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Compassionate Drug Use

Compassionate Drug Use. Group 4 Andrew Nicklawsky, Sarah Cusick, Zequing Lu, Anna Nordell, Jun Park, Pamela Portschy, Dongiuan Xu, Dariya Yegorova. Definitions. Treatment of a seriously ill patient with a new, unapproved drug/proc edure/device when no other treatments are available

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Compassionate Drug Use

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  1. Compassionate Drug Use Group 4 Andrew Nicklawsky, Sarah Cusick, Zequing Lu, Anna Nordell, Jun Park, Pamela Portschy, Dongiuan Xu, Dariya Yegorova

  2. Definitions • Treatment of a seriously ill patient with a new, unapproved drug/procedure/device when no other treatments are available • Legal – first approved in 1987 • Two ways/programsthrough which patients can access Drugs: • Expanded Access • Single Patient Access

  3. EXPANDED ACCESS • Drug in Phase III of clinical trial – patients who were not able to enroll initially, or at all, can access drug • FDA allows Expanded Access if it can demonstrated to work in some capacity • 21 CFR 312.300 (Subpart I)

  4. Expanded Access (Cont.) • “Expanded access, sometimes called "compassionate use," is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options. “ (www.fda.gov) • The need for expanded access is recognized by patient advocacy groups, physicians, and medical researchers

  5. SINGLE PATIENT ACCESS • Patients who did not enter in the clinical trial and were unable to qualify for expanded access may lobby for use of the drug • Doctor must request drug • If company allows use, doctor and company ask for FDA approval

  6. JUSTIFICATION • Life-threatening diseases where the patient’s prognosis is extremely poor in the near future • No comparable or satisfactory therapeutic alternatives • Access and administration of the drug is tightly controlled by the FDA and the patient’s physician • Companies are not required to make their drug available through expanded access, or to make more of a drug for that purpose.

  7. Arguments to Support Existing Programs

  8. Who Benefits: • Patients with the following characteristics may be able to receive drugs that are not yet available on the open market • Advanced disease • Established treatments did not work • Ineligible for current clinical trials • Benefit outweighs the potential risk

  9. EXAMPLE • Clinical Trial for Iressa – oral agent with little toxicity • Iressa showed promised in reducing tumors in patients with nonsmall cell lung cancer (75% of lung cancers fall in this category) • 10% of subjects enrolled in trial had dramatic results during Phase I • Opened up to expanded access • 20,000 patients were enrolled who had nonsmall lung cancer and were unresponsive or could not take chemo

  10. What about patient safety?

  11. Safeguards in Place for Current Programs • Tight control and oversight by the FDA and physicians • Companies not obligated to provide the drug • Limited amounts of drug, costly to produce extra

  12. Safeguards (cont.) • Both the FDA and company need to be in agreement that the drug can be used outside of trial

  13. Benefit to Researchers • Gain a better understanding of the medication it a real world clinical setting • See potential effects/side effects not evident through tightly controlled trials • Generate hypotheses for future trials

  14. Conclusion • Expanded Access Programs provide hope for those who may have none • Can help to provide additional information on both the positive, and unfortunately, negative effects of experimental medical procedures, drugs, and devices • Critics often point to the higher number of deaths and adverse events that occur • Fail to take into account the fact that expanded access programs target population

  15. REFERENCES • http://www.policymed.com/2013/06/fda-expanded-access-to-investigational-drugs-for-treatment-use-draft-guidance.html • http://www.cancer.org/treatment/treatmentsandsideeffects/clinicaltrials/compassionate-drug-use • https://humansubjects.stanford.edu/research/documents/ExpandedAccess_GUI01019.pdf • http://www.idispharma.com/sites/default/files/uploads/Life-Science-Leader.pdf • http://dash.harvard.edu/bitstream/handle/1/8965551/Benjamin%20Rossen%20course%20and%203L%20Paper%20-%20Proposed%20Rules%20to%20Expa.pdf?sequence=1 • https://www.nlm.nih.gov/services/ctexpaccess.html • http://www.foxnews.com/health/2014/03/26/dying-25-year-old-fights-for-compassionate-use-access-to-cancer-treatment/ • https://www.nlm.nih.gov/services/ctexpaccess.html • National Academy of Sciences report, 1991 - Expanding access to investigational therapies for HIV infection and AIDS

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