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SUMMIT ON CANCER CLINICAL TRIALS - V Dilimmas and Complexities of Compassionate Use

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?

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SUMMIT ON CANCER CLINICAL TRIALS - V Dilimmas and Complexities of Compassionate Use

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  1. SUMMIT ON CANCER CLINICAL TRIALS - V Dilimmas and Complexities of Compassionate Use

  2. 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?

  3. 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

  4. 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

  5. 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

  6. What is the probability of benefit from prescription drugs? • Commonly prescribed drugs have limited efficacy/response • ACE-1 10-30% • beta-blockers 15-35% • SSRIs 10-25% • 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

  7. 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% partialresponse of ~ 186 day duration

  8. 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

  9. 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

  10. 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?

  11. 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.”

  12. 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?

  13. 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

  14. 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

  15. 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.”

  16. 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?

  17. Thank you • Questions via e-mail? • bob.erwin@lsbc.com

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