Predicting Stem Cell Activity to Ensure Safe and Effective Therapies
Human multipotent stromal cells (MSCs) being prepared for use as therapies to treat human diseases or medical conditions- but have serious limitations:
- As of January 2018, no MSC-based clinical trials have resulted in FDA-approved treatments
- Significant challenge in ensuring MSCs will work together to perform the same desired function when they are administered to patients
FDA scientists developing ways to improve predictability
- FDA’s MSC Consortium trying to develop methods to predict with more certainty how manufactured or isolated MSCs will behave in patients
- Predict MSC behavior stimulated by growth factors
- Powerful imaging technologies to monitor and analyze changes in size and shape of many thousands of cells in a matter of hours
- Applicable to development and FDA approval of potential stem-cell based products
FDA activities related to the ongoing post-market review of Essure and FDA’s commitment to keep women informed
Active role in providing Essure consumers with up-to-date, evidence-based information to help informed medical decisions
- Met with consumers affected by Essure, a method of permanent birth control
- >750,000 women worldwide have received implant
- Some developed significant medical problems e.g. abdominal pain, abnormal uterine bleeding, device migration
FDA Steps
- Monitor product safety and effectiveness since approval in 2002
- Convened medical experts panel n the fall of 2015 to provide advice on understanding concerns
- Ordered manufacturer, Bayer, to conduct postmarketing study to better evaluate safety profile
- Boxed Warning and Patient Decision Checklist added to labeling
- Ongoing activities posted on Essure website
FDA continues to believe that Essure may be appropriate for some women
- Based on current information
- Recognize serious problems associated with use
- Consider regulatory options that appropriately balance benefits and risks based on new information
- Continue to communicate publicly, share learnings, help women make informed decisions
CDER: Regulatory Science
CDER has launched new video series on major challenges in drug evaluation and development and how to address
- Improving Drug Review with Data Standards. Learn how data standards can support the integration of real world evidence into the drug development process and patient-centric decisions
- Patient-Focused Drug Development. Explore how the voice of the patient critically impacts drug development.
- Real World Evidence. Learn how FDA is collaborating with external stakeholders to identify and evaluate previously unreported adverse reactions and support early detection of safety signals
- The Importance of Partnerships and Consortia. Explore how partnerships and consortia can enable us to address challenging regulatory science questions
- CDER Scientists – In Their Own Words. Investigators describe their roles in FDA’s regulatory science program, and how their research advances drug development and the public health
Image credit: FDA