• RF Quarterly
    Feature ArticlesFeature Articles

    RF Quarterly, December 2021: Key takeaways from RAPS Convergence 2021

    Welcome to the December issue of RF Quarterly which focuses on key takeaways from RAPS Convergence 2021, including patient-focused approaches in regulatory decision making and drug development, the transition from convergence to harmonization for global pharmaceutical regulatory requirements, pediatric drug development, and regulatory and clinical strategies.   Patient perspective and global harmonization There is increasing recognition of the importance of patients...
  • RF Quarterly
    Feature ArticlesFeature Articles

    Patient-reported outcomes and patient preference information in regulatory decision making

    This article discusses the patient-focused trends in collecting patient experience data (PED), such as patient-reported outcomes (PROs) and patient preference information (PPI), for use in regulatory decision making and drug development. The authors cover current programs initiated by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and discuss how the agencies and sponsors can collaborate to elevate the role of PROs and PPI in informing regula...
  • Regulatory NewsRegulatory News

    FDA releases draft guidance on assessment of PROs for cancer trials

    The US Food and Drug Administration (FDA) has released a draft guidance that recommends how sponsors can collect “meaningful” patient-reported outcome (PRO) measurements to assess results from cancer clinical trials.   The draft guidance focuses on a set of “core” PRO measures that can be used to gather data on patients’ symptoms, symptomatic adverse events and physical function and is specific to registration trials for anti-cancer treatments intended to demonstrate...
  • Regulatory NewsRegulatory News

    FDA Draft Guidance on Patient Experience Data Incomplete, Critics Argue

    Industry group BIO, the National Association for Rare Disorders (NORD) and the National Center for Health Research (NCHR) called for clarification on how regulators will use draft guidances proposed by external stakeholders on patient experience data. The comments on the US Food and Drug Administration (FDA) draft guidance from December 2018 on developing and submitting proposed draft guidances related to patient experience data showed some consensus around including ...
  • Regulatory NewsRegulatory News

    FDA Finalizes Guidance on PRO Assessment Tool for COPD Trials

    The US Food and Drug Administration (FDA) on Monday finalized guidance to help sponsors developing drugs to treat chronic obstructive pulmonary disease (COPD) to use the St. George’s Respiratory Questionnaire (SGRQ), a patient-reported outcome measure (PRO) assessment tool, in clinical trials. The 8-page guidance, which was revised in draft form in May 2016, notes that sponsors can can use the SGRQ as a PRO assessment of efficacy in submissions to investigational new ...
  • Regulatory NewsRegulatory News

    CDRH Sees Spike in Pre-Market Submissions With Patient-Reported Outcome Measures

    The US Food and Drug Administration’s (FDA) Center for Devices and Radiological Health (CDRH) on Tuesday issued its first report on patient-reported outcomes (PROs), noting a significant uptick in PRO data submitted in 2017. Over a six-year period, the report says CDRH observed a more than 500% increase in the number of pre-market submissions that include PRO measures. And in FY 2017, more than 75% of approved, pivotal original and new study investigational device...
  • Regulatory NewsRegulatory News

    FDA Holds First Patient Engagement Advisory Committee Meeting

    The US Food and Drug Administration's (FDA) Patient Engagement Advisory Committee (PEAC) met for the first time on Wednesday and Thursday to give the agency input from  patients on clinical trials for medical devices. The meeting focused on a number of key issues related to clinical trials, with the goal of increasing patient input on trial design; patient recruitment, enrollment and retention; and communicating results back to patients. Patient Engagement at FDA FDA ...
  • Regulatory NewsRegulatory News

    House Reps Seek More Transparency on Novartis-CMS Pricing Deal for Newly Approved CAR-T Therapy

    Rep. Lloyd Doggett (D-TX) and other House Democrats on Wednesday sent a letter to Seema Verma, administrator of the Centers for Medicare & Medicaid Services (CMS), seeking to find out more information about an outcomes-based payment approach for Novartis’ newly approved chimeric antigen receptor T-cell (CAR-T) treatment Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). The outcomes-based pay...
  • Feature ArticlesFeature Articles

    Wearable Technologies and Mobile Health Devices: Changing Medical Research and Delivering Better Patient Outcomes

    This article discusses how wearable technologies and mobile health devices are changing medical practice and improving patient outcomes with the potential to lower research and development costs and impact reimbursement. Introduction Wearable technologies and mobile Health (mHealth) devices are increasingly helping patients report meaningful outcomes and manage their conditions while enhancing their quality of life. Wearable technologies are "smart" electronic devices...
  • Regulatory NewsRegulatory News

    Ulcerative Colitis Drugs: FDA Offers Draft Guidance on Clinical Development

    Pharmaceutical companies looking to understand FDA’s current thinking on efficacy endpoints for clinical trials to develop new ulcerative colitis (UC) treatments will be interested in new draft guidance released Friday. And though the guidance spells out what primary endpoints should be used, the agency also bemoans the fact that there are not well-defined and reliable clinician-reported, patient-reported and observer-reported outcome instruments for clinical trials. Ba...
  • Regulatory NewsRegulatory News

    Researchers: Patient Reported Outcomes for Heart Failure Fail to Meet FDA Criteria

    A team of researchers say that no currently available patient-reported outcomes for heart failure meet the US Food and Drug Administration's (FDA) criteria for supporting product approval. In a recently published review in the Journal of the American College of Cardiologists , the researchers checked the patient-reported outcomes against nine criteria adapted from the 14 listed in FDA's 2009 guidance, Patient-Reported Outcome Measures: Use in Medical Product Developme...
  • Regulatory NewsRegulatory News

    FDA Grapples With Lesser-Known Costs of Clinical Trials Transparency

    US medical device regulators soon plan to hold a public hearing to discuss how the results of cardiovascular outcomes trials (CVOTs) can be kept confidential until they are completed. Background The meeting, previewed by the US Food and Drug Administration (FDA) in a 14 July 2014 Federal Register notice, comes after years of the agency placing more of an emphasis on mandatory post-market approval trials. Those trials are meant to assuage outstanding regulatory concer...