FDA Views: EvGen, Expedited Reviews, Continuous Manufacturing, FDA-China partnership – Drug and Device Digest

FDA BRIEF: Week of April 18, 2016

FDA Voice


What We Mean When We Talk About EvGen

Part I: Laying the Foundation for a National System for Evidence Generation

shermanRachel E. Sherman, M.D., M.P.H., Associate Deputy Commissioner for Medical Products and Tobacco


califfRobert M. Califf, M.D.,  Commissioner 

  • High-quality evidence derived from appropriately designed randomized controlled trials result in better outcomes for patients
  • However, most clinical practice guideline recommendations are not based on this standard
  • Therefore, FDA considering a national system for evidence generation (EvGen)
  • Foundational concepts—interoperability and connectivity
  • Interoperability : For use by patients, consumers, professional groups, payers, the medical products industry, and health systems by creating standardized approaches
  • Connectivity: Enable collaboration among patients, clinicians, hospital systems, health insurance organizations by leveraging all available data to provide answers to important public health questions.



FDA’s Breakthrough Therapy Designation and Expedited Review Programs



Richard Moscicki, M.D., Deputy Director for Science Operations, CDER, FDA 


  • Drug candidates in early development that have the potential to be breakthroughs
  • Intended to treat a serious condition and has preliminary clinical evidence
  • Receive enhanced communication and input from CDER
  • By March 2016: 342 requests; 111 granted


  • Drug candidate for serious condition and advantageous over available therapies
  • Use of surrogate endpoint- change indicators likely to predict clinical benefit
  • Require post-approval to confirm clinical clinical benefit

FAST TRACK designation

  • Drug candidates for serious condition
  • Non-clinical demonstration of potential to address an unmet medical need.
  • Obtained very early in drug development

All can lead to:


  • For very serious condition, trials have been completed, significant improvement in safety, effectiveness or both
  • Reduced FDA review time – but review standards not compromised

Areas of overlap:

Breakthrough Therapy drug would also receive a Priority Review and could receive an Accelerated Approval as part of its path to patients


Continuous Manufacturing Has a Strong Impact on Drug Quality



By: Lawrence Yu, Ph.D. Deputy Director, Office of Pharmaceutical Quality, CDER



  • Little change in pharmaceutical production over the last 50 years
  • Traditional  “batch” technology — many stops and starts in a series of steps
  • New technology — continuous manufacturing — faster production, reliable products
  • Decreases manufacturing costs with possible lower drug prices

Some manufacturers building continuous manufacturing into their processes

  • VERTEX: Orkambi (lumacaftor/ivacaftor) – cystic fibrosis drug
  • JANSSEN: Prezista (darunavir)- HIV-1 infection

Draft Guidance by Emerging Technology Team – ETT

ETT.JPG Reviewed in this blog


Strengthening Partnerships: FDA’s China Office Engages in Key Outreach with Chinese Provincial FDA, Academia, and Industry

chinaBy: Leigh Verbois, Ph.D., Director of FDA’s China Office in the Office of International Programs

独木不成林,单弦不成音  ‘A single tree makes no forest, one string makes no music.’


  • China – home to a significant number of FDA-regulated medical product manufacturers
  • Mission to collaborate with Chinese regulators, industry and academia to ensure medical products manufactured for the U.S. market are safe and effective
  • FDA China office met with key leaders and experts across the Yangtze river delta

SHANGHAI: Students and faculty at East China University of Science and Technology’s School of Pharmacy and China Pharmaceutical University, .U.S. Embassy Consul General on  FDA priorities in the region.

NANJING: Jiangsu FDA, regional regulator, to share information on  U.S. safety standards

SUZHOU: Town hall meeting with  China’s Association for Medical Device Industry

HANGZHOU: Provincial FDA officials on collaboration on future medical device and drugs

ZHEJIANG: Industry roundtable on pharmaceuticals and Zhejiang FDA




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