrine of deference to agency actions.
With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.