1. Market Research
  2. > Pharmaceutical
  3. > Therapy Market Trends
Charting the Orphan Drug Development Pipeline

Charting the Orphan Drug Development Pipeline

  • April 2017
  • ID: 4883739
  • Format: PDF
  • By Firstword Pharma


Table of Contents

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

Get Industry Insights. Simply.

  • Latest reports & slideshows with insights from top research analysts
  • 150+ Million searchable statistics with tables, figures & datasets
  • More than 25,000 trusted sources
  • Single User License — provides access to the report by one individual.
  • Department License — allows you to share the report with up to 5 users
  • Site License — allows the report to be shared amongst all employees in a defined country
  • Corporate License — allows for complete access, globally.
Ahmad helps you find the right report:

The research specialist advised us on the best content for our needs and provided a great report and follow-up, thanks very much we shall look at ReportLinker in the future.

Kate Merrick

Global Marketing Manager at
Eurotherm by Schneider Electric

We were impressed with the support that ReportLinker’s research specialists’ team provided. The report we purchased was useful and provided exactly what we want.

Category Manager at

ReportLinker gave access to reliable and useful data while avoiding dispersing resources and spending too much time on unnecessary research.

Executive Director at
PwC Advisory

The customer service was fast, responsive, and 100% professional in all my dealings (...) If we have more research needs, I'll certainly prioritize working with ReportLinker!

Scott Griffith

Vice President Marketing at
Maurice Sporting Goods

The research specialist provided prompt, helpful instructions for accessing ReportLinker's product. He also followed up to make sure everything went smoothly and to ensure an easy transition to the next stage of my research

Jessica P Huffman

Research Associate at
American Transportation Research Institute

Excellent customer service. Very responsive and fast.

Director, Corporate Strategy at

I reached out to ReportLinker for a detailed market study on the Air Treatment industry. The quality of the report, the research specialist’s willingness to solve my queries exceeded my expectations. I would definitely recommend ReportLinker for in-depth industry information.

Mariana Mendoza

Global Platform Senior Manager at
Whirlpool Corporation

Thanks! I like what you've provided and will certainly come back if I need to do further research works.

Bee Hin Png

CEO at
LDR Pte Ltd

The research specialist advised us on the best content for our needs and provided a great report and follow-up, thanks very much we shall look at ReportLinker in the future.

Kate Merrick

Global Marketing Manager at
Eurotherm by Schneider Electric

  • How we can help
    • I am not sure if the report I am interested in will fulfill my needs. Can you help me?
    • Yes, of course. You can call us at +33(0) 4 37 65 17 03 or drop us an email at researchadvisor@reportlinker.com to let us know more about your requirements.
    • We buy reports often - can ReportLinker get me any benefits?
    • Yes. Set up a call with a Senior Research Advisor to learn more - researchadvisor@reportlinker.com or +33(0) 4 37 65 17 03.
    • I have had negative experiences with market research reports before. How can you avoid this from happening again?
    • We advise all clients to read the TOC and Summary and list your questions so that we can get more insight for you before you make any purchase decision. A research advisor will accompany you so that you can compare samples and reports from different sources, and choose the study that is right for you.

  • Report Delivery
    • How and when I will receive my Report?
    • Most reports are delivered right away in a pdf format, while others are accessed via a secure link and access codes. Do note that sometimes reports are sent within a 12 hour period, depending on the time zones. However, you can contact us to escalate this. Should you need a hard copy, you can check if this option is offered for the particular report, and pay the related fees.
  • Payment conditions
    • What payment methods do you accept?
      1. Credit card : VISA, American Express, Mastercard, or
      2. You can download an invoice to pay by wire transfer, check, or via a Purchase Order from your company, or
      3. You can pay via a Check made out in US Dollars, Euros, or British Pounds for the full amount made payable to ReportLinker
    • What are ReportLinker’s Payment Terms?
    • All payments must normally be submitted within 30 days. However, you can let us know if you need extended time.
    • Are Taxes and duties included?
    • All companies based in France must pay a 20% tax per report. The same applies to all individuals based in the EU. All EU companies must supply their VAT number when purchasing to avoid this charge.
    • I’m not satisfied. Can I be refunded?
    • No. Once your order has been processed and the publisher has received a notification to send you the report, we cannot issue any refund or cancel any order. As these are not ‘traditional’ products that can be returned, reports that are dispatched are considered to be ‘consumed’.
  • User license
    • The license that you should acquire depends on the number of persons that need to access the report. This can range from Single User (only one person will have the right to read or access the report), or Department License (up to 5 persons), to Site License (a group of persons based in the same company location), or Corporate License (the entire company personnel based worldwide). However, as publishers have different terms and conditions, we can look into this for you.
Purchase Reports From Reputable Market Research Publishers
Endometriosis - Pipeline Review, H2 2019

Endometriosis - Pipeline Review, H2 2019

  • $ 2000
  • November 2019

Endometriosis - Pipeline Review, H2 2019SummaryThis latest Pharmaceutical and Healthcare disease pipeline guide Endometriosis - Pipeline Review, H2 2019, provides an overview of the Endometriosis (Women’s ...

Choroidal Neovascularization - Pipeline Review, H2 2019

Choroidal Neovascularization - Pipeline Review, H2 2019

  • $ 2000
  • November 2019

Choroidal Neovascularization - Pipeline Review, H2 2019SummaryThis latest Pharmaceutical and Healthcare disease pipeline guide Choroidal Neovascularization - Pipeline Review, H2 2019, provides an overview ...

Mild Cognitive Impairment - Pipeline Review, H2 2019

Mild Cognitive Impairment - Pipeline Review, H2 2019

  • $ 2000
  • November 2019

Mild Cognitive Impairment - Pipeline Review, H2 2019SummaryThe latest Pharmaceutical and Healthcare disease pipeline guide Mild Cognitive Impairment - Pipeline Review, H2 2019, provides an overview of ...


Reportlinker.com © Copyright 2019. All rights reserved.

ReportLinker simplifies how Analysts and Decision Makers get industry data for their business.

Make sure you don’t miss any news and follow us on