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Charting the Orphan Drug Development Pipeline

Product Code:
s_596200829
Publication Date:
April 2017
Format:
PDF
Price:
€675

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 >

 

Executive Summary

This FirstWord Dossier report provides a comprehensive review of the orphan drug development pipeline, focusing on those products that have been granted orphan drug designation by the US Food and Drug Administration (FDA) and/or European Medicines Agency (EMA). The information for this report was gathered mainly from publically available sources of information, including (but not limited to) the EMA, the FDA, the US National Organization for Rare Diseases, Orpha.net, pharmaceutical company pipelines and reports, ClinicalTrials.gov, various organisations and patient support groups that focus on rare diseases, and the World Health Organization (WHO). Information has been organised by the authors into the following therapy areas:

  • 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

Where possible, classification into therapy areas has followed the WHO’s Anatomical Therapy Classification (ATC) system. For novel developmental drugs that are not yet included on the ATC list, we have been guided by rare disease organisations. Please note that where a rare disease affects more than one system in the body, drugs are classified into the therapy area that is considered the most appropriate. For example, while cystic fibrosis can affect multiple systems and organs depending on the gene mutations that cause it, it has been classified as respiratory due to the build up of mucus in the lungs being a major symptom that adversely affects quality of life in a significant proportion of people who suffer from this condition. Data for clinical trials have been collected for active studies in Phases I, II and III that are industry sponsored. These are generally studies that have been designed to collect the data required by regulatory authorities for marketing authorisation. Additional studies may be under way, including long-term follow-up studies, patient access studies or investigator-initiated trials that have not been allocated a development Phase on the Clinicaltrials.gov website; these have generally not been included in this report.

©Doctor’s Guide Publishing Limited. This executive summary is provided for commercial evaluation purposes only. It can be shared with colleagues for this purpose but cannot be reproduced, extracted or published without the express permission of the publisher.

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

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

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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