Pharmiweb ChannelsAll | PharmaCo | Clinical Research | R&D/BioTech | Sales/Mktg | Healthcare | Recruitment | Pharmacy | Medical Comms RSS Feed RSS Feeds


Press Release

Global Orphan and Rare Dermatological Diseases Markets, 2018 - Neglected Therapy Area with High Levels of Genericization

Research and Markets
Posted on: 15 Oct 18
Global Orphan and Rare Dermatological Diseases Markets, 2018 - Neglected Therapy Area with High Levels of Genericization

PR Newswire

DUBLIN, Oct. 15, 2018

DUBLIN, Oct. 15, 2018 /PRNewswire/ --

The "Orphan and Rare Dermatological Diseases - First-in-Class Therapies Demonstrate Potential Disease-Modifying Effects in Areas of High Unmet Need Such as Epidermolysis Bullosa" report has been added to's offering.

Dermatology is a highly diverse therapy area that deals with diseases of the skin, hair and nails. Over 3,000 distinct dermatological conditions are thought to exist, ranging from the rare diseases such as systemic sclerosis (scleroderma), an autoimmune disorder, to very common conditions such as acne vulgaris. The scope of dermatological disorders is diverse in terms of severity and clinical presentation. Many of these disorders are associated with significant quality of life impairments, particularly if the disease is insufficiently controlled.

In particular, disease visibility can have a profoundly negative impact on patient confidence. As many dermatological conditions are chronic in nature, these symptoms can also increase the risk of developing psychosocial comorbidities - such as depression and anxiety - over time.

Unmet need within the Orphan and Rare Dermatology market is extremely high with some diseases having no effective treatments. The market is highly genericized and dominated to a large extent by products acting on hormones and their receptors. Most of these are corticosteroids and are used for symptom management with no disease-modifying effects. There is a significant unmet need for more efficacious disease-modifying treatments and safer treatment options across Orphan and Rare dermatological diseases, as physicians often cite poor efficacy, low patient compliance and problematic safety profiles as issues associated with the long-term use of available treatments.

This report covers all orphan and rare dermatology disorders, but there is a particular focus on six key indications, Systemic Sclerosis (Scleroderma), Alopecia, Epidermolysis Bullosa (EB), Pemphigus Vulgaris, Vitiligo and Cutaneous Lupus Erythematosus (CLE), as these conditions have the largest pipelines within the therapy area.


  • There is a strong need for innovative new therapies across the orphan and rare dermatology market. How are orphan and rare dermatological diseases currently managed? What are the greatest unmet needs within this market?
  • There are 262 pipeline products in development across all orphan and rare dermatological diseases. How does the composition of the pipeline compare with that of the existing market? Which molecular targets are most frequently acted upon by pipeline drugs? How do products in development for the key indications differ in terms of molecule type?
  • Over one-third of pipeline products with a disclosed molecular target are first-in-class. How does the proportion of first-in-class products in development differ in terms of stage of development, molecule type and molecular target class? Which are the most promising first-in-class targets?
  • The deals landscape has become more active in recent years. Which indications and products have attracted the highest deal values?

Reasons to buy

This report will allow you to:

  • Appreciate the current clinical and commercial landscapes by considering disease pathogenesis, etiology, epidemiology, symptoms, diagnosis and treatment options.
  • Visualize the composition of the market in terms of dominant molecule types and molecular targets, highlighting what the current unmet needs are and how they can be addressed. This knowledge allows a competitive understanding of gaps in the current market.
  • Recognize innovative pipeline trends by analyzing therapies by stage of development, molecule type and molecular target.
  • Assess the therapeutic potential of first-in-class molecular targets. Using a proprietary matrix, first-in-class targets have been assessed and ranked according to clinical potential. Promising first-in-class targets have been reviewed in greater detail.
  • Consider first-in-class pipeline products with no prior involvement in licensing and co-development deals that may represent potential investment opportunities.

