Drug Development Overview Quiz

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.