New Report Shows Past Drug Failures Aid in the Fight Against Cancer

A new report examines investigational cancer medicines that did not succeed in clinical trials. These so-called “failures” have helped advance the treatment of this devastating set of diseases. #PhRMA

John CastellaniOctober 8, 2014

This week PhRMA released a new report, “Researching Cancer Medicines: Setbacks and Stepping Stones,” which examines the investigational cancer medicines that did not succeed in clinical trials and how these so-called “failures” have helped advance the treatment of this devastating set of diseases.

While it may sound counterintuitive, research setbacks are central to advancing our knowledge of cancer. In our new report, we examine three difficult-to-treat cancers – melanoma, lung cancer, and brain cancer. In the past 16 years, there have been a total of 338 unsuccessful investigational drugs and only 20 FDA-approved medicines across the three cancers.

  • Melanoma – Seven medicines have been approved, whereas 96 investigational medicines did not make it through clinical trials. These setbacks have begun to bear fruit with several “game changing” medicines approved in recent years for the most deadly form of the disease.
  • Lung Cancer – Ten medicines have been approved compared to the “failure” of 167 potential treatments. We are beginning to see innovative new approaches that are improving life for patients with lung cancer and also providing hope for more to come.
  • Brain Cancer – Three new medicines have been approved, while another 75 investigational medicines were unsuccessful in the development process. In brain cancer we have seen progress but we still have a long way to go. These numbers show the level of effort researchers are making and their commitment to persevere.

The report illustrates the immense challenges in developing medicines to treat cancer. On average, it takes 10 to 15 years and more than $2.6 billion to bring a new medicine from the research pipeline to patients. Cancer medicines take an average of 1.5 years even longer to develop. While most investigational medicines never make it to market, every success – and every “failure” – builds on previous setbacks and advances to improve patients’ lives.

Despite the challenges, we have reason to be optimistic about the future of cancer treatment. Earlier this week, we released a report which found that there are 771 medicines and vaccines to treat or prevent cancer in clinical trials or awaiting FDA review. Another report on innovation in the biopharmaceutical pipeline found that 80 percent of cancer medicines in the pipeline globally are potentially first-in-class treatments – meaning that they use a new and unique mechanism for treating a medical condition.

Our industry’s significant investment in research, $51.1 billion in 2013 alone, is part of the reason I’m proud to join leaders in the cancer community at the Turning the Tide Against Cancer National Conference tomorrow in Washington, DC. This is an opportunity for health care stakeholders – health care professionals, academia, patient groups, government and industry – to discuss ways to improve cancer care and promote innovation. At this conference, I have the distinct pleasure of announcing two grants from the PhRMA Foundation to support new research measuring the benefits of cancer treatment innovation and the value these advances bring to cancer patients.

We hope you join us in celebrating the tremendous progress that has been made in improving treatment for cancer patients.

For more information on the conference or to view the live webcast of the meeting, please visit

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


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