Charting the Orphan Drug Development Pipeline

Charting the Orphan Drug Development Pipeline


Which are the most exciting areas of orphan drug research and where are the best opportunities?

Global sales of drugs for orphan indications were estimated to be $114 billion in 2016, with sales forecast to rise to $209 billion by 2022. Orphan drugs enjoy favourable regulatory pathways, satisfy unmet clinical need and often command significant prices. No wonder that research efforts have intensified and the number of molecules gaining orphan drug designation is growing year-on-year. But which are the most promising therapies and conditions? Where is investment being made? Which companies/developers are setting the pace?

Charting the Orphan Drug Development Pipeline analyses 3,868 unique molecules from 2,257 developers to create the most comprehensive independent analysis of orphan drug research activity available. This comprehensive report, and its associated MS Excel™ data set, reveals developments in the context of EMA/FDA designation status, therapy areas, conditions, developers and clinical trial activity.

Two Extensive Information and Data Resources Included

  • Analysis Report: Organised by therapy area, the report, through more than 420 easy-to-read charts, tables and figures, identifies the designation status, conditions being targeted, the leading developers and the current/planned clinical trial landscape
  • MS Excel Data: This powerful resource allows you to really drill down into the data for key insights. All the data in the report is included in a well-structured MS Excel Spreadsheet for your own further analysis or incorporation into internal data analytics platforms.
This Unrivalled Resource Provides
  • A comprehensive overview of the market dynamics, legal definition and US/EU incentives for orphan drugs
  • An analysis of orphan drug designation trends 2015-2016 by therapy area in the US and EU
  • A review of FDA and EMA orphan drug designation status by therapy area
  • Extensive reviews by therapeutic category identifying FDA and EMA designation status with a focus on the number of orphan drug designations issued between January 2016 and March 2017
  • The orphan diseases and conditions that are attracting developer interest and research effort
  • At a glance overview of companies’ orphan drug portfolios
  • An overview of 1,019 trials from 207 sponsors covering the number of active, dormant, recruiting and completed clinical trials within each therapy area
Reasons to Buy

Key Report Facts
  • 290 Pages
  • 166 Tables
  • 257 Charts
  • 37213 Data Points
  • 20 Therapy Areas
  • 1000+ Conditions
  • 3868 Molecules
  • 2183 Companies
Key Benefits
  • Identify the trends and status of orphan drugs in 20 major therapy areas
  • Compare the difference in orphan drug designations between the FDA and the EMA
  • Examine the rare diseases which are attracting research interest and investment
  • Assess how a company’s orphan drug research programme indicates its ambitions
  • Understand how clinical trial activity is reflecting interest in key therapy areas and know who’s involved
  • Incorporate all the data into your internal data analytics systems.
Key Questions Answered By This Report
  • Cardiovascular: What is the leading target condition in the cardiovascular area?
  • Gastrointestinal: There are 9 drugs being researched for short bowel syndrome, but which companies are involved in this area?
  • Genitourinary: How many drugs received orphan drug designation from the EMA in 2016?
  • Haematology: Which 3 companies are leading the orphan drug research push?
  • Neuroscience: 12 orphan drugs are in Phase III trials – what are they and who are the developers?
  • Metabolic disease: What is the target enrolment to clinical trials for abatacept, osilodrostat and metyrapone in the metabolic disease area?
  • Oncology: What cancers are attracting the most developer interest and how is this reflected in the number of orphan drug designations?
Who Will Benefit from this Report?
  • Corporate leaders looking to position their company in niche orphan disease opportunity areas
  • Business development teams profiling potential acquisition or collaboration opportunities
  • Research managers planning clinical research programmes
  • Regulatory teams applying for and monitoring trends in FDA and EMA orphan drug designation
  • Competitive intelligence professionals tracking the activity of key competitors in their field of interest
  • Financial and consultancy professionals identifying investment opportunities.
Report Content Highlights
  • Overview
  • Orphan drug market dynamics
  • Legal definitions and incentives for orphan drug development in US and EU
  • Trends in orphan drug designation
  • For each therapy area covered
  • EU/US designated drugs
  • Orphan drugs designated in 2016-2017
  • Leading conditions
  • Leading developers
  • Clinical Trials: Molecules, sponsors and trial end dates
  • MS Excel Dataset Gives Instant Access to
  • FDA/EU Designation Date
  • Therapy Area
  • Condition
  • Developer
  • Brand (if relevant)
  • Molecule INN
  • Therapy Areas Covered
  • Anti-infective
  • Antiparasitic
  • Cardiovascular disorders
  • Cerebrovascular disorders
  • Dermatological
  • Gastrointestinal disorders
  • Genitourinary
  • Gynaecology and obstetrics
  • Haematology (blood and blood clotting)
  • Hormonal (excluding sex hormones)
  • Immunological disorders
  • Inflammatory disorders
  • Metabolic disorders
  • Musculoskeletal disorders
  • Neuroscience
  • Oncology
  • Ophthalmology
  • Poisoning
  • Respiratory disorders
  • Transplantation
About FirstWord

