Report Description of the APAC Orphan Drugs Market Analysis

Research Scope and Assumption

• The report provides the market value for the base year 2020 and a yearly forecast up to 2028 in terms of revenue (USD billion)


• The key industry dynamics, regulatory scenario, reimbursement scenario, major market trends, and drug markets are evaluated to understand their impacts on the demand for the forecast period. The growth rates were estimated using correlation, regression, and time-series analysis


• We have used the combination of top-down and bottom-up approach for market sizing, analyzing key regional markets, dynamics, and trends for various solutions, services, and end uses


• All market estimates and forecasts have been validated through primary interviews with the Key Industry Players (KIPs) and secondary analysis


• Inflation has not been accounted for in order to estimate and forecast the market


• Numbers may not add up due to rounding off

Reason to buy the report:

• Facilitate decision-making based on strong current and forecast data for APAC Orphan Drugs Market Analysis


• Develop strategies based on the latest regulatory framework


• Strategically analyze micro-markets with respect to individual growth trends, future prospects, and their contribution to the market


• Analyze competitive developments such as expansions, investments, mergers & acquisitions, new product developments, and research & developments in the APAC Orphan Drugs Market Analysis


• Analyze the opportunities in the market for stakeholders and draw a competitive landscape for market leaders


• To strategically profile key players and comprehensively analyze their market shares and core competencies


• We have technically sound team which do a deep dive research and also provide strategy based consulting analysis

APAC Orphan Drugs Market Analysis Executive Summary

Orphan drug can be defined as a pharmaceutical agent specifically designed to treat rare (orphaned) diseases. These diseases differ from usual diseases as their prevalence rate is very low and hence appeal to a very small patient population. Therefore, as compared to non-orphan drugs, these drugs do not guarantee feasible returns on investment. Cost associated with the development of these drugs is higher when compared with non-orphan drugs. The different indications for which orphan drugs are used include lymphoma, leukaemia, cystic fibrosis, etc.

Market Size and Key Findings

The APAC Orphan Drugs Market Analysis size stood at around USD 35.01 billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of 8.92% during the forecast period.

The Asia Pacific Orphan Drugs Market is projected to be worth USD 35.01 billion in 2021 and is estimated to grow at a CAGR of 8.92%, reaching USD xx billion by 2028.



Market Dynamics

Market Growth Drivers Analysis

A growing number of patients suffering from various rare diseases related to cancer, cardiovascular problems, and other rare diseases worldwide are outshining the growth rate of the orphan drugs market in the Asia Pacific. These are manufactured specifically with a new formula to treat rare diseases, and hence the demand for this market is overgrowing. Government organizations focused on improving the services in healthcare centers and producing quality drugs favoring the end users, the rise in the concern towards health care among every individual and their prevalence for early diagnosis is elevating the growth rate: of the APAC orphan drugs market.

Market Restraints

Stringent rules and regulations by the government over pharmaceutical companies in concern towards the public's safety, the manufacturing cost of orphan drugs is high compared to the standard drugs, and the lack of professionals in manufacturing drugs with the latest formula is the significant challenges to the market.

COVID-19 impact on APAC Orphan Drugs Market

The novel COVID-19 outburst had enabled certain pharmaceutical markets to be affected moderately due to the postponement of treatment and diagnosis of non-life-threatening diseases. This holds significantly true for individuals suffering from rare diseases as more healthcare and financial resources by various central governments were focused on the fight against the pandemic. This apparently brought down the sales of orphan drugs, negatively impacting the market share.

Competitive Landscape

Key Players

A few noteworthy companies operating in the APAC orphan drugs market profiled in this report are Novartis, GlaxoSmithKline, Roche, Alexion, Sanofi, Bristol Myers Squibb, Pfizer, Vertex, Celgene, and Merck.

Products in Pipeline

June 2020-Chiasma, Inc. announced that the company had received the FDA approval for their product offering of Mycapssa for the treatment of acromegaly, a rare disorder.

June 2020 - Agios Pharmaceuticals, Inc. announced that the company had received the FDA Orphan Drug Designation for their pipeline candidate of Mitapivat for the treatment of Thalassemia.

May 2020-AstraZeneca, and Daiichi Sankyo Company announced that the company had received the FDA Orphan Drug Designation for their product offering of Enhertu (trastuzumab deruxtecan) for the treatment of patients with gastric cancer, including gastroesophageal junction cancer.

Notable Recent Deals

• On May 22, 2018, the new NHC (National Health Commission), along with the CNDA (China National Drug Administration), and the three other agencies jointly published the first edition of the Rare Disease List (RDL). The RDL includes 121 diseases and is considered a huge milestone for regulation in rare diseases.

Healthcare Policies and Regulatory Landscape

Policy changes and Reimbursement scenario

In Asia, There is an acceleration in the development of legal frameworks in many countries which set forth general criteria to foster the registration and approvals of drugs for the treatment of rare conditions. The regulatory and legal framework of Orphan drugs in Asia is still recent and varies between countries, with some still lacking resources to provide systematic diagnostic or testing for rare conditions.

The Action Plan on Rare Diseases launched in late 2018 by the Asia-Pacific Economic Cooperation is expected to accelerate supportive actions to develop public engagement, disease registries, and patient access to clinical trials, and to reduce enrollment durations.

In 2018, the Asia-Pacific Economic Cooperation (APEC) launched an Action Plan on Rare Diseases, providing a framework for facilitating alignment of domestic policies, best practice systems and enhancing regional collaboration5. The main goal of the plan is to improve the economic and social inclusion of those affected by rare diseases, facilitate diagnostic and nation-wide patient registries, and raise public and political awareness on Orphan diseases issues.

As the Asian pharmaceutical markets grow, so too will opportunities for orphan drugs in Asia. Asian governments are becoming more aware of the importance of orphan drugs, and reimbursements for these products will increase in the future. Each Asian market, however, is different, and one must study each Asian countrys orphan drug regulations and markets to be successful.

Rare diseases need more attention due to lack of proper diagnosis and treatment. Treatment and prevention for rare diseases is considered as no mans land. The EU parliament should provide more benefits like tax incentive, special status, and reimbursement for these orphan drugs. This encouragement can bring in a revolution among the pharmaceutical and biotechnology companies for developing and marketing orphan drugs. EURODIS and other organizations are creating awareness on rare diseases and are also influencing governments in bringing legislation acts for better quality of life for these special people. EURORDIS and National Alliances have announced rare day on February 29th and dedicated this day to special people who are affected by rare diseases. Henceforth, 29th February will be called the rare disease day.