UK Orphan Diseases Drugs Market Analysis Report [2022-30] I Insights10

UK Orphan Diseases Drugs Market Analysis

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The UK Orphan Diseases Drugs Market is projected to grow from $4.025 Bn in 2022 to $7.617 Bn by 2030, registering a CAGR of 8.30% during the forecast period of 2022 – 2030. There are an estimated 3 Mn people in the UK living with a rare disease, which is equivalent to around 5% of the population. Key players in the UK orphan drug market include Sanofi, Pfizer, and GlaxoSmithKline.

ID: IN10GBPH019 CATEGORY: Pharmaceuticals GEOGRAPHY: UK AUTHOR: Shivam Zalke

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UK Orphan Diseases Drugs Market Executive Summary

The UK Orphan Diseases Drugs Market is projected to grow from $4.025 Bn in 2022 to $7.617 Bn by 2030, registering a CAGR of 8.30% during the forecast period of 2022 - 2030.

The UK orphan drug market is growing due to increased R&D investment and regulatory incentives for developing treatments for rare diseases. The market is expected to continue its growth trend due to the high unmet medical need in the orphan disease space, the increased prevalence of rare diseases, and the availability of new technologies to diagnose and treat these diseases. Key players in the UK orphan drug market include Sanofi, Pfizer, and GlaxoSmithKline.

There are an estimated 3 Mn people in the UK living with a rare disease, which is equivalent to around 5% of the population. Over 7,000 rare diseases have been identified, with new ones being discovered regularly. Despite their high prevalence, only around 5% of rare diseases have an approved treatment. This highlights the significant unmet medical need in the orphan disease space.

uk orphan diseases drugs market analysis

Market Dynamics

Market Drivers

The key drivers of the UK orphan diseases drugs market are:

High unmet medical need: The large number of people affected by rare diseases and the limited number of treatments available for these diseases has resulted in a high unmet medical need in the orphan disease space
Increased R&D investment: The market has seen increased investment in R&D for the development of treatments for rare diseases, as pharmaceutical companies recognize the potential for significant returns in this niche market
Regulatory incentives: The UK and EU provide regulatory incentives for the development of treatments for rare diseases, such as extended exclusivity periods and fast-track approval processes
Advancements in technology: New technologies are helping to diagnose rare diseases earlier and more accurately, and are also enabling the development of new treatments for these diseases
Growing patient advocacy: Increased patient advocacy and awareness of rare diseases is driving demand for treatments and putting pressure on governments and the pharmaceutical industry to prioritize the development of treatments for these diseases

Competitive Landscape

Key Players

Some of the key players in the UK orphan diseases drugs market include:

Sanofi
Pfizer
GlaxoSmithKline
Roche
Novartis
AstraZeneca
Abbvie
Alexion Pharmaceuticals
Amgen
Biogen

These companies are actively investing in the development of treatments for rare diseases and have a significant presence in the UK market. Some of these companies have also acquired smaller niche players to expand their portfolio in the orphan disease space.

Healthcare Policies and Regulatory Landscape

The UK and EU have a supportive regulatory landscape for the development of treatments for rare diseases. Key policies and regulations that are driving the growth of the UK orphan diseases drugs market include:

Orphan Drug Designation: Companies can apply for orphan drug designation for their treatments, which provides regulatory incentives such as extended exclusivity periods and fast-track approval processes
Advanced Therapy Medicinal Product (ATMP) Regulation: The ATMP regulation provides a framework for the development and approval of advanced therapies such as gene therapies, stem cell therapies, and immunotherapies
Clinical Trials Regulation: The EU Clinical Trials Regulation provides a streamlined process for conducting clinical trials for treatments for rare diseases
Rare Disease Strategy: The UK government has a rare disease strategy in place that aims to improve diagnosis, treatment, and support for people with rare diseases
Research & Development Tax Credit: The UK provides tax credits for companies investing in R&D, including the development of treatments for rare diseases

These policies and regulations are helping to create a favorable environment for the development and approval of treatments for rare diseases, and are expected to continue to drive the growth of the UK orphan diseases drugs market.

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.