FirstWord is an innovative industry intelligence leader serving over 240,000 Pharma and MedTech professionals worldwide. FirstWord offers a range of products and services designed to help your company gain a competitive edge by making key business decisions with speed and confidence.

FirstWord Pharma PLUS is a personalised and comprehensive intelligence service delivering up-to-the-minute pharma news, insight, analysis and expert views of importance to your company’s success.

FirstWord Reports deliver timely, need-to-know intelligence about your products, your competitors and your markets. Covering biosimilars, market access, medical affairs, sales & marketing, technology and therapy areas, FirstWord Reports provide expert views and intelligence on the challenges facing pharma today.

1. Research objectives and methodology
2. Overview
.1 Market dynamics
2.2 Legal definitions and incentives for orphan drug development
2.2.1 Incentives in the US
2.2.2 Incentives in the EU
2.3 Trends in orphan drug designation
3. Anti-infective
3.1 Summary
3.2 EU/US designated drugs
3.2.1 Orphan drugs designated in 2016-2017
3.3 Leading conditions
3.4 Leading developers
3.5 Clinical trials
3.5.1 Molecules
3.5.2 Sponsors
3.5.3 Trial end dates
4. Antiparasitic
4.1 Summary
4.2 EU/US designated drugs
4.2.1 Orphan drugs designated in 2016-2017
4.3 Leading conditions
4.4 Leading developers
4.5 Clinical trails
4.5.1 Molecules
4.5.2 Sponsors
4.5.3 Trial end dates
5. Cardiovascular
5.1 Summary
5.2 EU/US designated drugs
5.2.1 Orphan drugs designated in 2016–2017
5.3 Leading conditions
5.4 Leading developers
5.5 Clinical trials
5.5.1 Molecules
5.5.2 Sponsors
5.5.3 Trial end dates
6. Cerebrovascular
6.1 Summary
6.2 EU/US designated drugs
6.2.1 Orphan drugs designated in 2016
6.3 Leading conditions
6.4 Leading developers
6.5 Clinical trials
6.5.1 Molecules
6.5.2 Sponsors
6.5.3 Trial end dates
7. Dermatological
7.1 Summary
7.2 EU/US designated drugs
7.2.1 Orphan drugs designated in 2016–2017
7.3 Leading conditions
7.4 Leading developers
7.5 Clinical trials
8. Gastrointestinal
8.1 Summary
8.2 EU/US designated drugs
8.2.1 Orphan drugs designated in 2016–2017
8.3 Leading conditions
8.4 Leading developers
8.5 Clinical trials
8.5.1 Molecules
8.5.2 Sponsors
8.5.3 Trial end dates
9. Genitourinary
9.1 Summary
9.2 EU/US designated drugs
9.2.1 Orphan drugs designated in 2016
9.3 Leading conditions
9.4 Leading developers
9.5 Clinical trials
9.6 Molecules
9.7 Sponsors
9.8 Trial end dates
10. Gynaecology/obstetrics
10.1 Summary
10.2 EU/US designated drugs
10.2.1 Orphan drugs designated in 2016–2017
10.3 Leading conditions
10.4 Leading developers
10.5 Clinical trials
10.5.1 Molecules
