Region in short:

• 17 individual countries with very heterogeneous healthcare systems.
• Mix of low-, middle-, and high-income countries, almost all of which offer a socialized healthcare system.
• Proportion of public spending compared to out-of-pocket spending can vary widely.

Rare diseases segment:

• A growing segment in the MENAT region, driven by genetic rare diseases, which are many times higher than in the West due to consanguinity.
• Funding for rare diseases and orphan medicines is generally stronger in the Gulf region, but North Africa, led by Algeria, and now Egypt with its SMA launch, form the next wave.
• Oncology is small comparatively to the regions' size due to its young population, but is still a very well-funded segment.
• Named Patient Programs, especially for rare and specialized products, exist primarily in the wealthier countries and are an attractive entry point on the path to full commercialization.

Region in short:

• The CEE region covers Central Europe, the Baltics, Eastern Europe, and South Eastern Europe.
• The focus is on nine countries: Bulgaria, Croatia, Czech Republic, Hungary, Lithuania, Poland, Romania, Slovakia, and Slovenia. These countries have some of the most developed healthcare systems in Europe.
• Poland was by far the leading pharmaceutical market in CEE in 2019, with sales of over €7.2 billion ($8 billion). Romania ranked second with pharmaceutical market sales of €3.1 billion ($3.5 billion) this year. In addition, the Czech Republic was around €3 billion ($3.3 billion).

Rare diseases segment:

• Reimbursement decisions are mostly made by the national health policy authorities.
• Access to reimbursed orphan drugs varies widely.
• The type of approval plays an important role in many CEE countries. In the Czech Republic, Lithuania and Slovakia, reimbursement status is significantly associated with the type of marketing authorization, whereas in Croatia, Estonia, Hungary and Lithuania, the type of disease is significantly associated with reimbursement status.
• The proportion of orphan drugs reimbursed is not significantly associated with GDP or GDP per capita.
• Croatia, Hungary, Lithuania, Romania and Slovakia have specific laws/policies for orphan drugs.

Region in short:

• A developing market, complicated and diverse in terms of regulatory, reimbursement, market, demographics and politics.
• Struggling to align its public spending.

Rare diseases segment:

• The region's health systems struggle to provide funds for complex, high-cost treatments.
• Brazil has pioneered in the development of specific regulations for rare diseases and gene therapies. However, these advances have had unintended consequences for the other institutions involved in the incorporation process, such as the increase in requests for inclusion of therapies in the public system.
• All Latin American countries have enshrined 'health for all' in their constitutions. This obliges private and public health services to procure prescribed treatment from abroad when it is not available locally.

Region in short:

• The Eastern Mediterranean markets of Cyprus, Greece and Malta are generally combined.
• All three markets are European Union countries, part of the EMA.
• Greece is by far the largest market by population and market size, accounting for over 90% of the potential.

Rare diseases segment:

• All three markets reimburse rare disease products.
• Greece, via the IFET, allows for procurement of unregistered and withdrawn products directly from manufacturers.
• Cyprus and Malta reimburse specialty therapies through main reimbursement systems.

Region in short:

• Australia is a government-reimbursed market although achieving reimbursement is extremely challenging and costly 

• SEA and NZ are predominantly private markets with minimal Government subsidy

• Core markets in SEA for high-cost medicines include Singapore, Malaysia, Brunei, Thailand and Vietnam

• In SEA high-cost medicines are largely inaccessible due to socio-economic factors

• Named Patient Access Programs are permitted across ST’s regions without any regulatory approvals, to enable early access to specialist therapies for life threatening conditions

Rare diseases segment:

• In Australia, Orphan Drug Designation may be granted for therapies to treat life-threatening or debilitating conditions affecting fewer than five in 10,000 people. The key advantage of an orphan designation is that regulatory submission fees are waived, and some reimbursement submission fees are waived for the first application. Orphan designation does not provide any market exclusivity benefits 

• In New Zealand, there is provision for Government funding of medicines via Pharmac, however it is widely established that opportunities are limited. In addition, some NZ private health insurers fund some specialty medicines for eligible members 

• In Singapore and other SEA countries, high-cost medicines are out of reach for most patients unless they are privately insured. Insured patients may receive medicines on a subsidized basis via Government or private insurance schemes.

Region in short:

Both Japan and Korea have universal healthcare systems funded by social / national health insurance, government subsidies, and patient co-pays.

The role of private health insurance is limited in Japan, but has a larger role in South Korea where patient co-pays are higher.

Rare disease segment:

Orphan Drug Designation is available in both Japan and South Korea and enables accelerated regulator review and premium pricing.

Local ethno-bridging and pivotal trials are often still required in Japan, although there is increasing flexibility extended to orphan drugs. In contrast, South Korea will often wave local trial requirements for orphan drugs.

Pricing and reimbursement is relatively generous in both Japan and South Korea.

In Japan, almost all new drugs receive reimbursement, and drug prices trend towards the EU4+ UK average. Orphan drugs can sometimes command pricing close to US levels.

In South Korea, reimbursement rates are high for orphan drugs – although not all new drugs are reimbursed – and prices are generally set at the lowest of a basket of reference countries (US, EU4+UK, Japan, Switzerland).

Named Patient Access Programs are not in use in Japan, although are in South Korea in serious or urgent cases.