Video: Every Case Tells a Story| Webinar: ACR/CHEST ILD Guidelines in Practice

An official publication of the ACR and the ARP serving rheumatologists and rheumatology professionals

  • Conditions
    • Axial Spondyloarthritis
    • Gout and Crystalline Arthritis
    • Myositis
    • Osteoarthritis and Bone Disorders
    • Pain Syndromes
    • Pediatric Conditions
    • Psoriatic Arthritis
    • Rheumatoid Arthritis
    • Sjögren’s Disease
    • Systemic Lupus Erythematosus
    • Systemic Sclerosis
    • Vasculitis
    • Other Rheumatic Conditions
  • FocusRheum
    • ANCA-Associated Vasculitis
    • Axial Spondyloarthritis
    • Gout
    • Psoriatic Arthritis
    • Rheumatoid Arthritis
    • Systemic Lupus Erythematosus
  • Guidance
    • Clinical Criteria/Guidelines
    • Ethics
    • Legal Updates
    • Legislation & Advocacy
    • Meeting Reports
      • ACR Convergence
      • Other ACR meetings
      • EULAR/Other
    • Research Rheum
  • Drug Updates
    • Analgesics
    • Biologics/DMARDs
  • Practice Support
    • Billing/Coding
    • EMRs
    • Facility
    • Insurance
    • QA/QI
    • Technology
    • Workforce
  • Opinion
    • Patient Perspective
    • Profiles
    • Rheuminations
      • Video
    • Speak Out Rheum
  • Career
    • ACR ExamRheum
    • Awards
    • Career Development
  • ACR
    • ACR Home
    • ACR Convergence
    • ACR Guidelines
    • Journals
      • ACR Open Rheumatology
      • Arthritis & Rheumatology
      • Arthritis Care & Research
    • From the College
    • Events/CME
    • President’s Perspective
  • Search

Efficacy Studied Following Accelerated Drug Approvals

Michele B. Kaufman, PharmD, BCGP  |  October 2, 2017

For the remaining seven indications (29%), studies were progressing according to target timelines. Clinical benefit had not yet been confirmed for eight indications that had been initially approved at least five years prior. The most common surrogate measure in the preapproval studies was disease response, because many drugs were cancer agents. Other surrogate measures included time-to-event outcomes, such as progression-free survival and time-to-sputum culture conversion; and change in baseline biomarker levels, such as liver iron concentration. Most requirements were for randomized clinical trials (n=25); the rest (n=13) were single-group studies, including long-term extensions of preapproval studies.

Among the 22 drugs with 24 indications granted FDA accelerated approval in the studied timeframe, efficacy was often confirmed in post-approval trials a minimum of three years after approval in most studies. The confirmatory and preapproval trials had similar study elements, including using surrogate measures as outcomes. Although this type of evaluation has many limitations, the information can be useful in evaluating post-marketing safety and effectiveness for use in patients.

ad goes here:advert-1
ADVERTISEMENT
SCROLL TO CONTINUE

Michele B. Kaufman, PharmD, BCGP, is a freelance medical writer based in New York City and a pharmacist at New York Presbyterian Lower Manhattan Hospital.

Reference

ad goes here:advert-2
ADVERTISEMENT
SCROLL TO CONTINUE
  1. Naci H, Smalley K, Kesselheim A. Characteristics of preapproval and postapproval Studies for drugs granted accelerated approval by the U.S. Food and Drug Administration. JAMA. 2017 Aug 15;318(7):626–636. doi: 10.1001/jama.2017.9415.

Page: 1 2 | Single Page
Share: 

Filed under:Drug Updates Tagged with:acceleratedFDAFDA approvalU.S. Food and Drug Administration (FDA)

Related Articles

    Rheumatology Drug Updates: Efficacy Studied Following Accelerated Drug Approvals; Plus Secukinumab Meets Benchmark for Psoriasis

    November 9, 2017

    Medications for serious or life-threatening conditions may receive accelerated approval from the U.S. Food and Drug Administration (FDA) by showing an effect on surrogate measures that are reasonably likely to predict a treatment’s clinical benefit. Post-approval confirmatory drug trials are then required to determine whether or not these effects translate into clinical improvements. In recent…

    Flimsy Evidence Behind Many FDA Approvals

    August 21, 2017

    (Reuters Health)—Many drugs granted accelerated approval by the U.S. Food and Drug Administration (FDA) lack clear evidence of safety and effectiveness, and the same is true for most high-risk medical devices, according to two new reports in JAMA, online Aug. 15. The Accelerated Approval pathway makes potentially promising investigational medicines available for use before the…

    FDA Provides 2020 Rheumatology Drug Update

    December 17, 2020

    Three FDA representatives discuss new drug indications, safety precautions and label changes, & an emergency program to rapidly evaluate existing immunomodulating therapies for use in COVID-19 patients.

    FDA Rheumatology Update: New Drug Approvals, Plus Expanded Drug Indications & Safety Concerns

    February 12, 2020

    Last year, the FDA was busy with new biologic and other drug approvals, new and expanded drug indications, and important safety updates relevant to rheumatology…

  • About Us
  • Meet the Editors
  • Issue Archives
  • Contribute
  • Advertise
  • Contact Us
  • Copyright © 2025 by John Wiley & Sons, Inc. All rights reserved, including rights for text and data mining and training of artificial technologies or similar technologies. ISSN 1931-3268 (print). ISSN 1931-3209 (online).
  • DEI Statement
  • Privacy Policy
  • Terms of Use
  • Cookie Preferences