Orphan Drugs: the Conundrum- Clinical Research Trials 2015

Orphan Drugs: The ConundrumOrphan Drugs Instructor: Dr Marybeth Regan Product ID: 500903 Level: Intermediate Description The Orphan Drug Act is intended to treat conditions affecting less than 200,000 patients in the US – about 1 in 1500 people. These cases are where there is no reasonable expectation that the developer will recover the costs of drug development and marketing via future sales of the drug in the US. The areas covered in this webinar are: Definition of the Orphan Drug Act Role of the FDA Understanding why the Orphan Drug Act has started a conundrum Why the varying views Good Intentions, Bad Results Competing in the Rare Disease Space Review of drugs passed that are Orphan Drugs Comparison of Rare Disease Drugs VS other drugs Benefits of the Orphan Drug Act VS drawbacks Why Should you Attend: The Orphan Drug Act was approved in 1983. The question that remains is whether the FDA is encouraging pharmaceutical companies to use its resources in the most efficient manner. According to Clinical Leader Magazine, there are approximately 7,000 different types of rare diseases and disorders affecting an estimated 350 million people worldwide. These diseases are different in many ways and there are the variations in patient treatment and gaps in care. The Rare Disease Impact Report mentions that it takes an average of seven years for a patient with a rare disease in the United States to receive a proper diagnosis. Between diagnosis and treatment, a patient typically visits up to eight different physicians and receives two to three misdiagnoses. Objectives of the Presentation: Understanding the objective of the Orphan Drug Act Understanding why the Orphan Drug Act has started a conundrum What is the role of the FDA as it pertains to Orphan Drugs? History of the results of the Orphan Drug Act What is the likeliest future? Who can Benefit: Benefit Managers Director of Human Resource Director of Access Director of Strategy Pharmacy Benefit Managers Representatives of