Benefits of Many Cancer Drugs Are Uncertain: Study

Drugs approved with limited evidence of effectiveness not supported by long-term patient outcomes, research finds

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The effects of expensive cancer drugs may fall short of achieving the long-term health benefits they’re designed to create, according to an extensive new study that examined dozens of clinical trials published in Clinical Drug Investigation.

Most Cancer Drug Benefits Are Uncertain

A team of researchers at the University of Gothenburg pored over claim reimbursement data in 40 clinical trials. While claim reports showed medicines to treat renal, lung, breast, and other cancers had therapeutic value, there was little evidence to support that the drugs were beneficial in the long run.

Results from just seven of the 40 trials showed that drugs played a significant role in extending patient survival rates. Only four were linked to improved quality of life.

Deeper analyses focused on an average 6.6-year follow-up from data collected between 2010 and 2020 in Sweden. Researchers looked at 22 drugs aimed at treating nine cancers: renal cell carcinoma, Hodgkin’s Lymphoma, chronic lymphocytic leukemia, chronic myeloid leukemia, melanoma, non-small cell lung cancer, breast cancer, ovarian fallopian tube cancer, and medullary thyroid cancer.

Only seven of the 22 drug indications had at least one trial showing conclusive evidence of overall survival rate and quality of life. The remaining 15 either didn’t include data on overall survival or quality of life, or they played no significant role in improving either.

“We have shown that the majority of the drugs launched with limited evidence still lack clear evidence of how they actually affect survival and quality of life in patients,” lead author Gabriella Chauca Strand, a doctoral student at Sahlgrenska Academy at the University of Gothenburg, said in a statement.

“The lack of confirmatory evidence for important patient outcomes is problematic and creates uncertainty about how these drugs actually contribute to meaningful patient benefit, and ultimately how effectively resources are being used within health care.”

Questions Raised

This raises the question of potential and significant flaws in the health care system. An estimated 18.1 million people worldwide had cancer (excluding nonmelanoma skin cancer) in 2020. To meet the treatment demand and quickly get drugs to market, most clinical trials focus on changes in biomarkers to prove that a medicine is successful but don’t include measures based on longevity. Reimbursement decisions are also based on cancer-specific biomarkers. Focusing solely on biomarkers isn’t enough to justify reimbursement from third-party payers, the authors argue in the paper.

The cost of cancer treatment places a tremendous financial burden on patients, families, and the health care system. According to a 2021 study, treatments and care for the top 15 leading cancers in the United States cost more than $156 billion in 2018. Another study, published in 2020, showed that the total cost of cancer in Europe was 199 billion euros (about $217 billion) in 2018. Health costs for all types of cancer care were 103 billion euros (about $112 billion), with 32 billion euros (about $35 billion) explicitly spent on cancer drugs.

Cancer is the second-leading cause of death in both the United States and Europe (after heart disease in the United States and diseases of the circulatory system in Europe).

Given the challenges of limited resources within health systems and the increasing disease burden, ensuring value for the cost should be an essential public health goal, the study authors wrote. Implementing expensive treatments with unclear effectiveness adversely affects not only patients with cancer, but it may also affect monies available for other patients suffering from disease, they wrote.

The study shows the urgent need for discussion about how cancer treatment is increasingly taking up limited resources within health systems and which drugs should be approved for reimbursement, the study authors concluded.