Breakthrough Business Models Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary (2009) / Chapter Skim
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1 Introduction and Overview
1 Introduction and Overview
Pages 1-6
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In addition, the number of new drug approvals has been slowly declining over the last 11 years -- from 53 new molecular entities approved in 1996, to an average of 28 per year between 1999 and 2005, and to a mere 17 in 2007. A recent editorial outlines many of these issues and concludes that "the conventional business model appears fallible" and that "both industry and academia are poorly positioned to respond in the [current]
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market exclusivity following approval of a designated orphan product. Patient groups, disease foundations, and philanthropic organizations have long recognized that the conventional drug development model is less effective in yielding treatments for rare and neglected diseases, and have therefore devised a range of financial and operational strategies for filling this gap. As a result, the outlook for the development of drugs for rare and neglected diseases is arguably far better today than was the case a decade ago.
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As outlined by workshop chair Nancy Sung, the workshop was designed to: • provide an overview of how drug development is financed; • review the state of orphan product development at FDA; • explore new models for funding translational research and tech nologies; and • examine and discuss the adequacy of the regulatory, legislative, and policy tools currently in place to help advance the development of drugs for rare and neglected diseases. The workshop presentations and discussions considered a range of strategies for reducing the risk to industry and venture capitalists of investing in the development of such therapies by filling critical funding gaps along the drug development pathway and pursuing highly targeted approaches to early-phase development.
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Establishing alliances is advantageous and can result in expanded access to innovative technology and to additional resources, including funding, intel lectual property, disease experts, and patient communities. Several speakers stated that relationship building is paramount, and a successful relationship requires that knowledge and intellectual property rights flow in both directions to enable acceleration, inno vation, translation, and deployment.
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• Chapter 4 is the first of four chapters offering specific examples of business models for the development of drugs for rare and neglected diseases. The funding models of four organizations are described: the Institute for OneWorld Health, a nonprofit phar maceutical company; Cystic Fibrosis Foundation Therapeutics, a disease foundation that functions as a virtual drug company; Genzyme, a for-profit biotechnology company; and Celtic Thera peutics, a global private equity firm that functions as a virtual pharmaceutical company.
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It reviews the regulatory tools available to assist with orphan drug development and approval processes, including fast track designa tion; accelerated approval; priority review; and early and frequent communication with FDA through such vehicles as Type A, B, or C formal meetings, special protocol assessments, or informal meetings. • Chapter 8 outlines areas identified as needing further discussion: (1)
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