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People who have Refsum Disease have a rare genetic disorder that stores too much phytanic acid in the body and hurts the nerves. If you have it, you may lose your hearing or sight, have trouble keeping your balance, or have heart problems. What changes the market for Refsum Disease is how rare it is, how few people have it, and how treatments for rare illnesses change over time.
The small number of people who have Refsum Disease is an important fact about the market. RD is very uncommon, so not many people have been diagnosed with it. Drug companies have a hard time because they can't use economies of scale or get a good return on the money they spend on making medicines. The fact that a drug is an orphan drug may encourage drug companies to work on possible treatments for RD, but the small size of the market is still a big worry.
As of January 2022, when the most up-to-date information was gathered, there aren't many known ways to treat Refsum Disease. RD patients don't have all of their medical needs met right now, so drug companies can look into and find new ways to treat them. Research and development that is going on all the time to find cures for Refsum Disease and make people's lives better has an effect on how the market works.
The label "orphan drug" is very important in shaping the market because it is given to possible medicines for Refsum Disease. With this label, drug companies can get tax breaks and exclusive access to a market. This makes them more likely to spend money on research and development for rare diseases. That there aren't many people with Refsum Disease doesn't stop people from making new treatments. This is because orphan drugs are backed by policymakers.
New treatments and ongoing research projects are always making things different for people with Refsum Disease. It is their goal to find new ways to treat RD as they learn more about its genetic and chemical reasons. How well studies in animals and people are going has an effect on how the market works. Refsum Disease might be handled in a different way if new, cutting-edge drugs come out.
The market for Refsum Disease is also changed when people around the world work together and speak up for patients. Firms that make medicines, schools that teach them, and political groups all need to work together to move research forward and make sure that people can get drugs quickly. To get people to know about Refsum Disease, change government choices, and make it easier for medicines to be developed, patient lobbying is very important.
The market factors are more difficult because it is hard to find Refsum Disease and make sure people can get the right medical care. Doctors and nurses may not know enough about RD to find cases quickly or correctly because they don't know enough about it. The price and ease of getting medicines can also affect access to them. This shows how important it is to have full healthcare laws and support systems.
Prices, how people pay, and the size of the market are some of the things that can make or break the business of treating Refsum Disease. Pharmaceutical companies still need to decide if it's worth their time to make and sell treatments for RD, even though not many people have it. The market will change as possible treatments go through animal tests and the legislative process.
Refsum Disease Market Highlights:
Refsum Disease Market Overview
The Refsum Disease Market Size was valued at USD 0.74 Billion in 2023. The Global Refsum Disease industry is projected to grow from USD 0.69 Billion in 2024 to USD 2.48 Billion by 2032, exhibiting a compound annual growth rate (CAGR) of 6.03% during the forecast period (2024 - 2032). Refsum disease is a genetic disorder which is inherited as an autosomal recessive trait.
The condition is characterized by progressive loss of vision (retinitis pigmentosa), failure of muscle coordination (ataxia), degenerative nerve disease (peripheral neuropathy), and dry, rough, scaly skin (ichthyosis). The accumulation of fatty acid (phytanic acid) in blood plasma and tissues is the main cause of refsum disease. This occurs due to malfunction of the enzyme producing gene that metabolizes phytanic acid. According to the U.S. National Library of Medicine, more than 90 % cases of refsum disease result from the mutations in PHYH gene, and the remaining cases are caused by mutations in a gene PEX7 gene.
Notably, increasing awareness among people is the key factor driving the refsum disease market. According to the National Organization of Rare Diseases, the age of onset of refsum disorder varies greatly. The disease can occur at any time from early childhood to nearly 50 years of age, but in most cases will have appeared by age 20. Males and females are equally affected in numbers.
Various other push factors such as increasing awareness among people, increasing government assistance, improving regulatory framework, and rising funding and reimbursement continuously contributing to the growth of the refsum disease market.
Despite these drivers, there are some issues associated with refsum disease market. Some of the challenges in research and development, and poor healthcare system in low and middle-income countries may hinder the growth of the market to some extent.
Intended Audience
- Pharmaceutical Companies
- Hospitals & Clinics
- Research and Development (R&D) Companies
- Diagnostic Laboratories
- Government Research Institute
- Academic Institutes and Universities
Refsum Disease Market Segmentations
The refsum disease market is segmented on the basis of type, diagnosis, treatment, and end-user.
On the basis of the type, the market is segmented into adult refsum disease, and others. The adult refsum disease is further segmented into adult refsum disease 1, and adult refsum disease 2.
On the basis of the diagnosis, the market is classified into physical examination, molecular genetic testing, prenatal screening tests, preimplantation genetic diagnosis, enzyme analysis, anosmia testing, and others. The molecular genetic testing is further segmented into genotyping, sequencing of gene, amplification or hybridization method, methylation-specific techniques, and others. The prenatal screening tests are further segmented into ultrasound, blood tests, chorionic villus sampling (CVS), amniocentesis, and others.
On the basis of the treatment, the market is classified as medical care, and surgical care. The medical care is further segmented into diet, plasmapheresis, and medication. The sub-segments of medication include keratolytics, and others.
On the basis of the end-user, the market is segmented into hospital, clinics, diagnostic centers, research & academic institutes, and others.
Refsum Disease Market Regional Analysis
The Americas dominate the refsum disease market owing to the rising awareness among people and high healthcare expenditure. According to the Centers for Disease Control and Prevention in 2015, the total health expenditure in the United States was reported to be USD 3.2 trillion and hospital care accounted for a share of 32.3%.
Europe holds the second position in the refsum disease market. It is expected that the support provided by the government bodies for research & development and improvement in reimbursement policies in healthcare is likely to drive the market of the European region.
The Asia Pacific is the fastest growing refsum disease market owing to a huge patient pool and developing healthcare technology. Healthcare expenditure is also improving in various Asia Pacific countries. According to the Australian Institute of Health and Welfare in the years 2015-2016, the total health expenditure was USD 170.4 billion, which is 3.6% higher than the expenditure of 2014-2015.
The Middle East & Africa holds the lowest market due to lack of technical knowledge and poor medical facilities.
Refsum Disease Market Key Players
Some of key the players in the refsum disease market are:
- Fresenius Kabi
- B. Braun Medical Inc.
- Medline Industries, Inc.
- Illumina, Inc.
- Igenomix
- Ceuta Healthcare Limited
- Cochlear Ltd.
- Cook
- Sonova
- William Demant Holding A/S
- Nurotron Biotechnology Co. Ltd.
- Agilent Technologies
- Bio-Rad Laboratories, Inc.
- Sequenom
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