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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? • When Should Compassionate Access Be Granted and Who Decides? 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 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 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 What is the probability of benefit from prescription drugs? • Commonly prescribed drugs have limited efficacy/response – – – – – ACE-1 beta-blockers SSRIs tricyclic AD statins 10-30% 15-35% 10-25% 20-50% 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 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 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 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 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? 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.” 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? 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 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 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.” 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? Thank you • Questions via e-mail? • [email protected]