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

 
  • Regulatory NewsRegulatory News

    Patient-Focused Drug Development: New FDA Draft Guidance Delves Into What’s Important

    The US Food and Drug Administration (FDA) on Monday published the second in a series of four guidance documents on patient-focused drug development, with this latest draft helping sponsors identify what is most important to patients with respect to their experience with disease burden and treatment. In determining what’s important, FDA recommends that sponsors use qualitative research methods, which can generate in-depth thoughts from patients in their own words through...
  • Regulatory NewsRegulatory News

    CDRH Works on Clinical Objectivity in Patient-Reported Outcomes

    A new study conducted by mathematical statisticians at the US Food and Drug Administration (FDA) bridges the gap between an objective endpoint and a patient-reported outcome (PRO). The research , led by statisticians Chul Ahn and Xin Fang at FDA’s Center for Devices and Radiological Health (CDRH), seeks to arrive at an acceptable level of probability for a PRO measure to reveal the “truth” of a subject’s disease or health condition. It comes at time when the science of ...
  • Regulatory NewsRegulatory News

    FDA to Focus on Autism, Psoriasis Under New Patient-Focused Program

    The US Food and Drug Administration (FDA) has released a pared-down list of diseases it plans to assess over the coming two years as part of its Patient-Focused Drug Development (PFDD) program—an integral part of the most recent authorization of the Prescription Drug User Fee Act (PDUFA). The PFDD was created in an attempt to bring patients into the regulatory process in a more systematic way. Instead of asking for patient feedback on a drug-by-drug basis, the PFDD asks ...
  • Regulatory NewsRegulatory News

    Patient-Focused Drug Development Tracker

    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. Those meetings have raised novel questions for regulators, who for the first time are asking patients and patient groups to ex...
  • Regulatory NewsRegulatory News

    Is FDA Listening Enough to Patients? Agency Wants Feedback

    When Congress passed the Food and Drug Administration Safety and Innovation Act (FDASIA) into law in 2012, its patient-centered provisions were among the biggest changes set to impact the culture of the US Food and Drug Administration (FDA). Now, two years later and with major planning already underway regarding the future of FDA's patient-centered activities, agency regulators are reaching out to the public to ask what more they can do to improve their efforts. Backgr...
  • 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...
  • Regulatory NewsRegulatory News

    Following Criticism, FDA to Focus on Female Sexual Disorders at Upcoming Patient Meeting

    The US Food and Drug Administration (FDA) this week quietly announced that it soon plans to hold a meeting on female sexual dysfunction (FSD) as part of a larger effort to bring patients into the drug development process to fine-tune its benefit-risk assessment process. Background The meeting will be the 11th under FDA's Patient-Focused Drug Development process—an initiative created under the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) to he...
  • 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...
  • 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...