Global Orphan Diseases Drugs Market Report 2022 to 2030

Global Orphan Diseases Drugs Market Analysis

$ 3999

This report presents a strategic analysis of the Global Orphan Diseases Drugs Market and a forecast for its development in the medium and long term. It provides a broad overview of the market dynamics, trends and insights, growth drivers and restraints, segmentation, competitive landscape, healthcare policies and regulatory framework, reimbursement scenario, challenges and future outlook. This is one of the most comprehensive reports about the Global Orphan Diseases Drugs Market, offering unmatched value, accuracy and expert insights.

ID: IN10GLPH019 CATEGORY: Pharmaceuticals GEOGRAPHY: Global

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Global Orphan Diseases Drugs Market Executive Summary

The world has achieved significant progress in terms of health during the 20th century. The advancements in health care over the last 50 years have been amazing; worldwide life expectancy has risen faster in this time than it has in the previous 5,000 years. The global healthcare industry continues to rise to the new challenges posed by the ongoing pandemic, which continues to consume the attention and resources of healthcare systems worldwide. They are continuing to improve their workforce's human experience by changing what, how, and where work is done, rapidly scaling virtual health services for patients, and forming partnerships to create and purchase the necessary vaccines, treatments, and supplies. Simultaneously, they continue to address the growing importance of healthcare disparities, sustainability, and the environment.

The pharmaceutical industry covers all aspects of medication research, development, production, and distribution. Pharma revenues worldwide reached $1.42 trillion in 2021 due to the market's rapid growth over the previous two decades. With the introduction of new technology as well as more economical and effective manufacturing techniques, the industry has experienced a significant transition. Additionally, the market growth has been positively driven by rising investment flow in this sector.

Orphan medications are brand-new pharmaceuticals created specifically for the treatment, diagnosis, and prevention of a variety of uncommon diseases or conditions. Rare diseases typically have a lower prevalence rate than common diseases, which results in a smaller market. Regulatory agencies have approved more than 500 orphan medications, but many uncommon diseases still have no effective treatments. The increased prevalence of rare diseases and the unmet demand for orphan medications have sparked additional research and development in this field.

Market Size and Key Findings

The global orphan drugs market is estimated to be valued at US$ 140.40 Bn in 2021 and is expected to exhibit a CAGR of 10.3% over the forecast period (2021-2030).

Global Orphan Diseases Drugs Market Size (In USD Bn) (2021-2030F)

Market Dynamics

Market Growth Drivers Analysis

The presence of a strong product pipeline and the imminent launches are expected to fuel market growth. Increasing approval and launch of new orphan drugs are anticipated to boost industry growth.

Market Restraints

The market growth could be hampered by the high cost associated with the research and development of pipeline candidates used for the treatment of rare diseases, which, in turn, contributes to the rise in drug prices. This can be attributed to factors such as the increased capital for conducting clinical trials through contract research organizations (CROs) and the risk of drug failure during the clinical trials.

Competitive Landscape

Key Players

Major players in Orphan Diseases Drugs Market are F. Hoffmann-La Roche Ltd.; Pfizer, Inc.; PTC Therapeutics; AstraZeneca; Novartis AG; Novo Nordisk; Bayer AG; AbbVie Inc.; Merck & Co. Inc.; Bristol Myers Squibb

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Orphan Diseases Drugs Market Segmentation

Therapeutic area

Cancer
Neurological condition
Cardiovascular condition
Musculoskeletal condition
Hematologic condition
Others

Route of administration

The injectable segment dominated the market and is expected to witness significant growth during the forecast period. This can be attributed to the launch of novel injectables for the treatment of rare diseases.

Oral
Injectable
Others

Drug Type

Biologics dominated the market and is expected to witness significant growth during the forecast period. The advancement in biotechnology and research techniques has facilitated the development of novel biologics. High target specificity and potential of biological drugs revolutionizing the treatments of several rare diseases are expected to fuel the segment growth.

Biologics
Biosimilars
Small molecule

Distribution Channel

The specialty pharmacy segment dominated the market. The dominance can be attributed to strategic initiatives undertaken by key players, such as the acquisition of specialty pharmacies for the distribution of their products.

Hospital Pharmacy
Specialty Pharmacy
Online Pharmacy

Methodology for Database Creation

Our database offers a comprehensive list of healthcare centers, meticulously curated to provide detailed information on a wide range of specialties and services. It includes top-tier hospitals, clinics, and diagnostic facilities across 30 countries and 24 specialties, ensuring users can find the healthcare services they need.

Additionally, we provide a comprehensive list of Key Opinion Leaders (KOLs) based on your requirements. Our curated list captures various crucial aspects of the KOLs, offering more than just general information. Whether you're looking to boost brand awareness, drive engagement, or launch a new product, our extensive list of KOLs ensures you have the right experts by your side. Covering 30 countries and 36 specialties, our database guarantees access to the best KOLs in the healthcare industry, supporting strategic decisions and enhancing your initiatives.

How Do We Get It?

Our database is created and maintained through a combination of secondary and primary research methodologies.

1. Secondary Research

With many years of experience in the healthcare field, we have our own rich proprietary data from various past projects. This historical data serves as the foundation for our database. Our continuous process of gathering data involves:

Analyzing historical proprietary data collected from multiple projects.
Regularly updating our existing data sets with new findings and trends.
Ensuring data consistency and accuracy through rigorous validation processes.

With extensive experience in the field, we have developed a proprietary GenAI-based technology that is uniquely tailored to our organization. This advanced technology enables us to scan a wide array of relevant information sources across the internet. Our data-gathering process includes:

Searching through academic conferences, published research, citations, and social media platforms
Collecting and compiling diverse data to build a comprehensive and detailed database
Continuously updating our database with new information to ensure its relevance and accuracy

2. Primary Research

To complement and validate our secondary data, we engage in primary research through local tie-ups and partnerships. This process involves:

Collaborating with local healthcare providers, hospitals, and clinics to gather real-time data.
Conducting surveys, interviews, and field studies to collect fresh data directly from the source.
Continuously refreshing our database to ensure that the information remains current and reliable.
Validating secondary data through cross-referencing with primary data to ensure accuracy and relevance.

Combining Secondary and Primary Research

By integrating both secondary and primary research methodologies, we ensure that our database is comprehensive, accurate, and up-to-date. The combined process involves:

Merging historical data from secondary research with real-time data from primary research.
Conducting thorough data validation and cleansing to remove inconsistencies and errors.
Organizing data into a structured format that is easily accessible and usable for various applications.
Continuously monitoring and updating the database to reflect the latest developments and trends in the healthcare field.

Through this meticulous process, we create a final database tailored to each region and domain within the healthcare industry. This approach ensures that our clients receive reliable and relevant data, empowering them to make informed decisions and drive innovation in their respective fields.