Scope of clinical research
Scope of clinical research
Prof K M Prasanna Kumar
Sr prof & Head-Endocrinology & Diabetes
M S Ramaiah Medical college
Are people guinea pigs and mice?
Men are not mice
Ethnic and racial variations
Pattern of clinical conditions/ management
Reporting of unusual clinical situations
Reporting of index case
A systematic study designed to evaluate a
product (drug, device, or biologic) using
human subjects, in the treatment, prevention,
or diagnosis of a disease or condition, as
determined by the product's benefits relative to
its risks. Clinical investigations can only be
conducted with the approval of the Food and
Drug Administration (FDA)/DCGI
Any investigation in human subjects intended
to determine the clinical pharmacological,
pharmacokinetic, and/or other
pharmacodynamic effects of an investigational
agent, and/or to identify any adverse reactions
to an investigational agent to assess the agent's
safety and efficacy.
Study of drug, biologic or device in human
subjects with the intent to discover potential
beneficial effects and/or determine its safety
and efficacy. Also called clinical study and
clinical investigation. This does not encompass
all the research that is carried out in the clinical
setting (e.g., health services research).
Invitaion from sponsor
Review of protocol
People participate in clinical research for a
variety of reasons. People who volunteer for
phase II and phase III trials can gain access to
promising drugs long before these compounds
are approved for the marketplace. They
typically will get excellent care from the
physicians during the course of the study. This
care also may be free.
Contract Research Organization (CRO)
A person or an organization (commercial,
academic or other) contracted by the sponsor to
perform one or more of a sponsor's studyrelated duties and functions
Good Clinical Practice (GCP)
International ethical and scientific quality
standard for designing, conducting,
monitoring, recording, auditing, analyzing and
reporting studies. Insures that the data
reported is credible and accurate, and that
subject's rights and confidentiality are
Institutional Review Board (IRB)
An independent group of professionals
designated to review and approve the clinical
protocol, informed consent forms, study
advertisements, and patient brochures, to
ensure that the study is safe and effective for
human participation. It is also the IRB's
responsibility to ensure that the study adheres
to the FDA‘/DCGI’s regulations.
A medical professional, usually a physician but
may also be a nurse, pharmacist or other health
care professional, under whose direction an
investigational drug is administered or
dispensed. A principal investigator is
responsible for the overall conduct of the
clinical trial at his/her site.
Case Report Form (CRF)
A record of pertinent information
collected on each subject during a
clinical trial, as outlined in the study
A Phase One Study:
Phase I studies are primarily concerned with
assessing the drug's safety.
This initial phase of testing in humans is
done in a small number of healthy
volunteers (20 to 100), who are usually paid
for participating in the study. The study is
designed to determine what happens to the
drug in the human body--how it is
absorbed, metabolized, and excreted.
About 70 percent of experimental drugs
pass this initial phase of testing.
Once a drug has been shown to be safe, it must be
tested for efficacy. This second phase of testing may
last from several months to two years, and involve up
to several hundred patients.
One group of patients will receive the experimental
drug, while a second "control" group will receive a
standard treatment or placebo.
Often these studies are "blinded"--neither the patients
nor the researchers know who is getting the
Only about one-third of experimental drugs
successfully complete both phase I and phase II studies
In a phase III study, a drug is tested in several
hundred to several thousand patients.
Provides thorough understanding of the
drug's effectiveness, benefits, and the range of
possible adverse reactions. Most phase III
studies are randomized and blinded trials.
70 to 90 % of drugs that enter phase III studies
successfully complete this phase of testing.
Once a phase III study is successfully
completed- approval for marketing the drug.
In late phase III/phase IV studies,
pharmaceutical companies have several
(1) studies often compare a drug with other
drugs already in the market;
(2) studies are often designed to monitor a
drug's long-term effectiveness and impact on a
patient's quality of life; and
(3) many studies are designed to determine the
cost-effectiveness of a drug therapy relative to
other traditional and new therapies.
Funding for clinical research comes from both
the federal government (through the National
Institutes of Health) and private industry
(pharmaceutical and biotech companies).
The sponsor of the research hires physicians,
who may work in a wide variety of health-care
settings, to conduct the clinical trial.
Physicians are typically paid on a per-patient
basis. The medical care is often provided free to
Global Pharmaceutical marketUS $ 427 billion
R&D costs-US $ 60-65 billion
70% of R&D money goes towards drug development
Clinical Trail Research-70% of time&money spentUS $ 13 billion
Focus on reducing costs of clinical development
Contract Research Organizations gaining
Timely clinical trails extremely important,on
account of steadily increasing number of global
Increasing global trend towards collaboration and
Market today is estimated at US $9 billion
Age of globalization
Share resources and benefits
Global pharmaceutical industry undergoing a
New technologies in R&D,health care
costs,consumerism and uniform IP laws are
making companies to rethink and re-align
Focus on R&D productivity, reducing
development time and controlling overall R&D
Indian pharmaceutical industry fast growing
Health care market US $ 40-50 billion
Global in outlook,focus on R&D and NNDS
Tremendous potential for Clinical Research
Potential opportunity US $ 2 billion by 2010
Between 1991-2001 performed clinical research is
estimated US $ 70-80 million
India is getting to be known as a hot
destination for executing clinical trials.
In this mad melee, what are the determinants
How real is the promise of clinical research in
India and what will make or break a new
entrant in this business?
for contract pharma research and development
its huge treatment naïve population,
human resources- Annually 27000 doctors
qualify in India
rules/laws by regulatory authorities, and
Changing economic environment.
Present- 400-500 PI in India
Year 2010 India needs 2000-3000 PI
500-1000 Centres of excellence in clinical
1,00,000 study coordinators, monitors, project
Medical coleges- 254 –offer MBBS
Large hospitals, corporate, private, govt
Centres of international standards for
inspection, site selection.
2000-2005 - 3-5 billion $
2010- 10 bilion $
3% of global clinical research –India
Next 5 years 15% of global clinical research to
be out sourced to India.
Are we ready?
Quality of data?
Speed of recruitment
India-China 20% of world population
Knowledge base to skill based
Contract research to architects of protocoldesign & devt.
Language-English vs China, Japan
Patient diversity- North , South- West-East
1.136 billion- 5% sickness/disease-Inexhaustible
Hospitals, Qualified Doctors
Nurses, beds,Graduates, universities, Colleges
2 largest number of technically qualified people in
Urban vs rural
China- 1 major language- many dialects
India- 24 major languages, 1600 dialects
Many don’t have script
Cost of translation
Infra structure- Air connectivityElectricity-Fax- Internet
Training, acridition- PI, CI, study coordinators,
Poor, illeterate patients
Doctors consider CR as second rate profession
Record keepingElectronic data capture
Infra structure improvement
COE for clinical research
Train CRA, PI, CI, Monitors-Research
Your role- CRA- project manager- VP- own a
CR centre of excellence.