Orphan drugs

Drug Discovery in a Multi-Omics World By Mike Furness Collectively rare diseases affect millions of people worldwide, but the number of people with any one condition is very small. Finding a cure is difficult as the market is not large enough to justify a big investment in drug development. This is all about to change […]

Therapeutic Drug Repurposing, Repositioning and Rescue Part IV: Financial model and analysis By David M. Kauppi and Dr Stephen Naylor There is a growing consensus that Drug Repurposing, Repositioning and Rescue (DRPx) impacts all stakeholders involved in the therapeutic drug sector. In part, this is due to the fact that the pharmaceutical industry accrues ~25% […]

Therapeutic Drug Repurposing, Repositioning and Rescue Part III: Market exclusivity using Intellectual Property and regulatory pathways By Dr Stephen Naylor, David M. Kauppi and Judge M. Schonfeld There is a growing consensus that Drug Repurposing, Repositioning and Rescue (DRPx) can impact the prescription drug industry for all concerned stakeholders. In part this is due to […]

BioPharma and CROs Collaborating In Early Drug Discovery By Dr John Montana and Cornelis E.C.A. Hop BioPharma and CROs are riding the latest wave – discovery research – together with a vigour and gusto that might have seemed implausible five years ago. How these collaborations are structured, and how well they function, can vary widely, […]

Therapeutic Drug Repurposing, Repositioning and Rescue Part II: Business Review By Dr Stephen Naylor, David M. Kauppi and Judge M. Schonfeld There is an emerging consensus that the impact of Drug Repurposing, Repositioning and Rescue (DRPx) on the pharmaceutical industry is real and sustainable. The activity and productivity of DRPx focused companies as well as […]

Data-mining Open Source Databases for Drug Repositioning Using Graph-Based Technique By Dr Ken McGarry & Ukeme Daniel The analysis of ‘Big Data’ has great potential in drug discovery; however complications arise in integrating this data in a principled and coherent way. An important statistical tool to manage complexity is that of graph theory, which is […]

Therapeutic Drug Repurposing, Repositioning and Rescue Part I: Overview By Dr Stephen Naylor & Judge M. Schonfeld The pharmaceutical industry is still beleaguered by escalating costs, stagnant productivity and protracted timelines as it struggles to bring therapeutic drugs to market. This situation has been compounded by a ravenous generic drug sector, and patients that have […]

Principles for Winning Digital Health Strategies By Dr Thilo Kaltenbach In a recent study on digital health in the pharmaceutical industry, management consultancy Arthur D. Little provided an industry snapshot of pharmaceutical companies’ goals for 2020 and their progress so far. The study indicated that by 2020, the business model of the pharmaceutical industry would […]

Reconfiguring Drug Discovery Through Innovative Partnerships By Dr Robert Grundy, Iain James, Chas Bountra and Timothy Harrison Attrition rates in drug discovery and development remain stubbornly high, despite large investments by the pharmaceutical industry. The cost of developing a new drug has been estimated at between $1.3 billion and upwards of $4 billion, if one […]

The Drug Repositioning Revolution: Rescuing & Repurposing Drugs By Dr Monika Green and Dr Kate Hudson-Farmer Cost containment and improving ways of treating people are key drivers for healthcare systems. Unfortunately, these do not often go hand in hand. Finding novel therapeutics with enhanced efficacy is both time consuming and costly – and offers low […]

Healthcare and Life Sciences undergoing disruptive change by linked data and semantic technologies By Hans Constandt From early days the sharing of knowledge has been one of the main forces driving science and innovation. Yet latterly, the pharmaceutical industry with its fear of loss of intellectual property has stalled the engine that has given so […]

There are approximately 7,000 rare diseases, which from a regulatory perspective are defined as those diseases where there are less than 200,000 patients in the US or that affect no more than five in 10,000 of the general population in the EU. Such diseases usually have a genetic basis, often affecting patients early in childhood, […]

New Tools Increase Accessibility to High Content Screening Assays By Dr John Comley There have been major improvements in the instrument offerings and tools that enable high content screening (HCS) over the past decade. In this review we examine the status of high content screening assays today; the motivation to purchase assays kits versus developing […]

Stem Cells: Cell-Based Assays and the World of Small Molecules By Dr Christina N. Ramirez, Dr Dana C. Duré and Dr Hakim Djaballah Stem cells remain a hot topic in academia and industry alike, and with the potential to cause a paradigm shift where many believe in their ability to differentiate into a variety of […]

By Dr Lesley J Millatt, Dr Rmy Hanf and Dr Dean W Hum According to the International Diabetes Federation, more than 285 million people (6.4% of the world population) are currently estimated to suffer from diabetes, a figure which is expected to rise to 438 million (7.8%) by 2030. Moreover, the World Health Organisation calculates […]

The Post Genomic Era: What Does It All Mean? By Dr Chris Torrance Information may no longer be a bottleneck to understanding and tackling complex genetic diseases such as cancer. For those who cut their research teeth not so very long ago, before PCR was thought of by Kary Mullis, this is truly an amazing […]