Rare Disease Treatment Market Size & Share Analysis - Growth Trends & Forecasts (2024 - 2029)

Rare Disease Treatment Market Analysis

The Rare Disease Treatment Market size is estimated at USD 216.65 billion in 2024, and is expected to reach USD 380.62 billion by 2029, growing at a CAGR of 11.93% during the forecast period (2024-2029).

The COVID-19 pandemic had a profound impact on the rare disease treatment market owing to the cancellations of elective procedures, including treatment and diagnostic procedures, worldwide. For instance, a review article published in the Frontiers in Public Health Journal in April 2021 reported the challenges faced by patients affected by rare diseases. The article concluded that regular clinical services, counseling, and therapies were extremely inadequate. Additionally, clinicians, researchers, and scientists working on rare genetic diseases faced extra challenges due to the pandemic since research projects and clinical trials for rare and genetic diseases were suspended during the COVID-19 pandemic. However, the market has been recovering following the lifting of restrictions. The market is expected to show a stable growth rate during the forecast period.

In addition, the increase in the number of rare disease cases and initiatives favoring vaccinations, rising R&D activities for novel therapeutics and drugs, and an increase in the number of new drug launches and favorable government policies are actively affecting the growth of the market studied.

The definition of rare diseases has distinctions in different parts of the world. The United States Food and Drug Administration defines a rare disease as a disease or condition that affects less than 200,000 people in the United States. The European Commission Public Health states that a rare disease affects no more than one person in 2000 in the European Union. According to Health Canada, the rare disease affects less than five persons in 10,000 in Canada. According to an article published in March 2021, over 6,000 different rare diseases are thought to exist, and more than 70% of rare diseases are hereditary, and the majority of them have a chronic course. According to the February 2022 update of rarediseaseday.org, about 300 million people around the world are living with rare diseases, which is quite high. Thus, the demand for effective and advanced treatment solutions for rare diseases is expected to increase due to the high burden and growing awareness about these diseases. This is expected to fuel growth in the market studied over the forecast period.

Various governments around the world have been formulating several initiatives for the treatment of rare diseases. For instance, the Indian government's Ministry of Health and Family Welfare's National Policy for Rare Diseases 2021 aimed at lowering the incidence and prevalence of rare diseases based on an integrated and comprehensive preventive strategy. This encompasses awareness generation, premarital, post-marital, pre-conception, and post-conception screening and counseling programs to prevent births of children with rare diseases, and within the constraints on resources and competing health care priorities, enable access to affordable health care to patients of rare diseases who are amenable to one-time treatment or relatively low-cost therapy.

Additionally, in May 2022, the Center for Drug Evaluation and Research (CDER) of the US FDA launched a new program for expediting the development of new treatment options for rare diseases, the Accelerating Rare disease Cures (ARC) Program. Such developments are expected to propel the market's growth during the forecast period.

Additionally, the innovation in treatment and increasing product approvals, along with partnerships and acquisitions by key players are helping in the market's growth. For instance, in May 2022, the European Medicines Agency (EMA) approved xenpozyme (olipudasealfa), a therapy for the treatment of non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), a rare and progressive genetic disease. EMA specified that xenpozyme was the first ASMD-specific treatment in the European Union developed to replace patients' deficient or defective enzyme, acid sphingomyelinase (ASM), and thereby reduce fat accumulation within cells and relieve some of the symptoms of the disease. Such approvals are expected to augment the market's growth during the forecast period.

The increase in the necessity of rare disease treatment worldwide is expected to propel the growth of the rare disease treatment market. However, a lack of awareness regarding rare disease treatment and the high cost of treatment is expected to hinder the market's growth during the forecast period.

Rare Disease Treatment Market Trends

This section covers the major market trends shaping the Rare Disease Treatment Market according to our research experts:

The Biologics Segment is Expected to Hold a Major Market Share in the Rare Disease Treatment Market

Rare Disease Treatment Industry Segmentation

As per the scope of this report, rare diseases are diseases that affect only a small population compared to the general population. Rare diseases are region specific in that a rare disease could be rare in one region while it is common in another. Rare diseases may be chronic or incurable, though many short-term medical conditions are also rare diseases.

The rare disease treatment market is segmented by drug type (biologics and non-biologics), therapeutic area (genetic diseases, neurological diseases, oncology, infectious diseases, cardiovascular diseases, and other therapeutic areas), mode of administration (oral, injection, and other modes of administration) and geography (North America, Europe, Asia-Pacific, Middle East and Africa, and South America). The report also covers the estimated market sizes and trends of 17 countries across major regions globally. The report offers values (in USD million) for the above segments.