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Pivotal Clinical Trial Costs Estimated at $19 Million

A new analysis has found it costs an average of $19 million to get new drugs through pivotal clinical trials and onto the market.
Researchers at the Institute for Safe Medication Practices and Johns Hopkins University reviewed 59 separate drugs or therapies approved between 2015 and 2016 and analyzed their costs based on estimates from IQVIA software. In what may be the first study of its kind, they found costs varied widely, from $12 million to $33 million per trial. A glaring exception: cardiovascular drug trials, which averaged more than $157 million.
The researchers said one lesson learned is that trials for novel drugs don’t have to break the bank.
“For years, there have been discussions about how expensive clinical trials are and can we afford to keep doing them at this level but no one had any price tags,” lead study author Thomas J. Moore told CenterWatch. This “actually puts a price tag on what it costs to run these trials.”
Tufts University’s Center for the Study of Drug Development periodically reviews the costs for all – including failed — clinical trials. Its most recent estimate, published in 2016, was that it took nearly $2.6 billion to get a single treatment to market.
Moore and his colleagues focused only on studies — most of them Phase III trials — that led directly to FDA approval.
Moore says he was motivated in part by critics urging regulators to trim the length of time of clinical trials, noting he was concerned that pressure to cut costs might lead policy mak­ers to sacrifice scientific rigor.
But it turns out the cost of pivotal trials are relatively reasonable.
“This is the price of evidence,” he says. “That’s telling me that you wouldn’t gain much by reducing requirements for approval and you might lose a lot.”
Moore and his colleagues identified three key elements likely to drive up costs. If a trial:

  • Needs large patient pools;
  • Requires the control group to take an active drug not a placebo; and
  • Focuses on clinical rather than on surrogate endpoints.

The most expensive trials involved cardiovascular drugs, the research team found. Those were followed by cancer drugs (more than $45 million), digestive system drugs (more than $29 million), central nervous system drugs (nearly $26 million) and dermatology drugs (nearly $25 million).
“The most striking finding we saw was that the most expensive trials were conducted because the drug under study was similar to an already proven drug for a similar ailment,” Moore says.
One such drug, the combo sacubitril-valsartan for chronic heart failure, checked in at nearly $347 million, Moore and his colleagues found.
Researchers not only had to prove the drug’s clinical benefit but that it was no worse than rival warfin. That meant more complicated trial designs and more patients, Moore said.
The team’s findings were published in JAMA Internal Medicine.