Navigation Links
Initiating Rare Disease Drug Discovery Projects
Date:9/19/2012

NEW YORK, Sept. 19, 2012 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

Initiating Rare Disease Drug Discovery Projects

http://www.reportlinker.com/p0968583/Initiating-Rare-Disease-Drug-Discovery-Projects.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 issues that need to be considered in the earliest stages of embarking on a rare disease project.Understand why rare diseases make attractive targets for launching new drug discovery projects.Identify the key challenges that are presented by low patient prevalences.Assess how the research strategy chosen can be influenced by the disease prevalence.Understand the importance of considering issues of similarity versus other orphan drugs.Compare the scenarios in which repurposing existing drugs offers advantages over the development of novel drugs, and vice versa.While disease prevalence has an impact on the commercial viability of a new treatment, returns are achievable even for ultra rare conditions. Rare diseases can provide sufficient opportunities for commercialization of multiple therapeutic agents. This is the case even for some ultra rare conditions such as Gaucher disease and Fabry disease.Both clinical experts and patient groups provide valuable resources in understanding a rare disease and in identifying potential patients for clinical studies.Many opportunities remain to develop treatments for untreated rare diseases. However, ultra rare diseases with high infant mortality present major challenges.Can more than one drug be a commercial success for treating rare indications?What are the best ways of identifying patients for recruitment into clinical trials?Why it is necessary to consider logistic issues in planning clinical studies?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?

 

About the author

•Disclaimer

EXECUTIVE SUMMARY

•Choosing rare diseases to target

•Preclinical development

Introduction

•Key points regarding rare disease drug discovery

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

Appendix

•Scope

•Methodology

•Glossary/abbreviations

•Bibliography/references

 

FIGURES

•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

 

Companies Mentioned

Amlin plc, Diageo plc, GlaxoSmithKline Plc, Hutchison 3G UK Limited, Informa plc, Sanofi, WPP Group plc

 

To order this report:

Drug_Discovery_and_Development Industry: Initiating Rare Disease Drug Discovery Projects

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. Clinical Trial at Evergreen Healthcare Shows Intestinal Gel Reduces Symptoms of Advanced Parkinsons Disease
2. New post-hoc analyses examined Neupro® effects on pain, depressed mood, apathy and fatigue in patients with Parkinsons disease
3. New Clinical Endocrinology Guidelines Support Lp-PLA2 Measurement For Risk Assessment of Coronary Artery Disease
4. Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
5. Chef Spike Mendelsohn Dishes on How to Turn Down the Heat on Acid Reflux Disease
6. New Published Guidelines to Help Physicians Diagnose and Treat Lupus Kidney Disease
7. The ALS Therapy Development Institute Partners with Neurotune to Investigate Potential Treatments for Motor Neuron Disease
8. Rigel Advances Asthma Programs: Two Inhaled Drug Candidates Take Aim at Acute and Chronic Lung Disease
9. Ceregene Parkinsons Disease Study Demonstrates Long-term Neurturin Expression and Enhanced Dopamine Activity 4 Years Following Delivery of CERE-120 (AAV2-neurturin)
10. UCB Proudly Reinforces Long-Standing Commitment to Crohns Disease Community at Digestive Disease Week 2012
11. Receptos to Deliver Scientific Presentation at Digestive Disease Week 2012 about the Therapeutic Role of RPC1063 in Inflammatory Bowel Disease
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/27/2017)... , March 27, 2017  Sanderling Ventures, ... by Ethicon, a division of Johnson & Johnson. Torax ... for the treatment of gastro-esophageal reflux disease (GERD). ... augmentation (MSA) technology and the procedure is currently ... Torax Medical was founded by Sanderling ...
(Date:3/27/2017)... March 27, 2017 FinancialBuzz.com News Commentary  ... Based on New Frontier ... states where cannabis is legal will generate $655 million in taxes ... from cannabis specific taxes, such as Washington State,s ... be earned from state sales taxes that are applied on all ...
(Date:3/27/2017)... /PRNewswire/ - INVICTUS MD STRATEGIES CORP. ("Invictus MD" or ... is pleased to announce it has received conditional approval ... TSX Venture Exchange.  Receiving the conditional ... significant achievements for Invictus-MD. Some of which include: ... Laboratories Inc. ("AB Labs"), a Licensed Producer under the ...
Breaking Medicine Technology:
(Date:3/28/2017)... ... March 28, 2017 , ... The Executives, Staff & Clients ... to for the Toys for Tots Literacy Campaign at their Semi-Annual Graduation and Fundraiser ... $70 billion, the U.S. ranks at number 14 internationally in literacy. Statistically, a direct ...
(Date:3/28/2017)... ... March 28, 2017 , ... Public relations pros work hard ... variety of business channels. , While many results are clear, much of PR is ... , When it comes to measurement, firms should always take an all-inclusive approach ...
(Date:3/28/2017)... ... March 28, 2017 , ... Thank you to all who attended Capio Partners ... This event was exclusive to providers and offered an opportunity to collaborate and network ... the Manchester Grand Hyatt where attendees gathered for a lively discussion on trends and ...
(Date:3/27/2017)... ... March 27, 2017 , ... ... braces. People who want straight teeth without the extensive time commitment and aesthetic ... FL, without acquiring a referral. A custom-designed series of virtually invisible aligners are ...
(Date:3/27/2017)... ... 2017 , ... This is the second major release since ... 2014. It is the culmination of collaborative efforts by members of the OSEHRA ... Member Zato Health co-funded the ONC certification and provided key technical support throughout ...
Breaking Medicine News(10 mins):