L17 Regulating Pharmaceuticals

Question 1

Regulatory Science

Answer 1

the science of developing new tools, standards and approaches to assess the safety, efficacy, quality and performance of all FDA-regulated products

Question 2

Regulatory affairs

Answer 2

an area of work that involves responsibility for checking whether a business is following official rules or laws

Question 3

What we do - regulatory affairs

Answer 3

1. work in regulated enviro.
2. health industry is regulated by federal and state laws, regulations, codes, statements, guidelines, standards and quality management systems
3. ensure a corporate entity complies with all the above pertaining to their business
4. advise corporate entity on regulatory climate
5. work with health regulator in jurisdiction where they want to do business, intend to market products

Question 4

Two pathways

Answer 4

see onenote

1. ethics and research governance
2. regulatory agencies/competent authorities

Question 5

Change in regulation

Answer 5

• regulations can change based on real events or through proactive planning by regulators
• change (usually) only happens after consultation with Industry EXCEPT in the case of safety/warnings
• usually a transition period for implementation because change often effects R&D/commercial spend

Question 6

Jurisdictional Requirements

Answer 6

see onenote

Question 7

Marketing Authorisation

Answer 7

see onenote

Drug approvals up due to breakthrough designations/orphans

Sponsor - pharmaceutical company, biotech company, corporate company that funds the work
- The sponsor is responsible for everything unless they sell it off

Question 8

Therapeutic Goods Administration (TGA)

Answer 8

see onenote

• therapeutic goods for marketing must be on Australian Register of Therapeutic Goods (ARTG)
• application process 225 calendar days
• AUST R = registered, higher risk
• AUST L = listed, lower risk
• L or R depends on poisons scheduling of ingredients, therapeutic claims, type of product and degree of medical oversight
• investigational products are exempt

Question 9

Therapeutic goods (TG) - what are they?
(corresponds to two slide)

Answer 9

• pharmaceuticals
• biological as medicines
• devices
• combination products e.g. asthma inhaler

Defined in the Therapeutic Goods Act 
Interpretation: 
- therapeutic use
- ingredient in manufacture of a therapeutic good
- container/closure system

Question 10

Biologicals

Answer 10

TG made from, or that contains human cells or human tissues, or live cells, tissues or organs

Question 11

Therapeutic use - TGA

Answer 11

see onenote

Defined broadly as products for use in humans in connection with…

Question 12

What is a medical device?

Answer 12

see onenote

E.g. contact lenses, doesn’t cause biological responses, it is an “implant”

Question 13

TGA Pyramid

Answer 13

see onenote diagram

Question 14

Ingredients - Poisons Scheduling

Answer 14

see onenote slides

Poisons scheduling = What category any compound sits in terms of risk

• The higher the number, the higher the risk
• product specific and risk

Question 15

Global Regulatory Considerations - 3 poitns

Answer 15

1. quality - manufacturing
2. safety - toxicology
3. efficacy - does it work?

Question 16

Drug Development Process

Answer 16

see onenote

1. discovery and development
2. preclinical research
3. clinical research
4. FDA review
5. FDA, post-market, safety monitoring

Question 17

Drug Development Considerations

Answer 17

see onenote

1. is it safe?
2. is it better?
3. how does it compare to standard of care?
4. does it work?
5. breakthrough?
6. can you sell it?
7. should it be on the Pharmaceutical Benefit Scheme (PBS) or Medical Services Advisory Committee (MSAC)?

Question 18

GXPs - Guidelines setting out Principles

Answer 18

see onenote slides

GMP - good manufacturing practice, Quality

GLP - good laboratory practice, governs lab practice and procedures, Safety

GCP - good clinical practice, clinical trial conduct, efficacy

Question 19

CMC

Answer 19

see onenote

Chemistry, Manufacturing, Controls

There are regulations for each excipients as well - not just regulations for active ingredients

Question 20

Preclinical research

Answer 20

• still required although minimising animal use

standard toxicology batteries include

1. repeat dose (rodent/non-rodent e.g. non-human primate)
2. safety pharmacology
3. in vitro/in vivo mutagenesis
4. chronic carcinogenicity
5. others, product specific

animal data not overridden by human data HOWEVER, may be omitted if justified

Question 21

Physiologically-based Pharmacokinetic (PBPK) modelling

Answer 21

see onenote diagram

Question 22

Does the regulation stop? No

Answer 22

1. post-marketing commitments
2. ongoing quality control for every batch
3. sponsor provides Periodic Safety updates
4. therapeutic claims (advertising code)
5. therapeutic use - new product
6. regulatory surveillance
7. pharmacovigilance

Question 23

Action by the TGA

Answer 23

decrease in the amount of codeine supplied to Australians

Question 24

Regulatory Surveillance - Story

Answer 24

see onenote

Question 25

TGA vs FDA

Answer 25

see onenote table

Question 26

Summary

Answer 26

1. regulatory requirements are harmonised 2. different jurisdiction have different laws 3. three regulatory principles of: Quality, Safety and Efficacy 4. GLP, GMP, GCP 5. animal and human drug data important 6. risk-based approach to regulation 7. regulation driven by public events and risk 8. surveillance leads to change