Many people are unaware of just how complex and long-winded the route of bringing a drug to the market really is. From first laboratory discovery to the clinical research testing phases and then eventually being granted a license to become available for purchase, the whole process typically takes up to 12 years and costs over $1.4 billion dollars.
That being said, very few experimental drugs being tested will ever make it onto the pharmacy shelves. In this article is a brief overview of each of the main stages of the drug development process, from lab through to market approval.
The drug development process begins in a laboratory, where researchers who have gained funding from research bodies or pharmaceutical industry players are undergoing research on the biology of a disease. They must first understand how the cell and biological processes work. This enables them to identify molecules or compounds which could have the ability to act against an identified “target”, such as a gene or protein involved in a disease.
Thousands upon thousands of new chemical compounds are discovered and tested in order to determine whether they have the potential for something more. However, very few of these will ever make it to the human testing stage (5 in 5,000).
Any potential new drug will initially be comprehensively tested for safety and efficacy. This includes both in the lab, using computational models and cells (in vitro), and on animals (in vivo).
These studies are somewhat small but they must give a very detailed analysis of dosing and toxicity levels to fully establish whether the drug can safely be tested in humans. This takes several years and the majority of candidates will be rejected.
Before medications, healthcare devices, or therapies are fully approved for the market, they must all pass strict testing which is assessed during clinical trials. The aim of these trials is to prove that they are safe for human consumption and effective at treating the disease.
There are three phases to every clinical drug trial. In fact, every single new medicine must pass through each phase before being allowed on the market.
This is the first stage of testing, which means that the experimental drug or treatment has not yet been tested on human subjects. There are typically only a small number (i.e. 20 – 100) of healthy volunteers aged between 18 and 45 taking part in Phase I trials. All subjects are provided with compensation for their time.
During this stage, the aim is to establish if the drug is safe for human consumption while determining the optimal dosage and discovering any side effects. Only once the results are positive in healthy people will the drug be tested in people with the disease (Phase II). Many proposed drug treatments will not make it to the next phase.
This stage is used to assess how well the drug works in treating a specific condition or disease and will involve a larger number of patients with that particular condition. This phase is also where the best method of taking the drug is determined (i.e. tablet, injection, spray and so forth). Here, the drug is compared with a standard treatment or placebo treatment and the effects assessed.
The aim of this stage, which can take place over several years and across many countries, is to evaluate how effective the drug is for treating people with the disease, monitor its side effects, and collect essential safety information. Phase III testing will involve a much larger group of participants, often in the thousands.
Only once a drug has successfully passed the Phase III clinical trial stage and has proven itself to be effective and safe will it be passed onto the regulatory bodies across the different countries for review. The regulatory authority will then assess its safety, efficacy, and quality to determine whether the drug can be made available to the public.
If the product is successful, then a marketing authorization or license will be granted. In the US, this body is the Food and Drug Administration (FDA), and in the UK it is the Medicines and Healthcare Products Regulatory Agency (MHRA).
On many occasions, an on-going phase of testing is conducted even after the drug has been approved. This is done in order to discover more about it in terms of long-term benefits, side effects, and risks so that safety continues to be a top priority.
As you can see, every drug available to purchase must pass through a long and stringent process of testing and regulatory requirements in order to prove that it is a safe and effective treatment option.
Did you know how long and multifaceted the medicine development process is before this? Sound off in the comments below.