The Success Rate of Clinical Trials Is Higher than We Thought

clinical trials success rate2 1

Instead of less than 10% of drugs reaching the market, a new study has found that almost 15% of new compounds pass clinical trials – which still suggests that it is pretty tough.

Clinical trials give us a chance to test how a new drug or therapy interacts with the body and whether it is any good at curing disease before they are launched. Previous studies estimated that the likelihood of a drug at Phase I making it all the way to the market was between 3 and 9%. However, a new study carried out by researchers at Massachusetts Institute of Technology (MIT), and published in Biostatistics, has found that the outlook may not be quite so bleak, with a success rate of closer to 14%.

The study followed more than 21,000 compounds through the clinic between 2000 and 2015. The group at MIT defined key characteristics, including whether a compound was undergoing Phase I, II, or III clinical trials so that a diverse range of studies could be analyzed in the study. Once the fate of each of the compounds had been discovered, the researchers calculated the probability of success (POS) for all drugs and indications, which is used by researchers and investors to decide whether or not to pursue a particular drug.

The study coordinators defined the drug development pathway was defined as the development of a drug for a specific indication.

Overall, 13.8% of all drug development programs were eventually approved, which is higher than the 10.4% reported in a previous, smaller study. The study also found that there was an increased POS at each of the clinical trial phases, particularly at from Phase II to III, which increased from 32.4% in observed in a previous study to 58.3%. 

But the numbers vary greatly between indications. In the case of infectious diseases, 1 in 3 drugs was approved, while around 3% of cancer drugs succeeded. This is despite suggestions that too many cancer drugs are making it through the clinic. A study carried out by King’s College London and the London School of Economics found that the majority of EMA-approved cancer drugs had not improved survival or quality of life during clinical trials. In addition, of those that did improve survival, the benefits were limited, with patients surviving for an extra 5.8 months maximum. Therefore, should the rate of cancer drugs being approved be even lower than the MIT study indicates?

The POS for the different therapeutic areas between 2005 and 2015. Ophthalmology has the highest rate of success, while oncology has the lowest.

Although the mortality rate from cancer has declined, it remains one of the biggest causes of mortality in the world. This has drawn in research groups, biotechs, and pharma companies, all desperate to make a difference. However, it isn’t worth expediting the approval of new drugs if they are not going to be able to save lives once they’re on the market.

Whether it is 9% or 14%, this still feels like a pretty low return on what will be a considerable investment by pharma and biotech companies that enter the clinic. This points to the need for better technology to help research groups at the early stages of development. Maybe the use of artificial intelligence to support drug discovery will see these numbers improve in the coming years.

Images – SUWIT NGAOKAEW /; Lo, A et al (2018) Biostatistics

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