Rare Diseases and Orphan Drugs [antikvár]

Foreword Dr. Berman's book, Rare Diseases and Orphan Drugs: Keys to Understanding and Treating the Common Diseases, addresses a topic of great importance at this particular moment in research history. Recent advances in the molecular biology of disease have taught us that the genetic changes in the common diseases are complex and that there is remarkable variation among affected individuals in the clinical presentation and in the genetic signature of common diseases. Research scientists are beginning to recognize that the common diseases are best conceived as aggregates of many different rare diseases. To benefit from our newly acquired knowledge of the genetics of common diseases, we will need to understand how treatments for the rare diseases will apply to subsets of the common diseases. Breakthrough discoveries among the rare diseases are now viewed as opportunities to understand and treat the common diseases. Hence, there is an increasing emphasis, coming from government, academia, and private research organizations, to increase funding for rare diseases research and orphan drug development. Worldwide, oiphan drugs are being developed and approved at a rapid rate. In the United States, expedited programs adopted by the FDA should continue to move potential products through the research and development continuum toward approval for safe and effective products. Streamlined programs such as Fast Track, Breakthrough Therapy, Accelerated Approval, and Priority Review bring optimism to patients and their families for the quick approval of new products. Additional regulatory approaches and incentives have been expanded and include the rare pediatric and tropical diseases priority review vouchers. Repurposed products now qualify for orphan product incentives at the FDA. Compounds included in the Best Pharmaceuticals for Children Act program are eligible for a 6-month extension to existing exclusivity as an incentive to expand the indications for use from the adults to the pediatric population. For antibiotics, a newer incentive program, Generating Antibiotic Incentives Now (GAIN), and authorizing legislation add 5 years to existing exclusivity for products considered new chemical entities and those included under the Orphan Drug Act. The FDA now uses expert consultants to facilitate applications for orphan drugs while still in the pre-approval stage. The success of clinical trials for orphan drugs can be credited, in no small part, to the willingness of individuals with rare diseases to participate in clinical trials. In the U.S., more than 2,900 active Orphan Product Designations have been made, and 50 additional designations have been provided thus far in 2014. There were 258 Orphan Product Designations in 2013. Obtaining the Orphan Product