[Reporter’s View] High-priced drugs: efficacy vs. efficiency

The launch of high-priced anticancer drugs and treatments for rare diseases will continue. There is no question that the efficacy of new drugs is improving, with a growing number of therapies extending survival or even offering the possibility of a cure.

The problem lies in how, for whom, and by what criteria these treatments should be used. This question is generating considerable debate in practice.

According to the collective views of oncology specialists, the current reimbursement framework is producing paradoxical outcomes.

This is because drugs for cancers with large patient populations are failing to secure reimbursement approval, even when they demonstrate strong clinical efficacy, simply because of concerns over financial sustainability. More problematic than the negative reimbursement decision itself is the lack of explanation regarding the rationale and criteria behind such decisions.

In the past, in addition to the improvement in overall survival (OS), the contribution of domestic patients to clinical trials was a key evaluation factor during approval or reimbursement review for new drugs. If domestic patients contributed to the clinical results, this was also partially reflected in the approval and reimbursement decision-making process.

Recently, however, a clear shift has emerged. Regardless of clinical benefit, drugs targeting cancers with large patient populations tend to be disadvantaged in reimbursement discussions, while coverage is increasingly concentrated on rare cancers with smaller patient populations. This perception is widely shared among clinicians.

If reimbursement policies have been adjusted due to financial constraints, the criteria and reasoning behind those decisions must be explained more clearly. Otherwise, patients are left unable to understand why the treatment they need is denied reimbursement.

Clinicians are well aware that healthcare resources are limited. However, there is growing concern over whether those limited resources are truly being allocated to where they are most needed.

For example, during cancer follow-up care, CT, MRI, and PET scans are often repeated without clear clinical justification. Streamlining expenditures in these areas could free up a substantial amount of funding for essential treatments.

The national health screening system also warrants reconsideration. Currently, cancer patients receive the same health screening notifications as the general population, leading to redundant examinations.

Despite cancer patients undergoing regular follow-up and monitoring at hospitals, the central data system fails to administratively distinguish them. Simply excluding cancer patients from routine general health screenings could significantly reduce unnecessary expenditures.

The government states that the ongoing drug price reforms and reimbursement system improvements are intended to safeguard the National Health Insurance finances and ultimately save patients with cancer and rare diseases. The direction itself, aimed at improving access to new drugs, cannot be denied. However, it is clear that before restricting treatment access, citing limited resources, we must first examine whether there are areas where public funds are being wasted inefficiently.

In the era of high-priced innovative medicines, reimbursement decisions are not merely about reducing or expanding reimbursement. They are about choosing between efficacy and efficiency. If patient survival and access to treatment are truly the guiding principles, then the priority should be eliminating unnecessary spending and systemic inefficiencies.