Fig. 9.10: Types of biostatistics

INDA and NDA 137

Biostatistics is the most appropriate methods for the collection, presentation, analysis,

and interpretation of data. Based on the analysis of statistical methods, decisions are

made.

Application of Biostatistics

After the Kefauver Harris Drug Amendments, it is compulsory for the firms to prove

the safety of the drug with suitable statistical evidence. Statistics is involved in all

stages of drug development, i.e. from drug discovery to post-marketing processes

(Fig. 9.11).

Application of biostatistics at the clinical stage: Stage 3

Clinical testing includes step by step process. At the initial stage, the pharmacokinetics

and pharmacodynamics of the drug is studied. At the next stage, the efficiency of the

drug is studied. The efficiency studies are done with the help of statistics. Next stage

is the confirmatory stage, at which most of the studies are based on statistical analysis.

The trial design is also prepared by using statistics. The risk–benefit analysis and

cost-effectiveness of drug are also calculated with the help of statistics.

Application of biostatistics at non-clinical stage: Stages 1, 2, 4, 5

At stage 1 and stage 2 biostatistics has application in the following processes:

i. Screening of drug product

ii. Chemical development

iii. Drug delivery process designing

iv. Assay development

v. Formulation development

vi. Prediction of rare disease

vii. Identification of biomarkers

viii. Quantification of gene expression

ix. In physiology and anatomy

x. Data presentation for FDA submissions

xi. Management of clinical studies

Fig. 9.11: Different stages at which statistics is involved

138 Industrial Pharmacy II

At stage 4 and stage 5 biostatistics has application in the following processes:

i. Rate of market expansion

ii. Calculate brand impact

iii. Create value of the drug

iv. Calculate optimized resources

v. Maximize the value of the launch

vi. Improve work efficiency

vii. Improve customer service

viii. Determine key factors influencing the target population

ix. Determine competitors efficiency

x. Designing successful strategies to overcome challenges

9.6 DATA PRESENTATION FOR FDA SUBMISSIONS

The FDA accepts electronic submissions. The electronic submission's regulatory process

is divided into four steps:

1. Early planning and management of the project

2. Document preparation

3. Dossier publishing

4. Dossier submission

During the planning stage applicants are recommended to use the following tools:

i. Checklist: This includes the name of each document that is needed to be submitted

with all information.

ii. Glossary: Applicants should create a list of a general glossary.

iii. Template: It is a standard file format document which contains pre-defined layout,

text, style and graphics.

iv. Timeline table: Timeline management is one of the most important tasks in the

submission planning phase. Applicant must update a timeline table or a Gantt

chart that also includes the role and responsibility of each person involved in the

process.

File formats used for electronic documents are:

i. Version: One should be able to read all the PDF files with the Acrobat Reader

version 3.0 with the search plug in.

ii. Fonts: Used only True Type or Adobe Type 1 font of black color for text and blue

color for hypertext link. Avoid using light colors for text.