Ultra Market Research | United Kingdom Orphan Drug Market
United Kingdom Orphan Drug Market
Report ID : 1037
Category : United-Kingdom(UK)
No Of Pages : 145
Published on: March 2025
Status: Published
Format :
Key Question Answer
Global Market Outlook
In-depth analysis of global and regional trends
Analyze and identify the major players in the market, their market share, key developments, etc.
To understand the capability of the major players based on products offered, financials, and strategies.
Identify disrupting products, companies, and trends.
To identify opportunities in the market.
Analyze the regional penetration of players, products, and services in the market.
Comparison of major players financial performance.
Evaluate strategies adopted by major players.
Recommendations
United Kingdom Orphan Drug Market
Introduction
The Orphan Drug Market of the United Kingdom is defined as the market for drugs that are specifically designed for the rare disease, or orphan disease, that is limited to a low percentage of the population. Orphan drugs are usually challenging to produce with their small market base, but these are vital to patients suffering from such rare diseases. The use of these drugs is to offer targeted therapeutic choices for conditions like orphan cancers, genetic disorders, and neurological disorders. Current trends in the UK orphan drug sector indicate a rise in approvals of drugs, fuelled by advancements in biotechnology and a more supportive regulatory framework. The UK government and the European Medicines Agency have made the orphan drug development process easier, providing incentives like longer market exclusivity. Based on market size, the UK orphan drug market is growing at a strong rate and is projected to touch new heights in the future as more investments are being made in research and development. The present situation reflects a hopeful future, with an emphasis on targeted therapies and personalized medicine.
There are a number of drivers contributing to the development of the United Kingdom orphan drug market. To begin with, the greater occurrence of rare diseases within the population has created a greater demand for specialized drugs. Advances in biotechnology and personalized medicine are facilitating the creation of more effective orphan drugs. In addition, the regulatory schemes of the UK and Europe, e.g., the Orphan Drug Designation, offer such incentives as tax relief, prolonged patent exclusivity, and expedited approvals that it becomes easier for companies to invest in developing orphan drugs. Furthermore, growing consciousness regarding rare diseases and availability of research funds fuel the expansion of the market. Finally, the UK government and private establishments increasing healthcare expenditure in order to enhance access to such treatment is a central role in the growth of the market.
Restraints
Notwithstanding the encouraging perspective, a number of challenges pose the growth of the UK orphan drug market. The cost of orphan drugs is one of the biggest challenges and tends to make it less affordable for patients. Furthermore, the low volume of patients poses a challenge towards attaining economies of scale, leading to even higher costs. Regulatory obstacles, while streamlined, remain a challenge to fast-track approval in certain instances. The insufficient data on long-term safety and efficacy for most orphan drugs also raise concerns among regulators and healthcare providers. Finally, the UK and European reimbursement and pricing models are restrictive, impacting market access and profitability for pharmaceutical companies.
Opportunity
The United Kingdom market for orphan drugs offers many opportunities for expansion. With the development of biotechnology and genomics, new innovative treatments are emerging, like gene therapies and biologics, providing new means of treatment for once untreatable orphan diseases. The UK government's ongoing investment in healthcare infrastructure and research, and also partnerships with pharmaceutical firms, provides a conducive environment for the development of orphan drugs. Furthermore, the growing emphasis on individualized medicine presents immense promise for precision care designed to accommodate specific genetic profiles. The orphan drugs market is poised to grow as medical systems mature into more accessible sources for these life-saving medicines. A better diagnostic capability will also identify rare diseases at an earlier stage, further boosting demand for targeted therapies.
Trend
One of the emerging trends in the UK orphan drug market is the growth of personalized medicine, which entails the customization of treatment against specific patients according to their genetic profile. This trend is driven by the revolution in gene sequencing technologies, which are simplifying the identification of rare genetic diseases and treating them with specialized treatments. In addition, there has been an increase in the adoption of gene and cell therapies as new replacement alternatives to conventional drug therapies, which hold the potential for lasting cures. Another major trend is the rising number of collaborations between pharmaceutical firms and academic research centers in a bid to hasten orphan drug development. The increasing application of digital health technologies and telemedicine is also enhancing access to the treatment of rare diseases, especially in rural locations. With the regulatory environment improving, increasingly orphan drugs are likely to find their way to the market and provide new avenues of treatment to patients suffering from rare diseases.
Gene Therapy for Cystic Fibrosis - Pre-Regulatory Gene Therapy for Duchenne Muscular Dystrophy - Regulatory CRISPR-Cas9-based treatments for various genetic disorders - Pipeline
Key Target Audience
Pharmaceutical Companies Biotechnology Companies Healthcare Providers Research Institutions Government Health Agencies Rare Disease Advocacy Groups Hospitals and Clinics Investors and Venture Capitalists Regulatory Bodies Orphan Drug Manufacturers
The market is experiencing substantial growth, with increasing approvals for orphan drugs, and is expected to continue expanding due to ongoing research and government support.
