The Development of a New Medicine

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


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


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


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


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


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


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


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


Interaction with

health authorities

Review of registration

documentation by regulatory authorities



trials in patients

(early development)

Interaction with

health authorities



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.

Health & Medicine

The Development of a New Medicine