Clinical trial failures: help from the Rare Diseases

In June, 2014, my book, entitled Rare Diseases and Orphan Drugs: Keys to Understanding and Treating the Common Diseases was published by Elsevier. The book builds the argument that our best chance of curing the common diseases will come from studying and curing the rare diseases. For a variety of reasons, clinical trials for rare diseases tend to have much greater likelihood of success than clinical trials on common diseases. Moreover, treatments developed for the rare diseases will almost always find some value in the treatment of one or more common diseases [a major theme discussed developed in the book]. Here is a short excerpt from Chapter 14. It can be difficult or impossible to enroll all the patients required for a clinical trial. In an analysis of 500 planned cancer trials, 40% of trials failed to accrue the minimum necessary number of patients. Of cancer trials that have passed through preclinical, phase I clinical, and phase II clinical trials, three out of five failed to achieve the necessary patient enrollment to move into the final phase III clinical trial [12]. Most clinical trials for cardiovascular disease, diabetes, or depression are designed to be even larger than cancer trials [12]. Overall, about 95% of drugs that move through the clinical trial gauntlet will fail [13]. Of the 5% of drugs that pass, their value may be minimal. To pass a clinical trial, a drug must have proven efficacy. It need not be curative; only effective. Of the drugs that pass cl...
Source: Specified Life - Category: Pathologists Tags: ancillary studies clinical trials common disease complex disease orphan disease orphan drugs rare disease reproducibility statistical significance trial design Source Type: blogs