Orphan drugs are medications developed specifically to treat rare diseases affecting a small percentage of the population.
High drug costs, limited patient populations, regulatory hurdles, and reimbursement issues are key challenges.
The UK government offers incentives such as extended market exclusivity, fast-track approval, and funding for research to encourage orphan drug development.
Personalized medicine, gene therapies, and partnerships between pharmaceutical companies and research institutions are some of the key trends driving the market forward.
1. Introduction 1.1 Report Description 1.2 Research Methodology 1.2.1 Data Mining 1.2.2 Market Modeling and Forecasting 1.2.3 Data Validation 1.2.4 Industry Analysis
2. Market Dashboard
3. Market Overview 3.1 Market Definition and Scope 3.2 Market Segmentation 3.2.1 By Treatment Types 3.2.1.1 Chemotherapy - Induction Phase - Consolidation Phase - Maintenance Phase 3.2.1.2 Targeted Therapies - Tyrosine Kinase Inhibitors - Antibody-Drug Conjugates - Others 3.2.1.3 Immunotherapies - CAR-T Therapy - Monoclonal Antibodies 3.2.2 By Patient Demographics 3.2.2.1 Children - Infants - Toddlers and Young Children 3.2.2.2 Adults - Young Adults - Seniors 3.2.3 By Distribution Channels 3.2.3.1 Hospital Pharmacies - Tertiary Care Centers - Pediatric Oncology Units 3.2.3.2 Retail Pharmacies - Local Pharmacies - Chain Pharmacies 3.2.3.3 Online Pharmacies 3.2.4 By Therapeutic Applications 3.2.4.1 First-Line Treatment - Chemotherapy-Only Regimens - Targeted Therapies 3.2.4.2 Relapse or Refractory ALL - CAR-T Therapies - Experimental Treatments 3.3 Market Dynamics 3.3.1 Drivers 3.3.2 Restraints 3.3.3 Opportunities 3.3.4 Challenges
5. United Kingdom Orphan Drug Market by Patient Demographics 5.1 Introduction 5.2 Market Size and Growth Rate by Patient Demographics (2024-2030) 5.2.1 Children - Infants - Toddlers and Young Children 5.2.2 Adults - Young Adults - Seniors
6. United Kingdom Orphan Drug Market by Distribution Channels 6.1 Introduction 6.2 Market Size and Growth Rate by Distribution Channels (2024-2030) 6.2.1 Hospital Pharmacies - Tertiary Care Centers - Pediatric Oncology Units 6.2.2 Retail Pharmacies - Local Pharmacies - Chain Pharmacies 6.2.3 Online Pharmacies
7. United Kingdom Orphan Drug Market by Therapeutic Applications 7.1 Introduction 7.2 Market Size and Growth Rate by Therapeutic Applications (2024-2030) 7.2.1 First-Line Treatment - Chemotherapy-Only Regimens - Targeted Therapies 7.2.2 Relapse or Refractory ALL - CAR-T Therapies - Experimental Treatments
10. Appendix 10.1 List of Tables Table: United Kingdom Orphan Drug Market Size by Treatment Types (2024-2030) Table: Chemotherapy Market Size by Sub-type (2024-2030) Table: Targeted Therapies Market Size by Sub-type (2024-2030) Table: Immunotherapies Market Size by Sub-type (2024-2030) Table: United Kingdom Orphan Drug Market Size by Patient Demographics (2024-2030) Table: Children Market Size by Sub-type (2024-2030) Table: Adults Market Size by Sub-type (2024-2030) Table: United Kingdom Orphan Drug Market Size by Distribution Channels (2024-2030) Table: Hospital Pharmacies Market Size by Sub-type (2024-2030) Table: Retail Pharmacies Market Size by Sub-type (2024-2030) Table: United Kingdom Orphan Drug Market Size by Therapeutic Applications (2024-2030) Table: First-Line Treatment Market Size by Sub-type (2024-2030) Table: Relapse or Refractory ALL Market Size by Sub-type (2024-2030) 10.2 List of Figures Figure: United Kingdom Orphan Drug Market Size and Growth Rate by Treatment Types (2024-2030) Figure: Chemotherapy Market Growth Rate by Sub-type (2024-2030) Figure: Targeted Therapies Market Growth Rate by Sub-type (2024-2030) Figure: Immunotherapies Market Growth Rate by Sub-type (2024-2030) Figure: United Kingdom Orphan Drug Market Size and Growth Rate by Patient Demographics (2024-2030) Figure: Children Market Growth Rate by Sub-type (2024-2030) Figure: Adults Market Growth Rate by Sub-type (2024-2030) Figure: United Kingdom Orphan Drug Market Size and Growth Rate by Distribution Channels (2024-2030) Figure: Hospital Pharmacies Market Growth Rate by Sub-type (2024-2030) Figure: Retail Pharmacies Market Growth Rate by Sub-type (2024-2030) Figure: United Kingdom Orphan Drug Market Size and Growth Rate by Therapeutic Applications (2024-2030) Tables: Financial Overview of Key Companies Table: Amgen Inc. Orphan Drug Financial Overview Table: Novartis AG Orphan Drug Financial Overview Table: Pfizer Inc. Orphan Drug Financial Overview Table: Gilead Sciences Orphan Drug Financial Overview Table: Kite Pharma Orphan Drug Financial Overview Table: Takeda Pharmaceutical Company Orphan Drug Financial Overview Table: Bristol-Myers Squibb Orphan Drug Financial Overview Table: Jazz Pharmaceuticals Orphan Drug Financial Overview Table: AbbVie Inc. Orphan Drug Financial Overview Table: Sanofi Orphan Drug Financial Overview Table: Autolus Therapeutics Orphan Drug Financial Overview Table: Cellectis Orphan Drug Financial Overview Table: Allogene Therapeutics Orphan Drug Financial Overview Table: Celgene Corporation Orphan Drug Financial Overview Table: Eli Lilly and Company Orphan Drug Financial Overview
