Volt Pharma Associates White Paper: Is Orphan Drug Status the Answer to Everyone's Prayers?

London, UK – October 7th, 2014 – There are an estimated 7,000 rare diseases affecting approximately 350 million people globally. 80% of these are of genetic origin and around 50% of those affected are children. These conditions are mostly chronic, degenerative or life-threatening and in serious need of treatment. Recent advances in the understanding of molecular processes and genetic testing have led to a better understanding of the underlying science and enabled viable approaches to be identified. There is no doubt that orphan drug development can provide significant opportunities for pharmaceutical companies. The costs and time needed to get to registration may be substantially smaller, and there are many risks that can be mitigated by a clear understanding of the process and changing environment. Claude Houet, Head of Practice Pharma & Life Sciences of Volt Pharma Associates, explains that there are benefits to both small and large companies when it comes to developing orphan drugs: “For large companies there may be research tax credits, funding grants and waivers of regulatory fees; clinical trials are smaller and cheaper; patients and their treating physicians are easy to find; new effective drugs can achieve premium pricing; small numbers mean marketing costs are reduced; and overall, profit margins are far higher than drugs for more common conditions and there is a period of exclusivity – unless a demonstrably better drug comes along.” “For small companies you won’t need to build a development organisation; regulatory authorities commit to the requirements for registration; the size of the clinical trials and, therefore, the development time and costs are a small fraction of that required for a ‘blockbuster.’ The potential for a rapid progression to market makes an orphan drug development an attractive option for a venture capital company as it means a possibility of an early exit with significant return on their investment.” Orphan drug development is still a risky venture. The small number of patients, despite the premium price, may not lead to high revenues and there is a significant risk of failure to reach Proof of Concept. The Volt Pharma Associates White Paper on Orphan Drugs outlines both the risks and opportunities, so that readers can make a well-informed decision in regards to the pursuit of ventures that have great potential to save lives, and retain profitability. A free download of the White Paper is available HERE (http://www.volt.eu.com/pharma-associates/downloads) and you don’t need to hand over any contact information. Claude says, “It is important to us that access is given freely to anyone who wishes to read this information. Our contact details are on the paper for those who would like to learn more or ask questions, so please do not hesitate to get in touch.” Volt Pharma Associates (VPA) is a division of Volt in Europe, a Volt Information Sciences company. VPA supports life sciences companies, investors and academic institutes in their development of new products, technologies and innovative approaches. ------------------------------------------------------------------------------- About Volt Founded in New York in 1950, Volt Information Sciences Inc. is a leading global talent, technology and consulting organisation. The European operations were established in 1978 and are focused on connecting talented people and respected organisations through innovative workforce solutions. Volt provides award-winning contract and permanent recruitment services, managed service programmes and professional services across a range of industries and skill areas, including IT, Telecommunications, Life Sciences, Engineering, Finance & Accountancy and Sales & Marketing. Volt’s Managed Programmes and Recruitment Process Outsourcing solutions are individually designed to meet each client's needs, including requisition management, sourcing, supplier management, candidate screening, offer/hire and on-boarding. We provide services across multiple countries in Europe, the Middle-East and Asia. For more information visit www.volt.eu.com. Contact: Rebecca Nixon, Senior Marketing Executive (EMEA), 020 7648 4814, [email protected] ------------------------------------------------------------------------------- About Volt Pharma Associates Volt Pharma Associates (VPA) supports life sciences companies, investors and academic institutes in their development of new products, technologies and innovative approaches. VPA’s aim is to help solve its clients’ product research, development and commercialisation challenges so they can achieve their business goals. VPA comprises a team of world-class pharmaceutical industry experts, each with an established track record and acknowledged expertise in their field.

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