A5059639133- Chronic Lymphocytic Leukemia Research
- PI3K/AKT/mTOR signaling in cancer
- Genomics and Rare Diseases
- Vascular Malformations and Hemangiomas
- Pharmaceutical studies and practices
- Adenosine and Purinergic Signaling
- Pharmaceutical Economics and Policy
- CRISPR and Genetic Engineering
- Biochemical and Molecular Research
- Biosimilars and Bioanalytical Methods
- Statistical Methods in Clinical Trials
- Anesthesia and Sedative Agents
Center for Drug Evaluation and Research
2020-2023
United States Food and Drug Administration
2020-2023
Abstract On April 5, 2022, FDA granted accelerated approval to alpelisib for the treatment of adult and pediatric patients 2 years age older with severe manifestations PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy. Efficacy was evaluated using real-world data (RWD) from EPIK-P1 (NCT04285723), a single-arm clinical study in or life-threatening PROS received as part an expanded access program (EAP) compassionate use. The primary endpoint confirmed radiologic response...
Abstract Background The small patient populations inherent to rare genetic diseases present many challenges the traditional drug development paradigm. One major challenge is generating sufficient data in early phase studies inform dose selection for later and optimization clinical use of drug. However, optimizing benefit-risk profile drugs through appropriate during critical all drugs, including those being developed treat diseases. Recognizing conducting finding disease importance...
<div>Abstract<p>On April 5, 2022, FDA granted accelerated approval to alpelisib for the treatment of adult and pediatric patients 2 years age older with severe manifestations PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy. Efficacy was evaluated using real-world data (RWD) from EPIK-P1 (NCT04285723), a single-arm clinical study in or life-threatening PROS received as part an expanded access program (EAP) compassionate use. The primary endpoint confirmed...
<div>Abstract<p>On April 5, 2022, FDA granted accelerated approval to alpelisib for the treatment of adult and pediatric patients 2 years age older with severe manifestations PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy. Efficacy was evaluated using real-world data (RWD) from EPIK-P1 (NCT04285723), a single-arm clinical study in or life-threatening PROS received as part an expanded access program (EAP) compassionate use. The primary endpoint confirmed...
A key goal in drug development is optimized dosing for patients. Interactions between developers and regulatory scientists throughout are important the optimization of serve as a forum to discuss approaches optimal dosing, such precision or individualized dosing. To date, there has not been systematic assessment advice provided by US Food Drug Administration (FDA) from an perspective. Here, we reviewed FDA recommendations on dose selection efficacy trials at end-of-phase meetings 76 new...