FDA News and Views: Stem Cell Therapies Enforcement, Real -World Evidence, KEYTRUDA alert, Drug Manufacturing Oversight, AIDS Look Back, – Drug and Device Digest

FDA BRIEF: August 28, 2017


 FDA’s new policy steps and enforcement efforts to ensure proper oversight of stem cell therapies and regenerative medicine

Promising new field involving pathways involved in tissue damage and regeneration with potential applications across a wide range of diseases and conditions

  • However, ‘unscrupulous actors’  making corrupt, assurances to patients based on unproven and dangerous products
  • Promote unproven, illegal, expensive treatments that offer little hope, and pose significant risks to the health and safety

Stepped Up Enforcement

  • Steps in Florida and California to address troubling products being marketed
  • Will take additional actions in the coming months
  • New working group to pursue unscrupulous clinics

Efficient Regulation

  • Promote science-based policy to expedite development of innovative, scientifically proven regenerative cell therapies
  • New framework  based on current risk-based, flexible regulatory framework and provisions of the Cures Act
  • Issue compliance policy for current product developers to interact with FDA to determine marketing authorization needs


mom and child, 3 people, baby

Use of RealWorld Evidence to Support Regulatory Decision-Making for Medical Devices

Final guidance on the Use of RealWorld Evidence to Support Regulatory Decision-Making for Medical Devices

  • Clarifies how realworld data may be sufficient for use in regulatory decisions,
  • Clarifies evaluation plan to determine relevancy and reliability
  • Clarifies when Investigational Device Exemption (IDE) may be needed to collect and use realworld data

Guidance is cornerstone of strategic priority to build a national evaluation system for health technology (NEST)


Related image

FDA Alerts Healthcare Professionals and Oncology Clinical Investigators about KEYTRUDA (pembrolizumab) in Multiple Myeloma

Inform public, health care professionals, and oncology clinical investigators about risks with KEYTRUDA® (pembrolizumab)

  • In combination with dexamethasone and an immunomodulatory agent (lenalidomide or pomalidomide) for the treatment of patients with multiple myeloma
  • Based on data review from two clinical trials – KEYNOTE-183 and KEYNOTE-185
  • Interim results demonstrated an increased risk of death for patients
  • Patients on KEYTRUDA® for an approved use : Melanoma, Lung Cancer, Head and Neck Cancer, Classical Hodgkin Lymphoma, Urothelial Carcinoma, Microsatellite Instability-High (MSI-H) Cancer


New Steps To Strengthen FDA’s Inspection And Oversight Of Drug Manufacturing

CDER and the Office of Regulatory Affairs (ORA) implementing a new, concept of operations agreement

  • integrate drug review programs with facility evaluations and inspections
  • “Integrated Quality Assessment”  team for closer risk assessments for drug substance, drug product, manufacturing processes, and facilities
  •  ORA reorganized into program-aligned commodity areas mirroring FDA’s centers and industries
  • New model will cover Pre- and Post-Approval Inspections, Surveillance Inspections, and For-Cause inspections at domestic and international drug manufacturing facilities


Commemorating 20 Years AIDS poster

A Look Back: The AIDS Crisis and FDA … 30 Years Later

Office of AIDS and Special Health Issues established in 1994 at the height of the AIDS epidemic

  • Advocacy group ACTUP (AIDS Coalition to Unleash Power) functionally closed down the FDA in 1988
  • Individuals continually called up various offices of FDA complaining that no one was paying attention to AIDs
  • There were “fax attacks” – to disrupt “business as usual,”
  • FDA responded to these needs

FDA engagement with AIDS Community particularly the patients

  • Patients played an important role in AIDS drug development
  • Pushed for use of accelerated approval based on viral load – a surrogate endpoint
  • Pushed for expanded access
  • Highlighted that policy had to move in tandem with science
  • Participated in Advisory Committee Meetings

In 1996, when protease inhibitors were approved, everything turned around


Image Credit: FDA

Scroll to Top