Transcript Investigational Drugs

Investigational Drugs
in the hospital
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What is Investigational Drug?
Investigational or experimental drugs are new drugs that have
not yet been approved by the FDA or approved drugs that have
not yet been approved for a new use, and are in the process of
being tested for safety and effectiveness.

new drugs

old drugs- new indications, new combinations, new route, new
dosage

investigational use of commercially available drugs
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Investigational New Drug
Applications (INDs)

The industry must submit a form called Investigational New
Drug IND to the FDA
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The form include: chemical information, preclinical data
including animal studies, detailed description of planned
clinical trials.

Purpose: to get approval to begin clinical trials in humans.
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It can only be filed after the study sponsor has identified the
pharmacological profile of the drug and has results from both
acute and short term toxicity studies in animals.
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Investigational New Drug
Applications (INDs)

NO experimental agents administered to patients for
research without an IND
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All IND sponsors have obligations which are specified in the
FDA regulations
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New Agent Development
process
Preclinical
studies
(Animals)
IND
Clinical
studies
(Humans- 3
phases)
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Preclinical Development
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Pre-Clinical Drug testing
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Animal Pharmacology and Toxicology Studies
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Permit an assessment as to whether the product is reasonably
safe for initial testing in humans.
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Provide the starting dose for clinical trials & prediction of the
likely effects of the agent on normal tissue.
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Must be performed before IND submission
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The clinical Investigation
 Divided
to three phases
 Aim: producing
substantial proof for the safety and
efficacy of the drugs
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The clinical Investigation
Phase I

Purpose: to determine pharmacokinetics,
pharmacodynamics, preferred route, safe dosage range,
toxicity information.
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Population : 20-80 healthy adult volunteers with no preexisting conditions, or in patients who have exhausted all
other options (cancer patients, AID’s patients)
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Duration: over 6 months -1 year
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Doses increased gradually until tolerable adverse events
&/or clear sign of therapeutic activity
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The clinical Investigation
Phase II
 Purpose: to
determine whether the new agent has
activity against particular disease
 Population: 100-200
 Duration: over
patients
2 years
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The clinical Investigation
Phase III
 Purpose: Further
define efficacy, safety, and
treating dose.
 Population
 Trials
: 600-1000 patients
usually multicenter studies
 Duration: Last
3+ years
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 FDA
should receive constant reports on the
progress of each Phase
 If
hazard the patient, discontinue the clinical
testing.
 If
significant activity is observed in any disease
during Phase II, controlled trials conducted to
compare the new therapy with the available or
standard therapy
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New Drug Application NDA

After completion of phase 3 trial (safe & effective),
sponsor submits NDA to the FDA requesting approval
for marketing to be available to patients and
physicians.
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Includes: pre-clinical data, clinical data,2 well
designed controlled clinical trials info, manufacturing
methods, kinetics, pharmacology, product quality
assurance, relevant foreign clinical testing, published
reports, proposed package insert for drug.
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Phase IV (Post Surveillance
Studies)
 Drug
is on the market.
 Purpose: gather
more data on safety and
efficacy and identify an advantage over other
therapies
 These
are conducted for the approved
indication, but may evaluate:
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
different doses
effects of extended therapy
drug’s safety in other populations (pregnancy, children, elderly)
New indications
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Treatment INDs
(investigational new drugs )
 to
make certain investigational new drugs
available to desperately ill patients before the
FDA approves them for marketing.
 These
treatment INDs used if:
- no comparable drug available
- immediately life threatening or serious
disease
- drug under investigation & shows promise of
therapeutic benefit
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Institutional Review Board
Approval IRB
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An administrative body established to protect
the rights and welfare of human subjects
(including patients) recurited to participate in
research activities
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IRB has the authority to approve, require
modification or disapprove all research
including humans.

Information related to the study proposal is
submitted to the IRB
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Informed Consents
A
document should signed by the patient
 Purpose: to
educate the patient about the
objectives of clinical trial including the known risk &
benefit of each therapy or procedure and any
alternative therapies available
 Investigational
agents may be used emergently or
not under the format of a clinical trial.
 Informed
consent and IRB approval or notification
is required for emergent use.
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Single- and Double- Blind
studies
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Evaluate an investigational drug by comparing its action to a
placebo or controlled drug.
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Single-blind Study: patient is unaware of drug therapy
course.
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Double-blind Study: the patient, nurse and physician are
unaware of which therapy the patient receives.
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Better
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Drug therapy are randomized
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Randomization helps to eliminate the bias
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The role of Hospital Pharmacist
in Investigational drug
Basic Activities:
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registration of investigational drug in hospital
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procurement
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Storage, preparing, & dispensing
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maintenance of records
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Provision of drug information
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Reordering Investigational Drugs
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The role of Hospital Pharmacist
in Investigational drug
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Disposal of Used or Unused drug
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Computerization of drug ordering and control
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IRB Membership
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Pharmacotherapeutic monitoring of invest. Therapy
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Patient counseling and education
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Data manager
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Adverse Drug Reaction Reporting
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