As patients who are receiving treatments via expanded access for diseases are outside of the controlled environment of a clinical trial, their experiences may be considered to be more "real-world." A multi-national chart review has shown that the success of treatments given through expanded access was mirrored in clinical trials, then after approval and mass [...]
Recent research of stem cells in mice has produced a more precise way to deliver chemotherapy to the tumor microenvironment of metastatic cancer. As opposed to targeting cancer cells directly, as other stem cell research has aimed to achieve, this delivery method targets the cells in their home, which could have a greater effect on [...]
Could wider inclusion of minority patients in clinical trials actually lower the cost of healthcare overall?
Lack of accurate data regarding potential side effects or differences in effectiveness in treatments between white Americans and patients of different ethnic backgrounds often leads to less affordable options of care. When drugs have been studied, and minorities left out, treatments that are less effective could lead to greater numbers of hospitalizations, driving up the [...]
New Waiver in IRB Process Speeds FDA Single Patient Expanded Access Approval; Updated Information available on WCGF website.
On October 3, 2017, Dr. Scott Gottlieb, FDA Commissioner, testified before the US House of Representatives Subcommittee on Health, Committee on Energy and Commerce. He announced a significant change to the way that IRB may review single patient expanded access request involving investigational drugs or biologics. This change, which permits an IRB chair or designee [...]
How could a new treatment, given previously under expanded access, successfully treat a serious disease of the eye?
A treatment for neurotrophic keratitis, initially given under expanded access, has been approved for use in European countries. The treatment, a genetically engineered nerve growth factor, has been shown to heal the corneas of affected patients, including children. http://bit.ly/2yjsygw
PRINCETON, N.J. Nov. 7, 2017—WCG Foundation has received $50,000 from Johnson & Johnson to help desperately ill patients get access to experimental medicines.
The gift supports WCGF’s efforts to help patients navigate the Food and Drug Administration’s expanded access program, which makes investigational drugs available to patients who have exhausted all other treatment and clinical trial options.Learn More >
Researchers funded by the National Institutes of Health (NIH) have completed an atlas of human DNA that influence gene expression. Gene expression is shown through observable traits like eye color or disease risk; this research has shown how some genes are turned off and on. Previously, such information was not available at this scale. However, [...]
Expanded access allows patients to receive innovative new drugs. How often does the FDA approve patients’ requests?
The FDA's expanded access program has allowed many desperately ill patients to receive experimental treatments in an effort to save lives. Just last fiscal year, over 1,700 requests were made by patients to access experimental treatments. The Food and Drug Administration's Center for Drug Evaluation and Research and their Center for Biologics Evaluation and Research combined [...]
What is a .@US_FDA priority review voucher and how does it expand opportunity for #medicalresearch of #rarediseases?
The priority review voucher program incentivizes pharmaceutical companies to research drugs for very rare diseases. When a drug developer researches a rare disease, a voucher is received by that organization from the FDA to move another treatment, often for a more common disease, through the regulatory process more quickly. How does this program work? http://bit.ly/2f5RXjm
What kind of information can be gained from a mock clinical trial? How can this procedure help us learn about diversifying clinical trials?
Two populations in the United States - one in Altoona, Pennsylvania, the other in Atlanta, Georgia - of similar incomes, but different racial backgrounds, were enrolled in a mock clinical trial to gauge participation given several variables. Some similarities, such as requests for background information, arose in both populations. But differences in requests for amenities such [...]