There is a very long and expensive development and approval process before a drug makes it to market. Most cancer drugs follow a similar overarching drug development path. This traditional pathway can take up to 15 years, but efforts have been made by industry to reduce this timeline.
All drugs start in the lab – the lab may be private, or in a university, hospital or specialist cancer center – but generally this early research is no longer done by pharmaceutical companies. This starting point is called basic or pre-clinical research. There are usually 3 stages before a drug can be approved:
- Pre-clinical, or basic research
- Approvals and funding process
- Clinical trials
1. Pre-clinical, or basic research
Researchers or doctors have an idea of something they would like to test, a question they would like answered. This could come from laboratory work, other trial results or experience with patients. After initial testing, any promising compounds undergo further study in the laboratory on animals such as mice, or on biopsied tissue. This pre-clinical stage can take several years. For example, the University of Edinburgh project that we are funding will take up to 3 years to complete its lab work. If results from basic research studies are positive, the new drug or approach will then begin to be tested on people in a clinical trial.
2. Approvals and funding process
All trials and studies go through an approval and funding process. This includes:
- Peer review. The detailed plan which outlines how the trial will run is called the protocol, and this will be reviewed by an external group which includes scientists, doctors and patients. This reviewal process by these external individuals is known as peer review. They will look at the trial design, whether the team has thought about all the possible issues and who they will recruit. Patient Information Sheets (PIS) are also developed at this point for recruitment.
- Ethical approval. The trial protocol and PIS are then reviewed by a Research Ethics Committee – they decide if the trial is safe and ethical, and whether it can go ahead or not.
- Registering the trial. There are a number of different international registries, including the WHO registry and clinicaltrials.gov
3. Clinical trials
Clinical trials aim to provide reliable evidence about how effective treatments are and whether they should be made available to patients. For patients who have often run out of other options, they offer access to experimental treatments.
Traditional Randomised Control Trials, considered by many to be ‘gold standard’, compare a new drug with standard care or a placebo. In most cancer drug trials, some patients will receive a new or repurposed drug, while others will have a standard treatment, such as chemotherapy for comparison purposes as cancer patients can’t afford to have no treatment at all. In other types of trials, especially for advanced or less common cancers which have few or no approved treatments, only the experimental drug is given.
Patients on trials are closely monitored, any side effects are recorded and patients will be treated to alleviate them.
Clinical trial results are used to help governing bodies decide whether the treatment should be available for use on the NHS. The more trials that can recruit patients quickly and be completed, the faster new drugs will become available.