Press Release

Past Drug Failures Help Drive Advances in War on Cancer

PhRMA October 7, 2014

Washington, D.C. (October 7, 2014) — Today, the Pharmaceutical Research and Manufacturers of America (PhRMA) released a new report, “Researching Cancer Medicines: Setbacks and Stepping Stones,”  which highlights the number of investigational cancer medicines that did not succeed in clinical trials and how these so-called “failures” are a critical part of the drug development process. 

The report illustrates the immense challenges in bringing new medicines to patients with cancer, and explores the factors that contributed to both the approvals of new treatments and those that “failed” between 1998 and 2014. The report focuses on three cancers that are particularly difficult to treat: melanoma, lung cancer and brain cancer.

Subscribe ButtonKey findings include:

  • 96 potential treatments for melanoma did not make it through clinical trials, but paved the way for 7 approved medicines, a nearly 14:1 ratio of “failures” to “successes.”
  • Ten medicines have been approved to treat lung cancer, whereas 167 other potential treatments did not make it through clinical trials.
  • Only 3 new medicines have been approved to treat brain cancer, while another 75 investigational medicines were unsuccessful in the development process.

Despite these challenges, America’s biopharmaceutical companies continue to invest in research to develop new treatments.  According to a new report by PhRMA, there are 771 cancer medicines and vaccines either in clinical trials or awaiting review by the U.S. Food and Drug Administration (FDA). Of these medicines, more than 50 are for the treatment of melanoma, 98 for lung cancer and 47 for brain cancer.

“While it may sound counterintuitive, research setbacks are instrumental to furthering efforts to better understand a disease and how to treat it. They are also an indication of the incredible difficulty in developing medicines to treat cancer,” said PhRMA President and CEO John J. Castellani.  “These setbacks serve as a reminder that to make progress, we need a public policy framework that supports drug development in combination with promising science so that we can bring important innovations to patients.”

Significant advancements in the treatment of diseases like cancer are typically the result of cumulative innovation over time, rather than a single breakthrough in treatment.  Every success – and every “failure” – builds on previous advances to improve patients’ lives. Research has shown that cancer is actually a set of more than 200 extremely complex diseases and discovering medicines that effectively treat each one is a difficult task.

“While it is incredibly disappointing to see a promising new drug candidate eliminated from the pipeline, researchers take immeasurable learnings from every setback and build upon each one to develop effective therapies for patients,” said Castellani.

The release of the “Researching Cancer Medicines: Setbacks and Stepping Stones,” report comes in advance of the Turning the Tide Against Cancer National Conference on October 9, 2014. At the meeting, Castellani will join other health care stakeholders to discuss ways to improve cancer care and promote innovation. For more information, please visit

To learn more about the discovery and development process, from initial research to delivery of life-saving medicines, please visit

View the full “Researching Cancer Medicines: Setbacks and Stepping Stones” report. 

About PhRMA

The Pharmaceutical Research and Manufacturers of America (PhRMA) represents the country’s leading innovative biopharmaceutical research and biotechnology companies, which are devoted to discovering and developing medicines that enable patients to live longer, healthier, and more productive lives. Since 2000, PhRMA member companies have invested more than $550 billion in the search for new treatments and cures, including an estimated $51.1 billion in 2013 alone.

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