Global Rare Disease Drug Market Size By Therapy Area (Oncology, Hematology), By Drug Type (Biological, Non-biological), By Distribution Channel (Hospital Pharmacy, Retail Pharmacy), By Geographic Scope And Forecast

Rare Disease Drug Market Size And Forecast

Rare Disease Drug Market size is growing at a moderate pace with substantial growth rates over the last few years and is estimated that the market will grow significantly in the forecasted period i.e. 2022 to 2030.

The Rare Disease Drug Market is really taking off, mainly because there's way more focus on research and making new therapeutic drugs. Plus, all this product development means we're seeing cooler, tech-driven gadgets being used to figure out those tricky rare genetic disorders. And let's not forget, governments are stepping up with policies to get the word out about rare disease drugs, so more people are getting the treatments they need. Want the full scoop? The Global Rare Disease Drug Market report gives you a complete look at everything happening in the market. It breaks down all the important stuff – segments, trends, what's helping and hurting the market, who the big players are, and basically, anything that's making a difference.

Global Rare Disease Drug Market Definition

A rare disease is defined as a disease that affects only a small proportion of the population. In some parts of the world, an orphan disease is a rare disease that lacks a large enough market to gain support and resources for discovering treatments for it, unless the government grants economically advantageous conditions for developing and selling such treatments. Rare Disease drugs are those that have been created or sold for this purpose. Because most rare diseases are genetic, they are present throughout a person’s life, even if symptoms do not appear immediately.

Many rare diseases manifest themselves early in life, and approximately 30% of children with rare diseases die before reaching the age of five. Ribose-5-phosphate isomerase deficiency is the rarest genetic condition known, with only four people diagnosed in 27 years. There is no single cut-off number for determining whether a disease is rare. A disease may be rare in one part of the world or among one group of people, but common in another.

So, what exactly is a rare disease? Well, it's tricky! There's no one universally agreed-upon answer. Some folks define it simply by how few people have it. Others think about things like, "Can we even treat it properly?" or "How bad is the disease, really?" For example, in the U.S., the Rare Diseases Act of 2002 says it's "any disease or condition that affects fewer than 200,000 people," basically one in 1,500. Funny enough, that's almost the same definition used in the Orphan Drug Act of 1983, which was created to encourage research and hopefully find some cures!

So, what exactly is a "rare disease?" Well, in Japan, it's something that impacts fewer than 50,000 folks, or roughly one out of every 2,500. But then you have the European Commission on Public Health, which says they're “life-threatening or chronically debilitating disorders of such low prevalence that extraordinary coordinated efforts are necessary to address them”. They define "low prevalence" as affecting fewer than one in 2,000 people. It's not just about being statistically rare, though; if a condition isn't life-threatening, chronically debilitating, or lacking adequate treatment, it usually doesn't count. You'll see similar definitions floating around in medical research and used by different countries' health plans, with the numbers bouncing around quite a bit, from one in 1,000 to one in 200,000.

Global Rare Disease Drug Market Overview

So, what's making the Rare Disease Drug Market tick? Well, a few thingswe're seeing more and more cases of rare diseases around the world. Plus, governments are putting favorable policies in place, and companies are launching a ton of new drugs. Don't forget all the R&D going into new treatments and drugs! All this really fuels growth because, frankly, rare diseases are on the rise. Did you know that, according to an article in the Journal of Rare Disorders (2020), there are around 7,000 known rare disorders, and a whopping 70% of them don't have any treatment? That means there's huge potential for growth as researchers work to fill those gaps. And get thisthe Global Genes Project says that rare diseases impact around 300 million people globally.

Because of the high prevalence of people suffering from rare disorders, special drugs will be required, accelerating the adoption of rare disease drugs. To drive global market growth, R&D initiatives are being expanded. Increased R&D investments by prominent players for rare disease drug development of novel product offerings is one of the critical driving factors prevailing in the global market. Since the public’s awareness and understanding of rare diseases have grown, several key clinical-stage biopharmaceutical companies and established market players have a strong pipeline of rare disease drugs in various stages of clinical trials.

This growing interest in rare disorder therapeutics is because major pharmaceutical breakthroughs resulting in blockbuster drug developments are more likely in rare disorders than in traditional pharmaceutical portfolios. The restraints for the market growth are a Lack of awareness regarding rare disease treatment and the High Cost of the drugs and therapy. Whereas the is growth potential in untapped emerging nations and growth in novel indications for known rare disease drugs.

