RAPS is closely monitoring developments in the Coronavirus (COVID-19) outbreak. See our public safety page for the latest updates.

 
  • Regulatory NewsRegulatory News

    Stakeholders call for regulatory clarity in rare disease research network

    Stakeholders weighing in on a proposed rare disease clinical trials network called for regulatory clarity, smart use of existing resources, and a move toward harmonized trial standards and assessments.   As part of the launch of the US Food and Drug Administration (FDA)’s Rare Disease Cures Accelerator , the agency asked for stakeholder input on how FDA and other agencies can achieve a more cooperative approach in supporting the drug development pipeline for rare dise...
  • Regulatory NewsRegulatory News

    FDA officials update on orphan drugs, gene therapies at DIA

    Officials from the US Food and Drug Administration (FDA) discussed the agency’s recent efforts to support the development of products to treat rare diseases during a session at DIA’s Global Annual Meeting on Wednesday.   Orphan and rare pediatric disease designations   While the number of products approved to treat rare diseases has increased over the last decade, the vast majority of rare diseases lack approved treatment options.   Janet Maynard, director of F...
  • Regulatory NewsRegulatory News

    FDA seeks input on rare disease clinical trials network

    To further the establishment of a rare disease clinical trials network, the US Food and Drug Administration (FDA) is asking for input from the public and a broad array of stakeholders.   In a press release, the agency noted that it is “looking to provide a more cooperative approach” in supporting the drug development pipeline for rare diseases.   The call for information comes as the FDA is launching a Rare Disease Cures Accelerator . This accelerator will establi...
  • Regulatory NewsRegulatory News

    Rare Diseases: FDA Awards Grants for 21 Clinical, Natural History Studies

    The US Food and Drug Administration (FDA) on Friday awarded $32 million in research grants to fund 15 clinical studies and six natural history studies for rare diseases. The grants are being funded through FDA's orphan products grants program , with $22 million going to the clinical studies and $9.8 million in combined funding from FDA and the National Center for Advancing Translational Sciences' (NCATS) Therapeutics for Rare and Neglected Diseases program going to fund...
  • Regulatory NewsRegulatory News

    FDA Calls for End to Priority Review Vouchers as GAO Says Too Early to Gauge Effectiveness

    The US Government Accountability Office (GAO) said Wednesday in a new report that it’s still too early to assess whether the Food and Drug Administration’s (FDA) three-year-old pediatric priority review voucher (PRV) program has stimulated the development of drugs to treat or prevent rare pediatric diseases. The PRV program offers vouchers that companies can win for gaining approval of new rare pediatric treatments affecting fewer than 200,000 people, more than half o...
  • Regulatory NewsRegulatory News

    Expert Committee: FDA Should Allow Mitochondrial Replacement Trials Under Certain Conditions

    While the US Food and Drug Administration (FDA) sits on the fence over whether to approve preclinical or clinical trials using mitochondrial replacement techniques (MRT) to help prevent the transmission of certain diseases passed from mother to child, the Institute of Medicine (IOM) of the National Academies of Sciences, Engineering, and Medicine came out with a new report on Wednesday detailing how it believes FDA should allow such trials and regulate them. Background ...
  • Regulatory NewsRegulatory News

    Public Health Groups Propose Global Drug Innovation Fund

    Two health advocacy groups, Médecins Sans Frontières (MSF) and the Drugs for Neglected Diseases initiative (DNDi) are urging public health authorities to create a global fund to tackle what they call "deadly gaps in innovation." Citing the rising threat of antimicrobial resistance (AMR), emerging infectious diseases and neglected tropical diseases (NTDs), the two organizations say the pharmaceutical industry is failing to address certain public health challenges. Back...
  • Regulatory NewsRegulatory News

    EMA Says Companies Still Need to Report Side Effects of Donated Drugs

    Drug makers are frequently involved in relief and humanitarian aid efforts, often through donating their products. However, in light of recent outbreaks of neglected tropical diseases such as Ebola, the World Health Organization (WHO) has asked the European Medicines Agency (EMA) to clarify its drug safety monitoring requirements for companies that donate medicines outside the EU to treat neglected tropical diseases. According to WHO, neglected tropical diseases are a...
  • Regulatory NewsRegulatory News

    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.” ...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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,...