summit on cancer clinical trials - v dilimmas and complexities of compassionate use
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SUMMIT ON CANCER CLINICAL TRIALS - V Dilimmas and Complexities of Compassionate Use. What is Compassionate Access?. Expanded Access Protocols and Single Patient Treatment INDs. Expectation of Benefit vs. False Hope Is There a Conflict with Clinical Trials? - PowerPoint PPT PresentationTRANSCRIPT
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SUMMIT ON CANCER CLINICAL TRIALS - VDilimmas and Complexities of Compassionate Use
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What is Compassionate Access?
• Expanded Access Protocols and Single Patient Treatment INDs.
• Expectation of Benefit vs. False Hope
• Is There a Conflict with Clinical Trials?
• When Should Compassionate Access Be Granted and Who Decides?
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What is Compassionate Access?
• Expanded Access Protocol – A treatment regimen for a predefined group of patients
• Single Patient Treatment IND – A treatment plan for an individual who does not meet the eligibility requirements of the clinical trial protocol
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When is Expanded Access Appropriate?
• Expanded access protocols should be based on a realistic possibility of therapeutic benefit
• Protocol should be designed so that it does not conflict with Phase III trial enrollment
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When is Single Patient Treatment Appropriate?
• Experimental agent has not been (and may never be) tested for efficacy in a particular patient’s type of cancer or specific medical condition and the patient is not eligible for a clinical trial
• Treatment use of an investigational drug may be the only opportunity for an extension of life
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What is the probability of benefit from prescription drugs?• Commonly prescribed drugs have limited
efficacy/response– ACE-1 10-30%– beta-blockers 15-35%– SSRIs10-25%– tricyclic AD 20-50%– statins 10-60%
• Significant occurrence of adverse reactions– 2.1 million people hospitalized annually due to
adverse drug reaction– Of those, 106,000 end in fatality
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What is the probability of benefit from an experimental cancer drug?
• Unknown in the best of circumstances• Improbable in the typical context of
compassionate access• Example: C225 produced a 22% partial
response of ~ 186 day duration
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Reasons for Publicity About Treatment Advances
• Peer-reviewed results are of clinical practice significance
• Peer-reviewed results are preliminary/speculative and interesting
• Political agenda (NCI, HHS looking for funding from Congress
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More Reasons for Publicity About Treatment Advances
• Announcement designed to promote a product or a company
• Announcement mandated by government for nonmedical reason (SEC compliance)
• Inadvertent misrepresentation of science by the press
• Fraud
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Optimism About Experimental Drugs
• With currently approved drugs: Hope is for high probability of benefit with possibility of harm; Reality is certainty of harm with possibility of benefit.
• Is risk – benefit consideration an individual decision or a group decision?
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Notes from ODAC: Reasons to Deny Compassionate Access
• “Unbridled treatment use of investigational drugs may interfere with enrollment in clinical trials to evaluate the safety and effectiveness of new drugs.”
• “Sponsors and FDA may be concerned that patients may refuse to enroll in a randomized trial designed to compare standard treatment to experimental treatment if the experimental treatment is available outside of trials.”
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Does Compassionate Access Interfere With or Complement Clinical Trials?
• Policies regarding eligibility for clinical trials and compassionate access
• Compassionate access and accelerated enrollment in pivotal trials
• Insights into broader uses of experimental drug?
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Compassionate Access and Crossover Design
• A crossover design can speed accrual
• If the experimental agent is a winner, crossover will not obscure effectiveness
• If the experimental agent is a winner, compassionate access helps individuals and adds to understanding safety
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Notes from ODAC: Reasons to Deny Compassionate Access
• “Sponsors may not have sufficient drug supply to support widespread treatment use.”
• Investment required to supply a new drug during trials and after FDA approval
• Fairness in rationing– No policy– Lottery– Sickest patient first– Most likely to benefit first
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Notes from ODAC: Reasons to Deny Compassionate Access
• “Sponsors may worry that adverse events from treatment use reported in patients who have a poor performance will have an adverse impact on drug development.”
• Are insights into safety resulting from compassionate access important?
• Policy and “the greater good.”
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Treatment Use During Clinical Research: Is There Conflict?
• Realistic Expectation of Benefit versus False Hope
• Faster or Slower to Market
• What is Fair and to Whom?
• Who Decides and How?