Summary

Dermatology is a highly diverse therapy area, in terms of severity and clinical presentation, which deals with diseases of the skin, hair and nails. Whilst an orphan disease is a disease that is neglected in terms of research and funding and in the US, a disease is considered to be rare if it affects fewer than 200,000 people.

Many orphan and rare dermatology 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. However, the current therapeutics market is a highly genericized therapy area and there exists a significant unmet need for more efficacious and safer treatment options that treat the underlying causes of disease as opposed to managing the symptoms.

This report covers all orphan and rare dermatology disorders, but there is a particular focus on six key diseases, systemic sclerosis (scleroderma), alopecia, epidermolysis bullosa, pemphigus vulgaris, vitiligo and cutaneous ...

Summary

Orphan and rare dermatological diseases are considerably diverse in terms of pathophysiology, clinical presentation and disease severity. Many of these conditions are extremely debilitating and can even be life-threatening in some cases.

Disease visibility can also have a profoundly negative impact on patient confidence. 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 disorder...