FDA BRIEF: Week of October 17, 2016
21 FDA grants to stimulate product development for rare diseases
AWARD of 21 new clinical trial research grants totaling > $23 million over 4 years
- Boost development of products for rare diseases
- To Principal Investigators from academia and industry (drugs and devices)
- For 21 different rare diseases with little or no available treatment
- Through the Orphan Products Clinical Trials Grants Program
- For clinical studies evaluating product safety and effectiveness contributing to FDA approval
FUNDING
- 24 % for cancer particularly devastating brain cancer
- 43% for pediatric patients as young as newborns
- 1 medical device for neuroprosthesis
- Funding rate: 31%
Introducing FDA’s Emerging Sciences Idea Portal: Please Help Us Predict the Future
By: Donna L. Mendrick, Ph.D., Associate Director for Regulatory Activities, NCTR
EMERGING SCIENCES WORKING GROUP formed in 2015
- Representatives from FDA product and research centers & offices
- Leverage scientific expertise and resources to conduct long-range horizon scanning
- Emerging issues and cross-cutting scientific advances
- FDA preparedness to proactively address emerging issues and scientific advances
GOAL
- Identify areas not yet addressed in current products e.g. brain-computer interfaces
- Develop science-based planning, programs, policies, reporting, communication
EXPERTISE
- Cast a wide net – Within and outside FDA
- Experts in private sector
PORTAL
- For science and technology experts outside of government
- Submit predictions on the next new things in their field of specialization – will barely show up on a web search
- NOT looking for advances that are already under discussion.
by:
Jonathan C. Goldsmith, M.D., FACP, Associate Director, Rare Diseases Program, CDER
Sandra Kweder, M.D., Deputy Director, EU Office, EMA Liaison
GLOBAL COLLABORATION
- FDA collaborates with other countries and international regulatory agencies for global development programs
- European Medicines Agency (EMA) is valuable collaborator : 4,500 scientists, supervises medicines for > 500 million people in 31 countries
FDA-EMA COLLABORATION
- > 10 years
- Work in groups called “clusters”
- Summarized in EMA website.
RECENT CLUSTER:RARE DISEASE
- To advance treatments for patients with rare diseases
- Share information and review aspects of rare disease drug development programs
- Identification and validation of trial end points
- Potential trial designs
- Flexibility in evaluation of drug development programs
- Expediting review and approval
RECENT CLUSTER: PATIENT ENGAGEMENT
- Incorporate the patient’s involvement and viewpoint in the drug development process
- Develop common goals of expanding patient engagement activities
- Finding patients as spokespersons
- Train selected patients to effectively participate in agency activities
- Report impact of patient involvement
FDA & NAtional Cyber Security Awareness Month
National Cybersecurity Awareness Month. Proclaimed by President Obama each year
FDA POSITION:
- Medical device cyber safety is large and shared responsibility
- Requires diligence from all stakeholders: manufacturers, government agencies, health care organizations, health care professionals, cybersecurity researchers, and medical device user
FDA PARTNERSHIPS:
- Public and private sector organizations
- Department of Health and Human Services, National Health Information Sharing and Analysis Center (NH-ISAC), Medical Device Innovation, Safety, and Security Consortium (MDISS), Department of Homeland Security’s Industrial Control Systems Cyber Emergency Response Team (ICS-CERT)
FDA RECOMMENDATIONS: Employing cybersecurity best practices and good cyber hygiene
RESOURCES: Stop.Think.Connect.™ campaign, CDRH website.
