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90% of clinical drugs never launch
The odds
Making a drug typically takes about a decade of academic research, preclinical development, and clinical research, and that is when it works. The vast majority never make it into the clinic at all.
A fast exception
Against those odds, the first medicine for Pompe disease built by Novazym took just 18 months from start to finish. One of its founders puts that down partly to luck: there were a thousand reasons it shouldn't have succeeded, and some element of right time and right place came together. At Amicus, far more programs have failed than have succeeded.
Biotech is not for the faint of heart, and the odds are why: companies spend years and millions of dollars on a program, and the drug often turns out to be the wrong one.
Why do most drugs fail in clinical trials?
A 2022 review in Acta Pharmaceutica Sinica B examined why clinical drug development fails. It attributed 40 to 50 percent of failures to lack of clinical efficacy, about 30 percent to unmanageable toxicity, 10 to 15 percent to poor drug-like properties, and about 10 percent to commercial or strategic reasons. Animal models of disease often predict human results poorly, which is why many drugs look promising before reaching people.
Why does drug development take a decade?
Drug candidates move through preclinical testing and then three clinical phases, with safety first, effectiveness next, and large confirming trials last. Each phase can take one to several years because trials must follow patients long enough to see effects. After the trials, the US FDA's standard review goal for a new drug application is ten months after a 60-day filing period.
How do rare disease drugs move faster?
The US Orphan Drug Act of 1983 created incentives for rare disease drugs, and the FDA later added pathways such as fast track, priority review, and accelerated approval. Rare disease trials can be smaller because few patients exist, and regulators may accept measures that show a change in the disease. Before the law, fewer than ten orphan drugs had reached the market in the previous decade, and hundreds have been approved since.
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