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 pricey and time-consuming. even though huge pharmaceutical businesses are able to come up with the money for the price of improvement simply because they could count on a wide go back on funding, firms constructing medicinal drugs to regard infrequent and overlooked illnesses are not able to depend upon 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 ignored illnesses and Individualized Therapies," which sought to discover new and cutting edge suggestions for constructing medicines for infrequent and overlooked ailments.
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Additional resources for Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary
Example text
Once the mechanism of a disease DIVERSE FUNDING MODELS 31 is understood, moreover, a proposed therapy must be testable. Cerezyme reverses the effect of Gaucher, but there are a number of diseases whose damage is not reversible, and the goal of a therapy is to slow the progression of the disease. 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.
A therapy would have to be disease modifying and/or lifesaving. And it would have to be testable—a challenge given the small populations involved when one is dealing with rare diseases. Post-approval, Genzyme is committed to optimizing patient care, as opposed to simply convincing physicians that the treatment for their Gaucher patients is Ceredase. 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.
What are the truly neglected diseases? With 127 compounds in development, HIV/AIDS is not a neglected disease. For malaria there are only 30 drugs in development, many of which are in the early stages and have a high risk of failure. For tuberculosis (TB), and particularly drug-resistant TB, only 22 candidate drugs are in the pipeline. For truly neglected diseases, such as human African trypanosomiasis, Chagas disease, and dengue fever, the pipeline is very limited, including 5, 7, and 8 compounds, respectively.