FDA approval lowers, but doesn’t eliminate disparities in immunotherapy use

Regulatory NewsRegulatory News | 30 June 2022 |  By 

Racial, ethnic and sociodemographic disparities that are present in clinical trials and compassionate use agreements for cancer immunotherapy during the pre-approval period are reduced, but not eliminated after the US Food and Drug Administration (FDA) approves these cancer treatments, according to recent research published in JAMA Network Open.
“The time frame leading up to FDA approval is an important period in which to observe disparities,” Theresa Ermer, MD, of the section of thoracic surgery in the department of surgery at Yale School of Medicine in New Haven, CT, and colleagues wrote in their study. “The clinical trials that defined the safety and efficacy of these innovative treatments were accruing patients during this period and were likely a major route for patients in the preapproval era to access immunotherapy. As a result, disparities in the preapproval era likely reflect a lack of representativeness in the immunotherapy clinical trials.”
While immunotherapy use increased post-approval and this led to some narrowing of disparities, some persisted and new disparities emerged, the authors noted, “Suggesting that FDA approval alone does not ensure the optimal administration of novel treatments in the US.”
Ermer and colleagues analyzed immunotherapy use 4 years prior to approval of immune checkpoint inhibitors and 3 years postapproval among a cohort of 402,689 patients (mean age, 68 years old) in the National Cancer Database between January 2007 and December 2018, categorizing patients based on health, sociodemographic, and socioeconomic information. Of these, 347,233 patients had stage 4 non-small cell lung cancer (NSCLC), 43,714 patients had renal cell carcinoma (RCC), and 11,742 patients had melanoma. The cohort consisted of patients who were mostly white (83.4%) non-Hispanic (93.3%) patients, with a minority in the database reporting as Black (11.8%), another race (4.1%) or Hispanic (3.9%).
The researchers found 3.2% of patients with NSCLC, 4.8% of patients with RCC, and 8.6% of patients with melanoma underwent immunotherapy prior to FDA approval. In the months following FDA approval, 15.6% of patients with NSCLC, 19.7% of patients with RCC, and 19.3% of patients with melanoma received immunotherapy. “For each tumor type, there was a progressive increase in the use of immunotherapy leading up to FDA approval, then a substantial increase in use in the years after FDA approval,” Ermer and colleagues said.
Overall, immunotherapy use significantly increased among race and ethnicity models for patients with NSCLC (odds ratio, 5.59; 95% confidence interval, 5.43–5.75; P < .001), RCC (OR, 5.06; 95% CI, 4.72–5.43; P < .001) and melanoma (OR, 2.66; 95% CI, 2.37–2.98; P < .001).
However, racial and ethnic disparities still persisted: compared with white patients, immunotherapy use was significantly lower in the post-approval period among Black patients with NSCLC (OR, 0.78; 95% CI, 0.71–0.85; P < .001) and RCC (OR, 0.74; 95% CI, 0.66–0.82; P < .001), and Hispanic patients with NSCLC (OR, 0.79; 95% CI, 0.67–0.92), RCC (OR, 0.70; 95% CI, 0.62-0.79; P < .001), and melanoma (OR, 0.64; 95% CI, 0.43-0.93; P = .02).
Insurance status also impacted immunotherapy use, with a significantly lower likelihood of immunotherapy use among non-insured patients with NSCLC (OR, 0.54; 95% CI, 0.50­–0.58; P < .001), RCC (OR, 0.48; 95% CI, 0.39–0.58; P < .001), and melanoma (OR, 0.48; 95% CI, 0.37-0.61; P < .001) compared with privately insured patients.
While a partial reduction in disparities could potentially be explained by increased access to immunotherapies, the authors said, persistence of disparities may be a sign of “unmitigated access barriers” for these patient populations such as a delay in payer coverage following approval as well as socioeconomic characteristics.
“Many of the challenges outlined (eg, travel distance) had disproportionate consequences for socioeconomically disadvantaged patients and were likely incompletely mitigated by FDA approval,” they said.
JAMA Netw Open Ermer et al.


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