Module 9 2024

03/09/2024

Efficacy vs Effectiveness

- When pre-licensure efficacy trials are not feasible or not ethical - Regulatory authorities accept to grant the license based on immunogenicity data

But generally require post- licensure effectiveness studies = “real -life efficacy” (less restrictive entry criteria)

• Comparison of a vaccinated population vs a non-vaccinated population

• Vaccinated communities vs unvaccinated communities (e.g. community randomized trial)

• Cases in vaccinated subjects vs cases in matched controls (case control study)

• Historical control : observe decrease in the incidence of the disease after introduction of a given vaccine in the Universal Mass Vaccination (UMV) programme

The Organisation for Professionals in Regulatory Affairs

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Vaccine Regulatory Framework - USA, EU and WHO (1)

USA (FDA)

Guidelines (https://www.fda.gov/vaccines-blood-biologics/biologics-guidances/vaccine-and-related biological-product-guidances) “General Principles for the Development of Vaccines to Protect Against Global Infectious Diseases” “Content and Format of Chemistry, Manufacturing and Controls Information and Establishment Description Information for a Vaccine or Related Product” Guidances on post-market safety reporting for vaccines Guidances for the evaluation of combination vaccines, viral vaccines, plasmid DNA vaccines Plus, specific guidances on Influenza , Therapeutic Cancer , and COVID-19 vaccines Under "scientific guidelines"/"Multidisciplinary: vaccines". Link: https://www.ema.europa.eu/en/human-regulatory overview/research-development/scientific-guidelines/multidisciplinary-guidelines/multidisciplinary-vaccines Adjuvants in vaccines for human use Clinical evaluation of new vaccines (new version effective from 1 August 23) + draft addendum on immunocompromised individuals A series of guidelines on influenza vaccines Pharmaceutical and biological aspects of combined vaccines Product information for vaccines Preclinical pharmacological and toxicological testing of vaccines (note: now deleted and there is reference to the WHO guideline on nonclinical evaluation of vaccines) Quality, non-clinical and clinical aspects of live recombinant viral vectored vaccines Also on Pharmacovigilance of Vaccines:

Vx under Center for Biologic Evaluation and Research ( CBER ) with assessors and experts in the organisation

W HAT TO LOOK FOR…

EU (EMA)

No CBER equivalent. EMA is a network of experts from across Europe. Vx managed by EMA’s Vaccines and Therapies for Infectious Disease

W HAT TO LOOK FOR…

http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/12/WC500157839.pdf

The Organisation for Professionals in Regulatory Affairs

(1)

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