Can't get it in time, can't afford it, can't keep it up: how to unlock the triple bind of rare-disease medication

Can't get it in time

R&D, launch, and reimbursement have never been on the same timeline. Even when a rare-disease drug has already been approved abroad, or has shown clear clinical value, patients may still wait months or even years because of local review, price negotiation, and National Health Insurance listing procedures.

In recent years Europe has gradually institutionalized this gray zone. Since July 2021, France has used two mechanisms—Accès précoce (early access) and Accès compassionnel (compassionate use)—to replace the old ATU/RTU framework, allowing patients with certain serious or rare diseases to obtain treatment under rigorous conditions before formal launch and reimbursement are complete, while simultaneously accumulating real-world data. At the EU level, Article 83 of Regulation (EC) No 726/2004 provides the legal basis for "compassionate use," with the EMA/CHMP issuing opinions to help member states maintain a degree of consistency in system design.

For Taiwan, what is truly missing is not the goodwill of a single case, but a bridge that can systematically catch patients. When a patient has been diagnosed, the physician has a treatment direction, and the drug is not entirely nonexistent, the task of medical services is not just to "wait for policy," but to help patients string together diagnosis, referral, application, medication communication, and continued follow-up into a workable path.

Can't afford it

The sharpest reality in rare-disease treatment is price. Taking Taiwan's publicly available information as an example, a single dose of Zolgensma costs about NT$49 million, one dose of AADC gene therapy is upward of NT$100 million, and one course of CAR-T also costs about NT$14 million. Under traditional annual budgets and the global budget system, this kind of one-time, high-cost expenditure is easily seen as an unabsorbable cost.

As a result, international payers have begun to develop different tools, such as outcomes-based agreements (OBA), installment payments, annuity-style payments, guarantee schemes, and data-collection-based reimbursement designs. The core logic is all the same: to rewrite an immediately incurred drug price into a trackable, verifiable payment relationship that can be adjusted according to treatment outcomes. This is not only financial engineering but also a prerequisite for giving high-priced therapies a chance to enter the real medical system.

On the patient side, "can't afford it" is often not just the drug price itself. From cross-hospital referrals after diagnosis, testing, medication education, and side-effect monitoring, to family communication and follow-up visit arrangements, all are hidden costs that determine whether treatment can truly begin. Rare-disease care therefore needs not just subsidies, but a medical service that integrates financial assistance, treatment-course management, and care coordination.
 

Can't keep it up

Even once a patient has started medication, they may still get stuck on the next question: how to keep it going? From the end of a clinical trial, to formal launch, to the completion of National Health Insurance decisions, there is often a long window in between. For patients who need long-term maintenance treatment, the most frightening thing is not that treatment has not yet begun, but that after treatment starts it is interrupted because of a failure to bridge between systems.

Some countries have already combined early access with real-world-evidence collection, allowing patients' medication data during the transition period to flow back into subsequent HTA and reimbursement assessments. This means "can't keep it up" is not necessarily just a budget problem; it can also be improved through system, data, and medical-service process design.

Here, the role PatientsForce can play is not just to help patients find a program, but to serve as a medical-service collaboration platform, connecting pharmaceutical companies, hospitals, physicians, nurses, case managers, and patients' families, so that patient support programs (PSPs), drug reimbursement, care-assistance arrangements, treatment-course tracking, and administrative processes are no longer scattered in separate silos. For rare-disease care, the value of this kind of role lies in reconnecting the previously fragmented segments of the journey—diagnosis, treatment initiation, resource application, continued follow-up—reducing the administrative burden on patients and the medical side while improving access and adherence.

Taiwan's next step

In 2000, Taiwan passed the Rare Disease Control and Orphan Drug Act, becoming the first country in Asia to legislate to protect the rights of rare-disease patients. But many advocacy efforts and analyses point out that since second-generation National Health Insurance, the average wait for a new rare-disease drug from application to inclusion in National Health Insurance coverage has stretched from about 5 months to about 30 months, and the reimbursement rate has clearly declined. In recent years Taiwan has tried to promote approaches such as accelerated review, parallel submission, the twin regenerative-medicine acts, and installment payments; but between "the drug exists" and "the patient can truly get it, afford it, and keep it up," a more complete bridging system is still missing.

PatientsForce's role in this gap should not merely be that of an administrative executor, but can be that of a medical service partner in the rare-disease care system: helping design individual case paths, treatment initiation, patient support program implementation, cross-hospital resource linkage, and continued follow-up, so that even beyond the system there is someone to truly catch the patient's needs.

When we talk about EAP, PSP, RWE, or innovative payment, on the surface we are talking about system tools; but for rare-disease families, what matters more is actually this: during the long wait, is there a service system that can truly connect medical care, resources, and support. This is also the piece that rare-disease care most needs to fill in next.