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  • Feature ArticlesFeature Articles

    Advanced therapies: ‘Trip hazards’ on the development pathway

    This article concerns the development of advanced therapies and the challenges and complexities of getting them through the developmental pathway, which the authors call “the trip.” The authors examine at strategic levels the importance of some of the fundamental building blocks for the development program and highlight some commonly encountered challenges (trip hazards) for cell and gene therapies and offer “bench-to-bedside” and chemistry, manufacturing, and controls (CM...
  • Regulatory NewsRegulatory News

    Hahn says COVID-19 vaccines will be reviewed in ‘real time’

    At an event marking the launch of Moderna’s Phase 3 clinical trial for its mRNA coronavirus vaccine in Miami on Monday, US Food and Drug Administration (FDA) Commissioner Stephen Hahn repeated assurances that his agency would not cut corners in approving a COVID-19 vaccine.   Pfizer also announced on Monday that it would begin a Phase 2/3 study of its COVID-19 vaccine developed in partnership with BioNTech. Both companies say they plan to enroll 30,000 participan...
  • Regulatory NewsRegulatory News

    Lawmakers ask FDA to outline COVID-19 vaccine approval process

    Key Democratic members in the US House of Representatives have called on Stephen Hahn, MD, commissioner of the US Food and Drug Administration (FDA), to brief their staff on how the agency plans to make decisions regarding the development, review and deployment of vaccines against the novel coronavirus SARS-CoV-2.   In a letter dated 18 June, Rep. Carolyn Maloney (D-NY), chairwoman of the House Committee on Oversight and Reform, Rep. Raja Krishnamoorthi (D-IL), chairma...
  • Feature ArticlesFeature Articles

    Bespoke therapies – opportunities, challenges, and hope

    This article discusses the advent of bespoke therapies, defined as the tailoring of medical treatment to the individual characteristics or symptoms and responses of a patient during all stages of care and as a new frontier beyond personalized medicine. The author covers the revolutionary genetic tools implementing such therapies and the clinical and nonclinical safety perspectives for bespoke therapies. The author concludes that with bespoke therapies we are entering a new...
  • Regulatory NewsRegulatory News

    Patient Groups, Industry Seek Changes to Rare Disease Drug Guidance

    Patient groups, drugmakers and other organizations are calling for changes to the US Food and Drug Administration’s (FDA) recently revised draft guidance on developing drugs to treat rare diseases.   The 24-page draft guidance was revised last February after first being published in 2015. The revision included updates to the agency’s approach to natural history studies, a discussion of issues for evaluating biomarkers for consideration as surrogate endpoints and a ne...
  • Regulatory NewsRegulatory News

    Asia Regulatory Roundup: Singapore’s HSA Apologizes After Vendor Stores Data in Vulnerable Repository

    Welcome to our Asia Regulatory Roundup, our weekly overview of the top regulatory news in Asia.   Singapore’s HSA Apologizes After Vendor Stores Data in Vulnerable Repository   Singapore’s Health Sciences Authority (HSA) has apologized to blood donors after a vendor stored their information in a vulnerable database. HSA learned the database lacked adequate safeguards from a cybersecurity expert and does not think other unauthorized people accessed the information...
  • Regulatory NewsRegulatory News

    FDA Finalizes Guidance on Nonclinical Drug Development for Serious Hematologic Disorders

    The US Food and Drug Administration (FDA) on Thursday finalized guidance on the nonclinical studies drugmakers should conduct when developing products to treat severely debilitating or life-threatening hematologic disorders (SDLTHDs).   FDA classifies SDLTHDs as hematologic conditions that cause shorter life expectancy or greatly diminished quality of life even with available treatments.   FDA notes that the guidance does not apply to hematologic cancers and covers...
  • Regulatory NewsRegulatory News

    FDA Drafts Guidance on Developing Drugs for Rare Chronic Digestive Disorder

    The US Food and Drug Administration (FDA) issued draft guidance on Wednesday to aid sponsors in the clinical development of drugs and biologics for the treatment of eosinophilic esophagitis (EoE). The 13-page draft guidance document describes the agency’s clinical trial recommendations for EoE drugs. These relate to trial population and design, safety and efficacy considerations, clinical outcomes assessments, as well as pediatric considerations. The clinical developmen...
  • Regulatory NewsRegulatory News

    Sanofi Raises Public Health Questions on Use of Nonsense Suffixes With New Vaccines

    A lot of ink has been spilled on the controversial random suffixes that the US Food and Drug Administration (FDA) is now tagging on the end of the nonproprietary names of biologics and biosimilars. Back in January 2017, FDA finalized a guidance document spelling out how this four-letter, FDA-designated meaningless suffix would be attached at the end of biologics’ and biosimilars’ nonproprietary names. Later in 2017 and 2018, companies began to realize FDA was only u...
  • Regulatory NewsRegulatory News

    EMA Recommends When Drugmakers Should Consult With Pediatric Research Network

    The European Medicines Agency (EMA) on Monday released recommendations from its European network of pediatric research (Enpr)-EMA working group on opportunities during various stages of development to consult with pediatric research groups.   “Enpr-EMA suggests that all companies consider using these opportunities in a spirit of shared learning about the best way to collaborate during [pediatric investigation plan] planning,” EMA writes, noting that companies can benef...
  • Regulatory NewsRegulatory News

    FDA Works to Reduce Dog Testing in Drug Development

    US Food and Drug Administration (FDA) researchers are working to develop a new informatics model to reduce the reliance on dogs to conduct studies in animal drug development.   The agency’s new study, which was proposed on Friday, is intended to aid animal drug developers in conducting certain research without the need for product testing on dogs.   Animal drug developers would be able to use the new model as a mechanism for comparing blood levels of certain oral...
  • Regulatory NewsRegulatory News

    FDA Finalizes Testicular Toxicity Guidance

    The US Food and Drug Administration (FDA) on Wednesday finalized guidance for drugmakers on identifying and evaluating early signals that could indicate whether a drug might have adverse effects on the testes and when clinical trials are necessary to assess those risks.   Specifically, the 14-page guidance lays out recommendations for identifying nonclinical signals that suggest risk of testicular toxicity; conducting nonclinical assessments to further evaluate the ris...