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Drug development process

The full drug development process is a lengthy and complicated one taking ten to twelve years. The cost of developing a new drug is over €800 million, about 60% of which is spent on necessary rigorous clinical trials. New drugs are selected from a range of many thousands of substances with the potential to treat the targeted condition. For a variety of reasons, fewer than one or two compounds per ten thousand tested actually make it to the market and are authorised for use in patients.

The table below sets out the different phases in a linear fashion over time. In reality this is becoming a complex and iterative process rather than a simple linear path, and while all the steps outlined below must take place, the sequence and cycles may vary.

Description Timescale

Where it all begins

A new drug starts as an idea or hypothesis based on exciting or unexplained observations. Sources for new ideas include scientific data and literature, scientific research undertaken at universities and university hospitals, patent information, patient focus groups, international symposia and explorative research in company laboratories.

Time in years

Research Phase

Lead Discovery and Lead Optimisation

The hypothesis is tested using biochemical methods and possibly animal testing in order to identify a lead compound (from a number of possible candidates). The lead compound is further optimised and characterised to determine how to produce it. During this time, animal models may be developed to reflect the disease in man as closely as possible to test the compound.

Manufacturing

The manufacturing process for the new drug is initiated and developed to produce it in sufficient quantities for pre-clinical testing and clinical trial purposes. The new drug must be ready for full manufacture before the start of Phase III trials. This phase continues throughout development.

Pre-clinical development

Pre-clinical development begins before clinical trials or testing in humans may begin, during which important safety and pharmacological data are collected. The main goals of pre-clinical studies are to determine the new drug's pharmacodynamics (PD), pharmacokinetics (PK), ADME, and toxicity using animals or human blood and tissues. Further pre-clinical development may continue as the new drug progresses through clinical trials.

Application for Investigational New Drug

An application for an IND is made to the FDA, EMEA and/or other regulatory agencies for permission to administer a new drug to humans in clinical trials.

Clinical development

Phase I clinical development

Phase I trials are conducted primarily to determine how the new drug works in humans and its safety profile, and to predict its dosage range. It typically involves between fifty and one hundred healthy volunteers.

Phase II clinical development

Phase II trials test for efficacy as well as safety and side effects in a group of between one hundred to three hundred patients with the condition for which the new drug is being developed.

Phase III clinical development

Phase III trials involves a much larger group of patients, between several hundred and several thousand, which will help determine if the new drug can be considered both safe and effective. It will usually involve a control group using standard treatment or a placebo as a comparison.

Product registration and regulatory approval

New Drug Application or New Biologics Application

When a product is considered safe and effective from Phase III trials, it must be authorised in each individual country before it can be marketed. All data generated about the molecule is collected and submitted to the regulatory authorities in the US (FDA), European Union (EMEA) and Japan (PMDA) and other countries which may require their own national approvals.

Marketing and Launch

A pre-marketing strategy may have been instigated as early as Phase I trials to ensure that the market’s needs are incorporated into the new drug’s overall development, but more usually during the later phases when clinical results are promoted at international symposia in order to develop an awareness amongst the medical community who will ultimately be prescribing the new product.  A salesforce will be trained and will begin an intense marketing campaign prior to launch.

Phase IV trials

Phase IV trials are conducted after a new drug has been granted a licence, approved and launched.  In these studies, the new drug is prescribed in an everyday healthcare environment using a much larger group of participants (two to five thousand patients). This enables new treatment uses for the new drug to be developed, comparisons with other treatments for the same condition to be made, and determination of the clinical effectiveness of the new drug in a wider variety of patient types, and more rare side effects, if any, may be detected.