• Regulatory NewsRegulatory News

    FDA Planning Five Guidance Documents to Help Children With Rare Diseases

    • 10 July 2014
    The US Food and Drug Administration (FDA) has released a new report indicating four core ways it plans to accelerate the development of treatments for pediatric rare diseases (PRD). The report, Complex Issues in Developing Drugs and Biological Products for Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases , was required under Section 510 of the Food and Drug Administration Safety and Innovation Act ( FDASIA ) and based off a...
  • Regulatory NewsRegulatory News

    What's a Rare Disease for Dogs? FDA Proposes Maintaining Current Levels for Major Species

    Drug regulation is in many ways as much about incentives as it is about requirements. Especially for drugs intended for smaller populations, an incentive—a free review voucher or a speedy review time, for example—may be the difference between a drug existing as a concept or as one available to consumers. Background In the US, patients have long benefited from the Orphan Drug Act , which defines a rare disease as one affecting fewer than 200,000 patients in a given year...
  • 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    A new draft guidance document published by the US Food and Drug Administration (FDA) aims to clarify the processes and procedures of meetings with its Office of Orphan Products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 ...
  • EMA, TGA Launch New 'Collaboration' to Accelerate Access for Orphan Medicines

    EU and Australian regulators have announced that they will begin sharing information about orphan medicines in an attempt to accelerate approval for new drugs intended for rare diseases. Background Orphan medicines are those intended for diseases or conditions which affect a small number of individuals-so-called "rare diseases"-and are thus less likely to have a ready market to incentivize investment in new therapies. The definition of "rare disease" differs throughout...
  • 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 ...
  • NCATS Calls for Regulatory Partners to Develop, Market Potential Rare Disease Treatment

    • 07 February 2013
    The National Center for Advancing Translational Sciences (NCATS) is the newest addition to the National Institute of Health (NIH), and perhaps the one center most closely aligned with the goal of regulatory affairs. Formed in 2011 as the brainchild of NIH Director Francis Collins, the center aims to bridge the so-called valley of death, making sure more discoveries are able to bridge the gap between bench and bedside. But with the exception of some announcements regardi...
  • FDA Proposes 'New Pathway' to Approve Drugs for Unmet Needs

    The US Food and Drug Administration (FDA) plans to hold a public meeting in February to discuss the formation of a "potential new pathway" intended to bring to market new products aimed at treating serious or life-threatening conditions for which there is an unmet need. The new pathway seems likely to take advantage of FDA's new draft guidance on enriched clinical trials , which states that small, targeted subpopulations of patients may be used as the basis of approval ...
  • FDA Training Session Set to Focus on Clinical Trials for Rare Disease Populations

    The US Food and Drug Administration (FDA) and the National Institutes of health (NIH) are launching a new training course to instruct sponsors of rare disease therapies on how to best conduct small clinical trials for drugs, biologics and medical devices. The course, " The Science of Small Clinical Trials ," is being coordinated by FDA and NIH's Office of Rare Diseases Research and National Center for Advancing Translational Sciences (NCATS). Because many diseases, and ...
  • 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...
  • EMA Recommends Vertex's Cystic Fibrosis Drug Kalydeco

    The European Medicines Agency (EMA) has recommended Vertex Pharmaceuticals' cystic fibrosis (CF) drug Kalydeco (ivacaftor), moving the drug one step closer to becoming the drug the first in Europe for the treatment of CF in patients with a specific gene mutation. The drug received the Committee for Medicinal Products for Human Use's (CHMP) recommendation on 25 May after being reviewed under a 150-day accelerated approval program. EMA notes Kalydeco is the eighth pro...