10.5.2 Sponsors
10.5.3 Trial end dates
11. Haematology
11.1 Summary
11.2 EU/US designated drugs
11.2.1 Orphan drugs designated in 2016–2017
11.3 Leading conditions
11.4 Leading developers
11.5 Clinical trials
11.5.1 Molecules
11.5.2 Sponsors
11.5.3 Trial end dates
12. Hormone therapy
12.1 Summary
12.2 EU/US designated drugs
12.2.1 Orphan drugs designated in 2016
12.3 Leading conditions
12.4 Leading developers
12.5 Clinical trials
12.5.1 Molecules
12.5.2 Sponsors
12.5.3 Trial end dates
13. Immunology
13.1 Summary
13.2 EU/US designated drugs
13.2.1 Orphan drugs designated in 2016–2017
13.3 Leading conditions
13.4 Leading developers
13.5 Clinical trials
13.5.1 Molecules
13.5.2 Sponsors
13.5.3 Trial end dates
14. Inflammatory disorders
14.1 Summary
14.2 EU/US designated drugs
14.2.1 Orphan drugs designated in 2016–2017
14.3 Leading conditions
14.4 Clinical trials
14.4.1 Molecules
14.4.2 Sponsors
14.4.3 Trial end dates
15. Metabolic disorders
15.1 Summary
15.2 EU/US designated drugs
15.2.1 Orphan drugs designated in 2016–2017
15.3 Leading conditions
15.4 Leading developers
15.5 Clinical trials
15.5.1 Molecules
15.5.2 Sponsors
15.5.3 Trial end dates
16. Musculoskeletal
16.1 Summary
16.2 EU/US designated drugs
16.2.1 Orphan drugs designated in 2016–2017
16.3 Leading conditions
16.4 Leading developers
16.5 Clinical trials
16.5.1 Molecules
16.5.2 Sponsors
16.5.3 Trial end dates
17. Neuroscience
17.1 Summary
17.2 EU/US designated drugs
17.2.1 Orphan drugs designated in 2016–2017
17.3 Leading conditions
17.4 Leading developers
17.5 Clinical trials
17.5.1 Molecules
17.5.2 Sponsors
17.5.3 Trial end dates
18. Oncology
18.1 Summary
18.2 EU/US designated drugs
18.2.1 Orphan drugs designated in 2016–2017
18.3 Leading conditions
18.4 Leading developers
18.5 Clinical trials
18.5.1 Molecules
18.5.2 Sponsors
18.5.3 Trial end dates
19. Ophthalmology
19.1 Summary
19.2 EU/US designated drugs
19.2.1 Orphan drugs designated in 2016–2017
19.3 Leading conditions
19.4 Leading developers
19.5 Clinical trials
19.5.1 Molecules
19.5.2 Sponsors
19.5.3 Trial end dates
20. Poisoning
20.1 Summary
20.2 EU/US designated drugs
20.2.1 Orphan drugs designated in 2016–2017
20.3 Leading conditions
20.4 Leading developers
20.5 Clinical trials
21. Respiratory
21.1 Summary
21.2 EU/US designated drugs
21.2.1 Orphan drugs designated in 2016–2017
21.3 Leading conditions
21.4 Leading developers
21.5 Clinical trials
21.5.1 Molecules
21.5.2 Sponsors
21.5.3 Trial end dates
22. Transplantation
22.1 Summary
22.2 EU/US designated drugs
22.2.1 Orphan drugs designated in 2016
22.3 Leading conditions
22.4 Leading developers
22.5 Clinical trials

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