Conferences

Manufacturers that do not answer all questions they are asked during joint clinical assessments under the upcoming EU HTA Regulation could have their applications deemed incomplete, says a co-chair of the JCA subgroup.

The European Medicines Agency is mulling how best to involve experts in its decision-making processes while avoiding potential conflicts of interest, says executive director Emer Cooke.

US and EU regulators have invited two companies to work with them on a pilot scheme for assessing drugs for rare genetic diseases, a move that the European Medicines Agency’s Emer Cooke said would help improve “global alignment.”

Lack of early patient involvement in trial design can lead to burdensome studies, inappropriate endpoints, and in some cases clinical failure, delegates heard at a recent regulatory meeting.

Pink Sheet infographic shows that while overall growth continues at the drugs and biologics centers, the US FDA still must add many employees to meet user fee-mandated hiring goals.

Amid a push for new incentives, the US FDA’s Kerry Jo Lee, associate director for rare diseases in the Center for Drug Evaluation and Research’s Office of New Drugs, said in an interview with the Pink Sheet that the tools ultra-rare sponsors need already are available.

Many platform designation requests have been from sponsors eager to cite other sponsors’ products, but CBER Director Peter Marks said in an interview with the Pink Sheet his office likely is years away from accepting those applications.

US FDA review divisions will determine whether the meeting is appropriate because there are no one-size-fits-all requirements.

Patient advocacy groups suggest US state and federal agencies are trying to exclude them from certain conversations due to drug industry connections and perceived bias. 

With the sheen of inevitability now scuffed off the BIOSECURE Act, diplomatic efforts to reduce US dependence on Chinese APIs are increasing in prominence. And the power of the legislation itself could be determined by litigation about a video app.

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.

BIO CEO John Crowley sat down with Pink Sheet at the industry’s annual convention to discuss his vision for a newly restructured BIO, his thoughts on the upcoming US elections and whether he’d ever lead another biotech. 

Firms can be more forceful in disputes with FDA now that Supreme Court has eliminated Chevron deference. Questions around whether an evidentiary standard has been met may be ripe for challenge, legal experts said, but they also caution that sponsors will face more uncertainty.

CBER Director Peter Marks outlines a streamlined process to approval for treating different mutations of the same gene. NCATS’ Philip Brooks tells the Pink Sheet the approach avoids the need to “start from scratch for every new mutation.”

The ability to quickly scour the internet for comments on drugs may make it challenging to weed out true adverse events from junk as Senate Republicans consider AI-related changes to FDA's authority.

The Center for Drug Evaluation and Research’s new Center for Clinical Trial Innovation (C3TI) may lead a “targeted” effort to update the FDA’s regulatory framework given advances in trial design and execution, CDER Director Patrizia Cavazzoni said. Industry already has asks.

Third-party screening of US-marketed drug products is worse than pointless if done with subpar test methods, agency tells PDA/FDA meeting. Meanwhile, release-testing with such methods has been triggering warning letters.

Third-party screening of US-marketed drug products is worse than pointless if done with subpar test methods, agency tells PDA/FDA meeting. Meanwhile, release-testing with such methods has been triggering warning letters.

Alkermes CRL is just the latest fallout from black-hole process.

Inter-associations working group suggests changes to the EU GMP Annex I revision to make it easier to understand by health authorities worldwide; the groups point out that the annex will be used globally and not just in Europe.

FDA is considering allowing MIDD meeting requests to be submitted more often than the current quarterly schedule, but agency must have a plan to deal with the likely increased demand, official says.

Focus of the campaign is the well-established pharmaceutical manufacturing sector, rather than cell and gene therapy production, the US FDA’s principal deputy commissioner said.

In admitting problems, the FDA creates a docket for comments and will convene a public workshop in the spring on why the clinical trial innovation is not more widespread.

Several FDA staffers presented at the RAPS Convergence conference via Zoom and pre-recorded video instead, illustrating that just the threat of a shutdown can scramble FDA plans.