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

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 awfully dear and time-consuming. even supposing huge pharmaceutical businesses are able to have enough money the price of improvement simply because they could anticipate a wide go back on funding, corporations constructing medications to regard infrequent and ignored ailments 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 enterprise types: Drug improvement for infrequent and missed illnesses and Individualized Therapies," which sought to discover new and cutting edge ideas for constructing medicinal drugs for infrequent and ignored ailments.

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For example, CFFT can save a business partner 6 months on the learning curve by having validated assays available at contract research organizations or by providing access to positive control compounds or cellular, antibody, or protein reagents. CFFT can also facilitate collaborations between a sponsor and academic partners, as well as access to intellectual property. And through regular advisory meetings, CFFT can assist in resolving issues and connect a sponsor with experts in pharmaceutical development.

Disease-preventing therapies, introduced at a very early age to prevent damage to the lung, include gene therapy, CFTR protein modulation, and restoration of ion transport. At the other end of the spectrum are disease-modifying therapies that help manage the manifestation and progression of the disease; they include drugs that thin and clear mucus, anti-­inflammatory and anti-infective drugs, products that can increase the success of lung transplantation, and nutritional supplements. CFFT, established in 2000, is a wholly owned nonprofit drug discovery and development subsidiary of the main foundation.

Genzyme knows this is a potentially lifesaving drug, and is in the process of scaling up production from 160 to 2,000 liters and ultimately to 4,000 liters. The company is providing the drug free in many places, including the United States, while it works through the process of scaling up production and meeting regulatory requirements. To date, ­Genzyme has invested more than $600 million in developing this drug. Indeed, cost and pricing represent a final, critical challenge for companies seeking to develop drugs to treat rare diseases.

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