The Asia-Pacific (APAC) region, home to over 60% of the world's population, holds immense potential for rare disease drugs, with an estimated 258 million people.1 The global rare disease market is expected to reach ~$370 billion by 2030,2 with North America capturing ~54% share. In the APAC region, it is far more difficult to launch rare disease treatments than it is to launch blockbuster drugs for common illnesses. The region's varying definitions of rare diseases, market access pathways, and funding sources (Figure 1) necessitate meticulous prelaunch planning.
Figure 1: Assessment of rare disease market maturity in APAC region, focusing on specific markets
Figure 2: Key challenges to launching rare disease treatments in APAC region
A good case study in this connection is an innovative thalassemia treatment that gained enormous success in the US market (over $800 million in sales and 50%+ annual growth) but struggled in most APAC countries.3 It failed to attain double-digit patient numbers despite a successful launch. This situation underscores a critical challenge: substantial disparities in rare disease identification and drug consumption across different regions. Addressing this challenge requires meticulous planning that factors in the maturity of healthcare systems, intricate regulatory landscapes, and limited funding mechanisms specific to rare diseases.
For multinational pharmaceutical companies venturing into the APAC rare disease market, success hinges on a well-defined prelaunch strategy. The following are three key pillars to build upon:
Multinational pharmaceutical companies must invest in in-depth feasibility studies to understand the specific needs of each target market. This includes:
Achieving patient access and affordability for treatment is a crucial component of successful drug launches. Here is how multinational pharmaceutical companies can achieve this goal:
Limited public resources often pose a significant challenge to the treatment of rare diseases in the APAC region. Here are a few potential strategies for navigating funding constraints:
A successful launch is just the first step. Multinational pharmaceutical companies must also focus on building long-term sustainability in the APAC rare disease market. To achieve this, consider the following strategic actions:
1. Asia Pathways - ADBI. (2020, July). Rare Diseases in Asia-Pacific Must Be Tackled Too.
2. Fortune Business Insights. (n.d.). Orphan Drugs Market Analysis, Insights and Forecast, 2020-2027.
3. IQVIA MIDAS data and past project experiences with leading rare disease pharmaceutical companies in the year 2023