Visiongain has published a new report entitled Rare Diseases Market Report 2023-2033: Forecasts by Diseases (Cancer, Metabolic Diseases, Neurological Conditions, Haematological Disorders, Infectious Diseases, Musculoskeletal Disorders, Cardiovascular Disorders, Others), by Type (Non-biologics, Biologics), by End-users (Specialty Pharmacies, Hospital Pharmacies, Retail Pharmacies), by Age (Adult, Paediatric) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies AND COVID-19 Impact and Recovery Pattern Analysis.

The rare diseases market was valued at US$188.58 billion in 2022 and is projected to grow at a CAGR of 12.27% during the forecast period 2023-2033.

In the modern world, science and technology offer an unprecedented chance to address to meet the unmet medical requirements of people who have a rare disease. Due to problems with accessibility, affordability, and availability of treatments, this potential is currently not translated into actual health benefits for the vast majority of people living with a rare disease. Coordinated, more strategic policies are needed to address these shortfalls and inequalities and ultimately improve the health outcomes and quality of life of people living with a rare disease.

How has COVID-19 had a Significant Impact on the Rare Diseases Market?
The COVID-19 pandemic in particular has brought many of the challenges faced by the rare disease community into sharp relief. It is vital that the government continues to support those living with and caring for those with rare diseases, many of whom are especially vulnerable to coronavirus through the current and any future pandemics. There will be opportunities to learn from the COVID-19 pandemic and the experiences of rare diseases patients and to reflect on this learning in shaping the broad commitments of this new framework and wider national responses to infectious disease outbreaks. Improving government efforts to increase awareness of rare diseases, along with the presence of advanced healthcare infrastructure in developed and emerging countries, are major drivers of rare disease growth in the rare diseases industry. In addition, advantageous payment policies in developing economies and advantageous medication clearance laws for the treatment of rare diseases are other important factors that are likely to accelerate growth over the projected period.

How will this Report Benefit you?
Visiongain’s 290-page report provides 107 tables and 186 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the rare diseases market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Rare Diseases. Get financial analysis of the overall market and different segments including diseases, type, end-users, age, and capture higher market share. We believe that there are strong opportunities in this fast-growing rare diseases market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.

What are the Current Market Drivers?

Gene Therapies Are a Promising Path to Treat Rare Diseases
Overall, the increasing prevalence of rare diseases, the potential of gene therapy to provide a one-time cure, and the lack of available therapies for many rare disorders are driving the demand for gene therapy in the treatment of rare diseases. While current gene therapy treatments may come at a high cost, advancements in the field are expected to lead to more affordable and accessible options in the future. Collaborative efforts between pharmaceutical companies and regulatory agencies are also expected to accelerate the development and approval of gene therapies for rare diseases.

Rare diseases affect a significant portion of the global population, with approximately 400 million people suffering from one or more of the over 6,000 rare diseases reported. These disorders can be caused by hereditary factors, pathogens, environmental conditions, or be degenerative or proliferative in nature. About 72% of rare diseases have a hereditary origin, and 70% of genetic rare diseases begin in childhood. Unfortunately, symptoms of rare diseases are often misdiagnosed and mistreated, leading to treatment delays and a decline in the patient's quality of life. However, gene therapy has emerged as a promising approach to treating rare diseases. The rising number of rare disease patients around the world is anticipated to drive the gene therapy application in this segment during the forecast period. Zolgensma is currently the costliest medication in the world, which is used to treat spinal muscular atrophy, a rare hereditary disease that affects nerve cells and causes muscle degeneration. The medication's one-time treatment for a young child costs $2.1 million. Despite the high cost of Zolgensma, several cell and gene therapies are expected to be available over the next ten years, with prices ranging from hundreds of thousands to millions of dollars for a single dose.

However, only a few hundred rare diseases currently have FDA-approved therapies, even though over 7,000 rare diseases are known to exist. Gene therapy is especially important for patients with rare disorders, as more than 80% of them have a documented monogenic (single-gene) cause. Unlike conventional small molecule medications that work by reducing symptoms, gene therapy has the potential to remedy structural genetic disorders, rather than merely treating symptoms.

Increasing Awareness to Drive Industry Growth
The media plays an increasingly important role in spreading awareness about the rare diseases and availability of therapeutics for such diseases. In conjunction with this, the development of low-income countries can lead more people being educated about the rare disease healthcare treatments. The increasing levels of awareness have promoted the benefits of treating rare diseases inducing cancer and genetic disorders, thus leading to the demand of novel drugs among the physicians.

Awareness campaigns by patient support groups play an important role in helping the public understand about the symptoms and challenges faced. In this regard, over that last 10 years there has been a noticeable increase in the efforts by the patient support groups. Persons living with genetic disorders have also become much more willing to share their stories on social media platforms. This is an important shift in how information about rare diseases is shared with the public as the patients themselves are the curators of content, and do not need to rely on media networks as they would have to do previously. By doing this, the public have gained much more meaningful insight into the daily challenges they face.

Where are the Market Opportunities?

Increasing Demand from Emerging Economies to Fuel Industry Expansion
As emerging economies continue to develop and grow, they will have more resources to ensure the secure supply of pharmaceutical drugs, which will increase the supply of rare diseases drugs within the country. This, in turn, will drive demand for these drugs, as the population in these countries grows, and healthcare expectations and standards of living increase. All of these factors will contribute to the expansion of the rare diseases drugs market.

Increasing Government Support to Influence Market Expansion
The government plays a pivotal role when it comes to the healthcare of the public. This is because government spending often influences how much money the health sector gets. If the government cuts the amount of funding to the health sector, then health providers will find it increasingly difficult to operate under such financial restraints. This will lead to a decrease in the number of rare diseases drugs that a healthcare establishment will be able to provide. On the other hand, a government also plays an active role funding the research into treating various mental disorders. This gives a large boost to the R&D efforts of pharmaceutical companies. It allows them to accelerate their trials or development and also develop rare diseases drugs that would have been too costly otherwise. Becoming the recommended drug (using it as the first line of treatment) is also a huge incentive for the pharmaceutical companies. This will allow them to engage with a larger potential market and also ensures that the country has adequate stock of drugs.

Competitive Landscape
The major players operating in the rare diseases market are AbbVie Inc., Alexion Pharmaceuticals, Inc. (AstraZeneca), Amgen Inc., Biogen Inc., Bristol-Myers Squibb Company, F. Hoffmann-La Roche Ltd., Johnson & Johnson Services, Inc., Merck & Co Inc., Novartis AG, Pfizer Inc., Sanofi S.A., Takeda Pharmaceuticals, and Teva Pharmaceuticals. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launch.

Recent Developments
• In February 2023, Janssen Pharmaceutical Companies of Johnson & Johnson announced positive topline results from the proof-of-concept Phase 2 open-label UNITY clinical trial for the treatment of pregnant adults at high risk for severe hemolytic disease of the fetus and new-born (HDFN). HDFN is a serious and rare condition which can cause life-threatening anaemia in the fetus.
• In November 2022, Alexion, AstraZeneca Rare Disease, completed the acquisition of LogicBio® Therapeutics, Inc. (NASDAQ: LOGC), a pioneering genomic medicine company. The acquisition creates an opportunity to accelerate Alexion’s growth in genomic medicines through unique technology, an experienced rare disease R&D team, and expertise in pre-clinical development.

Notes for Editors
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