OS Benefit Modest or Absent With Many High-Cost Therapies Approved for GI Malignancies

Overall survival benefits of FDA-approved treatment regimens are often short or in some cases, absent entirely, and are not correlated to treatment cost. These were the findings of an Irish study on access to novel agents for the treatment of gastrointestinal (GI) cancers presented at the 2021 Gastrointestinal Cancers Symposium.

The study looked at newly approved drugs in gastrointestinal oncology from 2010 to 2020 by first cataloging regimens that had been FDA-approved and then analyzing patient access to these drugs in the United States, United Kingdom, Europe, and Ireland.

By analyzing data from publicly available sources in each country/region, such as the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), the researchers catalogued all drugs approved for GI cancer treatment in each of the 4 studied areas over a 10-year period leading up to September 2020. The therapies included targeted agents, immune checkpoint inhibitors, and cytotoxic and hormone drugs. Using approval publications and updated survival analysis outcomes, the research team assessed the effect of each drug on overall survival (OS) and/or progression-free survival (PFS).

During the study period, 26 GI cancer treatment regimens were approved by the US FDA; 15 (57%) of these treatment interventions were also approved by the EMA, (median delay of 3 months after FDA approval). Of the FDA-approved regimens, only 31% and 27% were ultimately approved in the UK and Ireland, respectively.

The study found that 15 (58%) of the 26 regimens demonstrated an OS benefit; however, the median OS benefit was modest (2.3 months ± 3 months). Only 23% of the 11 remaining drugs demonstrated a PFS benefit.

Forty-three percent of FDA-approved regimens showed no OS benefit despite their high cost. The treatment regimens associated with an OS benefit were no different in cost than those that did not, with a median monthly expense of $13,065 and $13,758, respectively. The study authors also found no additional OS benefit with regimens that were more expensive.

The investigators concluded that less than one-third of patients in the UK and Ireland have access to FDA-approved medicines for GI malignancies approved in the past decade and that OS benefits of these regimens are often either brief or absent despite a treatment cost of more than $100,000 a year.

Disclosures: Some of the study authors disclosed financial relationships with the pharmaceutical industry and/or the medical device industry. For a full list of disclosures, please refer to the original study.

Reference

O Reilly DE, McLaughlin RA, Ronayne C, et al. Access to innovative cancer medicines in gastrointestinal oncology: 2010 through 2020. Poster presented at: 2021 Gastrointestinal Cancers Symposium; January 15-17, 2021. Abstract 469.

This article originally appeared on Cancer Therapy Advisor