BIOEQUIVALENCE / BIOAVAILABILITY

Refers to the relative amount of drug from an administered dosage form, which enters the systemic circulation & the rate at which it appears in the systemic circulation.

PHASE 1 and II STUDIES

To establish basic  safety and blood levels achieved with different  doses of the drug

To establish the drug’s effectiveness and dosage range, to determine drug kinetics, and to identify side effects

PHASE III and IV STUDIES

Phase III: Full-Scale Evaluation of the Treatment (Comparative clinical trial): is a planned experiment on human subjects.

To some people, the term “Clinical trial” is synonymous with such a full-scale phase III trial.

STATISTICAL DATA

“Statistics is the science of collection, organization, presentation, analysis and interpretation of numerical data”.

Statistics is the art and science of making  decisions in the face of uncertainty

MICRODOSING STUDIES

The action or practice of taking or administering very small amounts of a drug in order to test or benefit from its physiological action while minimizing undesirable side effects.

ETHICS IN CLINICAL RESEARCH

Any research study that prospectively assigns human participants or groups of humans to one or more health-related interventions to evaluate the effects on health outcome

Placebo Domain

In medicine, it typically refers to a response observed after inert or inactive treatments.

Louis Lasagna helped make placebo controlled trials as Gold Standard in 1982

Trials in Oncology

Objective tumor shrinkage, or tumor response, has been adopted as a standard end point to select new anticancer agents for further study and has played a role in the development of all drugs approved for use in cancer treatment to date.

Reg Dossier terminologies

What is Active Pharmaceutical Ingredient (API) : 

A substance used in a finished pharmaceutical product (FPP), intended to furnish pharmacological activity or to otherwise have direct effect in the diagnosis, cure, mitigation, treatment or prevention of disease, or to have direct effect in restoring, correcting or modifying physiological functions in human beings."

Reg parts

CHEM manufacturing controls: Declaration of the Composition, Description of the Formulation, Manufacture of the Drug product
Primary packaging material, Specification of the drug product, Test procedures, Results of the stability procedures, Evaluation of stability data. Shelf life of the drug product.

Scrupulous trial budgeting

The estimated amount of money that is required to accomplish the goal of a clinical trial or study. Study budgets are prepared overall and by each study year with costs linked to study tasks and patient recruitment. 

Clinical trials are assumed to be expensive, and efforts are underway to make them more efficient, e.g. through the direct use of routinely collected data in registries or electronic health records

Fast track approvals FDA

Accelerated Approval / Fast Track : Serious and life threatening diseases, Restricted distribution. Approval based on surrogate endpoints reasonably likely to predict clinical benefit, Contingent on follow up studies to confirm therapeutic benefits to patients

Priority review : Products with significant improvement in safety or effectiveness, comes under national programes Not limited to serious and life threatening diseases.

Guidelines for ICH E6 (R2) GCP

GCP is defined as a standard for the design, conduct, performance, monitoring, auditing, recording, analysis and reporting of clinical trials or studies, GCP compliance provides public assurance that the rights, safety and well being of human subjects involved in research are protected

NDA documentation with CDSCO

Forms: 44-Permission to import /trial/approval to manufacture or FDC, 45-Finished formulation import, 45 A - Import raw material, 46- approval to manufacture new drug.

FDA 505 B (2) Pathway

The 505(b)(2) NDA provides a potentially streamlined path for sponsors that have developed improvements to drug products that have
previously received FDA approval. Successful navigation of the process requires close communication with the FDA but the potential
benefits can be significant.

Medical script writing

In order to write effectively, you must identify your audience and decide what story you want them to learn.

Description of IP(s) and comparator Clinical Pharmacology, Indications, and justification for the route of administration.

Protocol writing

Be specific , no general and ambiguous statements, Be realistic , no unachievable aims.

What is your question:  Hypothesis, Why is your study important : Significance, How are you going to do it: Research design

Principles of GCP Nomenclature

Good clinical practice provides a framework of principles which aim to ensure the safety of research participants and the integrity and validity of data

The guideline was developed with consideration of the current good clinical practices of the European Union, Japan, and the United States, as well as those of Australia, Canada, the Nordic countries and the World Health Organization (WHO).

Status of REG affairs in India

Supporting Clinical Research Permitted simultaneous clinical trials from Phase II onwards, Indian CGP Guidelines (based on ICH GCP) - (ICMR) implemented, Move toward simplification of procedures, Reduction in timelines for regulatory approval, Tax breaks for R & D activities.

Drug development track

In India, PMS data used to be submitted to the Drugs Controller General of India (DCGI) within 2 years of launch. Now Periodic Safety Update Reports (PSURs) are filed at regular intervals as specified in the revised Schedule Y of the Drugs and Cosmetics Act.

HEOR or Pharmacoeconomics

Health economics and outcomes research (HEOR) is a growing field that provides important information for making healthcare coverage and access decision
Pharmacoeconomics study is primarily for the purpose of drug listing, competitiveness, pricing, and reimbursement.

Named patient Programme

A Named Patient Program (NPP) provides patients and physicians access to commercially approved medicines that are not available to them in their own country ( Managed access Programe)

Authority in the relevant country.

Concept on drug trials

Started in 1994, with Gemcitabine trial by Eli Lilly, Pfizer, started its clinical activity in 1995, In 2005-2006, Pfizer is conducting 35 trials, 22 global at nearly 80 sites

The veterans for global trials are Novartis, Roche, GSK, Eli Lilly & Pfizer whereas the leading CROs with focused activity are Quintiles, ICON, Pharmanet, Siro Covance, PPD. The emerging ones are IGATE, Neeman Medical International, OminCare.

CPP

The Certificate of Pharmaceutical Product (CPP), issued in the format as recommended by the World Health Organisation (WHO), is a document that is internationally recognised by national drug regulatory authorities for establishing the status of a pharmaceutical product under a national drug product licensing system.

CPPs are printed on security paper and contain the signature of the authorized FDA official, embossed federal seal and ribbon.

Patient Recruitment & Retention

Know your patients & learn how they expected to be treated , patient right awareness is high even in India In the country , the patients do expect the healthy attitudes , of the medical personnel Informed consent forms should be simple & clearly understood

Orphan Drugs

Approximately 7,000 rare diseases are known, but only about 5% have a FDA treatment approved 85 90% of rare diseases are
serious or life threatening

80% of rare diseases are genetic in origin

Safety Documents

PSURs are pharmacovigilance documents intended to provide an evaluation of the risk benefit balance of a medicinal product at
defined time points after its authorisation.

Critical analysis of the risk benefit balance of the product, taking into account new or emerging safety information

SDEA

A pharmacovigilance agreement (PVA) is a written agreement between the marketing authorisation holder (MAH) and a third party (to which the MAH has shared or activities regarding or impacting pharmacovigilance.

The main objective of pharmacovigilance agreement is to define a mechanism that ensures the flow of safety related information from the end customer to competent authorities via MAH.