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  • 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...
  • Regulatory NewsRegulatory News

    As FDA Takes Notice of Patients, Groups Trying to Influence Regulatory Policy do as Well

    An advocacy group focused on accelerating the discovery and approval of new therapies—and in particular therapies for under-served and rare diseases—has launched a new effort aimed at helping to bring patient perspectives into the regulatory process. Background In recent years, regulators and regulatory-minded groups alike have pressed to increase the involvement of patients in the regulatory process. The thinking is that patients are the best stakeholders to ask a...
  • Regulatory NewsRegulatory News

    Legislators Borrow From FDA's Approach in Seeking Patient Input About Regulatory Process

    House legislators are quickly moving ahead with a new plan that hopes to overhaul how the US regulatory system reviews new medical products, and are now soliciting input from a group that has not traditionally been at the center of the regulatory process: patient advocacy groups. Background In late April 2014, Reps. Fred Upton (R-MI) and Diana DeGette (D-CO) announced the launch of a new initiative called the " 21st Century Cures Initiative ," which they said would be a...
  • 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 ...
  • FDA Says Sponsors of Chronic Fatigue Syndrome Drugs can Rely on Patient-Reported Outcomes

    The US Food and Drug Administration (FDA) has released a new draft guidance document intended to expedite the development of drugs to treat chronic fatigue syndrome (also known as myalgic encephalomyelitis)-a poorly understood condition that has recently been subject to significant interest by FDA regulators. Background Chronic fatigue syndrome (CFS) affects patients by causing severe, persistent and often debilitating fatigue. Little is known about the underlying cause...
  • 2012: The Year of the Patient Registry?

    • 13 December 2012
    Patient groups, regulatory agencies and companies are increasingly finding use in the establishment of patient registries-databases of patient data usually specific to a particular disease, and often including genetic, health, demographic and other information. One of the earliest adopters of the registry model was the Cystic Fibrosis Foundation , which established a registry in the early 1970s to better assess information about the relatively small number of patients l...
  • RAPS' LatestRAPS' Latest

    Regulatory Experts Urge Caution With Communications Activities

    The Office of Prescription Drug Promotion (OPDP) and its Advertising and Promotional Labeling Branch (APLB) of the US Food and Drug Administration (FDA) have been busy. Over the past year, they have released multiple guidances, expanded the so-called "bad ad" program that asks healthcare professionals to police inappropriate promotional efforts, and issued a number of untitled and Warning Letters. During a panel discussion at 2012 RAPS: The Regulatory Convergence , regu...
  • NIH Launches New Registry for Down Syndrome Patients

    • 29 October 2012
    The US National Institutes of Health (NIH) has announced the establishment of a new patient registry for patients with Down syndrome, part of an effort to "facilitate contacts and information sharing among families, patients, researchers and parent groups." The registry could eventually have powerful research applications and regulatory significance. A similar registry operated by the Cystic Fibrosis (CF) foundation has been a driving force in product development, and ...
  • AHRQ Plans Patient-Based Adverse Event Reporting System

    • 12 September 2012
    Adverse event reporting could become more widespread under a new system proposed by the Agency for Healthcare Research and Quality (AHRQ). In a 10 September Federal Register posting , the agency, which acts to collect and analyze data about the quality and efficiency of care in the US, said it had submitted to the Office of Management and budget (OMB) a request to approve its prototype for a Consumer Reporting System for Patient Safety Events. AHRQ said the system is...
  • Trade Associations Want Time Before Sunshine Act Begins

    • 29 August 2012
    The Centers for Medicare and Medicaid Services (CMS) is prepared to publish a final rule for the Physicians Payments Sunshine Act , but some trade associations are asking for more time before implementation of that rule. According to CMIO , four life science trade associations sent a letter to CMS, insisting sufficient notice be given before collection of data begins. They are asking for 180-day grace period after the final rule is released before reporting is required...
  • FDA Looks to Establish Patient Registry to Assess Risks of Imaging Contrast Agents

    • 17 July 2012
    The US Food and Drug Administration (FDA) has announced its intent to establish a new patient registry to assess the postmarketing risks of several contrast agents used in magnetic resonance imaging and angiography (MRI/MRA). In an 18 July posting in the Federal Register , FDA noted six of the eight gadolinium-based contrast agents (GBCAs) used in MRIs and MRAs are associated with nephrogenic systemic fibrosis (NSF)-a condition that causes excessive connective tissue ...