Development of a New MedicineProvided by the IPHA Communications Department
May 2010
Supporting
Irish patientsand the
Irish economy
Research Activities
These include:
1. Combinatorial libraries – vast collections of novel compounds
2. Mass screening of compounds in assay systems
3. Molecular biology and modern genetics – role of genes in disease states
4. Recombinant DNA technology
5. Computer aided molecular modelling
Milestones in the Development
0 5 8 12 15
Years
Research and
Discovery
Compound discovered and patent application
made
Publication in
Scientific Journal
Beginning of Human
Trials
Registration
Submission of
registration dossier to regulatory authorities
Trials in Patients
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
Clinical Studies
0 5 8 12 15
Years
Research and
Discovery
Preparation of initial clinical plan. Selection
of clinical study locations
PHASE I
Human trials with
healthy volunteers to test
tolerability
PHASE II
Trials to determine
dose ranging,
safety and efficacy
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
PHASE III
Large scale trials to determine definitive safety and efficacy in
patients
Toxicology
0 5 8 12 15
Years
Research and
Discovery
Determine effects of
medicine in animals when administered over 2 to 13
weeks (depending upon length of planned use in
humans)Test to determine mutagenic potential
Longer term animal studies. Does the active substance
have long-term side effects?
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
Determine the effect
on impregnati
on and implantatio
n in animals
and whether
the active substance can affect the fetus
Determine the reproductive effect upon
future generations in
animals
Pharmacokinetics / Metabolism
0 5 8 12 15
Years
Research and
Discovery
Determination of how the medicine is
absorbed, distributed, metabolised and
excreted in animals
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
Determination of how and
to what extent the medicine is absorbed by
humans
Determination of how the medicine is distributed, metabolised, and excreted by humans
Determination of the effects of the medicine on specific populations such as the elderly, different
races and sexes.
Final construction of
the pharmacokinetic profile of the
medicine
Dosage Form
0 5 8 12 15
Years
Research and
Discovery
First dosage form for volunteer trials
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
Pre-formulation activities –
consultations with
pharmacists / determination
of physical and chemical properties of compound, e.g. particle
size
Development of
clinical trial formulation
Develop Market formulation based on known
characteristics of medicine and patient group involved,
e.g. age and condition. For example tablets, capsules, injectables or transdermal
patches
Process development for large scale
production of the dosage form. Testing
of stability of the dosage and
determination of shelf-life
Process validation and production of
the dosage form for launch
Active IngredientThe therapeutically active component in a medicine's final formulation that is responsible for its physiological or pharmacological action.
0 5 8 12 15
Years
Research and
Discovery
Batch 0 synthesis (1st non-
GMP batch)
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
Batch 1 synthesis (1st GMP
standard)
Batch 2 of GMP
Batch 4 using final method of synthesis
Batch 3 of GMP
Final production
concept
Batch 5 compound
produced in full scale
production equipment and
validation
Routine production
Marketing
0 5 8 12 15
Years
Research and
Discovery
Marketing input to
the design of clinical trials
especially in the
choice of comparat
or medicines
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
Early stage commercial assessment
of the medicine
taking into account
medical need and existing therapies on the market
Full development commercial assessment
Profiling – development of comparative studies on, for
example, specific patient groups and other similar medicines. Study
possible applications of the medicine to other diseases
Selection of a trade name
begins
Trade Name ™ chosen for the medicine
Medicine information
formulated into brochures and
videos. Displays at congresses and symposia
Pharmacoeconomics
0 5 8 12 15
Years
Research and
Discovery
Definition of the
healthcare costs
caused by the disease
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
Assessment of the
potential impact of the new
medicine on healthcare
costs Research for the
appropriate pharmacoecon-
omic and quality of life parameters
(linked to clinical trials Phase II)
Economic evaluation parallel to clinical trials to
determine the economic value of a new medicine, e.g. cost saving and cost-
effectiveness
Compilation and publication of the
pharmacoeconomic results
Regulatory Affairs
0 5 8 12 15
Years
Research and
Discovery
Application for trial
authorisation to begin
trials on healthy
volunteers
Early Developme
nt
Full Developme
nt
Pre-Market Activity
* GMP = Good Manufacturing PracticeThe above shows a general representation of the development of a new medicine. Various processes may differ from country to country and between different compounds.
Interaction with
health authorities
Review of registration
documentation by regulatory authorities
Application for trial
authorisation to begin
trials in patients
(early development)
Interaction with
health authorities
Application for trial
authorisation to begin
trials in patients
(full development)
Interaction with
health authorities
Formulation of medicine labelling and doctor/patient
medicine information
Compilation of
registration dossier
Interaction with
health authorities
Quick facts about Medicines Development
It takes an average of 10 to 12 years for a medicine to travel from the laboratory to the pharmacy shelf;
On average, only 1 out of 5,000 to 10,000 promising substances will survive extensive testing in the R&D phase to become approved as a quality, safe and efficient marketable product;
Several studies put the cost of researching and developing a new chemical entity €1,059 million;
Moreover around 70% of medicines that eventually reach the market do not provide sufficient return to recoup their R&D expenditure. As a consequence, the return on investment is highly dependent on a limited number of successful products.