Drug Shortages and Special Formulations: How Named-Patient Imports Bridge Treatment Gaps for Rare Disease Patients

Many patients with rare diseases or special medical needs face a recurring problem: the medicine they need — or the formulation they can actually take — is not available in Taiwan.

When a rare disease patient has no domestic treatment option, or lacks a suitable formulation (for example, an infant who cannot swallow tablets), named-patient importation offers a route to medicines not yet supplied locally. With the active guidance of the Taiwan Food and Drug Administration (TFDA) and close public-private collaboration, many patients facing complex cross-border regulatory procedures have been able to obtain the medicines they need in accordance with the law.

Controlled Substances Follow a Different Import Process

Yu-Han Lee, Deputy Director of the Early Access Office at PatientsForce, notes that many people assume a "personal-use drug import" application is enough to purchase medicines from abroad. But when a medicine involves controlled substances, the import process differs from that of ordinary drugs and must follow current regulations and the requirements of the competent authorities. "Sourcing medicines across borders is not a simple administrative filing," Lee emphasises. "A controlled substance must be applied for by a medical institution as a named-patient case, under the TFDA's professional guidance and oversight — that is what ensures the medicine reaches the patient legally and safely."

Complex Cross-Border Review, Bridged by Multi-Party Collaboration

Consider a young rare disease patient suffering from persistent dystonia and severely elevated intracranial pressure. Unable to swallow, the child urgently needed an imported oral-drop formulation not routinely stocked in Taiwan. Because the medicine required three-way review — by the TFDA, the International Narcotics Control Board (INCB), and the exporting country's authority — the administrative process was complex and the timeline critical.

During the cross-border application, facing delays in how international review messages were relayed, the TFDA case officers showed remarkable professionalism and efficiency, helping establish a real-time digital verification channel that connected Taiwan's system with international agencies. "It was precisely the TFDA's proactive communication and guidance that allowed the authorisation to be renewed before the permit deadline," Lee says. "The medicine arrived in Taiwan in time to keep the child on treatment."

Growing Demand for Early Access, Anchored in Institutional Safeguards

As precision medicine advances, demand for importing early-access medicines from abroad continues to grow. Applications still involve medical need, drug sourcing and cross-border regulatory procedures, and must be handled by medical institutions together with the competent authorities. Lee points out that the EAP (Early Access Program) mechanism is more than a regulatory procedure — it is the institutional bridge that ensures rare disease patients can obtain medicines under proper safeguards. Patients and industry alike should work within the regulators' framework so that cross-border named-patient applications proceed lawfully, under sound supervision and institutional support.

Reprint Notice

This article is reprinted with permission from Health News (健康醫療網). Original title: 「國內缺藥、特殊劑型怎麼辦？專案進口助罕病患者銜接用藥」, reported by Yi-Ning Huang.

Original article: https://www.healthnews.com.tw/article/69544