Rare Diseases Expertise

Rare Diseases:

Rare diseases are caused by genetic defects, environmental exposure during pregnancy or later in life in combination with genetic susceptibility.

As the number of rare diseases is huge and people affected with any particular rare disease is relatively small, a host of challenges surfaces and complicates the development of safe and effective drugs, biologics, and medical devices to prevent, diagnose, treat, or cure these conditions.

Rare Diseases Facts:

•  There are an estimated 6,000 rare diseases currently recognized in the US, and 8,000 in Europe
•  A disease or disorder is defined as rare in Europe when it affects fewer than 1 in 2000 and fewer than 200,00 Americans at any given time
•  Approx. 30Mn people in Europe and 25-30Mnin US may have affected by any of rare disease
•  80% of rare diseases are due to genetic origins while others are the result of infections (bacterial or viral), allergies and environmental causes
•  Since 1983, more than 350 medicines have been approved to treat rare diseases
•  Approx. 460 medicines for rare diseases are in late stages of the pipeline, either in clinical trials or awaiting FDA review

Key Questions:

•  Which rare diseases are companies targeting, and what are the recent alliances that have been established?
•  How big is market for any clinical condition of rare diseases and how it has been changing over the period of time?
•  How can companies leverage current initiatives to identify and develop commercially and clinically compelling drugs for rare diseases?
•  In developing drugs for rare diseases, what have been the approachesadopted by companies?Which offer the greatest potential for future success in this arena?
•  What resources are available to developers that seek to enter or grow their presence in rare diseases?

How We Help:

•  Assessment of the market dynamics and opportunity for a key drug in the market
•  Disease-area expert review encompasses a broad range of primary research
•  Evaluation of the likely positioning, receptivity, uptake and potential of emerging products
•  We cover KOL insight specific to rare disease

Business Impact:

•  Enabled client to gauge competitive landscape to understand the growing interest in developing new treatments for rare diseases
•  Opportunity assessment and identify the key challenges that are presented by low patient prevalence
•  Enabled client to review the regulatory environment governing the development of orphan drugs in different countries

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