Can't get it, can't afford it, can't stay on it: unlocking the triple barrier in rare-disease medication

By the PatientsForce editorial team | May 2026

Around 7,000 rare diseases are known worldwide, roughly 80% of them genetic — yet only a few hundred, about 5%, can be treated with medication. For most patients, the real challenge is not that no drug exists: even when it does, treatment is often stuck behind three barriers — can't get it, can't afford it, can't stay on it.

Can't get it

Development, market approval and reimbursement never run on the same timeline. Even when a rare-disease drug is approved abroad or has shown clear clinical value, patients may wait months or years for local review, price negotiation and national insurance listing.

Europe has been institutionalizing this grey zone. Since July 2021, France has replaced its old ATU/RTU framework with two mechanisms — Accès précoce (early access) and Accès compassionnel (compassionate access) — allowing patients with specific serious or rare diseases to receive treatment under strict conditions before launch and reimbursement are complete, while real-world data accumulates in parallel. At the EU level, Article 83 of Regulation (EC) No 726/2004 provides the legal basis for compassionate use, with EMA/CHMP opinions helping member states keep their frameworks broadly consistent.

What Taiwan lacks is not case-by-case goodwill but a bridge that can systematically carry patients across. When a patient is diagnosed, the physician has a treatment direction and the drug does exist somewhere, the task of a medical service is not to 'wait for policy' — it is to string diagnosis, referral, application, medication communication and continuous follow-up into a path that can actually be walked.

Can't afford it

The sharpest reality of rare-disease treatment is price. Public information in Taiwan shows that some rare-disease gene therapies cost tens of millions of NT dollars — in some cases over a hundred million — per dose, and a course of cell therapy can reach the ten-million level. Under traditional annual budgets and global caps, such one-off expenditures are easily deemed unabsorbable.

International payers have therefore developed new instruments — outcome-based agreements, installment payments, annuity-style payments, warranties and coverage-with-evidence designs. The core logic is the same: rewrite an immediate drug price into a payment relationship that is trackable, verifiable and adjustable to outcomes. This is not merely financial engineering; it is the precondition for high-cost therapies to enter real healthcare systems.

On the patient side, 'can't afford it' is rarely just the drug price. Cross-hospital referral after diagnosis, testing, medication education, side-effect monitoring, family communication and follow-up scheduling are all hidden costs that decide whether treatment truly starts. Rare-disease care therefore needs more than subsidies — it needs a medical service that integrates financial assistance, therapy management and care coordination.

Can't stay on it

Even when a patient starts treatment, the next question looms: how to continue? Between the end of a clinical trial, formal launch and the completion of reimbursement decisions there is often a long gap. For patients who need long-term therapy, the most frightening thing is not failing to start — it is starting and then being cut off by a failed institutional hand-over.

Some countries now combine early access with real-world evidence collection, so that data from the transition period flows back into subsequent HTA and reimbursement assessment. 'Can't stay on it' is not necessarily a budget problem alone; it can be improved through institutions, data and service-process design.

Here PatientsForce can play a role beyond helping patients find a program: as a medical service collaboration platform connecting pharma companies, hospitals, physicians, nurses, case managers and families, so that patient support programs (PSP), drug payment, care assistance, therapy tracking and administration are no longer fragmented. The value lies in reconnecting the broken segments of the journey — diagnosis, treatment initiation, resource application, continuous follow-up — lowering the administrative burden on patients and providers while raising accessibility and adherence.

Taiwan's next step

Taiwan passed the Rare Disease and Orphan Drug Act in 2000, the first country in Asia to legislate rare-disease rights. Yet multiple analyses point out that since the second-generation NHI, the average wait from application to reimbursement listing for new rare-disease drugs has stretched from about 5 months to about 30, and the reimbursement rate has clearly declined. Taiwan has tried accelerated review, parallel submission, the dual regenerative-medicine acts and installment payment — but between 'the drug exists' and 'patients can actually get it, afford it and stay on it', a more complete bridging system is still missing.

In this gap, PatientsForce should be more than an administrative executor — it can be the medical service partner of the rare-disease care system: designing case pathways, initiating treatment, introducing patient support programs, connecting cross-hospital resources and sustaining follow-up, so that someone truly catches patient needs outside the system.

When we talk about EAP, PSP, RWE or innovative payment, we appear to be discussing institutional tools. For rare-disease families, what matters more is whether, through the long wait, there is a service system that truly links medicine, resources and care. That is the piece rare-disease care most needs to fill next.