Navigation Links
Drug Discovery in Rare Diseases
Date:9/20/2012

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

 

Drug Discovery in Rare Diseases

http://www.reportlinker.com/p0968548/Drug-Discovery-in-Rare-Diseases.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Drug_Discovery_and_Development

 

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?

 

 

 

EXECUTIVE SUMMARY•Key findings

 

The growing interest in rare diseases•Summary

•Introduction

•What is a rare disease?

•Why target rare diseases?

 

Characterization of rare diseases•Summary

•Overview

•Rare diseases

•Very rare diseases

•Ultra rare diseases

•Useful resources

 

Orphan drug status•Summary

•Introduction

•Legislative distinctions - US

- Europe

- Japan

- Other markets

 

•Tax benefits

•Patient population subsetting

•Similarity

•Summary of key considerations

 

Choosing rare diseases to target•Summary

•Introduction

•Key issues

•Commercial potential - Prevalence

- Geographic distribution

- Disease understanding

- Available experts

- Current treatments

- What is similar?

 

•Conclusion

 

Preclinical development•Summary

•Introduction

•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

 

•Conclusions

 

Clinical issues•Summary

•Introduction

•Clinical trial design

•Access to patients - Geographic distribution

- Identifying patients

 

•Conclusions

 

Commercial considerations•Summary

•Introduction

•Identifying commercially promising opportunities - Prevalence

- Current treatments

- Competitive position

 

•Case studies - (Untitled sub-section)

- Gaucher disease

 

•Conclusions

 

Conclusions•Summary

•Introduction

•Checklist to consider

•Corporate strategies

•Conclusions

 

Appendix•Scope

•Methodology

•Glossary/abbreviations

•Bibliography/references

 

 

TABLES

•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

 

 

FIGURES

•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
Reportlinker
Email: nicolasbombourg@reportlinker.com
US: (805)652-2626
Intl: +1 805-652-2626

 


'/>"/>
SOURCE Reportlinker
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. ESTEVE Fosters R & D by Transferring its Center for Drug Discovery to the Science Park of Barcelona (PCB)
2. Initiating Rare Disease Drug Discovery Projects
3. Outsourcing in Drug Discovery: The Contract Research Organization (CRO) Market, 5th Edition
4. AstraZeneca And The Broad Institute Partner To Advance Discovery Of Antibacterial And Antiviral Agents
5. Drug Discovery Technologies, BCC Research
6. Antigen Discovery Inc. Awarded Phase I SBIR Grant to Develop a Fully Integrated Microfluidic Point of Care Diagnostic Device
7. Regulus Therapeutics Forms Strategic Alliance for Discovery, Development and Commercialization of microRNA Therapeutics with AstraZeneca
8. Zenobia Therapeutics Announce A Partnership With The Alzheimers Drug Discovery Foundation
9. Antigen Discovery, Inc. Awarded Phase II SBIR Grant to Identify Biomarkers Associated with Protection against Malaria
10. Omeros Announces Discovery in MASP-2 Program
11. Discovery Labs Receives US Patent for Method to Promote Mucus Clearance with Pulmonary Surfactant
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/14/2017)... -- ivWatch LLC, a medical device company focused on improving ... pleased to announce it was the Bronze Winner last ... Equipment at the 2017 Medical Design Excellence Awards (MDEAs), ... award was presented by Medical Device and Diagnostics Industry ... New York during MD&M East, the ...
(Date:6/13/2017)... 13, 2017 Zimmer Biomet Holdings, Inc. (NYSE and ... that the U.S. Food and Drug Administration (FDA) has notified ... relating to its Zhejiang, China manufacturing ... "The successful clearance of the Warning Letter related to ... is a measure of the progress we have made in ...
(Date:6/10/2017)... , June 9, 2017  Shane K. Burchfield, DPM, is recognized ... excellence as a Podiatrist in Alabama . ... Family First Foot Care. He brings over 20 years of experience, ... management and healthcare, to his role. ... Care, PC is pleased to welcome you to his ...
Breaking Medicine Technology:
(Date:6/25/2017)... ... ... June is Men’s Health Month and the focus is on prostate cancer. Second ... U.S. and the third most common cause of cancer related death today; lung cancer remains ... be diagnosed with prostate cancer during his lifetime. Those at highest risk are ...
(Date:6/24/2017)... ... 2017 , ... The weather is heating up and the days are getting ... be aware that the summer months provide more than warmer temperatures that are perfect ... be negatively affected from direct exposure to the sun. When it comes to the ...
(Date:6/23/2017)... ... ... By scoring 100% for fiscal management and accountability, the Arthritis National Research ... Charity Navigator, validating ANRF's work as a top charity in America. , This achievement ... earns ANRF a spot on their “ 10 Charities Worth Watching ” list as ...
(Date:6/23/2017)... ... 2017 , ... Dr. Ran Y. Rubinstein , a ... offering three new minimally invasive procedures to patients who want a younger and ... Rubinstein is excited to bring microneedling, microneedling facials, and platelet rich plasma (PRP) ...
(Date:6/23/2017)... ... June 23, 2017 , ... The Aesthetic Channel has recently highlighted Dr. ... come up with a proprietary technique that he calls the AuraLyft Facelift. ... For all ages, patients can expect to look refreshed, rejuvenated, and revitalized. , ...
Breaking Medicine News(10 mins):