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/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
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)
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 lifethreatening 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
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
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.
ARGUMENTS TO SUPPORT EXISTING PROGRAMS
WHO BENEFITS:
• Patients with the following characteristics may
be able to receive drugs that are not yet
available on the open market
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–
–
Advanced disease
Established treatments did not work
Ineligible for current clinical trials
Benefit outweighs the potential risk
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
WHAT ABOUT PATIENT SAFETY?
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
SAFEGUARDS (CONT.)
• Both the FDA and company need to be in
agreement that the drug can be used outside
of trial
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
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
REFERENCES
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http://www.policymed.com/2013/06/fda-expanded-access-toinvestigational-drugs-for-treatment-use-draft-guidance.html
http://www.cancer.org/treatment/treatmentsandsideeffects/clini
caltrials/compassionate-drug-use
https://humansubjects.stanford.edu/research/documents/Expa
ndedAccess_GUI01019.pdf
http://www.idispharma.com/sites/default/files/uploads/LifeScience-Leader.pdf
http://dash.harvard.edu/bitstream/handle/1/8965551/Benjamin
%20Rossen%20course%20and%203L%20Paper%20%20Proposed%20Rules%20to%20Expa.pdf?sequence=1
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https://www.nlm.nih.gov/services/ctexpaccess.html
http://www.foxnews.com/health/2014/03/26/dying-25-year-oldfights-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