June 2017 – Drug and Device Digest

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

group picture of ethnically diverse patients

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


FDA Case Study 1

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


Think it's easy becoming a generic drug in America? Think againFDA 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




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