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ACCELERATED APPROVAL

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Make Rapid Access to Medical Advances A Higher Priority. Moving Beyond ... System is Needed to Deliver Medical Advances to Seriously Ill Americans That Are ... – PowerPoint PPT presentation

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Title: ACCELERATED APPROVAL


1
ACCELERATED APPROVAL Moving Forward for Cancer
Patients
2
Accelerated Approvalin the Approval Process
  • Accelerated Approval (Subpart H)
  • Fast Track
  • Priority Review

3
Accelerated ApprovalWhat Cancer Patients Needed
  • Support of Accelerated Approval by Industry and
    FDA
  • Meaningful Participation by Industry
  • Effective Communication Between FDA and Industry
  • Adequate Supporting Clinical Trials
  • Regulatory Acceptance of Surrogate Endpoints
  • Realistic Evaluations of Risk/Benefit and
    Clinical Benefit
  • Flexibility at the FDA
  • Sense of Urgency at the FDA
  • Timely Approvals
  • Meaningful and Implementable Phase IV Trials

4
Accelerated ApprovalWhat Cancer Patients Are
Getting
  • Inconsistent Support of Accelerated Approval at
    FDA
  • Meaningful Participation by Industry
  • Insufficient Communication Between FDA and
    Industry
  • Inconsistent Supporting Clinical Trial Design
  • Limited Regulatory Acceptance of Surrogate
    Endpoints
  • Unrealistic Risk vs Benefit and Clinical Benefit
    Evaluations
  • Lack of Flexibility at the FDA
  • Sense of Urgency Rarely Evident
  • Some Timely Approvals / Too Many Delayed
    Approvals
  • Inconsistent Usefulness and Implementability of
    Phase IV Trials

5
Why Has Accelerated ApprovalUnderperformed for
Cancer Patients?
  • Over-Reliance on Statistics and Process Instead
    of on Recognizing and Approving Drugs that
    Represent Best Available Care Based on Realistic
    Risk/Benefit to Patients
  • Over-Emphasis on Potential Adverse Effects of
    Treatment When Compared to Disease-Related
    Morbidity and Mortality
  • Failure to Recognize Patients Right and Ability
    to Make Decisions About How They Try to Live
  • Result An Accelerated Approval Program That Has
    Not Lived Up to Expectations

6
How to Move Accelerated Approval Forward
  • Accept and Support Not Just the Letter But Also
    the Spirit of Accelerated Approval Intent
  • Standards for Accelerated Approval Should Not be
    Moved Closer to the Standards for Full Approval
  • Redefine Clinical Benefit to More Realistically
    Serve Patients With Life-Threatening Diseases
  • Defer More Decision-Making to the Physician and
    Patient in the Post-Accelerated Approval Setting
  • Recognize the Urgent Need for Timely Approvals
    Not Just Timely Reviews

7
Message for Todays Meeting
  • Patients Need Phase IV Trials or Phase IV
    Non-Clinical Studies That Provide Information for
    More Effective Use of New Drugs Not
    Confirmation of Efficacy Already Demonstrated in
    Previous Trials
  • Phase IV Clinical Trials Should be Ethical and
    Enrollable in a Post-Approval Setting Meaning
    That Randomized and Placebo-Controlled Trials
    Will Often Be Impracticable Use More Historical
    Controls
  • Challenges with Designing Post-Approval Trials
    Should Not Necessarily Be Considered a Problem
    with Accelerated Approval In Some Cases the
    Challenges Result from Problems with the
    Regulations and Policy
  • Make Rapid Access to Medical Advances A Higher
    Priority

8
Moving BeyondFast Track / Accelerated Approval
  • The Fast Track / Accelerated Approval Model Has
    Failed to Serve a Large Segment of Our Patient
    Population
  • A New Tiered Approval System is Needed to Deliver
    Medical Advances to Seriously Ill Americans That
    Are Being Abandoned by the Current System

9
Tiered Approval System
  • Tier 1 New Initial Approval Based on Limited
    Evidence of Safety and Efficacy Adequate to
    Support Determination that Treatment Represents
    Best Available Care for Patients with Terminal
    Diseases and No Other Options
  • Tier 2 Accelerated Approval Evidence of Safety
    and Effectiveness Based on Surrogate Endpoints
    Post-Approval Testing (Phase IV) Required to
    Confirm Clinical Benefit
  • Tier 3 Full Approval All Pre-Clinical and
    Clinical Testing Needed to Demonstrate Clinical
    Benefit Completed Post-Approval Testing Not
    Required

10
Tier 1 Initial Approval
  • Based on Limited Evidence of Safety and Activity
    From Phase I or Phase II Trials
  • Marketing Restricted to Patients With
    Life-Threatening Diseases, No Approved Treatment
    Options, and No Reasonable Access Through
    Clinical Trials or EAPs
  • Informed Consent Required
  • Continued Diligent Pursuit of Accelerated or Full
    Approval Required

11
Proof of Concept
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