cra demo presentation

7/28/2019 CRA Demo Presentation

1/17

11/11/04

Clinical Research and Development

in the Pharmaceutical andBiotechnology Industry

Robert Anderson, MHA, CCRA, CCRCP

Director, Clinical Trials AdministrationThe CRA Training Institute, Houston


2/17

11/11/04

Objectives

To understand the drug developmentprocess

To understand the phases and components

of the clinical research process To appreciate the history behind the

regulations in the clinical development

process

To understand the current regulations

involved with the clinical research process


3/17

11/11/04

Healthcare Industry Players

PharmaceuticalIndustry

Patient

Care

Physician

Hospital

Insurance

Company


4/17

11/11/04

Drug Discovery Process

Compound

Selection

Proof of Concept

Outcome2 4 Years

4 7 Years

Marketing

Introduction

*Key variable guiding

development time

Target Selection

Target Validation

& lead

optimization

Proof of Concept

Clinical Trials

Pre-clinical

Phase

Clinical Phases I, II

III, IIIB*

Drug

Registration

FDA Approval

Marketing

(Phase IV

Clinical Trials)

(5,000 10,000) (250)

(5)(1)


5/17

11/11/04

Clinical Trials: What Are They?

An organized research study designed to

investigate new methods of preventing,

detecting, diagnosing, or treating an illness

or disease (such as cancer).


6/17

11/11/04

The Players in Clinical Research

SponsorInvestigator

SitePatient

CRO

SMO

IRB


7/17

11/11/04

Clinical Trials Process and

Associated Regulatory Process

Phase IIIPre-NDA

Meeting

Phase

IVFile NDA Drug

Approval

Pre-IND Meeting

IND Application

Phase

I

Phase

II

Phase II

End of Phase II

Meeting


8/17


9/17


10/17

11/11/04

What is Involved in a Clinical Trial?

File IND application

Develop protocol

Submit to FDA for comment or

no action

Select investigational sites

based on # of patients

needed for the study

Regulatory requirements for

each trial at each site

1571 or 1572

PIs CV

Financial disclosure forms

Informed consent

IRB approval

Initiate site(s)

Sites enroll patients

Write study report

Patients cycle through study

Capture: Adverse events, vital

signs, study drug adherence,

QOL questionnaires captured

on Case Report Forms

Patients exit study

Data collected and cleaned

Sites closed

Add study to NDA


11/17

11/11/04

Clinical Trials Benefits & Risks

Possible Benefits of Trials Possible Risks of Trials Having access to potentially more

effective therapies than those currently

available

Receiving quality medical care from

leading physicians

Being closely monitored for possiblenegative effects

Sometimes receiving treatment at a

reduced rate or free of charge

Helping to further new research that may

result in significant medical advances

For patients in cancer therapy trialsassigned to control groups, they still

receive the top standard therapy available

today

Patients may not receive the therapy

under investigation (may receive a

placebo inactive pill instead)

The new therapy may not be more

effective than the standard, thoroughly

tested therapy In Phase I trails, not knowing the safety

consequences of the new therapy (risk is

less in Phase III trials)

New therapy may have unexpected,

possibly severe side effects or may be

less effective than standard of care Insurance companies may not cover all

costs of clinical trials


12/17

11/11/04

Clinical Trial Standard Language

Protocol The planned course of action for the clinical trial. The protocol is

established prior to the start of the trial and states the number of

participants, eligibility requirements, agents that will be used,

dosages, duration, how data is collected, etc.

Investigator A researcher in a clinical trial.

Sponsor The part of parties responsible for funding the clinical trial.

InstitutionalReview Board

(IRB)

An independent board of scientists, physicians, and nurses whoreview the clinical trial protocol to ensure patient safety.

Informed Consent A patients decision to participate in the clinical trial after being

informed of the potential benefits and risks of participation.

Participants may withdraw their consent at any time and leave the

trial.

Double blind Term used to describe a clinical trial in which neither the patient nor

the researcher knows which agents are being administered to

which patients. This helps prevent bias.

Invention group The group of participants receiving the new preventive or treatment

agent that is being evaluated in the clinical trial.


13/17

11/11/04

Clinical Trial Standard Language, continued

Control group The group of participants receiving a standard treatment or placebo

(see below) that is being compared to the new agent in the clinical

trial.

Randomization Assigning participants by chance to either the intervention group or

the control group. Randomization is often done with a computer.

Placebo An inactive substance that may be given to participant sin a clinicaltrial. Sometimes called a sugar pill.

Follow-up Monitoring of participants for a specified time after the clinical trial

is completed.

Prospective study A study of a group of patients that is conducted as they are

undergoing a treatment or preventive measure.

Retrospective

study

A study of a group of patients after they have already undergone a

treatment or preventive measure. Recall bias, unintentional

inaccurate reporting of certain information, can sometimes

influence a retrospective study.


14/17

11/11/04

Regulations often result in response to abuse of humanresearch subjects and concerns about the validity of dataand conclusions from clinical trials.

The primary vehicles for human subject protection areIRBs and informed consent.

The Declaration of Helsinki and the Belmont Report arecritical documents for the protection of human subjects inresearch.

The FDA, by means of PDUFA and FDAMA, has madesignificant gains in speeding the process of making new

drugs available for patients who need them. Current problems with clinical trials and trial oversight

may well lead to increased regulation.

History Behind Regulations of Clinical Trials


15/17

11/11/04

The FDA regulations pertaining to clinical trialsare found in 21 CFR Parts 11, 50, 54, 56, 312and 314.

The ICH Guidelines for Good Clinical Practice

should be followed in clinical trials. The FDA publishes many guidelines and

information sheets pertaining to the appropriateconduct of clinical trials.

Good clinical practices are the ethical andclinical standard for designing, conducting,analyzing, monitoring and reporting on clinicaltrials.

Regulations for Clinical Trials


16/17

11/11/04

Health Outcomes

Health outcomes studies examine the clinical, economic and quality-of-life outcomes of pharmacotherapy.

Health outcomes research expands upon the FDA-mandated efficacyand safety endpoints to give a fuller picture of the outcomesexperienced by a patient. It is a relatively new discipline that combinesa number of fields of study, including medicine, epidemiology, statistics,

economics and psychometrics. Early in development, companies may be interested in documenting the

epidemiology and cost burden of a particular disease state.

As a compound moves through to Phase II and II, behavioral,humanistic and economic endpoints may be incorporated intoregistration trials.

Concurrently, economic models may be created to quantify theeconomic benefit of the new therapy.

Once a compound is launched, a variety of research services may beutilized, including registries, Phase IIIb/IV comparative studies andclaims analyses.


17/17

11/11/04

Other Issues in the Clinical Research

Process

National Institutes of Health

Special populations

Data Safety Monitoring Boards Orphan drugs

cra demo presentation

Documents