Rare Genetic Disorders Treatment Market: Current Analysis and Forecast (2022-2028)

Rare genetic disorders, also known as orphan diseases, are defined as conditions that affect fewer than 200,000 people in the United States or fewer than 5 in 10,000 people in the European Union. While these diseases are individually rare, collectively they affect millions of people worldwide. The orphan drug market has been driven by government incentives, such as expedited regulatory review and approval processes, tax credits, and extended periods of market exclusivity.

The Rare Genetic Disorders Treatment Market is expected to grow at a strong CAGR of 11.9% during the forecast period owing to the rise in the incidences of rare genetic diseases. Globally the incidence of rare genetic diseases is increasing tremendously. According to the Genetic and Rare Diseases (GARD) Information Center in May 2022, 1 in 10 Americans (or 30 million people) have a rare disease, and there are approximately 7,000 known rare diseases. Moreover, as per data updated in September 2021 by the "GlobalGenes", more than 400 million people worldwide are impacted by rare diseases. 80% of rare diseases have been identified with genetic origins.

Based on application, the market is segmented into cancer, neurological disorders, cardiovascular diseases, metabolic disorders, hematology diseases, and others. The cancer segment held a dominant share of the market in 2021 mainly due to the surge in the incidences of rare genetic cancers and advances in genomic sequencing technology for their early diagnosis. In addition to this, several pharmaceutical players are actively investing in research and development to identify new drug candidates, thus having a positive impact on the segment growth in the coming years. For instance, in November 2022, Unravel Biosciences entered into a collaboration with Spanios to discover therapeutic targets for rare cancer. Under the partnership, Unravel Biosciences will leverage its artificial intelligence (AI) gene network-based computational model for predicting the therapeutic compounds and underlying new targets depending on the transcriptomic information.

By drug type, the market is bifurcated into biologics & biosimilars, and non-biologics. The biologics & biosimilar segment held a dominant share of the market in 2021 owing to their high specificity towards the disease. Biologics & biosimilars drugs are increasingly being used for the treatment of rare genetic disorders, including hematology diseases. Biologics are drugs that are derived from living cells, and include proteins, antibodies, and other complex molecules. Biosimilars are similar to biologics, but are developed after the patent for the original biologic drug has expired. Several biologics have beend approved for the management of rare genetic disorders including Rituximab, Adalimumab, zynteglo, and others. Apart from this, rising product pipelines and their encouraging response in clinical trials is also suggesting positive future growth of this segment. For instance, in 2020, Regenreon Pharmaceuticals announced encouraging results from LUMINA-1, a 44-patient, Phase 2, double-blind placebo-controlled trial evaluating garetosmab (REGN2477) in patients with fibrodysplasia ossificans progressiva (FOP).

On the basis of distribution channel, the market is segmented into hospital pharmacy, online pharmacy, and retail pharmacy. The online pharmacy is expected to witness high CAGR during the forecast period. Online pharmacies are also gaining considerable momentum in the market owing to their convenience. Consumers can order medications from the comfort of their own homes and have them delivered directly to their doorsteps. This is especially beneficial for those who live in rural areas or have mobility issues, as it eliminates the need for them to travel to a physical pharmacy. Moreover, online pharmacies often offer lower prices than traditional brick-and-mortar pharmacies due to lower overhead costs. In addition, online pharmacies may offer discounts, promotions, and loyalty programs to attract and retain customers. According to a survey conducted by the National Community Pharmacists Association, 61% of respondents said that cost was the primary reason they chose to use an online pharmacy.

For a better understanding of the market adoption of the rare genetic disorders treatment industry, the market is analyzed based on its worldwide presence in the countries such as North America (U.S., Canada, Rest of North America), Europe (Germany, U.K., France, Spain, Italy, Rest of Europe), Asia-Pacific (China, Japan, India, Rest of Asia-Pacific), Rest of World. The Asia Pacific market is expected to register highest growth rate in the forecast period due to an increase in awareness regarding rare diseases, rising healthcare expenditure, and favorable government policies. Moreover, the surge in the incidences of rare diseases in the region and the availability of therapies is also driving the growth of the market. For instance, According to the World Economic Forum, around 200 million people are living with rare diseases in Asia Pacific.

Some of the major players operating in the market include: F. Hoffmann-La Roche Ltd.; Pfizer, Inc.; AstraZeneca; Bristol Myers Squibb; Sanofi; Novartis AG; AbbVie Inc.; GlaxoSmithKline Plc; Amgen Inc.; and Johnson & Johnson Inc.