Charting the Orphan Drug Development Pipeline

March 2017 | | ID: C1EEC0573FCEN
FirstWord

Abstracts

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

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

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Contents

1. RESEARCH OBJECTIVES AND METHODOLOGY

2. OVERVIEW

2.1 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