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Scientific Session

Rheumatoid arthritis (RA)
General Topic: Clinical Science

15W127: Distinguished Investigator: The Evolution of Clinical Research in Vasculitis: Lessons Learned for the Study of Rare Diseases

Wednesday, November 15, 2023
9:15 AM – 10:15 AM PT
Location: Room 6F/6C
CE: 1

In Person On Demand

    Speaker(s)

  • Peter Merkel, MD, MPH

    University of Pennsylvania
    Philadelphia, PA, United States

    Disclosure(s): AbbVie/Abbott: Grant/Research Support (Ongoing); Amgen: Consultant (Ongoing), Grant/Research Support (Ongoing); ArGenx: Consultant (Ongoing); AstraZeneca: Consultant (Ongoing), Grant/Research Support (Ongoing); Boehringer-Ingelheim: Consultant (Ongoing), Grant/Research Support (Ongoing); Bristol-Myers Squibb(BMS): Consultant (Ongoing), Grant/Research Support (Ongoing); Cabaletta: Consultant (Ongoing); CSL Behring: Consultant (Ongoing); Eicos: Grant/Research Support (Ongoing); Electra: Grant/Research Support (Ongoing); Genentech: Grant/Research Support (Ongoing); GlaxoSmithKlein(GSK): Consultant (Ongoing), Grant/Research Support (Ongoing); HiBio: Consultant (Ongoing); InflaRx: Consultant (Ongoing), Grant/Research Support (Ongoing); Janssen: Consultant (Ongoing); Jubilant: Consultant (Ongoing); Kyverna: Consultant (Ongoing), Stock options or bond holdings in a for-profit corporation or self-directed pension plan (Ongoing); MiroBio: Consultant (Ongoing); Neutrolis: Grant/Research Support (Ongoing); Novartis: Consultant (Ongoing); NS Pharma: Consultant (Ongoing); Q32: Consultant (Ongoing); Regeneron: Consultant (Ongoing); Sanofi: Consultant (Ongoing); Sparrow: Consultant (Ongoing); Takeda: Consultant (Ongoing), Grant/Research Support (Ongoing); UpToDate: Royalties (Ongoing); Visterra: Consultant (Ongoing)

This session will review the changes in the conduct of clinical research in the last 30 years, including cohort studies and clinical trials, that led to significant progress in our understanding of these diseases and success in developing effective therapies for several forms of vasculitis. These changes include advances in outcome measures, use of various study designs to address small sample sizes, creation of large research networks, partnerships with the biopharmaceutical industry, increased engagement of patient research partners and patient advocacy groups, and success in obtaining research funding. The accomplishes made and challenges encountered in this work offer guidance to the study of other rare diseases.

Learning Objectives:

  • Review the history of clinical research in vasculitis.
  • Describe the many elements of research design utilized to study vasculitis.
  • Outline the innovative aspects of research infrastructure and implementation that help advance research in vasculitis.
  • Summarize the lessons learned in the study of vasculitis that are applicable to research in other rare systemic rheumatic diseases.

Presentations:

  • 9:15 AM – 10:00 AM PT
    The Evolution of Clinical Research in Vasculitis: Lessons Learned for the Study of Rare Diseases

    Speaker: Peter A. Merkel, MD, MPH – University of Pennsylvania

  • 10:00 AM – 10:15 AM PT
    Q&A