Advanced Technologies

There is “a lot of flexibility” in the European Medicines Agency’s reflection paper on the use of artificial intelligence during drug development, which is principles-driven rather than setting rigid recommendations, says the agency’s Florian Lasch.

The FDA is developing several structures and a broad group of experts across disciplines to help craft artificial intelligence policy. But the proliferation of AI-related initiatives raises the question of who, ultimately, will make decisions about when novel applications of AI are acceptable.

Many of the comments were very helpful in improving the document in relation to both form and content, the European Medicines Agency said of its newly published reflection paper on the use of artificial intelligence during the drug development, marketing authorization and post-authorization phases.

The five-year roadmap aims to expand support for AI research and development in essential health care and new drug development, as well as advance medical data usage systems and enable its safe use.

Draft recommendation for investigators to maintain a task log of activities that local health care providers perform was removed from final guidance on decentralized clinical trials. Other changes include more detail on US FDA oversight and what HCPs can and cannot do in such studies.

The Australian teletrials program has surpassed expectations at its mid-way point and is being used by drug and device sponsors looking to enhance trial participant recruitment and retention by enabling access to rural, regional and remote areas.

The implementation phase of the European Health Data Space is an opportunity to address “critical aspects” of the new regulation that require clarification, says pharmaceutical industry federation EFPIA.

Europe’s Innovative Health Initiative, a public-private funding partnership, has put out a call for a research project that could help analyze the use of regulatory sandboxes in health care innovation in addition to three others.

Meeting the regulatory gold standard for drug candidates in ultra-rare diseases can be impractical, a regulatory expert says, but greater collaboration and shared insights from regulatory reviews could help find a viable path forward.

Decentralized manufacturing methods for cell and gene therapies will be critical for improving patient access to treatments, but sponsors must prepare to demonstrate “comparability” with centralized manufacturing.

Hemgenix has now secured reimbursement in several European markets, with more talks ongoing. While the path to reimbursement has not always been easy, innovative access deals have helped to smooth the way.

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.”

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.

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.”

New draft guidance defines the necessary components of a platform technology designation request, including that sponsors demonstrate how it will save the FDA review time.

CBER’s Nicole Verdun said the FDA is using the principles of the platform approach in situations that don’t qualify for the agency’s new incentive.