 What do you see when you go to the drugstore shelves full of pills to help with all sorts of ailments from headaches to allergies? What we don't see are the years of research that led to that little bottle on the shelf or the hundreds of drugs that were tried and tested along the way Let's walk through the very long and expensive but thorough journey of getting a new medicine to the shelf Shining a spotlight on the challenges faced at every stage But first what is a drug? For this discussion, we're talking about small organic molecules two great examples of these are aspirin and caffeine Drugs such as these work by binding to what is called a target The drug tweaks the activity of the target making it more or less active. So how do scientists find drugs? Some drugs such as caffeine come from nature But others like aspirin are made by chemists But it can be difficult to figure out which molecular shapes are most likely to be successful drugs So what to do? Well a common method is to make hundreds of thousands of different compounds and then sort them to find ones that bind specifically to the target and can tweak its activity This is called a high throughput screening if you're lucky you get a few hit compounds These hits usually need to be further chemically optimized, which is a tricky process However compounds that do make it through are tested for safety and efficacy in animals such as mice Many drugs that work in a test tube or Petra dish don't work when given to an animal or have adverse effects If the drug makes it through this filter step, which is another valley of death for drugs Researchers investigate the best way to deliver the medicine to people in a pill with a patch in an injection How does the body absorb, distribute, break down and excrete the drug? By this point many years have gone by After establishing efficacy and safety in animals an Investigational new drug IND application can now be filed with the FDA the food and drug administration If the FDA approves the drug can move into human clinical trials The first of these are phase one clinical trials to establish any adverse effects of the drug in humans If safety is established a phase two clinical trial is next to see if the drug is effective in Treating the disease in a relatively small number of patients There are many important decisions to make who should get the medicine for how long does it work better than current treatments? How will we know if it really works? These trials are usually performed in a blind fashion Which means neither the patient nor the physician know whether an experimental drug or a control Meaning no drug or a known drug is being administered If the phase two trial data shows patient safety and efficacy then a phase three trial is conducted These trials require a larger number of patients cost millions of dollars and can take years However, if the data is positive a new drug application or NDA is submitted to FDA And if approved the drug can finally be marketed to treat patients Sometimes for special cases such as epidemics life-threatening diseases or unmet medical needs Medicines can be fast-tracked an option developed by the FDA in 1988 at the height of the AIDS epidemic Naturally, even after a drug is on market drug safety and efficacy continue to be monitored Sometimes rare side effects only show up when many people take the medicine Phew, that was a lot of work to make a drug, but the truth is that it's even more involved Prior to starting drug discovery Researchers often in universities spend years or decades trying to understand a disease and to find potential relevant drug targets We still don't know enough about many diseases, which is why we don't have good drugs for Alzheimer's or Huntington's disease Although the road to a new drug is a lot of hard work time and money I think you'd agree that the drug discovery process gives us and our loved ones an opportunity to have healthy and happy lives And it gives us a sense of security that much testing has been done when we pick up those pills from the pharmacy