Key Topics Covered:

1 Table of Contents

1.1 List of Tables
1.2 List of Figures

2 Executive Summary

2.1 Neglected Therapy Area with High Levels of Genericization
2.2 Diverse Pipeline Demonstrates Potential to Address Unmet Needs
2.3 Increase in Strategic Consolidations in Recent Years

3 The Case for Innovation

3.1 Growing Opportunities for Biologic Products
3.2 Diversification of Molecular Targets
3.3 Innovative First-in-Class Product Developments Remain Attractive
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Innovation
3.5 Sustained Innovation in Orphan and Rare Dermatological Diseases

4 Clinical and Commercial Landscape

4.1 Overview of the Dermatology Market
4.2 Overview of Orphan and Rare Diseases
4.3 Symptoms
4.4 Diagnosis
4.5 Etiology
4.6 Pathophysiology
4.7 Epidemiology
4.8 Co-morbidities and Complications
4.9 Treatment
4.10 Overview of Marketed Products

5 Assessment of Pipeline Product Innovation

5.1 Overview
5.2 Pipeline by Stage of Development and Molecule Type
5.2.1 Overall Pipeline
5.2.2 Pipeline by Key Indications
5.3 Pipeline by Molecular Target
5.3.1 Overall Pipeline
5.3.2 Pipeline for Key Indications
5.4 Comparative Distribution of Programs between the Market and Pipeline by Molecular Target Class
5.5 Comparative Distribution of First-in-Class and Non-First-in-Class Pipeline Programs by Molecular Target Class
5.6 Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Programs by Stage of Development, Molecule Type and Molecular Target Class
5.7 Ratio of First-in-Class Programs to First-in-Class Molecular Targets by Stage of Development, Molecule Type and Molecular Target Class
5.8 List of All Pipeline Programs

6 Signaling Network, Disease Causation and Innovation Alignment

6.1 Complexity of Signaling Networks in Orphan and Rare Dermatological Diseases
6.2 Signaling Pathways and First-in-Class Molecular Target Integration
6.3 First-in-Class Molecular Target Matrix Assessment

7 First-in-Class Molecular Target Evaluation

7.1 Pipeline Programs Targeting Collagen type XVII (COL17A1)
7.2 Pipeline Programs Targeting Collagen type VII, alpha 1 (COL7A1)
7.3 Pipeline Programs Targeting Interleukin 2 Receptor Subunit Beta (IL2RB)
7.4 Pipeline Programs Targeting Desmoglein 1 (DSG1)
7.5 Pipeline Programs Targeting Nacht LRR and PYD Domains Containing Protein 3 (NLRP3)
7.6 Pipeline Programs Targeting Interleukin 3 Receptor Subunit Alpha (IL3RA)
7.7 Pipeline Programs Targeting Non-Receptor Tyrosine Protein Kinase TYK2 (TYK2)
7.8 Pipeline Programs Targeting Interleukin 6 Receptor Subunit Beta (IL6ST)
7.9 Pipeline Programs Targeting Potassium Voltage-Gated Channel Subfamily A Member 3 (KCNA3)
7.10 Pipeline Programs Targeting Kallikrein 7 (KLK7)
7.11 Pipeline Programs Targeting Eotaxin (CCL11)
7.12 Pipeline Programs Targeting Interleukin 2 (IL2)

8 Strategic Consolidations

8.1 Industry-wide First-in-Class Deals
8.2 Licensing Deals
8.2.1 Deals by Region, Value and Year
8.2.2 Deals by Key Indication and Value
8.2.3 Deals by Stage of Development and Value
8.2.4 Deals by Molecule Type and Molecular Target
8.2.5 List of Deals with Disclosed Deal Values
8.3 Co-development Deals
8.3.1 Deals by Region, Value and Year
8.3.2 Deals by Indication and Value
8.3.3 Deals by Stage of Development and Value
8.3.4 Deals by Molecule Type and Molecular Target
8.3.5 List of Deals with Disclosed Deal Values
8.4 First-in-Class Programs with and without Prior Licensing or Co-development Deal Involvement

9 Appendix

For more information about this report visit

Did you know that we also offer Custom Research? Visit our Custom Research page to learn more and schedule a meeting with our Custom Research Manager.

Media Contact:

Research and Markets
Laura Wood, Senior Manager

For E.S.T Office Hours Call +1-917-300-0470
For U.S./CAN Toll Free Call +1-800-526-8630
For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907
Fax (outside U.S.): +353-1-481-1716

View original content:

SOURCE Research and Markets

PR Newswire

Last updated on: 15/10/2018

Site Map | Privacy & Security | Cookies | Terms and Conditions is Europe's leading industry-sponsored portal for the Pharmaceutical sector, providing the latest jobs, news, features and events listings.
The information provided on is designed to support, not replace, the relationship that exists between a patient/site visitor and his/her physician.