• SKIP TO CONTENT
  • SKIP NAVIGATION
  • Patient Resources
    • COVID-19 Patient Resource Center
    • Clinical Trials
    • Search Clinical Trials
    • Patient Notification System
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
  • Professional Resources
    • Research Center Profiles
    • Clinical Trial Listings
    • Market Research
    • FDA Approved Drugs
    • Training Guides
    • Books
    • eLearning
    • Events
    • Newsletters
    • White Papers
    • SOPs
    • eCFR and Guidances
  • White Papers
  • Trial Listings
  • Advertise
  • COVID-19
  • iConnect
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » PPMD calls for new collaborative approach to benefit/risk analysis with FDA

PPMD calls for new collaborative approach to benefit/risk analysis with FDA

October 9, 2013
CenterWatch Staff

Parent Project Muscular Dystrophy (PPMD), a patient advocacy organization fighting to end Duchenne muscular dystrophy, has proposed a collaboration with the FDA to initiate a rare disease benefit/risk pilot program using Duchenne as the initial therapeutic area.

PPMD issued a paper, Benefit-Risk Assessments in Rare Disorders: The Case for Therapeutic Development in Duchenne Muscular Dystrophy as the Prototype for New Approaches, which explores the benefit/risk paradigms used in both the U.S. and Europe, including patient survey data from a recent PPMD study, and offers recommendations for ways to modify these frameworks to accommodate the unique needs of rare disease communities.

"A robust and functioning system to evaluate and consider the benefits and risks of a potential therapy is critical to the process of reviewing and approving novel therapies," said Pat Furlong, parent project muscular dystrophy founding president and CEO. "It is absolutely essential that reviewers and regulators fully understand the perspective of the patients and parents impacted by such decisions. We also aspire to understand from regulators how to better provide such patient perspectives for their considerations."

While the evaluation of a product's potential benefits and risks is similar whether the product is intended to treat a highly prevalent and well-understood condition or a rare disease, the nature of rare diseases necessitates that special considerations be made.

For example, rare diseases are often not as well-characterized because they lack large patient populations studied over long periods of time, and many rare diseases do not have any approved therapies or means of prevention to serve as points of reference. To compensate for these deficiencies, regulators and other stakeholders must embrace modified assessment frameworks that protect patient safety while at the same time supporting innovation and efforts to develop life-enhancing and life-saving medications.

"Duchenne offers the FDA the ideal candidate for a rare disease benefit/risk pilot program. While Duchenne is a rare and fatal disease with no approved treatments, the disease progression is steadily becoming better understood and a number of potential therapies are in various stages of the development and clinical trial process," said Furlong. "As FDA prepares to receive these Duchenne drug applications, a Duchenne benefit/risk pilot and complementary research and development initiatives with government, industry and academia are vital to our success. PPMD is committed to creating the unique tools and delivering the strategic advocacy that only the patient community can provide to this vital work."

PPMD recently completed the first-ever rigorous scientific survey of benefit/risk expectations of nearly 120 Duchenne parents/guardians, producing a unique data resource to help power the proposed pilot. Data from the study illustrates a relatively high level of risk tolerance if the potential benefit is slowing or stopping the progression of muscle weakness. The preliminary survey data was presented to FDA leadership in a meeting in July, and PPMD continues to work with the agency to apply this approach to new knowledge.

"Our survey and now this paper are very much in line with FDA's patient-focused drug development initiative. Undertaking a rare disease benefit/risk assessment pilot program with Duchenne as the initial focus will help achieve continued progress in our fight to end Duchenne and provide a framework and foundation upon which similar rare disease benefit/risk programs can be built," Furlong said.

Upcoming Events

  • 16Feb

    Fundamentals of FDA Inspection Management: Reduce Anxiety, Increase Inspection Success

  • 21May

    WCG MAGI Clinical Research Conference – 2023 East

Featured Products

  • Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

    Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

  • Surviving an FDA GCP Inspection

    Surviving an FDA GCP Inspection: Resources for Investigators, Sponsors, CROs and IRBs

Featured Stories

  • Revamp-360x240.png

    Califf Calls for Major Evidence Generation Revamp, Experts’ Opinions Differ

  • AskTheExpertsGreen-360x240.png

    Ask the Experts: Managing Investigational Products

  • SurveywBlueBackground-360x240.png

    Survey Outlines Site Challenges, Successes on Diversity

  • PatientCentricity-360x240.png

    Site Spotlight: DM Clinical Shows Patient Centricity Doesn’t Have to Break the Bank

Standard Operating Procedures for Risk-Based Monitoring of Clinical Trials

The information you need to adapt your monitoring plan to changing times.

Learn More Here
  • About Us
  • Contact Us
  • Privacy Policy
  • Do Not Sell or Share My Data

Footer Logo

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

Phone 617.948.5100 – Toll free 866.219.3440

Copyright © 2023. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing