Cancer accounts for more than one-quarter of new drug approvals, analysis finds

In Clinical Trials by Barbara Jacoby

By: Alaric DeArment


Overall, the report, from the Tufts Center for the Study of Drug Development, found that new cancer drug approvals since 2010 have increased by 4 percent since the 1980s.

More than one-quarter of the drugs that have received Food and Drug Administration approval have been for cancers, according to a new analysis.

The analysis, by the Tufts University Center for the Study of Drug Development in Boston, found that since 2010, cancer drugs account for 27 percent of all new drug approvals in the U.S., which itself represents a 4 percent increase from the 1980s. It was included in the Tufts CSDD Impact Report, which the center released on Tuesday.

Other findings included that clinical development time for cancer drug approvals between 1999 and 2018 was 9 percent longer than for drugs in other disease areas, and that regulatory approval phase time for the approvals during the same period was 48 percent shorter for cancer drugs than for non-cancer drugs. Drugs in hematological cancers also experienced longer clinical development and approval phase times than drugs for solid tumors – 8.8 years, compared with 7.5 years. Cancer drugs overall were likelier to receive priority ratings and orphan drug status from the FDA than non-cancer drugs.

“New approaches to development helped to drive the surge in new oncology products, including improvements in clinical trial design, novel drug formats and a focus on new and validated targets,” said Tufts CSDD director of economic analysis Joseph DiMasi in a statement. “Those efforts appear to have paid off, as cancer patients today have many more effective treatment options.”

A large number of the drugs that have entered the market in the last few years have done so thanks to winning accelerated approval from the FDA. This includes a number of new therapies with new molecular targets and even entirely new mechanisms of action. For example, 2017 saw the accelerated approval of the first CAR-T cell therapies, namely Novartis’ Kymriah (tisagenlecleucel) and Gilead Sciences’ Yescarta (axicabtagene ciloleucel) – the latter of which is approved for diffuse large B-cell lymphoma and the former of which is approved for DLBCL and pediatric acute lymphoblastic leukemia. That same year, the agency gave a supplemental approval to Merck & Co.’s Keytruda (pembrolizumab) that marked the first approval for a tissue-agnostic, biomarker-based indication, namely microsatellite instability-high and mismatch repair-deficient tumors. Loxo Oncology’s – and now Eli Lilly & Co.’s – Vitrakvi (larotrectinib) was the first to win such a biomarker-based label as its sole indication.

Still, some drug approvals have been controversial as well. The FDA’s decision to grant accelerated approval to Karyopharm Therapeutics’ Xpovio (selinexor) for penta-refractory multiple myeloma drew criticism from many people in the medical community.