In today’s fast-paced world of scientific discovery, cutting-edge technology is revolutionizing the way we approach complex biological problems. One such innovation is in silico modeling, a method that harnesses the power of computer simulations to model biological systems. But what exactly is in silico modeling? How does it work, and why is it becoming a…
Tag: fda 16 results
The FDA’s Center for Drug Evaluation and Research issued a structured, meticulous roadmap to advance the clinical development of psychedelic drugs, highlighting key considerations in regulatory frameworks, ethical conduct, chemistry manufacturing controls (CMC), and clinical studies to ensure safety and efficacy in addressing psychiatric and substance use disorders. In this blog, Premier Research dissects the…
Oncology & Hematology
Rare Oncology and the FDA: Taking the Guesswork Out of Expedited Pathways
Rare cancers account for 25-30 percent of all new cancer diagnoses and 25 percent of cancer deaths.1 With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Faced with limited treatment options, researchers, clinicians, and patients may be…
One of the most common formulation issues that sponsors of new drug or biologic products face is the qualification of novel excipients. Although novel excipients can be a part of any new drug application (NDA) or biologics license application (BLA) development program, they seem to be more common with the 505(b)(2) pathway, since many companies…
Cell & Gene Therapy
PDUFA VII: Implications for Developers of Cell and Gene Therapies
One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). PDUFA VII, as the reauthorization is commonly known, will cover the fiscal years 2023-2027. Many of the advancements included in PDUFA VII are driven in large part by an increase in…
Oncology & Hematology
New FDA Draft Guidance Provides Insights on Use of PROs in Oncology Trials
Incorporating patient-reported outcomes (PROs) into clinical trials can help sponsors better understand patients’ symptoms and how a therapy will affect their quality of life, and these insights can be particularly valuable in oncology research. However, there has been a lack of guidance to help sponsors ensure consistency and quality when incorporating PROs into cancer drug…
Drug development is a resource-intensive endeavor. Seeking input from the U.S. Food and Drug Administration throughout the journey can help optimize those resources and maximize the likelihood of regulatory approval. When preparing to submit a new drug application (NDA), a pre-NDA meeting with the FDA can be a critical step in ensuring the submission of…
A clinical hold from the U.S. Food and Drug Administration can significantly prolong the time and increase the cost of drug development, which is particularly concerning for emerging/small biotech and specialty pharma companies.
Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases.[1] The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research and development in this area with more than 20 vouchers granted to date, but it…
The Electronic Common Technical Document (eCTD) is the standard format for submitting applications, amendments, supplements, and reports to the U.S. Food & Drug Administration (FDA). The eCTD harmonizes the regulatory review process for global drug development, as its structure is based on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use…