Disparities in cancer immunotherapy use persist after FDA approval
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Various sociodemographic and socioeconomic factors, including race/ethnicity and insurance status, correlated with receipt of immunotherapy among patients with melanoma and kidney and lung cancers.
The findings, published in JAMA Network Open, showed FDA approval led to an increase in immunotherapy use overall; however, key differences remained across patient cohorts.
Rationale and methods
“We wanted to understand which patients were receiving innovative treatments prior to FDA approval — a timeframe that is particularly important, as access to innovative treatment can be quite limited and largely represents clinical trial participation,” Daniel J. Boffa, MD, MBA, professor and chief in the division of thoracic surgery at Yale School of Medicine, told Healio. “Any disparities in the preapproval era could reflect differences in clinical trial participation, which could impact the representativeness and applicability of clinical trial findings. We were also interested in the reaction of clinicians and patients to FDA approval and to understand patterns of utilization once approval had mitigated some of the access barriers.”
Boffa and colleagues evaluated data of 402,689 patients (median age, 68 years; 55.9% men) with stage IV non-small cell lung cancer (n = 347,233), renal cell carcinoma (n = 43,714) and melanoma (n = 11,742) included in the National Cancer Database between 2007 and 2018. Most patients (83.4%) were white, 11.8% were Black and 3.9% were Hispanic.
Researchers pooled data on patient health and sociodemographic and socioeconomic characteristics, and they used multivariable logistic regression modeling to assess the association of patient characteristics with receipt of immunotherapy in the 4 years before and the 3 years immediately after FDA approval.
Key findings
Results showed that before FDA approval, only 8.6% of patients with melanoma, 4.8% with renal cell carcinoma and 3.2% with NSCLC received immunotherapy, whereas after FDA approval, 19.3% of patients with melanoma, 19.7% with renal cell carcinoma and 15.6% with NSCLC received immunotherapy.
Researchers identified sociodemographic and socioeconomic characteristics associated with variable administration of immunotherapy before FDA approval. Among those with NSCLC, Black patients had a lower likelihood of receiving immunotherapy than white patients (OR = 0.78; 95% CI, 0.71-0.85), and uninsured patients with renal cell carcinoma had a lower likelihood than privately insured patients (OR = 0.31; 95% CI, 0.2-0.48).
Although disparities narrowed after FDA approval for Black patients with NSCLC (OR = 0.87; 95% CI, 0.83-0.91) and uninsured patients with renal cell carcinoma (OR = 0.6; 95% CI, 0.48-0.75), some gaps widened across socioeconomic subgroups. This included for patients with NSCLC in the lowest vs. highest income quartile (OR = 0.8; 95% CI, 0.76-0.83). In addition, new disparities emerged, specifically among Black patients with renal cell carcinoma (OR = 0.82; 95% CI, 0.72-0.93).
Limitations of the study included the fact that distribution of facility and regional characteristics in the National Cancer Database is not generalizable to the general population. In addition, some sociodemographic strata were underrepresented and some patient subsets were small, which limited the ability to fully understand race-associated disparities in the cohort.
Implications
“Although we are not able to examine clinical trial participation directly, the findings in the preapproval era support previously voiced concerns over potential disparities in clinical trial participation for innovative treatments,” Boffa said. “The Henrietta Lacks Enhancing Cancer Research Act, passed in 2021, is intended to fund research to broaden access of clinical trials to ‘racial and ethnic minorities, older, rural and lower income individuals.’ We interpret our findings as being consistent with the signals supporting the urgency of this research need.
“In addition,” he added, “there may be opportunities to enhance the equity of innovative treatment administration in the years that follow FDA approval. Hopefully, the findings of the Henrietta Lacks Act will also be leveraged to optimize care delivery outside of the clinical trial setting.”
For more information:
Daniel J. Boffa, MD, MBA, can be reached at Yale School of Medicine, P.O. Box 208062, New Haven, CT 06520; email: daniel.boffa@yale.edu.
Perspective
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Ariel Sindel, DO
Immunotherapy has become a mainstay of cancer treatment since the first checkpoint inhibitor received FDA approval in 2011. Since then, multiple checkpoint inhibitor drugs have been approved. Indications have expanded considerably and now include metastatic disease, adjuvant therapy and neoadjuvant therapy. These unique drugs offer some patients the ability to have a long-term response, making their use even more important.
In this manuscript, the authors sought to see the potential barriers for immunotherapy use both before FDA approval and after FDA approval. It has been well-documented that socioeconomic barriers exist in clinical trials and limit the enrollment of those of non-white races. This was reflected in their study, which showed that — among patients with NSCLC — Black patients had a lower likelihood of receiving immunotherapy than white patients (OR = 0.78). In addition, economic disparities also limit trial enrollment, as patients with renal cell carcinoma who were uninsured had a lower likelihood of receiving immunotherapy than those with privately insured patients (OR = 0.31). After FDA approval, the gap narrowed, as the OR for Black patients with NSCLC increased to 0.97 and the OR for uninsured patients with RCC increased to 0.6.
These data answer an important question, but they also leave us with many other questions. They help show that some barriers are improved after FDA approval; however, many patients still are left without access to these vital drugs. Is it because many oncologists aren’t made aware of new FDA indications? In 2021, there were 54 new FDA oncology approvals, including new drugs or new indications. Is there still a gap due to a delay between FDA approval and when insurance companies start accepting their use? How do we better bring these vital clinical trials to the community so the demographics can more accurately mirror the diversity of the real world and improve racial and socioeconomic disparities in trials?
In this golden age of oncology with new drugs or indications being approved monthly, it is more important than ever to work to overcome these barriers to give every patient an opportunity to receive cutting-edge treatments.
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