Research Methodology
The process of market research at Ultra Market Research is an iterative in application and usually follows following path. Information from secondary used to build data models, then results from data models are validated from primary participants. Then cycle repeats where, according to inputs from primary participants, additional secondary research is done, and new Information is again incorporated into data model. The process continues till desired level of Information is not generated
To calculate the market size, the report considers the revenue generated from the sales of Ultra Market Research providers. The revenue generated from the sales of Ultra Market Research has been calculated through primary and secondary research. The report also presents the key players operating in the Ultra Market Research market across the globe identified through secondary research and a corresponding detailed analysis of the top vendors in the market. The market size calculation also includes distribution channel segmentation determined using secondary sources and verified through primary sources.
Secondary Research
The secondary research source that are typically referred to include, but are not limited to:
Company websites, annual reports, financial reports, broker reports, investor presentations and SEC filings
Internal and external proprietary databases, relevant patent and regulatory databases
National government documents, statistical databases and market reports
News articles, press releases and web-casts specific to the companies operating in the market
The source for secondary research includes but is not limited to: Factiva, Hoovers and Statista
Inner Circle Represents – Stage of Research Process
Middle Circle Represents – Source of Information
Outer Circle Represents – Information Derived from that source
Primary Research We conduct primary interviews on an ongoing basis with industry participants and commentators in order to validate data and analysis. A typical research interview fulfills the following functions:
It provides first-hand Information on the market size, market trends, growth trends, competitive landscape, future outlook etc.
Helps in validating and strengthening the secondary research findings
Further develops the analysis team’s expertise and market understanding
Primary research involves E-mail interactions, telephonic interviews as well as face-to-face interviews for each market, category, segment and sub-segment across geographies
The participants who typically take part in such a process include, but are not limited to:
Industry participants: CEOs, VPs, marketing/ distribution channel managers, market intelligence managers and national sales managers
Purchasing managers, technical personnel, distributors and resellers
Outside experts: Investment bankers, valuation experts, research analysts specializing in specific markets
Key opinion leaders specializing in different areas corresponding to different industry distribution channels
Models Where no hard data is available, we use modeling and estimates in order to produce comprehensive data sets. A rigorous methodology is adopted in which the available hard data is cross referenced with the following data distribution channels to produce estimates:
Demographic data: Population split by segment
Macro-economic indicators: GDP, etc.
Industry indicators: Expenditure, distribution channel stage & infrastructure, sector growth and facilities.
Data is then cross checked by the expert panel.
2..1 Company Share Analysis Model
Company share analysis is used to derive the size of United States market. As well as study of revenues of companies for last three to five years also provide the base for forecasting the market size and its growth rate. This model is built in following steps: 2..2 Revenue Based Modeling
Revenue based models can be built in two ways – Top-Down or Bottom-Up irrespective of industry. Market size estimated from company share analysis acts as a validation point for bottom-up approach where as it acts as starting point for top-down approach.
2.1 Research Limitations Inflation is not a part of pricing in this report. Prices of United Kingdom Orphan Drug Market and its derivatives vary in each region and hence similar revenue ratio does not follow for each individual region. The same price for each distribution channel has been taken into account while estimating and forecasting market revenue Globally. Regional average price has been considered while breaking down this market by application in each region. This report provides market size of United Kingdom Orphan Drug Market for the past year and forecasts for the next six years. United Kingdom Orphan Drug Market size is given in terms of revenue Market revenue is defined in USD Million. Market numbers are given on the basis of different United Kingdom Orphan Drug Market categories. Market size and forecasts for each major application is provided in the context of Global market. The numbers provided in this report are derived on the basis of demand for United Kingdom Orphan Drug Market from different application industries in different regions.
