We use cookies to provide you with a better experience. By continuing to browse the site you are agreeing to our use of cookies in accordance with our Cookie Policy.
Accept
  • SKIP TO CONTENT
  • SKIP NAVIGATION
  • Drug & Device Products
    • FDAnews Books Library
    • Events
    • Form 483s Database
    • Publications
    • Webinar Training Pass
    • eCFR and Guidances
    • Books
  • Clinical Products
  • Advertising
  • White Papers
  • Contact Us
  • About Us
  • COVID-19
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » Drugmakers Comment on FDA’s Guidance on Natural Histories for Rare Diseases

Drugmakers Comment on FDA’s Guidance on Natural Histories for Rare Diseases

May 30, 2019

Drugmakers offered feedback on the FDA’s guidance for developing natural history studies for rare disease drug development, praising some parts of the guidance but calling for more details.

Lundbeck noted that many rare diseases have established disease registries made available by clinicians and institutions, calling on the agency to acknowledge that it may not always be feasible for sponsors to influence registry development. Establishing a free-standing prospective natural history study can be challenging in terms of recruitment as well, the drugmaker added.

The company also suggested electronic health records as a potential source for creating a control group. The agency should also consider how the guidance’s principles could apply to other vulnerable populations and pediatric development, Lundbeck said.

Merck asked the agency to clarify its expectations for data submissions. The company said it’s unclear if sponsors should submit only external control data or the whole natural history study.

View today's stories

Pharmaceuticals Regulatory Affairs

Upcoming Events

  • 08Sep

    Organizing Data and Document Archives: Finding a Needle in a Haystack for FDA Inspections

  • 14Sep

    Omnichannel Engagement: Are You Digitally Ready?

  • 15Sep

    Califf’s Post-PDUFA, Post-COVID FDA Agenda: Key Developments, Insights and Analysis

  • 20Sep

    Building a World-Class Pharmaceutical Advertising and Promotion Review Program

  • 21Sep

    The FDA’s Focus on Clinical Data Integrity Throughout Product Development and Approval

  • 22Sep

    FDA’s New Quality Management System Regulation: What the Proposed Harmonization with ISO 13485 Means for Devicemakers

Featured Products

  • FDA’s New Quality System Regulation: Transitioning from QSR to ISO 13485

    FDA’s New Quality System Regulation: Transitioning from QSR to ISO 13485

  • Selecting and Implementing Electronic Document Management Systems in the EU

    Selecting and Implementing Electronic Document Management Systems in the EU

Featured Stories

  • Organogenesis Gets 510(k) Clearance to Expand PuraPly Portfolio

  • FDA Grants Priority Review to sNDA for Lynparza

  • Visibly Gets 510(k) Clearance for Its Online Eye Test

  • Resilience Inks Collaboration Deal With Mayo Clinic Over Biotherapeutics

The Revised ICH E8: A Guide to New Clinical Trial Requirements

Learn More
  • Drug Products
    • Quality
    • Regulatory Affairs
    • GMPs
    • Inspections and Audits
    • Postmarket Safety
    • Submissions and Approvals
    • Research and Development
    • Commercial Operations
  • Device Products
    • Quality
    • Regulatory Affairs
    • QSR
    • Inspections and Audits
    • Postmarket Safety
    • Submissions and Approvals
    • Research and Development
    • Commercial Operations
  • Clinical Products
    • Trial Design
    • Data Integrity
    • GCP
    • Inspections and Audits
    • Transparency
  • Privacy Policy
  • Do Not Sell My Personal Information
Footer Logo

300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

Phone 703.538.7600 – Toll free 888.838.5578

Copyright © 2022. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing