research & pharmacy profession phcl 311 hadeel al-kofide m.sc

Research & Pharmacy Profession

PHCL 311

Research & Pharmacy Profession

PHCL 311

Hadeel Al-Kofide M.Sc

Why do we need research?Why do we need research?

• Promotion

• Better services to patients

• Better for our career

• Investigate problem

• Gain knowledge

• Prosper for the country/humanities…

Research & Pharmacy PracticeResearch & Pharmacy Practice

• Pharmacist is involved in the characterization, optimization &

selection of new drug candidates

• Pharmacists develops & tests new formulations & drug

delivery systems

• Pharmacists develops analytical methods for new active

principles & pharmaceutical forms

Different Research Fields in Pharmacy Profession

Different Research Fields in Pharmacy Profession

• Universities

• Research institutes

• Industrial (drug companies)

• Hospitals

• Others

Qualifications Needed to Work in Research Area

Qualifications Needed to Work in Research Area

• The normal path to a career in research is to gain high

academic achievement in the BPharm programme to earn

direct entry to PhD studies

• PhD studies can be undertaken entirely within a university or

jointly with industry or a research institute

Types of Studies Used in Hospital PharmacyTypes of Studies Used in Hospital Pharmacy

• Survey

• Clinical trials

• Questionnaires

• Case control

• Cross-sectional & follow-up approach

Important Elements of a Research PaperImportant Elements of a Research Paper

• Abstract

• Introduction

• Methodology

• Results

• Discussion

• Conclusion


Abstract

• An abstract is a brief summary of a research article, thesis,

review, conference proceeding or any in-depth analysis of a

particular subject or discipline

• It is often used to help the reader quickly ascertain the paper's

purpose

• When used, an abstract always appears at the beginning of a

manuscript, acting as the point-of-entry


Introduction

• Usually contains background information for the study:

Current treatment paradigms for the disease

Reasons why treatment under investigation may offer benefit

• Contains study objectives


Methodology

• Usually contains:

Study design: Randomization, blinding & controls

Study population (Inclusion & exclusion criteria)

Sample size

Data collection

Statistical analysis


Results

• Contains results (data) of the study

• Could be in written or table format

• No interpretation of data


Discussion

• Interpretation of data (explains why you are getting these

results) & how it relates to clinical practice

• Relates to previous studies in the same field

• Recommendation for future research

• Limitations?


Conclusion

• Summary of the results, usually 1 – 2 statements

• May be mentioned in the end of discussion section

Challenges That Face Research in Saudi Arabia

Challenges That Face Research in Saudi Arabia

• Group Discussion

Investigational Drugs & The Role of Pharmacists

PHCL 311

Investigational Drugs & The Role of Pharmacists

PHCL 311

Hadeel Al-Kofide MSc

Topics to be covered todayTopics to be covered today

• Definitions

• FDA regulations in regards to drug approval

• The clinical investigations

• Investigational review board

• The role of hospital pharmacists

• Single & double blinded studies

• Formulation & Development of A Dosage Form

• In summary

• Drug withdrawal

What is Investigational New Drug?What is Investigational New Drug?

• Investigational New Drug (IND) program is the means by

which a pharmaceutical company obtains permission to ship

use an investigational drug before a marketing application for

the drug has been approved. The FDA reviews the IND

application for safety to assure that research subjects will not

be subjected to unreasonable risk. If the application is

approved, the candidate drug usually enters a Phase 1 clinical

trial

Clinical Trial or StudyClinical Trial or Study

• Any investigation in human subjects that is intended to

discover or verify the clinical, pharmacological or other

pharmacodynamic effects of an investigational product, to

identify any adverse reactions or to study absorption,

distribution, metabolism & excretion of an investigational

product with an object of ascertaining its safety, efficacy or

both

Definitions

FDA RegulationsFDA Regulations

• Before an investigational drug be can be used in clinical

settings the sponsor must submit a Notice of Claimed

Investigational Exemption for a New Drug (FDA form 1571)

• It contains 15 points

FDA RegulationsFDA Regulations

1. Complete composition of the drug, its source & manufacturing

data

2. Results of all preclinical investigations

3. Background information of each clinical investigators (training,

experience & facilities)

4. An outline or protocol of the phases for clinical investigation

• Also other requirements (total of 15)

Drug DevelopmentDrug Development

1. Preclinical development

2. The clinical InvestigationExtensive research that is done on

compounds before therapeutic

indication found

Millions of compounds are tested

in an attempt to find one that may

have a pharmaceutical or medicinal

use

The Clinical InvestigationThe Clinical Investigation

• Phase 1

• Phase 2

• Phase 3

Phase 1Phase 1

• They are designed to determine a safe dose for phase 2 trials

• The purpose of these studies include: the determination of

human toxicity, absorption, metabolism, elimination,

pharmacodynamic, preferred route of administration & safe

dosage range

• It involves a small number of persons (20-80)

The Clinical Investigation

Phase 2Phase 2

• Limited number of patients (100-200)

• Designed to investigate drug efficacy & relative safety

• Want to determine if the new drug has activity against a

particular disease


Phase 3Phase 3

• These trials will be initiated if the information obtained from the

first two phases demonstrate reasonable assurance of safety &

effectiveness or suggests that the drug may have a potential

value outweighing possible hazards

• Done to determine a new drug’s safety, effectiveness & most

desirable dosage in treating a specific disease in a large group of

subjects

• Phase 3 trials are mostly conducted as a multi-centered trials


Institutional Review Board (IRB)Institutional Review Board (IRB)

• IRB is an administrative body established to protect the rights

& welfare of human subjects (including patients) recruited to

participate in research activity

• The IRB has the authority to approve, require modification (in

order to approve), or disapprove all research activities

involving humans that fall under 1 or more justification

criteria


• Justification criteria for IRB:

1. The research is sponsored by the organization

2. The research is conducted by or under the direction of any employee

or agent of the organization in connection with institutional

responsibility

3. The research interventions or interactions are performed at an

organization property &/or this research involves the use of this

institution's nonpublic information


• Information related to the study proposal is submitted to the

IRB investigators

• For submission usually use an IRB cover sheet

• Also must report any problems like serious adverse effects,

changes in the protocol that may affect the status of the

investigation & the willingness of patients to participate in it

The Role of Hospital PharmacistsThe Role of Hospital Pharmacists

• The extent of the role of hospital pharmacists in investigational

drugs depend upon:

Type of hospital

Hospital facilities

Equipments

Specialized services

Expertise of the pharmacy department


• The basic roles of hospital pharmacists in investigational drugs:

Registration

Control

Storage

Dispensing

Maintenance of disposition records

Drug information for every investigational drugs

In order to carry on these basic roles it is imperative that the

hospital adopt certain policies for the use of investigational drugs


• Although the FDA does not require that investigational drugs

be stored & dispensed through a hospital pharmacy, many

hospitals require that all clinical investigational drugs to be

stored & dispensed by the hospital pharmacy

• Some hospitals have established a policy that no

investigational drug be administered to a patient unless it bears

a pharmacy department control or registration number


• Some advantages of having a role for hospital pharmacies in

investigational drugs include:

Drugs can be stored, dispensed & accounted for

They can be observed for shelf-life & stability and then if

needed it could be sent back to the manufacturer

Investigational drug pharmacists are now being an essential part of the use of new

agents in different hospitals


• When the clinical investigator has written a clinical research

protocol they meet with the pharmacy to discuss the handling

& dispensing of the drug

• Certain points should be obtained at this stage


• Information obtained at first encounter with the investigator:

1. Name of the drug

2. Dosage form & strength

3. Pharmacology

4. Purpose of the investigation (including copy of the proposal

which was already approved by the FDA & other required

committees)



5. Route of administration

6. Dosage

7. Side effects, toxicity & any known antidote

8. Storage conditions

9. Stability of product



10.Manufacturer

11.Name of the principal investigators & co-investigators

authorized to prescribe

12.Nursing unit

13.Sponsor

14.How the drug will be written or called



15.Re-label or special label (with a caution saying: New Drug:

limited by Federal Law to Investigational Use)

16.Arrangement for obtaining additional drug along with

tentative utilization rate


• These initial information should be recorded on a special form

called Investigational Drug Service Request Form: IRB cover

sheet

• After the pharmacist meet with the physician the product is

labeled with a control number, a disposition record is prepared

& the drug is stored


• Then the clinician can prescribe the new drugs for

participating patients

• Before dispensing the medication pharmacy must check the

disposition record to see if the physician writing the order is

an authorized investigator

Single & Double Blinded StudiesSingle & Double Blinded Studies

• Pharmacists can be very helpful in establishing a single or

double blinded studies

• Single blinding

• Double blinding


• The role of pharmacist in single blinded studies:

Develop a simple system for coding drugs

Assigning a series of numbers or letters with the drug &

placebo

Example: compound name 429 assign series A, C & T for

the drug, & B, E & Y for placebo, how?


• The role of pharmacist in single blinded studies:

Sometimes it is requested to break the blinding for medical

emergencies, so if pharmacist was asked to break the

blinding he must determine the reason


• The role of pharmacist in double-blinded studies:

Use random generating tables, how?

Sometimes manufacturer does all the blinding & sends the

drug & placebo to be directly given to the patient

Formulation & Development of A Dosage Form

Formulation & Development of A Dosage Form

• If an investigator which to carry on a study but no

manufacturer is interested in sponsoring

• Pharmacists can formulate the new dosage form for this drug

• Must initially conduct studies to insure the identity, strength,

quality & purity of the compound

Expanded Role for Hospital PharmacistsExpanded Role for Hospital Pharmacists

• Other than the basic role they play in investigational drug

within a hospital they have additional value in:

Study blinding

Formulating a new dosage form

In SummaryIn Summary

• Pharmacists play a role in investigational drug development

• With their expertise they participate in labeling, storage &

dispensing of all drugs in the hospital

• Pharmacy is the logical place for the central repository for all

investigational drug information including the investigational

new drug number

WAIT….We want to talk a little about the drug

withdrawal

WAIT….We want to talk a little about the drug

withdrawal

FDA & Drug ApprovalFDA & Drug Approval

• During the 1980s & early 1990s, the FDA was criticized for

taking too long to review & approve drugs

• Then Congress, the FDA, & the pharmaceutical industry

negotiated the Prescription Drug User Fee Act (PDUFA) of

1992

• Under PDUFA, drug companies pay fees that allow the FDA

to add more resources & speed up drug review time

Myths About Drug Withdrawals & User FeesMyths About Drug Withdrawals & User Fees

The reality is that it's a rare occasion when a drug is taken off the market.

The drug withdrawal rate has been constant over the last two decades, when comparing the time before user fees & after, there has been no change in the rate of drug withdrawals (2.7% before user fees & 2.8% after user fees)

Faster reviews to get valuable & life-saving drugs on the market does not translate into safety shortcuts

It isn't that the same number of FDA reviewers are working faster

It also isn't the case that shorter FDA review times mean abbreviated clinical drug trials

Myths About Drug Withdrawals & User FeesMyths About Drug Withdrawals & User Fees

But Still FDA is Facing Serious Problems for Fast Drug Approving

But Still FDA is Facing Serious Problems for Fast Drug Approving

Example of Significant Drug WithdrawalsExample of Significant Drug Withdrawals

Drug name Withdrawn Remarks

Thalidomide 1950s–1960s

Withdrawn because of risk of teratogenicity; returned to market for use in leprosy & multiple myeloma under FDA orphan drug rules

Fen-phen (popular combination of fenfluramine & phentermine)

1997

Phentermine remains on the market, dexfenfluramine & fenfluramine – later withdrawn as caused heart valve disorder

Cerivastatin (Baycol, Lipobay)

2001Withdrawn because of risk of rhabdomyolysis

Rofecoxib (Vioxx)

2004Withdrawn because of risk of myocardial infarction

Thank youThank you

research & pharmacy profession phcl 311 hadeel al-kofide m.sc

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