Rare Disease Treatment Market

Rare Disease Treatment Market Outlook (2024 to 2034)

Fact.MR’s latest study reveals that the global rare disease treatment market size is forecasted to increase from a value of US$ 182.28 billion in 2024 to US$ 412.59 billion by the end of 2034. Market expansion has been projected at a CAGR of 8.5% from 2024 to 2034.

This market addresses medical conditions that affect a small number of people and are not so common. Given their rarity and complexity, these illnesses-often referred to as orphan diseases-have a cumulative effect on a comparatively small population, which presents a barrier to research and development. The combined prevalence of rare diseases is significant.

Increasing awareness and advocacy efforts, advancements in genetic and molecular research, and the creation of novel therapeutic approaches are some of the factors driving the market's growth. Due to developments in the field of genomics, precision medicine is emerging, providing new opportunities for targeted therapies catering to the unique genetic profiles of patients suffering from uncommon diseases.

Key Market Growth Drivers

• Primary factor for increase in the demand for rare disease treatment is the rising funding for research and development initiatives aimed at creating novel therapies for uncommon illnesses.
• Advancements in personalized medicine, genomics, and biotechnology are opening up opportunities for therapeutic discovery.
• Pharmaceutical companies are encouraged to develop treatments for rare diseases by favorable regulatory policies, such as fast-track approvals and orphan drug designations.
• The drug development and commercialization process is streamlined through these incentives.
• Demand for rare disease treatment is rising as a consequence of the use of various distribution channels, including online pharmacies, specialty pharmacies, and hospital pharmacies.
• Government policies that are supportive of the industry's growth are driving factors such as the rise in rare disease cases and vaccination campaigns, along with the launch of new medications and innovative therapeutics.
• Research in rare hemophilia factors and rare neurodegenerative disease treatments are also influencing the market.

Report Attributes	Details
Rare Disease Treatment Market Size (2024E)	US$ 182.28 Billion
Forecasted Market Value (2034F)	US$ 412.59 Billion
Global Market Growth Rate (2024 to 2034)	8.5% CAGR
Latin America Market Share (2024E)	8.9%
North America Market Share (2024E)	42%
Sales of Originator Drugs (2024E)	US$ 134.84 Billion
Key Companies Profiled	Novartis AG AbbVie Bristol Myers Squibb BioMarin Amgen Inc. PTC Therapeutics AstraZeneca GlaxoSmithKline Plc Amgen Inc. Pfizer Inc. Johnson & Johnson

What is Responsible for Increase in Rare Disease Treatment Solutions?

“Increasing Government Incentives Promoting Innovation in Healthcare for Improved Outcomes”

The market for rare disease treatment is exhibiting growth in developing countries due to rising demand for better healthcare services, large investments in infrastructure development, and the emergence of innovative practices in the healthcare sector. The demand for treatments for rare diseases is not just seen in developed nations but also in developing nations. The demand for rare disease treatments is rising due to factors such as government incentives to encourage innovation in rare disease treatment management.

What are the Hindrances to Market Growth?

“Limited Patient Pool and Dispersed and Diverse Patient Population”

As rare diseases affect a small fraction of the global population, it can be challenging for treatment providers and researchers to find enough patients who meet the inclusion and exclusion criteria for a given study. The rare disease patient pool is dispersed and diverse in terms of disease subtype, symptoms, stages, and previous medical treatment.

Country-wise Insights

The market for rare disease treatments in North America is projected to expand at a significant pace as a result of the country's high prevalence of rare diseases, the growing number of such patients receiving treatments, and improvements in rare disease treatment management. These are some of the key rare disease treatment market trends in the region.

What is Fueling Expansion of Rare Disease Treatment Market Size in the United States?

“Robust Healthcare System for Identification and Management of Rare Diseases”

Attribute	United States
Market Value (2024E)	US$ 63.37 Billion
Growth Rate (2024 to 2034)	8% CAGR
Projected Value (2034F)	US$ 137.12 Billion

High prevalence of rare diseases in the United States, including Huntington's disease, spina bifida, fragile X syndrome, Guillain-Barre syndrome, Crohn's disease, cystic fibrosis, and Duchenne muscular dystrophy, as well as the existence of a strong healthcare system for the diagnosis and treatment of rare diseases, are expected to contribute to the country’s dominance in the market for rare disease treatments.

• Around 200,000 people in the United States are being affected by a rare disease, according to the Food and Drug Administration.
• The burden of rare diseases is roughly 10 times greater than that of mass-market diseases on a per-patient per year (PPPY) basis, according to an article published in 2022 in Chiesi Global Rare Diseases.
• According to the Genetic and Rare Diseases (GARD) Information Center, one in every 10 Americans (or 30 million people) has a rare disease, with approximately 7,000 known rare diseases as of May 2022.

Which Factors are Conducive to Market Growth in Brazil?

