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    Perspective: A Modern Progressive Approval System for Rare Diseases

    • 16 April 2012
    The recent dramatic increase in the number of orphan drug designations has prompted patient groups, pharmaceutical companies, legislators and many other stakeholders to look for ways to accelerate the delivery of innovative new medicines to people with rare diseases. In particular, patients suffering from illnesses for which there are no adequate licensed therapies want access to promising new products earlier in the drug development cycle. Against this backdrop, there i...
  • Group Advocates Treating Superbugs Like Rare Diseases

    The research group Infectious Diseases Society of America (IDSA) is calling on the US Food and Drug Administration to classify superbugs as rare diseases in a bid to spur the development of new classes of antibiotics to treat increasingly drug-resistant strains of bacteria, reports Reuters . The research area has been plagued by a lack of investment, as drugmakers have been shying away from expensive investments in a historically unprofitable sector. The solution, ar...
  • FDA Looking to New Paradigms for Getting Drugs to Patients More Quickly

    The US Food and Drug Administration (FDA) is evaluating whether to upend several approval pathways, including the current over-the-counter (OTC) pathway with one that emphasizes more OTC medicines available to patients with chronic conditions, reports Bloomberg . FDA Commissioner Margaret Hamburg and Center for Drug Evaluation and Research Director Janet Woodcock said that FDA is looking to hold a public hearing in March to discuss the possibility of accelerating how ...
  • ULTRA Bill Introduced in the House Legislation Seeks to Permit Broader Use of Scientific Data to Support Surrogate Endpoints for 'Ultra Orphan' Drug Approvals

    • 03 January 2012
    Two congressmen have introduced legislation that would amend the Food, Drug and Cosmetic Act to provide an easier, accelerated approval pathway for drugs for "Ultra Orphan" diseases, which affect fewer than 6,000 people.  In comparison, "Orphan Diseases" affect fewer than 200,000 individuals. The bill, Unlocking Lifesaving Treatments for Rare-Diseases Act (ULTRA) (H.R. 3737), would allow for surrogate endpoints that is "reasonably likely, based on epidemiologic,...