Rare Disease Clinical Trials Market Size, Share & Trends Analysis Report By Therapeutic Area, By Phase, By Sponsor, By Region, And Segment Forecasts, 2023 To 2030

Industry Insights

The global market for rare disease clinical trials is anticipated to register significant growth over the forecast period. The main driving factors of the rare disease clinical trial market are the increasing prevalence of rare diseases, which has led to rising research, intermittent launches, and a large number of products in the pipeline. Also, government initiatives that encourage product development, such as the Orphan Drug Act, are anticipated to assist industry growth.

The COVID-19 pandemic has greatly impacted our ability to conduct clinical trials. Due to the pandemic, various clinical trials of rare diseases were halted and delayed, and participant enrollment was postponed. This resulted in the delayed arrival of new treatments and drugs developed for the treatment of rare diseases. However, clinical trials for rare diseases resumed by the second half of 2020, and effective treatment and drugs for them were initiated.

Genetic disorders dominated the market in terms of therapeutic areas. This is due to the large number of rare diseases that are hereditary or arise due to genetic mutations. Genetic disorders encompass a wide range of diseases, including spinal muscular atrophy and cystic fibrosis.

The North America region is expected to dominate the market over the forecast period due to the presence of major market players offering rare disease clinical trials, and the increasing prevalence of disease, which will increase the demand for drugs that will in turn increase clinical trials for drug development, and the R&D expenditures made by market players for the creation of novel therapeutics for rare diseases. According to the National Institutes of Health (NIH), there are more than 7,000 rare diseases, and they affect nearly 30 million Americans, or 1 in every 10 people.

The key market players include Takeda Pharmaceutical Company, F. Hoffmann-La Roche Ltd., Pfizer, Inc., AstraZeneca, Novartis AG, LabCorp Drug Development, IQVIA, Charles River Laboratories Inc., Icon PLC, and Parexel International Corporation. They are adopting various strategies like partnerships, collaboration, merger and acquisition, and expansion. For instance, In January 2022, Pfizer announced a collaboration with Beam Therapeutics Inc. to research in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle, and central nervous system.