“Government Healthcare Policies Supporting Efficient Management of Rare Diseases”

Attribute	Brazil
Market Value (2024E)	US$ 6.09 Billion
Growth Rate (2024 to 2034)	9.4% CAGR
Projected Value (2034F)	US$ 14.89 Billion

Demand for rare disease treatment in Brazil is estimated to reach US$ 6.08 billion in 2024. Brazil accounts for 38% of the rare disease treatment market share in Latin America in 2024. Brazil's healthcare system is made up of three private components, where patients pay for their medical care, a small paid health insurance component, and a large public component called SUS (Sistema Único de Saúde).

According to the Commonwealth Fund, approximately 75% of the population is dependent on SUS, which does not currently offer sufficient coverage for procedures involving genetic medicines. Brazil has developed a policy that is suitable for managing rare diseases, indicating the introduction of medical genetics into the public health system, thus resulting in rare disease treatment market growth in the country.

Category-wise Insights

Rare disease treatments are primarily categorized based on therapeutic area into genetic diseases, neurological diseases, oncology, infectious diseases, inflammatory diseases, metabolic diseases, pulmonary diseases, kidney diseases, musculoskeletal diseases, endocrine diseases, and topical diseases. Genetically mutated diseases occupy a leading market share of 28% in 2024.

Why are Rare Disease Treatment Solutions Widely Adopted for Addressing Genetic Disorders?

“Increased Public Awareness of Genetic Conditions and Advancements in Diagnostic Procedures”

Attribute	Genetic Disease
Segment Value (2024E)	US$ 51.66 Billion
Growth Rate (2024 to 2034)	8.7% CAGR
Projected Value (2034F)	US$ 118.76 Billion

Many individuals with rare diseases are affected by genetic mutations. This reflects the wide spectrum of such conditions. A 2021 trial identified 791 potential orphan drug candidates, with 192 patients experiencing genetic issues. The genetic testing market, integral to rare disease diagnosis, is expanding, driven by factors including increased public awareness, technological advancements, and improved accessibility.

Why Do Originator Drugs Account for a Leading Market Share?

“Sales of Originators Driven by Patent Protection and Ability to Offer Specialized Care”

Attribute	Originator Drugs
Segment Value (2024E)	US$ 134.84 Billion
Growth Rate (2024 to 2034)	8.9% CAGR
Projected Value (2034F)	US$ 316.96 Billion

Originator drugs are protected by a patent, giving the pharmaceutical company that discovered them exclusive rights. These medications are used to treat rare diseases for which there were no prior effective therapies. Since rare diseases are not commonly observed, effective treatment options are limited.

This gap is filled by originator medications, which offer focused care for particular hereditary or uncommon disorders.

Medication for spinal muscular atrophy, Gaucher disease, and cystic fibrosis are a few examples. Different classifications of originator drugs exist, such as biologics (complex molecules derived from living organisms) and organic compounds. Treatment providers for rare genetic disorders are increasingly utilizing biologics, such as monoclonal antibodies.

Competitive Landscape

Key players in the rare disease treatment market are Novartis AG, AbbVie, Bristol Myers Squibb, BioMarin, Amgen Inc., PTC Therapeutics, AstraZeneca, GlaxoSmithKline Plc, Amgen Inc., Pfizer Inc., and Johnson & Johnson.

• As per the Canadian Organization for Rare Disorders (CORD) Report 2021, approximately 1 in 12 individuals in Canada is impacted by rare diseases annually, with approximately 80% attributed to genetic mutations. Due to the substantial prevalence of rare diseases in the country, demand for effective treatment solutions is projected to rise over the forecast period.
• A study reported that as of May 2022, 30 million Americans, or 1 in 10 of the population, suffered from a rare disease, and there are about 7,000 known rare diseases, according to the Genetic and Rare Diseases (GARD) Information Center.
• In November 2023, the Union Health Ministry of India introduced generic medications to support the care and treatment of four conditions: Wilson's disease, Gaucher's disease, Tyrosinemia-Type 1, and Dravet- Lennox-Gastaut syndrome. This initiative aims to alleviate the burden on patients with rare diseases across India, offering these medications at a fraction of their current market prices, ranging from 60 to 100 times less expensive.

Segmentation of Rare Disease Treatment Market Research

• By Therapeutic Area :

  • Hematology
  • Genetic Diseases
  • Neurological Diseases
  • Oncology
  • Infectious Diseases
  • Inflammatory Diseases
  • Metabolic Diseases
  • Pulmonary Diseases
  • Kidney Diseases
  • Musculoskeletal Diseases
  • Endocrine Diseases
  • Topical Diseases

• By Drug Type :

  • Originator Drugs
  • Generic Drugs

• By Drug Class :

  • Biologics
  • Organic Compounds

• By Mode of Administration :

  • Injectables
  • Oral

• By Distribution Channel :

  • Hospital Pharmacies
  • Specialty Treatment Pharmacies
  • Retail Pharmacies

• By Region :

  • North America
  • Europe
  • Latin America
  • East Asia
  • South Asia & Oceania
  • Middle East & Africa