The regulatory strategy of global pharmaceutical companies is changing to enhance access to new drugs in the treatment landscape for rare and intractable diseases.

The trend is a 'regulatory science approach' that goes beyond simply meeting approval requirements. It now involves incorporating patient-reported treatment experience and quality-of-life (QoL) measures into regulatory and evaluation processes from the early stages of development.

In line with the current trend, UCB is restructuring its global regulatory strategies, focusing on a patient-centric approach across clinical design, evaluation variables, and regulatory cooperation.

DailyPharm met with Susanne Dorn, Senior Vice President and Head of Global Regulatory Affairs at UCB, to discuss the shift toward patient-centric regulatory strategies and the direction of collaboration in the Korean market.

Changes in regulatory strategy, strengthening patient-centric access

Susanne Dorn evaluated that the regulatory function within pharmaceutical companies is shifting from merely meeting requirements to a scientific discussion focused on delivering value to patients.

She said, "In rare and intractable diseases, it is difficult to secure sufficient evidence using conventional clinical trial designs alone," and explained. "Therefore, an approach that sets the actual difficulties and burdens experienced by patients and their families during the treatment process as variables in the clinical design stage is becoming critical."

According to Dorn, UCB is incorporating everyday patient experiences, such as the degree of functional recovery, the burden of taking medicine, and the long-term sustainability of treatment, into its clinical trial endpoints.

She said, "UCB sets investment priorities and establishes strategies to simultaneously meet regulatory requirements and deliver tangible value to patients during the drug development process," and emphasized, "The patient's story is not just a reference, but it is the motivation and starting point for why we must continue developing this therapy."

Dorn also noted the expansion of the regulatory environment toward Regulatory Science, shifting from an approval-procedure focus to one centered on scientific evaluation.

"Given the increasing complexity of diseases, it's crucial to set appropriate endpoints and find the optimal pathway from the product development stage," she stressed. "In areas where the total population is small, such as rare diseases or pediatrics, an efficient evaluation design that maintains scientific evidence is necessary."

It means that the importance of developing faster, more efficient evaluation methods while maintaining scientific integrity through collaboration with regulatory authorities aligns with the discussion of utilizing Real-World Data (RWD).

Dorn added, "Regulatory agencies in various countries are actively considering new evaluation methods like expedited approval, and UCB is collaborating closely with them."

"Korean approval success provides crucial global insights"

UCB's regulatory perspective is drawing attention because the company has recently achieved success in new drug approvals in Korea.

Starting with the psoriasis treatment Bimzelx (approved August 2024, reimbursed June 2025), the company has continued its success with the systemic myasthenia gravis treatments Zilbrysq (approved November 2024) and Rystiggo (approved April 2025).

Regarding these successes, Dorn said, "Korea is a vital country in the global market, and we are extremely proud of the achievements made in the short span of the last one or two years. We see the effort to resolve unmet needs through a patient-centric approach in neurology and immunology as highly exemplary."

UCB is pursuing collaborations that leverage the environments and strengths of each country in line with its global strategy. The recent experience in Korea is considered crucial for the execution of the global strategy.

"Based on these experiences, we are deliberating on how to implement successful cases from the U.S. and Europe even more effectively in the Korean market," she emphasized. "This visit to Asia is also intended to broadly understand institutional differences between countries and further strengthen global collaboration."

UCB is also currently preparing for the domestic approval of its Dravet syndrome treatment candidate. The drug has been selected as a target for the MFDS's Concurrent Approval-Evaluation-Negotiation Pilot Program, which is noteworthy.

Dorn stressed, "Dravet syndrome is a severe intractable disease that impacts the entire daily life of not only the children affected but also their caregivers and siblings. The treatment goal is not just to reduce seizure frequency and manage comorbidities but also to restore the family's quality of life."

She said, "Access to innovative treatments is not only a matter of drug price or budget discussions; it is a matter of the time remaining for patients," and added, "UCB will collaborate with Korea's institutional efforts and do its utmost to create an environment where innovative treatments genuinely change the lives of patients and their families."

Dorn concluded by stating that the global headquarters and the Korean branch will work closely together to expand patient access to patient-centric, innovative treatments continuously.

"Our goal is to create an environment where Korean patients can rapidly benefit from innovative treatments through efforts, including regulatory collaboration and clinical evidence generation," she added. "UCB will continue to accurately identify unmet patient needs and fulfill its role as a partner contributing to the improvement of the treatment environment."