Clinical Trials/R&D

The biotech plans a pivotal trial that would support accelerated approval of the Takeda-partnered antisense oligonucleotide, WVE-003, and will discuss it with regulators before the end of the year.

US FDA trial enrollment data show improving enrollment across broad demographic definitions, but stakeholders agree more attention is needed for complex subgroups. Could larger trials be part of the answer?

New draft guidance on clinical trial diversity action plans pushes for disproportionately high enrollment of traditionally underrepresented groups, while also asking companies to tailor global programs to US populations and consider diversity aspects that Congress did not outline. 

Pink Sheet spoke with leaders of a new consortium that hopes to make drug company-patient advocacy group partnerships more efficient and effective.

Meeting requests related to the Oncology Center of Excellence’s new dose optimization approach are increasing, along with sponsor questions, prompting the US FDA to determine how to clarify its intentions.

With the much-anticipated guidance still pending, OCE’s Tamy Kim reminds industry of the requirements and timing around avoiding the soon-to-be requirements on study enrollments.

AI-generated vaccines, fast-moving regulatory systems and scalable manufacturing platforms will be key in preparing for future pandemics, according to vaccine and immunology experts including the CEOs of CEPI and Gavi.

Companies should talk to FDA staff in between formal meetings if they need clarity on agency advice that is several years old or development programs hit a snag, Office of Therapeutic Products Director Nicole Verdun says.

The FDA does not appear likely to provide a calculation for sponsors to show clinical trial diversity, but agency officials seem split on how tough to enforce the new regulations intended to ensure all groups are adequately represented.

New guidance from the International Council for Harmonisation is designed to tackle problems companies face by having to meet different requirements around the world when evaluating the potential of DDIs for their investigational products.

Updated clinical trial reporting rules and stronger participant protections are among the several changes to research in Switzerland that will apply from November, while new regulations on clinical trial transparency will apply from March 2025.

As of today, drug developers can apply to take part in two pilot programs that will involve regulators across the EU strengthening their coordination when it comes to providing companies with advice on their applications for clinical trials and marketing authorization.

In an interview with the Pink Sheet, Troy Tazbaz, director of the US FDA's Digital Health Center of Excellence, says the agency lacks authority to regulate assurance labs, which would be used to help AI developers ensure their models are working correctly before submitting them for approval.

The European Medicines Agency has encouraged companies to seek “guidance and direction” early in the development process after it reported a significant fall in the number of companies that sought scientific advice and protocol assistance compared with the three previous years.

Oncology developers should ensure clinical development plans involving early-stage trials adding on to perioperative treatment regimens will show that new therapies are adding benefit in each phase, Oncology Center of Excellence Director Richard Pazdur said.

England’s chief scientific officer explains how the NHS Genomic Medicine Service has led to faster access to targeted cancer drugs and outlines the organization’s ongoing work in pharmacogenomics, liquid biopsies and personalized RNA vaccines.