Biomedical Data Science

Biologics License Applications (BLA)

Marketing Authorization Application (MAA)

Clinical Development Plan

A document describing the clinical studies that must be
performed during the development of a particular active
substance, device, procedure or treatment strategy, typically
with the intention of submitting the study data as part of an
application to the FDA for marketing authorization. The plan
should indicate appropriate decision points and allow for trial
modifications as knowledge accumulates.

Document, presentation, study meta data, project plan,
project database

Scientific:
describes science, targets, molecule, literature
Medical:
disease numbers, indications and their medical codes,
regulatory information, Health Authority information,
probability of success,
Clinical study data:
no. of subjects, treatment information, in & exclusion criteria,
sites, countries, drug supply, dosing
Resources:
financial data, competitor data, payers’ data, prizing

R&D portfolio planning involves the selection and prioritization
of research and development projects to assure an effective
and efficient use of the company's resources.

Portfolio Management system, document, presentation,
project plan, project database

See Development Plan and multiply, market data, additional
financial and resource data

CDP: Document, presentation, study meta data, project plan,
project database

Process of visualizing what future conditions or events are
probable, what their consequences or effects would be like,
and how to respond to, or benefit from, them.

Scenario Planning system, document, presentation, project
plan, project database

See Portfolio Planning and alter data with different senarios:
scientific success, competition, market, other external factors,
Output: timelines, financial & resource data.

A study in Humans defined by a Clinical Study Protocol,
reported in the Clinical Study Report, published on Clinical
Trial Portals.
The objective of clinical study is to establish the effect of an
intervention. Treatment effects are efficiently isolated by
controlling for bias and confounding and by minimizing
variation.

Documents (CDP, CSR, TMF, IC, IB, Publication, etc) and
Data (CRF, Lab, Diary, Statistics, Randomization, etc)

Scientific:
describes science, targets, molecule, literature
Medical:
disease numbers, indications and their medical codes,
regulatory information, Health Authority information,
probability of success,
Clinical study data:
no. of subjects, treatment information, in & exclusion criteria,
sites, countries, drug supply, dosing
Resources:
financial data, competitor data, payers’ data, prizing

Patient-reported outcomes

Patient-reported outcomes (PROs) are reports coming directly from patients

It stands in contrast to an outcome reported by someone else,
• such as a physician-reported outcome,
• a nurse-reported outcome,
• a clinical study or its diary
• and so on.

• how they feel or function in relation to a health condition and its therapy without interpretation by healthcare professionals or anyone else.

• PROs can relate to symptoms, signs, functional status, perceptions, or other aspects such as convenience and tolerability.

• PROs are not only important when more objective measures of disease outcome are not available but also to represent what is most important to patients about a condition and its treatment.

• PROs are often driven by public health institutions, payers, patient

• The US Food and Drug Administration (FDA) has issued formal Guidance to Industry on PROs in label claims[1] and the
European Medicines Agency (EMA) has produced a reflection paper on HRQoL.[2]

• Specific, often rare diseases
• Patient Education
• Health Information
• Patient Safety
• Families
• “promote safe storage and disposal of unwanted or expired medications”

• laboratory information management system (LIMS)
• aka. laboratory information system (LIS)
• aka. laboratory management system (LMS)

• software-based solution with features that support a modern laboratory's operations
• Challenges are:

• different data standards
• online exchange of data with CRO and Pharma systems
• confidentiality
• flexible architecture for rapid changing technologies and therefore validation
• global infrastructure

Sample management
• Instrument and application integration
• Electronic data exchang
• audit management
• fully track and maintain an audit trail
• barcode handling
• assign roles and groups that dictate access
• compliance
• follow regulatory standards that affect the laboratory
• customer relationship management
• handle the demographic information and communications for associated clients
• document management
• process and convert data to certain formats
• instrument calibration and maintenance
• schedule important maintenance and calibration of lab instruments and keep detailed records of such activities
• inventory and equipment management
Biomedical Data Science ● NoB AUG 2020 15
EXTERNAL Lab
Information
Management
External Lab
Systems
CRO Systems
Affiliate and
Partner Systems
Investigator Site
Systems/PCs
Health Authorities
Gateway
Patient Reported
Outcomes
• measure and record inventories of vital supplies and laboratory equipment
• manual and electronic data entry
• provide fast and reliable interfaces for data to be entered
• method management
• provide one location for all laboratory process and procedure (P&P)
• personnel and workload managementquality assurance and control
• gauge and control sample quality, data entry standards, and workflow
• reports
• time tracking
• calculate and maintain processing and handling times on chemical reactions
• traceability
• show audit trail and/or chain of custody of a sample
• workflows
• track a sample, a batch of samples, or a "lot" of batches through its lifecycle

Common Technical Document

External Study Data
• Health Authorities Gateways

Enterprise Content Management Systems

• systematic collection and organization of information that is to be used by a designated audience
• not a single technology nor a methodology nor a process but dynamic combination of strategies, methods, and tools
• to capture, manage, store, preserve, and deliver information supporting key organizational processes through its entire lifecycle

mainly based on HL7 and XML

• Study Data for Submission to CDER and CBER
• Study Design Standard & Study Participation Standard
• Study & Subject Data Standard
• CDISC - Clinical Data Interchange Standards Consortium
• Health Data Exchange standards
• HL7 - Health Level Seven

Stability Data Standard
Substance Registration System - Unique Ingredient Identifier (UNII)
Federal Medication Terminology hosted by NCI Enterprise Vocabulary Services
FDA Terminology hosted by NCI Enterprise Vocabulary Services

• Substance Library
• Sample Management & Libraries
• Biomarker Data
   • “physical” biomarkers
   • digital biomarkers
• Diagnostics Data
• Genetic Data

• Standardized Document Metadata, exchange across the Pharma Development process (eTMF, CTMS)
• Document Management (templates, document lifecycle, eSignature, archiving & retention)
• Dossier Compilation (ie. Publishing, eCTD, printing)
• Documentation and Product/Project Status Tracking
• Component based Document Authoring
• Business Intelligence and Knowledge Management
• Direct interfaces (ie. Investigator Portals, clinicaltrials.gov)
• Workflow Documentation“ (SOPs, Processes, etc) will be handled through the workflow repository
• Business documentation (i.e. strategy papers, controlling, etc)

• ‘Documentation’ derived from Document meaning official paper
• Document means material that provides official information or evidence or that serves as a record.
• The connotational meaning of documentation is processing of information.
• Documentation is that process that process by which the documents are ready to use in a stratified way i.e. the process of
classifying and annotating data, texts, photographs, etc.
• Documentation is a set of documents provided on paper or online or on digital or analogue media, such as, image, video,
audio, etc.
• This field best known as Information Service.

• systematized collection of patient and population electronically-stored health information in a digital format
• records can be shared across different health care settings
• records are shared through network-connected, enterprise-wide information systems or other information networks and
exchanges
• data including demographics, medical history, medication and allergies, immunization status, laboratory test results,
radiology images, vital signs, personal statistics like age and weight, and billing information

• data linked to medical diagnosis, such as images, lab values, genetics, biomarkers, etc
• certain types of diagnostic data can be linked to the efficiency of a treatment (“personalized medicine”)