Tag Archives: orphan

The Ethics of the Market Access Debate

Rogerio Vivaldi

At the core of the market access conversation lies the question of value. How much do we value two years added to the end of an 80-year-old’s life? How do we put a dollar amount on a medicine that prevents a rare disease patient, who may only live to age 20 regardless of receiving treatment, from losing her ability to walk? How can we define metrics to measure the cost of a child with a Read More >

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A Bright Future for Orphan Disease Indications

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In celebration of National Rare Disease Day BIO’s Industry Analysis team took a look at recently-approved drugs in the BioMedTracker database with an orphan indication designation. Our research found that 2012 saw the greatest number of approvals of therapies for orphan indications going back all the way to 2000 (This count includes previously approved drugs with new, orphan indication approvals).   Overall, 24 therapies were approved in orphan indications last year, including drugs for Anthrax, Gaucher’s Read More >

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Navigating the Rare Disease World

Susan Ruediger

The international orphan drug marketplace is expected to grow some 30 percent by 2014, fueled by the entrance of big pharmaceutical companies and U.S. legislation favoring development and coverage. This growth promises to improve the health of patients with debilitating and life-threatening disorders. Hoping to further the rare disease dialogue and relationship building opportunities, the 2013 Orphan Disease Forum will provide unique perspective on the orphan drug marketplace, regulatory considerations and much more. Operating within Read More >

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Orphan Drugs: Making Rare Diseases Rarer

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A panel at the BIO CEO & Investor Conference provided an analysis of the thriving orphan drug market opportunity featuring industry, clinical and regulatory experts specializing in rare diseases. The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline with hopes of achieving the Read More >

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FDASIA: A Step Forward for Patients

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Earlier this year, Congress passed and President Obama signed the FDA Safety and Innovation Act (FDASIA) which included a re-authorization of the Prescription Drug User Fee Act (PDUFA). PDUFA provides essential funding to the FDA by authorizing the agency to charge user fees to companies seeking to have products reviewed. Originally enacted in 1992, it must be reauthorized every 5 years. Sara Radcliffe, executive vice president of health at BIO, moderated a panel of experts and advocates Read More >

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