Global Policy Changes Affecting Rare Disease Access

The Rare360 Editorial Team
11 hours ago
4 min read

Around the world, governments are experimenting with new policy models, from expanded treatment programs to innovative reimbursement frameworks, to address one of the most persistent barriers in rare disease care: access to high-cost therapies.

Below are eight notable examples where governments or international bodies have recently implemented policies that affect access to rare disease diagnosis, treatment, or innovation. These developments span North America, Europe, and Asia, as well as global governance efforts, illustrating how different health systems are confronting the same structural challenge: affordability and equitable access.

Canada — National Strategy for Drugs for Rare Diseases

Canada launched a national rare disease drug strategy backed by up to $1.4 billion in federal funding over three years to improve access to treatments. The strategy supports provincial coverage of rare disease drugs, expanded screening and diagnostics, and improved coordination of treatment decisions across the country.

Impact

Expands drug coverage across provinces
Reduces disparities in access to therapies
Supports new diagnostic and data infrastructure

Why it matters

Canada’s strategy represents one of the most significant national investments in rare disease access in recent years. By coordinating federal and provincial efforts, the policy aims to reduce geographic inequities and ensure that patients across the country can benefit from new therapies.

United States — Multi-State Medicaid Effort to Cover Gene Therapies

Several U.S. states have joined a national initiative to expand Medicaid coverage for gene therapies for sickle cell disease. These therapies can cost more than $2 million per patient, presenting a major challenge for public health systems.

The federal-state program aims to make these therapies accessible through Medicaid, which covers a large portion of patients living with sickle cell disease.

Impact

Enables Medicaid programs to cover high-cost gene therapies
Links payment to treatment outcomes
Reduces financial risk for state health systems

Why it matters

Without public payer coverage, many breakthrough therapies remain theoretically available but practically inaccessible. New reimbursement models are increasingly necessary to ensure that transformative treatments reach patients who depend on public insurance programs.

India (Kerala) — Expanded SMA Treatment Coverage

The Indian state of Kerala expanded its CARE rare disease program to cover risdiplam treatment for spinal muscular atrophy (SMA) patients up to age 25, extending coverage beyond the previous pediatric limit.

Impact

Expands access for adolescents and young adults
Demonstrates regional government leadership in rare disease care
Acknowledges the lifelong nature of many genetic conditions

Why it matters

Rare diseases do not end in childhood. Expanding eligibility beyond pediatric patients reflects a growing recognition that sustainable rare disease policies must support individuals across their lifespan.

European Union — Reform of the Orphan Drug Regulation

The European Union is advancing reforms to its Orphan Drug and Pediatric Medicines Regulations as part of a broader overhaul of pharmaceutical legislation. The proposed reforms aim to rebalance incentives for drug development while improving patient access.

Proposals include:

Adjusting market exclusivity incentives
Strengthening evidence requirements
Encouraging development in underserved rare diseases

Impact

May reshape investment incentives in rare disease research and development
Could influence drug pricing and availability across EU member states

Why it matters

The EU orphan drug framework has been one of the most influential policy tools supporting rare disease innovation. Changes to this system could significantly affect how companies prioritize rare disease drug development and how quickly therapies reach patients across Europe.

United Kingdom — Innovative Medicines Fund

The United Kingdom expanded the Innovative Medicines Fund, a program designed to provide early access to promising therapies while additional clinical evidence is collected. Rare disease therapies are a key focus of the fund.

Impact

Accelerates access to therapies before full reimbursement decisions are made
Generates real-world evidence to support long-term coverage decisions

Why it matters

Rare disease treatments often reach the market with limited clinical trial data due to small patient populations. Managed access programs like the Innovative Medicines Fund help bridge the gap between regulatory approval and long-term reimbursement decisions.

Japan — Expanded Coverage for “Nanbyo” Rare Diseases

Japan continues to expand its Nanbyo (intractable disease) program, which provides government subsidies for patients living with designated rare diseases. The program now covers hundreds of conditions, helping offset treatment costs and providing access to specialized care.

Impact

Reduces financial burden on patients
Ensures nationwide access to specialized care

Why it matters

Japan’s approach demonstrates how long-term national programs can integrate rare disease support into broader health systems, providing both medical care and financial protection for patients and families.

Taiwan — National Rare Disease Subsidy Programs

Taiwan includes rare diseases within its National Health Insurance system, offering subsidies for treatments, medical care, and social support services.

Support programs also include:

Transportation assistance
Educational support
Tax deductions for families

Impact

Integrates medical and social care support
Reduces economic burden on families

Why it matters

Rare diseases often create financial challenges beyond medical costs. Taiwan’s approach illustrates how integrated support systems can address the broader social and economic realities faced by patients and caregivers.

World Health Organization — Global Rare Disease Resolution

In 2025, the World Health Assembly adopted a historic rare disease resolution, calling on countries to integrate rare diseases into national health planning and improve access to diagnosis and treatment.

The resolution also calls for the development of a 10-year global action plan focused on equity and access.

Impact

Elevates rare diseases as a global public health priority
Encourages countries to develop national rare disease strategies

Why it matters

For decades, rare diseases were largely absent from global health policy discussions. The WHO resolution signals growing international recognition that rare diseases are a public health issue requiring coordinated global action.

Key Trend Emerging Globally

Across these policy examples, governments are converging around three major strategies:

Innovative payment models for ultra-expensive gene therapies
National rare disease strategies to coordinate care and coverage
Global policy recognition of rare diseases as a public health priority

Together, these developments suggest that rare disease policy is moving beyond isolated initiatives toward system-level health policy reform worldwide.

Global Policy Changes Affecting Rare Disease Access

Canada — National Strategy for Drugs for Rare Diseases

United States — Multi-State Medicaid Effort to Cover Gene Therapies

India (Kerala) — Expanded SMA Treatment Coverage

European Union — Reform of the Orphan Drug Regulation

United Kingdom — Innovative Medicines Fund

Japan — Expanded Coverage for “Nanbyo” Rare Diseases

Taiwan — National Rare Disease Subsidy Programs

World Health Organization — Global Rare Disease Resolution

Key Trend Emerging Globally

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