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 Israeli market is expected to grow steadily, with a high penetration rate of innovative medicines.
• Key drivers are a strong network of academic and research institutes, R&D facilities and developed medical facilities.
• Israel spends 7.5% of its GDP on the public healthcare system, providing universal healthcare coverage to its entire population via four HMOs and a network of hospitals, community clinics and specialized doctors.
• Israel’s healthcare policy makers have been focusing on promoting healthy nutrition and lifestyle, preventive medicine and age-based screening standards.

Rare diseases segment:

• Reimbursement is done once a year through a national health basket committee that evaluates all new pharmaceuticals and technologies submitted.
• Focus on scientific research and a community-based healthcare distribution system has made Israel an ideal candidate for many research projects in rare diseases.
• No orphan drug designation in registration or in reimbursement, but there have been attempts for some years to change the status.
• For many rare diseases there are patient associations which push for acknowledgment, access of treatments and reimbursement. Some are also supported by specific HCPs.

Region in short:

• The African subcontinent, outside of North Africa, is rapidly evolving from its infancy compared to the rest of the global pharmaceutical market.
• Healthcare systems are highly fragmented, with very little public procurement outside of a few wealthy nations.
• South Africa and Nigeria lead the region in terms of overall pharmaceutical market size, but healthcare spending on orphan medicines is very limited across the continent.

Rare disease segment:

• Named Patient Programs are available across the continent. However, funding remains a limiting factor for their widespread implementation.
• South Africa's Section 21 provides a good pathway for orphan drugs, but procurement is expected to come from a private insurer.
• OOP and NGOs are likely to be the financiers of orphan medicines for the foreseeable future, with economies gradually expanding to cover specialty medicines.

Region in short:

• The Canadian market is growing steadily, driven by an aging population, the rising prevalence of chronic diseases, and the adoption of innovative therapies.
• Underpinned by a robust regulatory, health technology assessment and pricing regulation framework.
• Canadian healthcare system is defined by public and private sector involvement, leading to broader access to treatments and support for innovation, but also resulting in delayed access to healthcare systems and innovative therapies.
• Healthcare is a provincial responsibility, operating within a universal, publicly funded system called Medicare.

Rare disease segment:

• Canada currently has a number of organizations to support advocacy, education,
  research and access to medicines:
  - The Canadian Organization for Rare Disorders
  - Canadian Rare Disease Network
  - Quebec association for rare disease (www.rqmo.org and www.rare.quebec)  
• Governmental strategies: National Strategy for Drugs for Rare Diseases
• Health Canada’s Special Access Program (SAP) enables priority drug pathways and reviews.
• Patient advocacy plays a crucial role in shaping Canadian healthcare policies,
  improving access to care, and ensuring the voices of patients and caregivers
  are included in decision-making processes.