NorthWest Biotherapeutics Inc (NWBO)

[hyperopia]

International regulatory collaboration is definitely increasing. Have you heard of the International Coalition of Medicines Regulatory Authorities? (ICMRA) https://icmra.info/drupal/en/home

FDA's Marks hopes to align global regulators to boost gene therapy
By Patrick Wingrove
March 30, 2023

PHILADELPHIA, March 29 (Reuters) - U.S. Food and Drug Administration official Peter Marks said on Wednesday he hopes to align global regulations to boost commercialization for gene and cell therapies to get more treatments launched for patients with rare diseases.

“If you could have a way of having the EU, U.S., Japan, Canada, perhaps Switzerland all have approvals at close to the same time, that would give a market that would be four or five times as large as just the U.S.,” he said at the Reuters Pharma USA conference in Philadelphia.

Health regulators including the FDA and the European Medicines Agency have different rules for cell and gene therapies that make it harder to get treatments on their markets at similar times. That can make it difficult for companies to achieve commercial success, he said.

Marks said he wanted to create an international collaborative review between the FDA and the health regulators of those high-income countries to make it easier for drug companies to launch globally, calling it "regulatory convergence."

That process would help companies targeting ultra rare diseases where there are perhaps only 30 to 40 patients in the U.S. market but 100 or 150 across the world, Marks said.

Rare diseases in the U.S. are considered to be conditions that affect less than 200,000 people.

The FDA is moving to encourage use of disease related biomarkers that may predict efficacy as substitutes for extensive trials that definitively prove clinical benefit to help get accelerated approval of gene therapies for diseases with small patient populations, Marks announced last week.

He discussed the importance of getting these treatments to market faster using biomarkers for approval on Wednesday.
"If you could even take people who are severely affected and make them into people who are minimally affected, that would be a huge win," said Marks, the FDA’s director of the Center for Biologics Evaluation and Research.

The accelerated approval process would look different for different kinds of therapies depending on how many patients were affected, he said.

Despite the currently exorbitant prices for these types of therapies, Marks said he also wanted to educate people from lower- and middle-income countries on the promise of gene therapies.

https://www.reuters.com/business/healthcare-pharmaceuticals/fdas-marks-hopes-align-global-regulators-boost-gene-therapy-2023-03-29/

FDA announces completion of first joint assessment with EMA in ICMRA pilot
Regulatory News
 28 July 2023 
https://www.raps.org/news-and-articles/news-articles/2023/7/fda-announces-completion-of-first-joint-assessment


Regulators tout ICMRA pilots, adoption of ICH guidelines as major steps toward convergence
Regulatory News
26 January 2024 
https://www.raps.org/news-and-articles/news-articles/2024/1/regulators-tout-icmra-pilots,-adoption-of-ich-guid


Cell & Gene State of the Industry Briefing January 8, 2024

Expediting the Development of Cell and Gene Therapy
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)

Peter Marks addresses some of the recently reported CAR-T malignancies, small batch automated manufacturing, regulations for platform technologies to speed development based on prior approved evidence, accelerated approvals using biomarker based endpoints, increased global regulatory collaboration to approve gene and cell therapy products faster, START program helping smaller companies in the rare-disease space, and CBER aims to make 2024 the breakout year for addressing key challenges to the development of cell and gene therapies for rare diseases.

https://alliancerm.org/arm-event/sotibriefing/