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

2. 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/programs through which patients can access Drugs: Expanded Access Single Patient Access D EFINITIONS

3. 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) EXPANDED ACCESS

4. “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)www.fda.gov The need for expanded access is recognized by patient advocacy groups, physicians, and medical researchers E XPANDED A CCESS (C ONT.)

5. 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 SINGLE PATIENT ACCESS

6. 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. JUSTIFICATION

7. A RGUMENTS TO S UPPORT E XISTING P ROGRAMS

8. 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 W HO B ENEFITS :

9. 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 EXAMPLE

10. W HAT ABOUT PATIENT SAFETY ?

11. Tight control and oversight by the FDA and physicians Companies not obligated to provide the drug Limited amounts of drug, costly to produce extra S AFEGUARDS IN P LACE FOR C URRENT P ROGRAMS

12. Both the FDA and company need to be in agreement that the drug can be used outside of trial S AFEGUARDS ( CONT.)

13. 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 B ENEFIT TO R ESEARCHERS

14. ● 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 Conclusion

15. http://www.policymed.com/2013/06/fda-expanded-access-to- investigational-drugs-for-treatment-use-draft-guidance.html http://www.policymed.com/2013/06/fda-expanded-access-to- investigational-drugs-for-treatment-use-draft-guidance.html http://www.cancer.org/treatment/treatmentsandsideeffects/clini caltrials/compassionate-drug-use http://www.cancer.org/treatment/treatmentsandsideeffects/clini caltrials/compassionate-drug-use https://humansubjects.stanford.edu/research/documents/Expa ndedAccess_GUI01019.pdf https://humansubjects.stanford.edu/research/documents/Expa ndedAccess_GUI01019.pdf http://www.idispharma.com/sites/default/files/uploads/Life- Science-Leader.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 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/ 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 REFERENCES