Drug Development Overview Quiz

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Questions and Answers

What is the first stage in drug development?

Drug discovery

Post-clinical stage

Clinical stage

Preclinical stage (correct)

What expertise is typically involved in drug development?

Safety assessors (correct)

Financial management

Marketing

Human resources

What is the focus of the clinical stage in drug development?

Synthesizing new molecules

Pharmacovigilance

Clinical trials (correct)

Target site identification

Which process involves understanding diseases and identifying target sites?

Preclinical stage Signup and view all the answers

What area of expertise is responsible for ensuring drug safety in clinical trials?

Regulatory affairs Signup and view all the answers

What is the purpose of regulatory affairs in the context of gene editing on humans?

To protect the public by ensuring medicinal products are safe, efficacious, and of suitable quality Signup and view all the answers

What is the role of the International Council on Harmonisation (ICH) in product approval regulations?

To standardize regulatory requirements for product approval across different regions Signup and view all the answers

What is a common concern addressed by the 'Paediatric Investigation Plan' (PIP) requirement?

Effects of drugs on normal development processes in children Signup and view all the answers

What does the term 'Snake oil' or 'Patent remedies' refer to in the context of medicinal products?

Ineffective medicinal products Signup and view all the answers

In what way do UK, EU, and FDA differ in their approach to clinical studies on pediatric medicines compared to historical practices?

They now mandate clinical studies in children to address historical controversies and concerns Signup and view all the answers

Why was it historically controversial to conduct clinical studies of new drugs in children?

Concerns over the effects of drugs on normal development processes Signup and view all the answers

What is the purpose of Phase II clinical trials?

To establish preliminary efficacy of the IMP in patients Signup and view all the answers

How many subject groups with different doses are usually included in a Phase II study design?

3 Signup and view all the answers

Which regulatory status is needed for Phase III clinical trials in Europe or the UK?

Needs new or updated CTA Signup and view all the answers

What is the primary focus of Phase I clinical trials?

Evaluating safety and tolerability of the IMP Signup and view all the answers

Where are Phase III clinical trials usually conducted?

In hospital or clinical centers under full medical supervision Signup and view all the answers

How many subjects typically participate in Phase I clinical trials?

50 - 200 Signup and view all the answers

What is needed for Phase II clinical trials in the USA?

Needs Investigational New Drug Application (IND) Signup and view all the answers

What is the primary purpose of Phase III clinical trials?

Establishing final selection of dose(s) for marketing Signup and view all the answers

'Double-blind' refers to:

'Blinding' both the researchers and participants from knowing who is receiving treatment or placebo Signup and view all the answers

Study Notes

Drug Development Process

The process involves preclinical, clinical, and post-clinical stages.
Experts involved include basic research, safety assessors, research and development, clinical trials, regulatory affairs, and marketing.

Preclinical Stage

Understanding disease, identifying target site, synthesizing and ADME pharmacological testing of molecules.
Development of pharmaceutical products involves gene editing on humans and safety assessments.

Clinical Stage

Safety studies, clinical trials (Stages I, II, III, and post-market studies).
Clinical trials involve Phase I (non-therapeutic safety/tolerability), Phase II (therapeutic preliminary efficacy), and Phase III (crucial therapeutic).

Phase I Clinical Trials

Purpose: Assess safety and tolerability of the IMP in humans.
Timing: After 1 month Safety Assessment studies.
Regulatory status: Needs Investigational New Drug Application (IND) in the USA and Clinical Trials Application (CTA) in Europe or UK.
Subjects: Healthy volunteers and patients.
Number: 50-200.
Location: Conducted under full medical supervision in a hospital or clinical center.

Phase II Clinical Trials

Purpose: Establish preliminary efficacy of the IMP in patients and effective range of doses.
Timing: After Phase I studies and 6 month (rodent) and 9 month (non-rodent) Safety Assessment studies.
Regulatory status: Needs new or updated IND in the USA and CTA in Europe or UK.
Subjects: Patients with a defined disease and specific stage of disease progression.
Number: 100s.
Location: Conducted under full medical supervision in hospital or clinical centers.

Phase III Clinical Trials

Purpose: Confirm efficacy of the IMP in patients, final selection of dose(s) for marketing, and relative efficacy to the market leader.
Timing: After Phase II studies and DART and carcinogenicity Safety Assessment studies.
Regulatory status: Needs new or updated IND in the USA and CTA in Europe or UK.
Subjects: Patients with a defined disease and specific stage of disease progression.
Number: 1000s.
Location: Conducted under full medical supervision in hospital or clinical centers.

Phase IV Clinical Trials

Purpose: Post-market surveillance to gain more experience in more patients.
Timing: After approval and marketing.
Study design: Various studies, including bioequivalence and safety assessments.

Regulatory Affairs

Purpose: To safeguard public health and ensure medicinal products are safe, efficacious, and of suitable quality.
International Council on Harmonisation (ICH) harmonizes regulatory requirements for product approval.
ICH members: FDA, EU, Japan, and industry representatives.
"Common Technical Document" for final approval.

Paediatric Medicines

Historically, clinical studies of new drugs in children have been controversial and not usually performed.
UK, EU, and FDA now require clinical studies in children with a "Paediatric Investigation Plan" (PIP) and "waiver" if no therapeutic application in children.
6 months extra patent protection granted for paediatric studies.

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Description

Test your knowledge on the process of drug development, including quality assurance, legal framework, expertise involved, and different stages like preclinical, clinical, and post-clinical. Learn about disease identification, target site synthesis, pharmacological testing, and more.