Last month, the Massachusetts-based pharmaceutical company Vertex announced that the federal Food and Drug Administration had approved its new drug to treat Hepatitis C, Incivek. The drug took two decades to develop and is expected to bring huge profits to Vertex. Mansoor M. Amiji, Distinguished Professor and chair of the Department of Pharmaceutical Sciences at Northeastern University, said that new prescription drugs can cost more than $1.2 billion to develop, forcing many companies to focus almost exclusively on major drugs that will bring enormous profits.
How do pharmaceutical companies begin work on developing a new prescription drug, a process that can take decades?
It starts with understanding the biology of a disease, and then trying to intervene through the use of drugs. What you try to do is identify disease targets that you’d be able to affect through therapeutic intervention. Once you develop the target, you then come up with the molecules, which is basically the drug discovery process.
Essentially, you have a pyramid-type structure, with a large number of ideas potentially moving up the ladder. As a molecule gets higher and higher, these drugs start to show activity, but they also show a lot of problems. They may have a lot of toxicity issues, the doses they may need may be very, very high so they’re not realistic. You start to see attrition, which happens at the pre-clinical stage. At that point you come up with lead compounds, which you then take to the clinical stage.
What study continues if a drug is finally approved by the FDA – a one in 10,000 outcome – and released onto the market?
After the FDA approves a drug, there is a Phase 4 evaluation. After a drug is approved and is available to a large number of patients, obviously a much larger number than during testing, the data still needs to go back to the company to look at how the drug works.
This presents an opportunity to see how this drug works in a variety of populations. You can look at patient status or disease status, and you can see how it works based on different ethnicities, genders – many different variables. When drugs are taken off the market, it’s most likely because something that was missed during clinical trials.
Because it can take more than a billion dollars and several decades to develop a new drug, most pharmaceutical companies look to develop “blockbuster” treatments that can serve the largest population possible. What is being done to encourage the development of drugs that treat diseases affecting a smaller population?
That’s really one of the biggest challenges in pharmaceutical science and drug approval. Because the cost is so high and the process takes so much time, you really have to think of this as an investment — and any investor is looking for a positive return from that investment.
Fortunately in the United States, we have orphan drug laws. That is, if there are drugs that will only affect a small number of patients, companies may receive incentives from the federal government to develop those.
View selected publications of Mansoor Amiji in IRis, Northeastern’s digital archive.