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    Rare Disease Patient Group Commends EMA, Wants EU-Wide Access to Treatment

    A rare disease advocacy network has applauded the European Medicines Agency’s (EMA) efforts to incentivize the development of treatments for rare diseases , but says  the variance in treatment access across the EU is “unacceptable.” In an interview with EurActiv , Terkel Andersen, president of the advocacy group EURODIS, said that EMA and the European Commission (EC) are “doing their utmost to try to make rare diseases ‘attractive’ for the pharmaceutical industry.” ...
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    Applying Ethics to Conflicting Healthcare Philosophies: The Ebola Example and the RAPS Ethics Code

    The Ebola crisis has brought more than anxiety to the US; it has brought controversy in the form of conflicting treatment recommendations for individuals returning from African countries affected by hemorrhagic fever disease. How does a regulatory professional determine how to respond in the face of conflicting philosophies on best practice for developing a treatment or prevention protocol? This article looks at the Regulatory Affairs Professionals Society (RAPS) Co...
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    Leprosy––Not Yet Consigned to History

    The word leprosy brings to mind a gruesome disease that separates its sufferers from society and continues to strike fear into communities, much like Ebola. While the genome for leprosy has been sequenced and there is a drug cocktail that effectively cures the disease, its mode of transmission still is unknown. A disease that dates back through millennia, leprosy so far has eluded efforts to find the final piece of information that might eradicate it from the world per...
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    Orphan Drugs Around the World: The Regulations and Requirements You Need to Know

    "Orphan drugs" are product intended for use in the treatment of a "rare" disease—those diseases that occur infrequently or rarely in the general population of a specific location.  Rare diseases affect a limited number of people out of the whole population, and the definition varies on a country-by-country basis; it is country-specific and population-dependent.  It is estimated that between 5,000 and 8,000 (typically averaged to 7,000) distinct rare diseases exist today,...
  • FDA Guidance: How to Improve Your Meetings With the Office of Orphan Product Development

    A new draft guidance document published by the US Food and Drug Administration (FDA) aims to clarify the processes and procedures of meetings with its Office of Orphan Products Development (OOPD). Background OOPD's mission is to help facilitate the development of drugs, devices, biologics and other medical products to treat patients with rare diseases. Those diseases, as defined by the Orphan Drug Act of 1983, affect fewer than 200,000 patients in the US in any given ...
  • As US Population Grows, Rare Diseases Become Even Rarer

    In 1983, the population of the United States was approximately 233 million people. More than 30 years later , the US population is now closer to 313 million-an increase of 82 million people. But while the increase has been good for many things, it has been perversely disadvantageous for patients with rare diseases. Background In 1983, Congress passed the Orphan Drug Act , a piece of legislation meant to provide incentives for manufacturers to develop therapies to me...
  • FDA Announces $14 Million in Grants for Trials in Support of Rare Disease Therapies

    The US Food and Drug Administration (FDA) is often seen as a reactive entity-reacting, that is, to the information contained within applications in order to render an approval decision, or to postmarketing adverse event data to determine if a product remains safe for use. But as it illustrated in an announcement made on 21 October 2013, it can also be proactive when it needs to be. FDA said it will be issuing 21 grants through its Orphan Products Grants Program with the...
  • OECD Calls for Regulatory Harmonization to Support Global Clinical Trials System

    The Organization for Economic Co-operation and Development (OECD), a long-standing global governance group focused on economic issues, has called for countries around the globe to harmonize their clinical trials regulations in an attempt to save both costs and lives. Background In a statement, OECD said it was troubled by "increasingly complex and inconsistent clinical trial regulations," which it said were causing delays, increasing costs and leading to fewer clinical ...
  • In Landmark Reversal of Policy, Health Canada Announces Launch of Orphan Drug Policy

    Canadian Health regulators have announced the launch of a new orphan drug regulatory approach aimed at getting more treatments to patients with rare diseases. In a 3 October 2012 announcement, Leona Aglukkaq, Canada's minister of health, said the Canadian government was committing to a, "New approach that will better support the development and authorization of drugs for rare diseases and [the] launch of a new Web portal to assist patients in finding the information an...
  • FDA Grant to Support Neglected Orphan Disease Trials

    The US Food and Drug Administration (FDA) is looking to help fund the development of products intended to treat orphan diseases in areas where no treatments currently exist or the existing treatments are inferior. In a Federal Register post on 3 August, FDA noted the grant money will total $14.1 million, with $4.1 million going toward the development of between five and 10 new awards during the first year and the remaining $10 million supporting continuation awards...
  • Report: FDA Increasingly Playing Key Role in Global Health

    A report issued by the Global Health Technologies Coalition (GHTC), a coalition of 40 non-profit groups including the Bill & Melinda Gates Foundation, argues that the US Food and Drug Administration's (FDA) effects are being felt far beyond US borders. The US health regulatory agency's statutory mandate is ostensibly to ensure the safety and effectiveness of products used by American consumers, but it is "increasingly…playing a role in global health R&D,...
  • Hamburg: 'I Understand the Frustration' With Inefficient Regulations

    US Food and Drug Administration (FDA) Commissioner Margaret Hamburg spoke at the International Conference on Emerging Infectious Diseases, saying the agency is aware of the "dearth of new products" in the development pipeline to fight infectious diseases, and is doing everything it can to open up new and innovative regulatory pathways to speed products to market. The 11 March keynote speech by Hamburg-herself a former commissioner of health and physician-focused primaril...