Where do medicines come from? How does a medicine go from idea in a laboratory to the shelf in a pharmacy?
The average medicine on the market takes twelve years to develop, at a cost of over a billion dollars.
The journey begins with researchers investigating diseases on a cellular or molecular level. These researchers are looking for the biological processes behind the disease. They’ll look for weak points – a gene or protein that can be affected by an outside agent. Disrupting that biological process will attack the disease.
Once a weak point is identified, the researchers will look for a molecule or compound that can affect it. Besides testing its efficacy, they also need to test its safety. A molecule that can eradicate a disease, but will also cause catastrophic liver failure, isn’t fit for the public!
If a candidate proves effective and safe, the researchers can apply to the regulatory body of their country for permission to run a clinical trial on humans. There are generally three phases of human clinical trials, after which a company can apply for permission to bring a drug to market.
Phase 1 clinical trials test safety and pharmacology. A small number of healthy volunteers are given the prospective drug and closely monitored. If the drug proves toxic, or has side effects deemed too costly to people’s health, the phase 1 clinical trials are ended, and the drug is a failure.
Phase 2 tests how effective the drug is. This time, the volunteers are people with the condition the drug is meant to treat. The number of volunteers is small, though larger than phase 1. The trials will determine the optimal pathway – oral, intravenous, or other – and the most effective dose. If the drug proves ineffective, it will be classified a failure.
Phase 3 is the big test. Thousands of volunteers are recruited from all over the globe, and rigorous testing of safety, efficacy, and dosage size is performed. The goal is to confirm the findings of phase 2. With a positive result in phase 3 testing, the drug is ready to be submitted to the regulatory authorities. Even here, more than 10% of drugs who have reached this stage ultimately fail their phase 3 trials.
Once phase 3 is completed, a company can apply to a regulatory authority such as the FDA for approval to market the drug. This process is not a simple one, and occasionally the FDA can deny a drug even after it passes all three stages of clinical trials.
If the regulatory body approves, the drug is now ready for people to use.
Of course, so many different things go into each of these steps! Each stage takes years to carry out.
Above is the preclinical stage and phase 1 clinical trials for a drug, mapped out using Proggio. You can see that if it started today, they process would finish in Q3 2020 – that’s around two years time!
Many biotech companies use Proggio to manage this complex project. Proggio has a template for phase 1 clinical trials available, which can save researchers some time in their efforts. From exploratory research to pill in the medicine cabinet, biotechnology takes time, effort, perseverance, and a healthy dose of project management skill.