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4 Diverse Funding Models
Pages 19-41

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From page 19...
... Hale, and inspired her to found the Institute for OneWorld Health, a not-for-profit pharmaceutical company focused on neglected diseases in the developing world. Such diseases are not   Thissection is based on the presentation of Victoria Hale, Ph.D., Founder and Chair of the Board of Directors, Institute for OneWorld Health.
From page 20...
... (CFFT) Business Model "Venture philanthropy" Structure Wholly owned, nonprofit drug discovery and development subsidiary of the Cystic Fibrosis Foundation with eight staff members.
From page 21...
... • Post-approval, Genzyme is committed to optimizing patient care and access. CELTIC THERAPEUTICS, LLLP Business Model Global private equity firm Structure A "virtual pharmaceutical company" comprising a management company that runs a private equity fund and a biomedical development organization that devel continued
From page 22...
... Orphan Drug Act as diseases that affect fewer than 200,000 people in the United States. "Neglected" and tropical diseases have significant impact in the developing world, but all are rare diseases as defined in the U.S.
From page 23...
... Initial program funding was provided by the Bill and Melinda Gates Foundation. OneWorld Health now has the task of convincing new funders that there are worthwhile investments to be made in research and product development addressing neglected diseases.
From page 24...
... In the short term, OneWorld Health has demonstrated that people can work together through a not-for-profit company to develop a medicine for a neglected disease. For example, OneWorld Heath developed a new use for paromomycin, an antibiotic already on the market for 30 years, as a lifelong cure for visceral leishmaniasis, a parasitic infection. While regulatory approval of a new product or a new use of an existing product is necessary for success, it is not sufficient.
From page 25...
... Its primary mission is to convince biopharmaceutical companies to develop drugs for a disease that affects only 30,000 people in the United States and 70,000 worldwide. The primary strategy involves reducing the risk to development partners of entering the cystic fibrosis field and making products more attractive from a business perspective.
From page 26...
... –targeted candidates are in Phase III clinical trials. SOURCE: Wetmore, 2008.
From page 27...
... The clinical care centers in the network receive grant support from CFFT to ensure that they are not only providing excellent care, but also training their staff in the conduct of clinical trials. In addition, the network includes an independent data safety monitoring board whose members are familiar with the clinical development of drugs for cystic fibrosis.
From page 28...
... Principal investigators and staff have experience in the design and conduct of clinical trials for cystic fibrosis and access to patients for trial enrollment. NOTE: CF = cystic fibrosis; CFFT = Cystic Fibrosis Foundation Therapeutics, Inc.; HRCT = high-resolution computed tomography; Figure 4-2, R01292 IPFT = infant pulmonary function testing; NPD = nasal potential difference test.
From page 29...
... In 1993, the CFTR gene had recently been cloned, and venture capitalists were willing to invest $85 million in a Genzyme cystic fibrosis program over a 5-year period. Part of the reason, Meeker believes, was recognition not only that the Orphan Drug Act had opened doors for drug approval, but also that the model for Gaucher disease had shown that a company could make a viable business out of treating a small population.
From page 30...
... Thus the company seeks to facilitate an environment in which patients with Gaucher disease have a reasonable expectation of being seen and diagnosed by a disease expert so informed decisions can be made about therapy. Ultimately, accomplishing this means fostering the development of sustainable health care systems around the world capable of caring for patients with rare diseases.
From page 31...
... Indeed, cost and pricing represent a final, critical challenge for companies seeking to develop drugs to treat rare diseases. Given the success of Cerezyme, some might suppose that a company can develop a therapy for a rare disease, charge a high price, and be successful.
From page 32...
... Summary Whether a drug indication under study benefits a large or a small population, therapies must make a difference. A sustainable business model for the production of drugs to treat rare diseases is a shared responsibility between industry and the health care system.
From page 33...
... Furthermore, in Phase II many questions about compounds remain, and only 30–40 percent make it to the next phase, a fact that discourages many companies from investing in compounds that are in early development. Generally, a smaller biotechnology company that cannot afford to move forward with a compound on its own may discuss an agreement with a large pharmaceutical company, but such discussions may extend for up to 18 months, during which time the biotechnology company must continue to spend money and consume critical patent life of a potential product.
From page 34...
... The Celtic Therapeutics "virtual pharma" comprises a management company that runs a private equity fund and a biomedical development organization that manages the outsourcing of all components of product development. The development organization consists of a small core of very experienced drug development professionals who develop the firm's strategy for each product, and a small group of experts that manages product development execution through outside vendors and consultants, such as contract research organizations (CROs)
From page 35...
... IND POC NDA Basic Research Therapeutic Preclinical Exploratory Advanced Approval and Discovery Development Development Development Phase IV Initial – Robust "The Idea" "The Compound" "The Medicine" "Proof of "Regulatory "Medical Concept" Proof" Marketing" • Universities Biotechs and Emerging The Gap • Institutes • Governments Pharma • Foundations Institutes • Companies • Associations Universities Academic Medical Centers, Hospitals, Clinics Venture Capital Support 10 to 14 years FIGURE 4-3  Defining the gap in biomedical research, from idea to patent expiration. Celtic Therapeutics funds and facilitates the development of promising product candidates to the point of submission of an NDA or a major increase in value of the potential product.
From page 36...
... In conclusion, Corr suggested that, through collaboration across sectors and through new and innovative business models, it will be possible to address not only the issues related to rare and neglected diseases, but also global disparities in health.
From page 37...
... through a nonprofit charity failed in its primary goal because of an inability to bridge a different gap from that previously described. The costs of the chemistry, manufacturing, This section is based on the remarks of Doug Onsi, J.D., Venture Partner, HealthCare V ­ entures; Chaitan Khosla, Ph.D., Professor, Departments of Chemistry, Chemical Engineering, and Biochemistry, Stanford University; Mark Batshaw, M.D., Chief Academic Officer, Children's National Medical Center; Marleen Haffner, M.D., Ph.D., Executive Director, Global Regulatory Intelligence and Policy, Amgen; and Gail Cassell, Ph.D., Vice President, Scientific Affairs and Distinguished Lilly Research Scholar for Infectious Diseases, Eli Lilly and Company.
From page 38...
... Technically, the charity was successful as it helped identify a drug that is currently in clinical trials, and it also aided research on two biomarkers that could be used in those trials. Khosla cautioned that without the development of new business models for preclinical and early clinical drug development, the rare and neglected disease community will have difficulty developing new molecular entities, and will be limited to evaluating old entities with existing CMC capabilities and toxicology profiles.
From page 39...
... Dr. Cassell suggested that the United States cannot afford to be investing $29 billion in federal funds in biomedical research without fostering partnerships to develop drugs for rare diseases.
From page 40...
... Finally, Hale drew attention to the fact that some neglected diseases are more neglected than others. African sleeping sickness and visceral l ­eishmaniasis, for example, are at the bottom of the list when it comes to drug development efforts.
From page 41...
... Hale has observed that when poor people are very sick and fear they may die, they ask for injections instead of oral products because in their experience, oral products are of poor quality. This is why OneWorld Health developed an injectable intramuscular paromomycin for visceral leishmaniasis.


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