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:2/11/2016)... 2016  Governor Andrew M. Cuomo today announced a ... throughout Western New York . This ... Polytechnic Institute, includes a major expansion of Athenex,s North ... Buffalo , as well as the creation of ... Dunkirk . The combined projects are expected ...
(Date:2/11/2016)... , Feb. 11, 2016 AAIPharma ... provider of custom manufacturing and development services for ... sterile fill-finish capabilities and capacity in its ... in demand has driven several recent investments. ... it had one filling line with small-scale lyophilization. ...
(Date:2/11/2016)... -- Proliant Biologicals is proud to announce the opening of ... The facility is located on the North Island of ... Boone , Iowa.  The equipment design ... in the U.S. facility, with critical processing systems coming ... --> Boone , Iowa.  The ...
Breaking Medicine Technology:
(Date:2/12/2016)... ... ... Erlanger Agency has announced a new partnership in its ongoing community involvement ... the fight against breast cancer, fundraising for a local woman named Carmen, who is ... is a loving single mother of two boys who also serves as caregiver for ...
(Date:2/12/2016)... ... 2016 , ... Donor Network West, the organ procurement organization that heals lives ... San Ramon Regional Medical Center. Under the collaboration, the first of its kind, specific ... a more certain time frame for donor families for the recovery of organs. ...
(Date:2/12/2016)... ... February 12, 2016 , ... For Coast Dental dentist Everet Lake, ... and dental assistant Terrell Moore shortly before 7 a.m. to volunteer at Friday’s Dentistry ... donating their time and skills to help hundreds of uninsured and underinsured people receive ...
(Date:2/12/2016)... , ... February 12, 2016 , ... ... Cancer Care.” , The print component of “Revolutionizing Cancer Care” is ... New York, Washington DC/Baltimore, and Seattle, with a circulation of approximately 250,000 copies ...
(Date:2/12/2016)... ... February 12, 2016 , ... Atlantic ... for Health Plans and PBMs,” an upcoming Feb. 24 webinar that will discuss ... in big-dollar therapeutic categories, such as the $1,000-per-pill hepatitis C treatment Sovaldi and ...
Breaking Medicine News(10 mins):