Rare Disease Treatment Market Share

North America Market Analysis

As per the National Organization for Rare Disorders (NORD), more than 10,000 rare diseases together affect over 30 million Americans. Federal agencies including the NIH and FDA assign funding for rare and orphan disease R&D to support clinical trials in the region. In September 2024, Travere Therapeutics, Inc., received U.S. FDA approval for FILSPARI which aims to reduce the declination rate in kidney functioning among adults suffering from primary IgAN who are at risk of disease progression. In addition, U.S. reimbursement policies such as Medicare coverage and other coverage by private insurance companies, impact the commercial viability of these treatments positively.

The U.S. rare disease treatment market is growing majorly due to the presence of several prominent players. Owing to their presence, the country is witnessing a wide number of clinical trials, followed by medication and therapy launches as a positive outcome for the trials. In July 2022, the U.S. FDA approved Pfizer Inc.’s crizotinib for adult and pediatric patients of 1 year and above, suffering from ALK-positive IMT. These approvals from the regulatory bodies are further projected to lead to several product launches, further enhancing the rare disease treatment market growth in the upcoming years.

According to an article posted by the Government of Canada in March 2023, 1 out of 12 people in Canada suffer from a rare disease. It further states that the Minister of Health declared supportive measures of the first-ever National Strategy for Drugs for Rare Diseases. The government offered an investment of up to USD 1.5 billion over three years. This support from the government is projected to boost the market growth significantly in Canada.

APAC Market Statistics

APAC rare disease treatment market is witnessing a significant rise. As per a report dated July 2024, the Asia Pacific region is home to 60% of the global population. It has registered the highest number of people residing with a rare disease, with estimates indicating more than 258 million individuals. Noteworthy progress to advance access is underway across the APAC region, both in the higher-income and lower-income countries, and more technologically advanced countries such as Australia. In February 2020, the Australia government launched the country’s first National Strategic Action Plan for Rare Diseases. These factors are boosting the expansion of the market in several untouched areas in the region as well.

India rare disease treatment market is projected to witness notable growth owing to supporting policies from the local government for rare and orphan diseases. In March 2021, the Ministry of Health & Family Welfare announced the launch of the National Policy for Rare Diseases (NPRD). The policy includes 63 rare diseases on the reference of the Central Technical Committee for Rare Diseases (CTCRD). So far, 12 Centres of Excellence (CoEs) have been identified, including premier Government tertiary hospitals offering diagnostics, prevention, and treatment facilities for rare diseases. Per person monetary funding of up to Rs. 50 lakhs are offered for the treatment at the notified CoEs for Rare Diseases.

China rare disease treatment market is anticipated to garner a considerable share. According to the CEIBS, in February 2023, among the 7,000 worldwide confirmed rare diseases, over 1,400 cases have been diagnosed in China. NLM stated that in September 2023, the National Health Commission of China officially announced the Second List of Rare Diseases, comprising 86 rare diseases. The demand for therapies in the country is hence increasing. In November 2022, AstraZeneca introduced Soliris (eculizumab), through Alexion, for the treatment of PNH and aHUS in China for adults and children.