L5 Marketing Authorisation Flashcards
What 3 things were introduced after the falsified medicines directive 2011/62/EU?
Anti-tampering device
Safety features
Unique identfier
What do MAHs outline?
Specific legal responsibilities across whole life of the product
What are 4 things included in the MAH?
Requirement to launch product
Market product (legally)
Legally responsible for maintaining quality & safety throughout life cycle
Legally responsible for monitoring real-world performance & report findings
What is off label use?
Use of product outside of approved indications
How often must EU MAs be renewed?
After 5 years on a basis of re-evaluation of benefit-risk balance
What happens when MA is renewed?
Valid for an unlimited period unless CA proceeds with one additional 5 year renewal
What are MAHs post-marketing responsibilities?
Pharmacovigilance
What happenes if new data/info on medicines efficacy, safety or quality becomes available?
MAH has to alert CA & apply for necessary variations
What is Prescription only?
POM must be prescribed by a doctor & is not licensed for ‘free’ sale to general public
What is included in the list of criteria for OTC?
Indication suitable for self-diagnosis & self treatment
Clear instructions for use
Well defined maximum dose
Appropriate safety profile
What do accelerated approval programmes allow for?
Earlier approval of promising, novel/innovative medicines that will treat serious, life-threatening conditions & fill an unmet need
What is a surrogate endpoint?
A marker that strongly predicts clinical benefit but is not itself a measure of clinical benefit
What is the EU and US accelerated approval programmes?
EU = PRIME
US = FDA Accelerated Review
What are characteristics of Conditional MA?
Legal contract
Benefits have to be clear, apparent & immediate
Renewed each year
Valid for one year
What are 3 key post authorisation documents?
SmPC
PIL
PAR
What is the smPC?
Information resource for healthcare professionals
Based on clinical & preclinical development data
Written in standard format
Prepared b y MA
What is PIL?
Leaflet that is included in pack with a medicine
Patient friendly derived from SPC
Written by MA
What is the PAR?
Published by EMA/NCA for every medicine granted a MA following assessment procedure
Full, detailed scientific assessment report of medicines summarising data & regulators considerations, interpretations & scientific conclusions
Persistent
Where is ‘scientific advice’ available?
National, EU & FDA levels
What are Orphan Medicinal Products?
Products intended to treat diseases so rare that sponsors would be reluctant to develop them under ‘usual’ MA procedures
How is ‘Rare Disease’ defined in EMA & FDA?
FDA = condition that affects fewer than 200,000 people in US
EMA = condition that affects fewer than 5 in 10,000 in EEA
Whats an example of a rare disease?
CF, motor neuron disease, Tourette’s Syndrome
Is Orphan status a MA?
No
What are 5 incentives for Orphan Status?
Free scientific advice & protocol assistance
Significantly reduced fees
Centralised procedure for MA
R&D grants
10yr market exclusivity
