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Few newly approved cancer drugs change first-line treatments

But many are helpful for second-, third-line treatment courses.

Out of 207 new cancer drugs approved in half a decade, only 14% displaced standard-of-care medications.

However, most anti-cancer therapies (42%) approved in that same time period were approved for use as later-line drugs for second, third or later-line uses.

This isn’t a bad thing, but disappointing in that there weren’t more breakthrough drugs for first-line treatment, said Dr. David Benjamin, chief fellow in hematology and oncology at the University of California at Irvine.

“Cure is uncommon with second- and third-line treatments, but they can help prolong life,” he told CanadianHealthcareNetwork in an interview. He was lead author of a study investigating cancer drugs approved by the US FDA over a five-year period. The goal was to determine how many displaced standard-of-care therapies and how many were added to existing therapies or to fill breaks in systemic treatments in the metastatic setting, adjuvant setting, or maintenance setting. Findings were published in JAMA Network Open.

The past decade has been considered the ‘golden age of oncology’ because of the approval of new types of cancer therapies—especially immunotherapy such as checkpoint inhibitors and targeted therapies.

The study noted that despite obstacles triggered by the pandemic, the FDA “approved more new cancer medicines during 2020 than in the prior year.” Generally, the large numbers of cancer drugs being approved is a hopeful sign after a drought of several decades without new drug approvals for several cancers.

“Several anti-cancer drugs receive FDA approval each month, but it wasn’t clear how many really changed the treatment landscape for each tumor group and displaced the existing standard-of-care therapies, as opposed to simply providing an alternative treatment option,” Dr. Benjamin said.

The retrospective cross-sectional study used all landmark clinical trials which lead to FDA approval of cancer drugs between May 1, 2016, and May 31, 2021. Data were obtained from the FDA Oncology (Cancer)/Hematologic Malignancies Approval Notifications website.

The researchers evaluated all malignant hematology and oncology drug approvals to determine which newly approved drugs displaced the standard-of-care drugs. They also sorted the data according to those used in combination with previously approved drugs, those approved for use in the adjuvant or maintenance settings, and which ones were approved for use in the second-, third-, or later-line settings. Some drugs were counted more than once because they were approved for different cancers.


Lung therapy drugs had most approvals

Of the 207 approvals, 28 drugs (14%) were first-line therapies that displaced cancer therapies previously approved and considered to be the standard of care for their indication. Thirty-two drugs (15%) were first-line drug alternatives or new therapies which did not displace the existing standard of care. Sixty-one drugs (29%) were add-on therapies approved in addition to previously approved therapies or were approved for use in the maintenance and adjuvant settings.

Finally, 86 therapies (42%) were approved as later-line drugs for use in the second-, third-, or later-line settings.

Drugs for lung-related tumors had the largest number of approvals (37 new drugs), followed by other drugs for other common cancers such as genitourinary tumors (28 new drugs), leukemia (25), lymphoma (22), breast cancer (19), gastrointestinal cancers (14).

A number of the new cancer drugs displaced standard of care drugs and represent important success stories, the study said.

Read: Lung cancer detected earlier in breast cancer patients

Researchers concluded that “These later-line drugs may benefit patients with few alternatives but also add to cost of care and further delay palliative and comfort services. Further incentives to maximize competition in the cancer drug space warrant consideration.”

The findings aren’t particularly surprising, said Dr. Doreen Ezeife, a medical oncologist and clinical assistant professor of medicine at the University of Calgary. She was not part of the study.

While most don’t displace first-line standard-of-care treatments, “There is value in second and third-line treatments—our patients need these options available to them after the cancer grows resistance to the first treatments that we try,” she said in an interview.

She noted too, that some new first line drugs don’t displace currently used medications, but work as alternatives. And this has value because “Some patients can’t have the standard of care either because of pre-existing comorbidities, or the nature of their cancer. They would want to have other options,” she said.

Read: Chemotherapy doesn't increase the risk of death from COVID-19

Canada approves similar numbers of drugs as the US—but usually after they are already available there. The reason is because drug companies tend to seek approval from the three largest sectors first—the US, the Europe Union and Japan. Once they get approval in those areas, then they’ll apply for approval in smaller markets such as Canada.

But even once an application is received in Canada, the process takes about three months longer than the process at the FDA, according to research by Dr. Ezeife and colleagues.

“In Canada, after a drug is approved, it still needs to undergo Health Technology Assessment in order to be placed on individual provincial formularies. Those additional steps can further delay the accessibility of a drug for patients to use. Whereas in the US, after FDA approval the drug is generally accessible for patients,” Dr. Ezeife said.

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