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Genetic Diseases of Children The Role of Orphan Drugs Marlene E. Haffner, MD, MPH 9 March 2011 [email protected] 301 984 5729 First let’s talk about drug development • • • • Complex and failure prone process Takes 5 – 10 years or more Human genome opening many doors Many if not most genetic diseases of children are orphan diseases – prevalence of rare disease is less than 200,000 in the US by law New Drug Development IND Drug Discovery Animal Testing Preclinical Development 0.5 - 3 years 0.5 - 1 year NDA Human Testing Phase I Phase II Approval Post Marketing Phase III 2 - 5 years 6 months - 1 year 4 - 10 years $1.2 Billion 3 Orphan Drug Act • Amended the FD & C Act – signed in 1983 • Has been very successful – almost 400 new drugs developed since signing • Many more in the pipeline • Children recognized • Incentives in the ODA to offset cost of development for sponsors Characteristics of a Rare Disease • While < 200,000 by law, many if not most < 6000 patients in the US • Many Genetic Diseases – often heterogeneous • ~ 85% Serious or Life Threatening • Children comprise ~ 50% • Most are chronic Development of orphan products • Same as for non-orphan products • Orphan products need to be as safe and as effective as non-orphan products • Always must evaluate risk vs benefit • Much attention given to Orphan Products in the FDA In the realm of Genetic Diseases of Children • PKU - Kuvan • NAGS disease – Carbaglu • Urea Cycle Disorders – Buphenyl and Ammonol • SCID (ADA type) – Adagen • Tyrosinemia – Orfadin WOULD NOT HAVE BEEN DEVELOPED WITHOUT THE ORPHAN DRUG ACT INCENTIVES Many treatments, no cures • Cystic Fibrosis - many products to alleviate disease • Sickle Cell Disease – several alleviating treatments but no definitive therapy • Human Genome project and aftermath leading to more directed products • No Stem cell therapies yet • Lives have been significantly and positively altered ? Marlene E. Haffner, MD, MPH President & CEO 11616 Danville Drive Rockville, Maryland 20852 [email protected] 301 984 5729 - office 301 641 4268 - cell