[ASH 2024 Roundtable ②] Korean experts call for regulatory overhaul in blood cancer care

During the 2024 American Society of Hematology Annual Meeting, Korea Biomedical Review gathered experts from Korea and the U.S. to discuss the transformative impact of CAR-T cell therapy and bispecific antibodies, focusing on challenges faced in the Korean healthcare system. The discussion revealed complex systemic barriers while exploring potential solutions for broader access to these revolutionary treatments. The second part of the discussion is between three Korean hematology leaders from the Korean Society of Hematology.-- Ed.

SAN DIEGO, Calif. - By Lee Han-soo/Korea Biomedical Review correspondent - In a candid roundtable discussion, leading figures from the Korean Society of Hematology (KSH) highlighted critical challenges facing blood cancer treatment in Korea, particularly emphasizing the urgent need for more flexible drug approval and reimbursement policies to keep pace with rapid medical advances.

Professor Kim Seok-jin, Chairman of the KSH from Samsung Medical Center, expressed particular concern about the growing gap between medical progress and regulatory frameworks.

"The current regulatory environment isn't keeping pace with medical advancement," Kim said. "While we're seeing breakthrough treatments emerge globally, our institutional framework often creates barriers to accessing these innovations."

The discussion revealed a key issue in Korea’s regulatory process: the requirement for long-term follow-up data.

Professor Yoon Dok-hyun, Research Support Director of KSH, highlighted this challenge, questioning the ambiguity in regulatory requirements.

"We're facing a fundamental dilemma with the demand for long-term follow-up data," he explained. "The regulatory bodies keep requesting long-term data, but there's no clear definition of what constitutes 'long-term.'”

More importantly, what happens to patients who need these treatments while waiting for this undefined long-term data to accumulate, Yoon added.

Yoon further emphasized a critical concern regarding the management of newer cancer therapies, particularly bispecific antibodies.

"The emergence of bispecific antibodies has made cytokine release syndrome (CRS) a significant concern," he said. "Yet we face regulatory barriers in accessing essential medications to manage these complications."

He explained that while Actemra (ingredient: tocilizumab) has labeling for CAR-T cell therapy-induced CRS, it lacks approval for CRS resulting from bispecific antibody treatment, creating a significant treatment gap.

The discussion turned to systemic issues within the healthcare regulatory framework.

"This isn't just about a single medication," Yoon stressed. "We're seeing similar challenges with antiviral medications for herpes zoster prevention and other supportive care medications.”

As a result, doctors are often forced to prescribe these medications off-label, accepting the risk of insurance claim denials, he added.

Yoon stressed that the gap between medical advancement and regulatory adaptation is becoming increasingly problematic."

This regulatory hurdle has significant implications for patient access to innovative treatments. Professor Kim Hye-ry, Public Relations Director of KSH, shared a telling example involving Kineret (ingredient: anakinra), a critical supportive care medication for managing CRS.

Despite efforts to work with international pharmaceutical companies for relabeling and import, they've encountered resistance due to pricing concerns.

"When we approached the original manufacturer, they indicated they had no plans to enter the Korean market because of anticipated price reductions," Kim explained. "This creates a devastating gap in our treatment arsenal, particularly when managing serious complications from newer cancer therapies."

Yoon also highlighted the broader implications for Korea's position in global clinical research.

"Seoul currently ranks as the world's leading city for clinical trials, primarily due to our efficient patient enrollment and cost-effectiveness," he said. "However, if our regulatory framework continues to demand Korea-specific data beyond what's required by the FDA or EMA, we risk losing this position.”

This would not only impact our industrial advantages but, more importantly, would reduce our patients' access to innovative treatments through clinical trials, he added.

KSH to lead the charge in making policy changes

Kim, as KSH Chairman, outlined the society's renewed focus on addressing these challenges.

"Since July 2024, we've been actively engaging in policy seminars and dedicated policy sessions at our academic conferences,” he said. “While maintaining our academic excellence, we're now taking a more active role in addressing these practical challenges that affect both physicians and patients."

He elaborated on the society's shifting priorities.

"Historically, our society concentrated primarily on academic achievements and successful scientific conferences,” he said. “However, we've recognized the urgent need to expand our role.”

Today's healthcare environment demands that doctors become more actively involved in shaping policy and addressing real-world clinical challenges, he added.

However, he acknowledged the complexities of achieving meaningful change.

"While we're making every possible effort, we recognize that not all our recommendations can be immediately implemented,” the Chairman said. “From the government and regulatory perspective, there are multiple factors to consider.”

Nevertheless, KSH believes there needs to be greater weight given to expert opinions in the decision-making process, particularly when it comes to specialized fields like hematology, he added.

Kim concluded that the expertise of clinicians who regularly use these advanced therapies needs to be better integrated into the policy-making process.

"We're not just looking at individual drug approvals or reimbursement decisions, but at creating a more comprehensive framework that can adapt to emerging medical innovations while ensuring patient safety and healthcare system sustainability,” he added.