Global Rare Disease Drug Market Segmentation Analysis

The Global Rare Disease Drug Market is segmented on the basis of Therapy Area, Drug Type, Distribution Channel, And Geography.

Rare Disease Drug Market, By Therapy Area

• Oncology• Hematology• Neurology• Cardiovascular• Other Disease Types

So, when we look at what kinds of diseases these drugs target, we see the market broken down into things like Oncology (that's cancer stuff), Hematology (blood disorders), Neurology (brain and nerve problems), Cardiovascular (heart-related issues), and then just "Other Disease Types" to catch everything else. Right now, Oncology is the big kahuna when it comes to rare disease drug market share. That's because there are a ton of cancer drugs being developed by major companies, and honestly, a lot of rare disease drugs are focused on fighting cancer. But, keep an eye on Hematology! With all the new drugs hitting the market and getting the thumbs up from regulators, it's likely to become the second biggest piece of the pie. And don't forget Neurology – it's expected to grow pretty quickly too, especially with better treatments coming out for diseases like multiple sclerosis.

Rare Disease Drug Market, By Drug Type

• Biological• Non-biological

Based on Drug Type, the market is bifurcated into Biological and Non-biological. The overwhelming presence of biologic product offerings is one of the major factors responsible for the segment’s dominance. According to the United States Food and Drug Administration (U.S. FDA), the organization’s Orphan Products Development (OOPD) program has resulted in the development and marketing of over 600 drugs and biologic products for rare disorders since 1983. The non-biologics segment accounted for a smaller share of the global market and had a lower CAGR.

Rare Disease Drug Market, By Distribution Channel

• Hospital Pharmacy• Retail Pharmacy• Online Pharmacy• Others

Based on Distribution Channel, the market is bifurcated into Hospital Pharmacy, Retail Pharmacy, Online Pharmacy, and Others. During the forecast period, hospital pharmacies are expected to have the largest market share. The primary reason for this dominance is that a significant number of drugs must be administered intravenously by trained healthcare professionals in hospitals.

Rare Disease Drug Market, By Geography

• North America• Europe• Asia Pacific• Rest of the world

On the basis of Geography, the Global Rare Disease Drug Market is classified into North America, Europe, Asia Pacific, and Rest of the world. North America currently dominates the Rare Disease Drug Market and is expected to do so for the foreseeable future. The United States has the largest market share in North America. One reason for market growth is that in the United States, a drug designated as a Rare Disease drug receives seven years of marketing exclusivity upon FDA approval for a specific indication, as well as tax credits and a user fee waiver.

Key Players

The “Global Rare Disease Drug Market” study report will provide valuable insight with an emphasis on the global market. The major players in the market are Novartis AG, F Hoffmann-La Roche Ltd., Celgene Corporation, Bristol-Myers Squibb Company, Shire plc, Pfizer, Inc., Sanofi S.A., Bayer, Alexion Pharmaceuticals, Inc., Biogen, Inc., Abbvie Inc., Amgen Inc., Amryt Pharma Plc., GlaxoSmithKline Plc (Gsk), Johnson & Johnson (Janssen Global Services, Llc). The competitive landscape section also includes key development strategies, market share, and market ranking analysis of the above-mentioned players globally.

Key Developments

• In May 2020, AstraZeneca and Daiichi Sankyo Company announced that Enhertu (trastuzumab deruxtecan) received FDA Orphan Drug Designation for the treatment of patients with gastric cancer, including gastroesophageal junction cancer.

• In June 2020, Agios Pharmaceuticals, Inc. announced that their pipeline candidate Mitapivat for the treatment of Thalassemia had received FDA Orphan Drug Designation.

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Reasons to Purchase this Report

We take a deep dive into the market using both qualitative and quantitative analysis, breaking it down by segments considering everything from the economy to other important factors. You'll get clear data on market value (in USD Billion) for each segment and sub-segment. We'll also point out which region and segment are predicted to grow the fastest and really take the lead. Expect a geographic breakdown, highlighting product/service consumption in each area and the things influencing the market there. Our competitive landscape section ranks the major players and covers new service/product launches, partnerships, business expansions, and acquisitions over the last five years. You'll also find extensive company profiles, including company overviews, insights, product benchmarking, and SWOT analysis for the key players. We'll give you the current and future market outlook, considering recent developments like growth opportunities, drivers, challenges, and restraints in both emerging and developed regions. We provide an in-depth look at the market from different angles using Porter's five forces analysis, plus insight into the market's Value Chain. Finally, we cover market dynamics, potential growth opportunities, and include 6 months of post-sales analyst support to help you make sense of it all.

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