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  • Regulatory NewsRegulatory News

    Stakeholders seek clarity on FDA cell and gene therapy draft guidance

    Industry, medical societies and other stakeholders have weighed in on draft guidance from the US Food and Drug Administration (FDA) detailing how sponsors that want to study multiple versions of cell and gene therapies could combine them in a single “umbrella” trial.   The draft guidance, released in September 2021, outlined FDA’s proposed thinking for how this process would work. For cell and gene therapies intended to treat a single disease, sponsors would typically ...
  • Regulatory NewsRegulatory News

    Gene therapy developers can expect less hand-holding from OTAT

    A growing workload and insufficient staffing have prompted the US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) to institute new processes to expedite its reviews of new cell and gene therapies, said OTAT Director Wilson Bryan at a 6 December webinar sponsored by the Alliance for a Stronger FDA.   At the webinar, Bryan was asked to discuss the impact of the projected workload for OTAT, projections for approving new cell and gene t...
  • Regulatory NewsRegulatory News

    In US, unlicensed stem cell clinic numbers keep climbing

    Nearly 1,500 US businesses were engaged in direct-to-consumer marketing of unproven and unlicensed stem cell therapies in 2021, marking a significant uptick over the last five years.   The figure comes from an analysis published in the journal Cell Stem Cell that breaks down the number of clinics promoting various types of putative stem cell treatments in the United States between 2016 and 2021. As of March 31, 2021, there were 1,480 U.S. business operating 2,754 c...
  • Regulatory NewsRegulatory News

    For gene therapies, FDA drafts trial guidance, finalizes "sameness" for orphan exclusivity

      A new draft guidance from the US Food and Drug Administration (FDA) gives cell and gene therapy developers a detailed framework for the conduct of umbrella trials, offering the potential for enhanced speed and efficiency in early-stage clinical trials.   In the umbrella trials envisioned under the draft guidance, two or more versions of a cellular or gene therapy product would be studied for one specific disease using just one trial design, shared infrastructure, a...
  • Regulatory NewsRegulatory News

    Convergence: Inadequate testing of cell and gene therapies draws FDA concern

    The lack of consistent testing in measuring product quality for cell and gene therapies is a major deficiency found by US Food and Drug Administration (FDA) reviewers in its information requests (IRs) to manufacturers in their submission of biologics license applications (BLAs).   This observation was made by Kedest Tadesse, senior research manager for Agency IQ, which recently compiled a survey of deficiencies identified in IR letters in FDA’s review of five approved ...
  • Feature ArticlesFeature Articles

    Fostering diversity and talent: A Pfizer case study

    The Pfizer Global Regulatory Affairs (GRA) – Howard University College of Pharmacy (HUCOP) Advanced Pharmacy Practice Experience (APPE) program fulfills a requirement of the Doctor of Pharmacy (PharmD) curriculum and helps students explore biopharmaceutical industry career options and prepare for their career after graduation, such as pursuing postgraduate training and seeking employment. The HUCOP Preceptorship Program at Pfizer was designed to broadly increase awareness ...
  • Feature ArticlesFeature Articles

    Regulation of cell and gene therapy products in Japan

    In recent years, new cell and gene therapies have been developed and approved in a number of countries, including Japan. The Japanese government fully supports academia and the pharmaceutical industry in the research and development of these innovative therapies, and in 2019, revised the Act on Securing Quality, Efficacy, and Safety of Products Including Pharmaceuticals and Medical Devices to enact the conditional early approval system for accelerating the development and ...
  • Regulatory NewsRegulatory News

    Two more stem cell firms slapped with untitled letters

    Untitled letters issued to two firms for marketing unapproved stem cell products signal that some in the industry are undeterred by the US Food and Drug Administration’s (FDA) efforts to get these products approved or off the market.       The letters join four others from the Center for Biologics Evaluation and Research (CBER) in recent months to clinics marketing unapproved stem cell products after an enforcement grace period ended on 1 June. (RELATED: CBER targets...
  • Regulatory NewsRegulatory News

    CBER targets four stem cell firms with untitled letters

    The US Food and Drug Administration’s Center for Biologics Evaluation and Research (FDA’s CBER) recently issued untitled letters to four companies for marketing unapproved stem cell products, indicating that despite the end of an enforcement grace period, companies are still resisting the agency’s efforts to get these products approved.   Effective June 1, companies are required to file biologics license applications for stem cell therapies that claim to treat a diseas...
  • Regulatory NewsRegulatory News

    CBER Q&A addresses stem cell enforcement questions

    The US Food and Drug Administration (FDA) issued a question-and-answer document on Monday to address issues that may arise now that the agency's enforcement discretion policy against unauthorized marketing of these products has come to an end.   The agency’s Center for Biologics Evaluation and Research (CBER) ended a period of enforcement discretion against purveyors of unauthorized human cells, tissues, or cellular or tissue- based products (HCT/Ps) on 31 May 2021. Ac...
  • Regulatory NewsRegulatory News

    Recent ruling gives FDA ammunition against unapproved cell therapies

    A recent appeals court decision strengthens the US Food and Drug Administration’s efforts to crack down on clinics making unapproved stem cell therapies, the agency’s Melissa J. Mendoza said at a recent meeting.   The 11 th US Circuit Court of Appeals rendered a decision in USA vs US Stem Cell Clinic LLC , agreeing with the “FDA that these products did not fit the same surgical procedure exemption” and should be regulated as drugs, said Mendoza, deputy director of ...
  • Regulatory NewsRegulatory News

    FDA disappointed with slow uptake of approvals for stem cell therapies

    At a recent meeting, a US Food and Drug Administration official said the agency is “concerned” about the slow uptake from companies seeking regulatory approval to market and distribute stem cell therapies and is therefore stepping up their enforcement efforts against companies making unapproved therapies.   Wilson Bryan, director of FDA’s Office of Tissues and Advanced Therapies (OTAT) in FDA’s Center for Biologics Evaluation and Research, told attendees of an 8 June m...