Case 5 The pharmaceutical market 15-10-2022
How does a drug come into the market?
Drug development process
Stages of drug development
1. Pre-clinical studies: test tubes,
laboratory, animal testing
2. Clinical studies: Phase I + II + III
clinical trials
a. Phase 1: small group of
health volunteers. To
evaluate safety and
determine a safe dosage.
b. Phase 2: larger group of
volunteers and patients,
up to 200.
c. Phase 3: confirm findings
by research on a large
patient population with
randomized control (new
, drug vs usual care = gold standard). In this phase, also the efficacy, safety and
side-effects are observed.
3. Marketing authorization (EMA or DFA) and reimbursement
4. Pharmacovigilance (geneesmiddelenbewaking) (=product surveillance) testing long
term safety in real-life patients and monitor side-effects when the drug is on the
market
Market authorization
Licensing and regulatory approval are important parts within the pharmaceutical market.
Important parties who play a big role in licensing and regulatory approvals are:
- EMA: European Medicine Agency They operate on EU-level and observe three
criteria: efficacy, safety and quality and analyse the benefit-risk ration.
- FDA: The U.S. Food and Drug Administration.
- CHMP: EMA’s Committee for Medicinal Products for Human Use: assess applications
submitted by medicine developers and recommends whether or not a medicine
should be granted marketing authorization
- After market authorization, reimbursement criteria and pricing decisions are made.
Type of drugs
- Ethical drugs = prescription drugs = prescription needed
- OTC drugs = without prescription
- Brand drugs = produced and distributed with patent protection
- Generic drugs = without patent protection, opportunity to increase efficiency in
pharmaceutical market
- Me-too drugs = branded drug which is structurally similar to already known drugs, is
a way to get a new patent, innovative problem?
- Orphan drug = for rare diseases, regulatory support and market exclusivity
o Criteria:
Life threatening (chronically debilitating disease)
How does a drug come into the market?
Drug development process
Stages of drug development
1. Pre-clinical studies: test tubes,
laboratory, animal testing
2. Clinical studies: Phase I + II + III
clinical trials
a. Phase 1: small group of
health volunteers. To
evaluate safety and
determine a safe dosage.
b. Phase 2: larger group of
volunteers and patients,
up to 200.
c. Phase 3: confirm findings
by research on a large
patient population with
randomized control (new
, drug vs usual care = gold standard). In this phase, also the efficacy, safety and
side-effects are observed.
3. Marketing authorization (EMA or DFA) and reimbursement
4. Pharmacovigilance (geneesmiddelenbewaking) (=product surveillance) testing long
term safety in real-life patients and monitor side-effects when the drug is on the
market
Market authorization
Licensing and regulatory approval are important parts within the pharmaceutical market.
Important parties who play a big role in licensing and regulatory approvals are:
- EMA: European Medicine Agency They operate on EU-level and observe three
criteria: efficacy, safety and quality and analyse the benefit-risk ration.
- FDA: The U.S. Food and Drug Administration.
- CHMP: EMA’s Committee for Medicinal Products for Human Use: assess applications
submitted by medicine developers and recommends whether or not a medicine
should be granted marketing authorization
- After market authorization, reimbursement criteria and pricing decisions are made.
Type of drugs
- Ethical drugs = prescription drugs = prescription needed
- OTC drugs = without prescription
- Brand drugs = produced and distributed with patent protection
- Generic drugs = without patent protection, opportunity to increase efficiency in
pharmaceutical market
- Me-too drugs = branded drug which is structurally similar to already known drugs, is
a way to get a new patent, innovative problem?
- Orphan drug = for rare diseases, regulatory support and market exclusivity
o Criteria:
Life threatening (chronically debilitating disease)
