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Drug Discovery in Rare Diseases

NEW YORK, Sept. 20, 2012 /PRNewswire/ -- announces that a new market research report is available in its catalogue:


Drug Discovery in Rare Diseases


In comparison with major diseases, the targeting of rare diseases poses many different challenges, necessitating consideration of bespoke R&D strategies for drug discovery efforts to be successful. This report examines the role that low disease prevalence plays in determining the most suitable R&D path.Understand the growing interest in developing new treatments for rare diseases, and why low patient numbers do not preclude commercial viability.Review the regulatory environment governing the development of orphan drugs in different countries.Identify the key challenges that are presented by low patient prevalences.Assess how the research strategy chosen can be influenced by the disease prevalence.Compare the scenarios in which repurposing of existing drugs offers advantages over the development of novel drugs, and vice versa.Approximately 7,000 rare diseases have been identified, but only a very small proportion of these are currently well treated. Orphans represent a greater proportion of all new BLAs than they do of NMEs submitted as NDAs. Most orphan approvals are not first approvals of new drugs but are new orphan indications for previously approved drugs.It is possible to obtain orphan drug designation for conditions with a total prevalence greater than that defined by legislation, but only if medically justifiable subsets can be defined with a lower (overall) prevalence. Pediatric subsets are most commonly used.In the development of new treatments for rare diseases it is less critical to seek to optimize the pharmacokinetic properties of candidates than is the case for common chronic diseases, with parenteral delivery or frequent oral dosing being much more acceptable provided that efficacy is achieved.What impact on R&D strategy does disease prevalence have on moving from rare through very rare to ultra rare diseases?Can more than one drug be a commercial success for treating rare indications, and how do drug regulators view the question of drug similarity?What are the best ways of identifying patients for recruitment into clinical trials for drugs designed to treat rare diseases?What are the requirements for a clinical candidate to treat a rare disease?What factors determine whether a small-molecule or biologic strategy is most suitable when targeting a rare disease?






The growing interest in rare diseases•Summary


•What is a rare disease?

•Why target rare diseases?


Characterization of rare diseases•Summary


•Rare diseases

•Very rare diseases

•Ultra rare diseases

•Useful resources


Orphan drug status•Summary


•Legislative distinctions - US

- Europe

- Japan

- Other markets


•Tax benefits

•Patient population subsetting


•Summary of key considerations


Choosing rare diseases to target•Summary


•Key issues

•Commercial potential - Prevalence

- Geographic distribution

- Disease understanding

- Available experts

- Current treatments

- What is similar?




Preclinical development•Summary


•Screen or repurpose? - Repurposed

- Directed approaches

- Taking advantage of orphan drug status


•Other issues - Small molecule or biological?

- Biological test models

- Requirements of a clinical candidate




Clinical issues•Summary


•Clinical trial design

•Access to patients - Geographic distribution

- Identifying patients




Commercial considerations•Summary


•Identifying commercially promising opportunities - Prevalence

- Current treatments

- Competitive position


•Case studies - (Untitled sub-section)

- Gaucher disease






•Checklist to consider

•Corporate strategies










•Table: Timeline of international orphan drug legislation, 1983–2008

•Table: Comparison of orphan drug legislation in the US, Europe, and Japan

•Table: The FDA's definitions of similarity




•Figure: US FDA orphan approvals, 2001–11 (part 1)

•Figure: US FDA orphan approvals, 2001–11 (part 2)

•Figure: Rare diseases, medical need, and disease prevalence

•Figure: Distribution of US orphan drug approvals by disease prevalence (to 2010)

•Figure: European orphan drug approvals by disease prevalence (to 2010)

•Figure: Exploiting knowledge resources for rare disease research

•Figure: The organizational relationships within the EMA pertaining to orphan drugs

•Figure: Schematic relationship between rare disease prevalence and commercial returns

•Figure: Identifying suitable patients for clinical studies

•Figure: The influence of prevalence on research strategy

•Figure: Current exploitation of rare disease space

•Figure: "Similar" approved BCR-ABL inhibitors with orphan drug status

•Figure: Similar approved endothelin receptor antagonists with orphan drug status

•Figure: Selecting a rare disease to target

•Figure: Alternative strategies to identifying new treatments for rare diseases

•Figure: Strategic pathways for identifying development candidates

•Figure: Comparison of duration and size of clinical studies in developing drugs to treat major diseases (a), and rare diseases (b)

•Figure: Number of participants in European orphan drug clinical trials

•Figure: Comparing commercial opportunities

•Figure: Strategic options for developing drugs for already targeted rare diseases

•Figure: Different approaches to treating cystic fibrosis

•Figure: GlaxoSmithKline and Pfizer; exploiting internal and external resources in rare disease research




Companies Mentioned

Amlin plc, GlaxoSmithKline Plc, Hutchison 3G UK Limited, Recordati S.p.A., Sanofi, WPP Group plc




To order this report:

Drug_Discovery_and_Development Industry: Drug Discovery in Rare Diseases


Nicolas Bombourg
US: (805)652-2626
Intl: +1 805-652-2626


SOURCE Reportlinker
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