Cost-Effectiveness of Erdafinib vs. Chemotherapy for Advanced or Metastatic Urothelial Carcinoma from the Payer Perspectives in the US and China

Bladder cancer is a disease that affects the lining of the bladder, and when it spreads beyond that organ, it becomes a serious condition known as metastatic urothelial carcinoma. For decades, the standard approach to treating this advanced stage has been chemotherapy, a powerful but often difficult treatment that uses drugs to kill rapidly dividing cells. However, many patients do not respond well to chemotherapy, or their bodies stop responding after a few months. In recent years, doctors have turned to a different strategy for specific patients: targeted therapy. This approach looks for specific genetic changes inside the tumor cells, such as alterations in genes that control how cells grow, and uses drugs designed to block those specific signals. One such drug, erdafitinib, was approved to treat patients whose tumors carry these specific genetic changes and who have already tried other treatments without success. While this drug offers hope where other options have failed, it comes with a high price tag, raising a critical question for healthcare systems: does the extra benefit it provides justify the extra cost compared to standard chemotherapy?

Researchers from Peking University First Hospital set out to answer this question by building a detailed computer model to simulate the long-term outcomes for patients in two very different healthcare systems: the United States and China. They focused on patients with advanced bladder cancer who had specific genetic changes and whose disease had progressed after trying immune therapy. The team compared two paths: one group of patients receiving the new targeted drug, erdafitinib, and another group receiving standard chemotherapy. The model tracked how long patients lived, how much time they spent in good health, and the total medical costs incurred by payers, such as insurance companies or government health programs, over a ten-year period. The researchers fed the model with real data from a major clinical trial, adjusting for the different costs of drugs, hospital visits, and managing side effects in each country. They also accounted for the fact that patients who get sicker later in the course of treatment might receive different follow-up therapies.

The results of this simulation were clear and consistent across both nations. While the targeted drug did help patients live longer and spend more time in a state of good health compared to chemotherapy, the financial cost was disproportionately high. In the United States, the extra cost to gain one additional year of healthy life was calculated to be over one million dollars. In China, the figure was significantly lower but still far above what health systems typically consider a reasonable investment. The researchers found that under current pricing, the drug is not a cost-effective choice for payers in either country. This means that for every dollar spent, the health benefit gained is not enough to justify the expense when compared to the cheaper, albeit less effective, chemotherapy option. The study suggests that the drug's price is the primary barrier to its economic viability.

To understand how much the price would need to change to make the drug a sensible financial option, the researchers ran further simulations. They discovered that for erdafitinib to become cost-effective in the United States, its price would need to drop to roughly thirteen percent of its current cost. In China, the price would need to fall to about twenty-four percent of its current level. Even when the researchers removed the cost of the genetic testing required to find the right patients, the conclusion remained the same: the drug itself is too expensive relative to the health benefits it provides. The study also looked at the likelihood of the drug being a good value if the price were to fluctuate, and found that under current conditions, there is virtually zero chance it would be considered cost-effective.

This analysis does not suggest that the drug is ineffective or that patients should not receive it if they can afford it. The clinical data used in the model showed that erdafitinib extends life and delays disease progression better than chemotherapy for this specific group of patients. However, from the perspective of a healthcare system managing limited resources, the current price creates a gap between the clinical benefit and the economic value. The researchers emphasize that while the drug represents a significant medical breakthrough for a difficult-to-treat cancer, a substantial reduction in its price would be necessary to make it a sustainable option for widespread use in both the United States and China. The study concludes that without such a price adjustment, the drug remains an expensive treatment that, while beneficial to the individual, places a heavy burden on the collective healthcare budget.