• Regulatory NewsRegulatory News

    FDA prevented record number of drug shortages in 2021

    The US Food and Drug Administration (FDA) prevented 317 drug shortages in 2021, the highest number since the agency began tracking drug shortages in 2012, according to a recent report released by the agency.   The agency’s Center for Drug Evaluation and Research (CDER) has been tracking drug shortages since the Food and Drug Administration Safety and Innovation Act (FDASIA) was passed in 2012, which requires FDA to release an annual report to Congress; FDA’s Center f...
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    NASEM report: FDA should disclose drug and device manufacturing information

    To ensure resiliency in the medical supply chain, the US Food and Drug Administration (FDA) should publicly disclose the location of all manufacturing facilities that supply ingredients and parts for pharmaceuticals and medical devices approved in the US, according to a consensus study report from the National Academies of Science, Engineering and Medicine (NASEM).   This is one of seven recommendations in a 365-page report on building supply chain resiliency. The re...
  • RF Quarterly
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    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
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    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...
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    The RACE is on: Opportunities in pediatric oncology product development

    Pediatric oncology patients continue to have significant unmet medical need. Although the Pediatric Research Equity Act (PREA) mandated evaluation of new drugs for pediatric patients, the rarity and uniquity of pediatric cancers allowed for waivers or exemptions from PREA requirements. The passing of FDA Reauthorization Act amendments in 2017 included the Research Acceleration for Cure and Equity Act, which expands the scope of oncology products subject to PREA. Sponsors d...
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    Strategies for pediatric clinical trials and drug development

    This article discusses the considerations for designing and conducting pediatric clinical trials from a pediatrician’s perspective. The author covers pediatric subpopulations, issues related to age, endpoints, and pharmacokinetic considerations, while presenting practical solutions and ethical considerations. Patient and family‒centric approach to studies and engagement with patient advocacy groups are also discussed. The author concludes with some recommendations about pe...
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    FDA seeks new members for Patient Engagement Collaborative

    The US Food and Drug Administration is seeking eight new members for its Patient Engagement Collaborative (PEC), a group charged with gaining meaningful patient input into medical product development and regulatory decision making.   The 16-member panel includes patients, caregivers, and advocates who serve for 2 to 3 years. The panel is a joint effort between FDA and the Clinical Trials Transformation Initiative . Panel members are expected to participate in two to f...
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    EMA consults on ICH S12 guideline, shares comments on PFDD reflection paper

    The European Medicines Agency (EMA) on Thursday opened a public consultation on the International Council for Harmonisation’s (ICH) S12 guideline on nonclinical biodistribution (BD) considerations for gene therapies.   The S12 guideline recently reached Step 2b of the ICH process after being endorsed at the ICH Assembly virtual meeting earlier this month. (RELATED: ICH reports ‘significant milestones’ reached on guidelines ranging from impurity testing to eCTD standa...
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    Cures 2.0: Discussion draft signals impact on FDA, creation of ARPA-H

    More than four years after the 21 st Century Cures Act was signed into law, Reps. Diana DeGette (D-CO) and Fred Upton (R-MI) on Tuesday circulated a discussion draft of their proposed Cures 2.0 legislation, setting the stage for negotiations on the long-awaited legislative package.   In addition to proposing new programs and enhancements for the US Food and Drug Administration (FDA) and the Centers for Medicare and Medicaid Services (CMS), the 127-page draft bill ...
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    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...
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    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...
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    Patient Engagement in Device Trials: FDA Drafts Guidance

    The US Food and Drug Administration (FDA) on Monday released new draft guidance explaining how sponsors can improve the design and conduct of medical device clinical investigations by using patient experience, perspectives and other relevant information. The 10-page draft mainly addresses four questions: What approaches might sponsors use to engage patient advisors (defined by FDA as those who have dealt with or are dealing with a particular condition but are not inv...