The Orphan Drug Fact File 2019

Maximum Purchase:
1 unit
Publication Date:
September 2019
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The Orphan Drugs Pipeline: Everything you need to know about current contenders and new opportunities

By 2024, orphan drugs are expected to account for one fifth of worldwide prescription drug sales—some $242 billion in potential revenue. This huge growth area is a hive of activity for investors, big pharma, specialist developers and biotech companies all vying for their share. High-fliers such as Celgene's Revlimid, Merck & Co.'s Keytruda, and Bristol-Myers Squibb's Opdivo are already blazing a trail. So what high-potential opportunities are there for investors and drug developers now poised and ready to take the leap?

The Orphan Drug Fact File (2019) offers the most comprehensive independent analysis of orphan drug research activity available today. This detailed report delves into 11 therapy areas and reveals designation status, conditions, developers and clinical trial activity.

Key questions answered

  • What are the incentives for orphan drug development in the US and Europe?
  • What activity is happening in a specific disease area?
  • Which conditions are being targeting and what trials are underway?
  • What are the trial sizes and end dates by disease area?

What's included?

As well as a comprehensive report packed with charts, graphs and commentary, clients also receive the associated MS Excel™ data set. The report features the latest information from (but not limited to) the EMA, the FDA, the US National Organization for Rare Diseases,, pharmaceutical company pipelines and reports,, various organisations and patient support groups that focus on rare diseases, and the World Health Organization (WHO).

It is provided in two formats:

  • Analysis Report by therapy area: 160 charts and 102 tables plus explanatory text on leading conditions, key developers and clinical trials (including molecules, sponsors and end dates)
  • MS Excel data file: Drill down into the data to conduct your own analysis and incorporate it in your internal reports

Therapy areas covered

  • Anti-infectives
  • Cardiovascular disorders
  • Haematology (blood and blood clotting)
  • Immunology
  • Inflammatory disorders
  • Metabolic disorders
  • Musculoskeletal disorders
  • Neuroscience
  • Oncology
  • Ophthalmology
  • Respiratory disorders

How to use this report

  • Build understanding of orphan drug market dynamics and designation trends
  • Identify niche orphan disease opportunities and potential investment areas
  • Profile potential acquisition or collaboration opportunities
  • Plan orphan drug clinical research programmes
  • Monitor trends in FDA and EMA orphan drug designation
  • Track the activity of key competitors in specific disease areas

Why buy now?

With huge market growth predicted, competition in orphan drugs is intensifying along with the associated risks. In such a high stakes market, it pays to stay firmly on top of what's happening and know who's involved. This report brings together the latest information from a host of sources in order to shine a light on the orphan drugs pipeline as it stands today. Use it to support a case for investment, identify new opportunities, and plan ahead—knowing what the bigger picture looks like and who you're up against.

Why FirstWord ChartViews reports are different

  • Relevant: Organised so you can go straight to the information that's most important to you
  • Easy to navigate: Packed with charts and data tables to help you see the full picture
  • Comprehensive: We've collated all the latest information—so you don't have to