Developing A New Blockbuster Medicine: What Does It Take?

Any business in the medical arena that develops a blockbuster new medication is set to make a fortune. Companies that achieve these levels of success often see their share prices rocket tenfold, with massive dividends being paid to their chiefs.

But what does it take to get to this stage? How can the average company in the field achieve the heights of success and develop drugs that help the public? 

That’s the topic of this post. We look at the anatomy of breakthrough medical discoveries and what pharmaceutical firms can do to increase the odds of success. 

Look For Medical Needs

One of the reasons custom peptides have been so popular is because of the overarching medical need. These target multiple pathways, which is why they are so popular. 

It is admirable to target diseases with only a few patients and create “wonder” breakthroughs. However, these markets are often small and once the intervention has been used once, it isn’t liable for a second treatment. 

Therefore, look for opportunities in the most popular areas. Research diseases that are likely to be front and centre in years to come and research interventions that might deal with them. 

Conduct The Basic Science

Once you’ve done that, the next step is to move on to basic science. You need to have a fundamental understanding of what’s going wrong in the body and what might fix it. 

Sometimes, the answer will jump out at you immediately. Other times, it’s a struggle to figure out which pathways to target, or which chemicals are even relevant. The beauty of basic science is its ability to illuminate the problem. Once you know more, you can make better decisions about the path to chart forward. 

Test Libraries Of Compounds

If you have an idea which drugs might target the right pathways, test libraries of compounds. Keep running through them to see which works best in your situation and what’s most likely to work. Usually, you’ll wind up with a handful of substances that seem to work, at least in a petri dish. 

You can also try using machine learning to simulate the effects of compounds on the body. Again, the longer the list, the more likely you are to find something that works. 

Once you have a shortlist of viable compounds, the focus shifts from discovery to formulation and delivery, turning the active ingredient into a stable, easy-to-administer product. This is where modern pharmaceutical technology comes into play. Automated systems like capsule filling machines have become essential for producing precise, reproducible doses at scale. Understanding the capsule filling machine working principle helps researchers and manufacturers ensure that every capsule is filled accurately and consistently before clinical trials even begin.

Start Clinical Trials

Unfortunately, you can’t really get far in the medical testing world without clinical trials. These are the gateway you need to go through before agencies will approve your drugs for use by patients. 

Phase I trials get the ball rolling. These are small-scale studies to address safety issues (not efficacy). The next step is to move on to Phase II trials with a larger group of people. These test the effectiveness of the drug and the side effects it produces. 

Phase II trials are the final step and tend to be the most expensive, often employing extensive randomisation and including thousands of participants. 

Completing these can lead to regulatory approval and getting the go-ahead to sell the drug more widely. Of course, all this requires building a superstar team of clinicians and scientific experts to produce something that works.