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Charting the Orphan Drug Development Pipeline

Product Code:
Publication Date:
April 2017

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?

Easily contrast and compare 3,868 orphan drug pipeline candidates >

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.

Critically identify orphan drug development pressure points and opportunity areas >

Two Extensive Information and Data Resources Included

1. 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.

2. 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

Get these features now >

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.

This report and the accompanying MS Excel spreadsheet are available now – place your order today >

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?

Get answers to your orphan drug development questions >

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


  • 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

This critical intelligence is available for immediate download — order your copy now

Need more information? Contact a consultant for an executive summary and sample pages from the report.


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