FDA News: Sickle Cell Research, Office of Orphan Products Development (OOPD), Biomarker Qualification Case Study, Generic Drug Access, Bodybuilding Products
FDA BRIEF: Week of June 19, 2017
Celebrate World Sickle Cell Day by Learning About Clinical Trials
Sickle Cell Disease is most common inherited blood disorder in US
- Chronic condition with no cure
- Can cause severe pain, organ damage, stroke
- One way to help advance scientific discovery is through clinical trials
Learn more about clinical trials in videos
- Brings awareness to importance of having diverse participants in clinical trials
- Help ensure medical products are safe and effective for everyone
Office of Orphan Products Development (OOPD)
To promote the development of products for the treatment, diagnosis, and prevention of rare diseases and conditions
- Drugs, Devices, Biologics, Medical Foods
Designation Programs
- Orphan Drug Designation
- Rare Pediatric Disease Designation
- Humanitarian Use Device Designation
Grant Programs
- Orphan Products Grants
- Orphan Products Natural History Grants
- Pediatric Device Consortia Grants
Medical Device Humanitarian Use Device (HUD) and Humanitarian Device Exemption (HDE) programs
- Legislative History
- Process
- Training
Case Study for Biomarker Qualification Program
CDER Biomarker Qualification Program works with external stakeholders to develop biomarkers for drug development process
- Fictional case studies part of an educational series on qualifying biomarkers
- Intended to help patients, patient advocacy groups, health professionals, small businesses, and pharmaceutical and clinical innovators learn more
- Provide “real-world” examples
Case Study on fictional biomarker of drug-induced kidney injury in rats
- Understand role of biomarker qualification in drug development
- Understand validation studies necessary to support qualification
- Understand collaborative efforts in qualification for a specific context of use
FDA Working to Lift Barriers to Generic Drug Competition
By: Scott Gottlieb, M.D., FDA Commissioner, @SGottliebFDA
FDA doesn’t have direct role in drug pricing – but taking steps to help address access
- Expediting approval of lower-cost, generic medicines.
- Facilitating increased competition for prescription drugs
Drug Competition Action Plan Federal Register
- Understand ‘gaming’ of FDA’s rules to create obstacles to generic access
- Blocking availability of branded products for comparative testing
- Misusing REMS by having single shared program to delay generic entry
Policy and programmatic changes to address issues
- Using FDA authorities more forcefully
- Collaborate with sister agencies
- Coordinate with Federal Trade Commission in identifying and publicizing anti-competitive practices
- Simultaneously strike a careful balance between pharmaceutical innovation and access to lower cost generic products
FDA issues warning about body-building products labeled to contain steroid and steroid-like substances
Illegally marketed products labeled to contain steroid and steroid-like substances and promoted to increase muscle mass and strength
Illegally marketed as dietary supplements
- Unapproved drugs without FDA review of safety and effectiveness
- Safety assessment based on more than seven years of adverse event reports
- Risk of serious liver injury and other adverse health consequences including kidney injury, increased risk of heart attack and stroke, shrinkage of testes, male infertility