Breakthrough Business Models: Drug Development for Rare and by Theresa Wizeman, Sally Robinson, Robert Giffin, Development,

By Theresa Wizeman, Sally Robinson, Robert Giffin, Development, and Translation Forum on Drug Discovery, Institute of Medicine

The method for constructing new drug and biologic items is very dear and time-consuming. even if huge pharmaceutical businesses are able to have enough money the price of improvement simply because they could anticipate a wide go back on funding, businesses constructing medicines to regard infrequent and missed illnesses are not able to depend on such returns. On June 23, 2008, the Institute of Medicine's discussion board on Drug Discovery, improvement, and Translation held a public workshop, "Breakthrough company types: Drug improvement for infrequent and missed illnesses and Individualized Therapies," which sought to discover new and cutting edge techniques for constructing medications for infrequent and missed ailments.

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Today under Gates Foundation leadership, neglected tropical dis- 24 BREAKTHROUGH BUSINESS MODELS eases are generally acknowledged to be not-for-profit territory with respect to intellectual property. What Defines Success? 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.  While regulatory approval of a new product or a new use of an existing product is necessary for success, it is not sufficient.

Haffner explained that while this voucher may encourage sponsors to develop drugs that are expected to yield a small return on investment, more will need to be done. Haffner also raised the issue of funding for the OOPD grants program, stressing that at $14 million it is very small, and the need is much greater than the program can meet. 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.

Demonstrating decreased progression is very different statistically from showing reversal. Another challenge, alluded to earlier, is the practicality of running a clinical trial when the patient population is extremely small. One example is Niemann-Pick B disease, a genetic condition, much like Gaucher, affecting 500 to 1,000 patients worldwide. Genzyme began a Phase I trial of a therapy for this condition in January 2007 at a single center in New York City. Patients are also being screened in the United Kingdom, Germany, Chile, Brazil, Saudi Arabia, and New Zealand.

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