• Regulatory NewsRegulatory News

    FDA Seeks Patient Insight Into Autism, Depression and 14 Other Conditions

    The US Food and Drug Administration (FDA) has announced a second batch of meetings intended to facilitate the development of drugs for neglected conditions by taking into account the perspective of the patients suffering from the conditions. Background In recent years, some patient groups—and in particular groups representing patients suffering from serious diseases with few or no suitable treatments—have begun to express their concerns that FDA is too stringent when it...
  • Feature ArticlesFeature Articles

    Filling a Regulatory Void: Patient Advocates Submit Guidance for Duchenne Muscular Dystrophy

    Muscular dystrophy (MD)––a group of diseases that cause progressive muscle weakness due to defects in muscle proteins ––has a range of complex features that make treatments elusive. MD exists in many forms (Duchenne MD is the most common); animal models do not accurately reflect human disease; and it is difficult to define and measure the progression of this slow disease. Because MD can be life-threatening, new disease-modifying (serving to slow or halt progression) an...
  • Regulatory NewsRegulatory News

    FDA to Focus on Blood, Platelet Disorders at Upcoming Meeting With Patients

    US regulators have announced they will soon hold their tenth patient-focused drug development meeting, this time focusing on a collection of blood and platelet disorders that includes hemophilia A, hemophilia B, and von Willebrand disease. Background The meetings are required under the Food and Drug Administration Safety and Innovation Act (FDASIA) , Section X of which called for the US Food and Drug Administration (FDA) to meet with patients in order to better un...
  • Regulatory NewsRegulatory News

    FDA to Focus on Idiopathic Pulmonary Fibrosis at Upcoming Patient Meeting

    Since the passage of the  Food and Drug Administration Safety and Innovation Act (FDASIA)  in 2012, the US Food and Drug Administration (FDA) has been publicly meeting with patients who suffer from debilitating conditions without adequate treatment options in the hopes of better understanding their wants and needs in the drug development process. Now the agency is gearing up for its ninth patient-focused drug development meeting, this time focusing on the views of p...
  • New Patient-Focused Drug Development Meeting to Concentrate on Neurologic Disorders

    Since the passage of the  Food and Drug Administration Safety and Innovation Act (FDASIA)  in 2012, the US Food and Drug Administration (FDA) has been publicly meeting with patients who suffer from debilitating conditions without adequate treatment options in the hopes of better understanding their wants and needs in the drug development process. Now the agency is gearing up for its eighth meeting for patients with neurologic manifestations of inborn errors of ...
  • EMA Looks to Formalize Involvement of Patients Within the Regulatory Process

    There is perhaps no bigger trend among established regulatory regimes around the world at this time than increasing the involvement of patients in the regulatory process. The US Precedent In the US, this involvement has been growing since the early 1990s, when the AIDS crisis energized a movement among patient groups to press the US Food and Drug Administration (FDA) for faster and less risk-averse approvals. In recent years, this involvement has grown, with most-if n...
  • Fourth Patient-Focused Drug Development Meeting Announced With Focus on Common Questions

    The US Food and Drug Administration (FDA) has today announced the details of its fourth patient-focused drug development meeting, this time focusing on narcolepsy, a condition characterized by an patient's inability to regulate sleep habits. Background Under the  Food and Drug Administration Safety and Innovation Act  ( FDASIA ) of 2012, FDA was charged with setting up a " Patient-Focused Drug Development Initiative ," which is set to seek out opinions from pa...
  • Lung Cancer Focus of Upcoming Patient-Focused Drug Development Meeting

    The US Food and Drug Administration (FDA) has announced its third meeting on patient-focused drug development, this time focusing on the views of patients with lung cancer and seeking to identify unmet needs in its first patient population with cancer. Background Under the  Food and Drug Administration Safety and Innovation Act  of 2012, FDA was charged with setting up a " Patient-Focused Drug Development Initiative ," which is set to seek out opinions from ...
  • Patients First: FDA's New Website Aims to Make Regulations Clear to Everyone, Invite Involvement

    Patients are, as regulators and industry frequently remind each other, at the heart of every regulatory process and decision. But it can sometimes be difficult to find patients in the regulatory process itself. Now, thanks to a new website just launched by the US Food and Drug Administration (FDA), that paradigm stands to change in a major way. Power (of Understanding) to the Patients The new website, FDA's Patient Network , was launched on 23 April 2013, and seems gea...
  • Overestimated Efficacy, Underestimated Risks Guide Patient Treatment Options

    • 13 November 2012
    Patients and physicians alike often have lofty expectations and aspirations about the effectiveness of a prospective treatment. But, as a new article in the Annals of Family Medicine argues, those expectations contribute significantly to both the increasing costs of healthcare and avoidable harms suffered by patients. At the core of the healthcare cost crisis right now is an unavoidable fact, writes Steven Woolf, MD: The cost of obtaining healthcare, and in particular ...
  • Report: Compassionate Use Exemptions Being Granted in Increasing Numbers

    • 24 October 2012
    A so-called "compassionate use" program launched by the US Food and Drug Administration (FDA) is helping increasing numbers of patients access potentially life-saving drugs at early stages in their development process, reports the Wall Street Journal . The compassionate use program is relatively straightforward: FDA allows manufacturers to provide some patients-generally only those with highly advanced and likely fatal diseases-with drug compounds still under early-...
  • PCORI: Industry Likely Skewing Trial Outcomes to Detriment of Patients, Regulators

    • 26 July 2012
    It's an age-old question: Are life science companies influencing the outcomes of clinical trials? While it may not have a clear-cut answer, a newly-formed semi-governmental agency has announced it's looking to at least partially address it by focusing on how to make clinical trials more responsive to the needs of patients-a development with potentially huge implications for the pharmaceutical and medical device industries. The Patient-Centered Outcomes Research Institute...