scholarly journals Open label multi-centre trial on efficacy and tolerability of microionized isotretinion therapy in treatment of moderate to severe acne vulgaris

2020 ◽  
Vol 7 (1) ◽  
pp. 7
Author(s):  
Bela Shah ◽  
Neha Jangid ◽  
Aswini A. Naidu
Keyword(s):  
Acne Vulgaris ◽  
Open Label ◽  
Total N ◽  
Severe Acne ◽  
Drug Tolerability ◽  
Satisfactory Outcome ◽  
United States Food ◽  
States Food ◽  
Us Fda ◽  
Isotretinoin Therapy

<p class="abstract"><strong>Background:</strong> Isotretinion was approved by United States Food and Drug Administration (US FDA) in 1982 for the treatment of severe recalcitrant nodulocystic acne. Its conventional recommended dose has been 0.5-1.0 mg/kg body weight per day for 16-32 weeks, with a maximum cumulative dose of 120 mg/kg. Objective of the study was to assess the efficacy and tolerability of 24 week of once daily micronized isotretinoin therapy in the treatment of moderate to severe acne vulgaris was conducted.</p><p class="abstract"><strong>Methods:</strong> Total n=580 of patients were included with 249 (43%) male and 331 (57%) females. Patients were assessed at baseline 6, 12 and 24 weeks and 12 weeks post treatment follow-up based on the assessment of severity of acne vulgaris using global acne grading system (GAGS), assessment of improvement in lesion counts, global assessment of overall efficacy by doctor (based on overall assessment) and by patients (based on overall relief in symptoms) and overall assessment of drug tolerability.<strong></strong></p><p class="abstract"><strong>Results:</strong> In terms of improvement in lesions, excellent results from 4% (22) in week 6th has move to 34% (193) in week 24th. In terms of global efficacy examined by doctors, very effective results i.e. 40% (234) in 12th week has moved to 70% (403) in 24th week. In terms of drug tolerability, excellent results have moved from 119 patients to 190 patients by end of the study.</p><p class="abstract"><strong>Conclusions:</strong> Hence the micronized isotretinoin therapy had overall satisfactory outcome in the treatment of patients with moderate to severe acne vulgaris (grade 2, 3 and 4).</p>

scholarly journals Ravulizumab: a novel C5 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria

2019 ◽  
Vol 10 ◽  
pp. 204062071987472 ◽  
Author(s):  
Robert M. Stern ◽  
Nathan T. Connell
Keyword(s):  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Bone Marrow Failure ◽  
Phase Iii ◽  
The European Union ◽  
Regulatory Review ◽  
Dosing Schedule ◽  
The Us ◽  
United States Food ◽  
States Food ◽  
Us Fda

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare stem cell disorder characterized by hemolytic anemia, bone marrow failure, and thrombosis. Until recently, the complement inhibitor, eculizumab, was the only United States Food and Drug Administration (US FDA)-approved therapy for the treatment of PNH. Although effective, eculizumab requires a frequent dosing schedule that can be burdensome for some patients and increases the risk of breakthrough intravascular hemolysis. Ravulizumab, an eculizumab-like monoclonal antibody engineered to have a longer half-life, is intended to provide the same benefits as eculizumab but with a more convenient and effective dosing schedule. In two recently published phase III non-inferiority trials, ravulizumab was found to be non-inferior to eculizumab both in efficacy and safety for the treatment of patients with PNH. Based on these results, ravulizumab was approved by the US FDA on 21 December 2018 and is currently under regulatory review in both the European Union and Japan.

Improving immune-related adverse event management in a thoracic clinic.

2017 ◽  
Vol 35 (8_suppl) ◽  
pp. 74-74
Author(s):  
Meghan Shea ◽  
Deepa Rangachari ◽  
Daniel Botelho Costa ◽  
Aya Sato-DiLorenzo ◽  
Jessica A. Zerillo
Keyword(s):  
Adverse Events ◽  
Response Rate ◽  
Package Insert ◽  
Retrospective Chart Review ◽  
High Response Rate ◽  
Event Management ◽  
Metastatic Nsclc ◽  
United States Food ◽  
States Food ◽  
Us Fda

74 Background: Widespread use of immunotherapeutic agents has transformed the profile of adverse events associated with systemic cancer therapy. Management of immune-related adverse events (IRAEs) is contingent upon grading severity using the National Cancer Institute’s Common Terminology Criteria for Adverse Events (CTCAE). Nivolumab and Pembrolizumab were recently approved for metastatic non-small cell lung cancer (NSCLC). United States Food and Drug Administration (US FDA)-approved package label inserts provide guidance on IRAE management and are predicated on CTCAE grade, including when to discontinue drug. Currently, clinicians in the thoracic oncology group are documenting CTCAE grade of IRAEs infrequently, and management is varied. Methods: A retrospective chart review of baseline data revealed 45 patients (8 on Pembrolizumab, 37 on Nivolumab) who initiated immunotherapy for metastatic NSCLC between March 2015 and August 2016. A team of clinicians developed a process map from diagnosis of IRAE to initiation of toxicity management. Physicians were surveyed. The team’s aim is by February 1, 2017, at least 50% of patients who develop an IRAE on immunotherapy for metastatic NSCLC have documentation of toxicity grade using the CTCAE criteria. Results: The physician survey response rate was 12 of 16 (75%). Physicians reported not using grade to guide management of IRAEs over two thirds (67%) of the time. Time to look up CTCAE criteria and knowing that grade is needed ranked as the top barriers. At baseline, 18 of 45 (40%) patients had 22 IRAEs, of which 6 IRAEs (27%) had grading documented; all graded IRAEs (100%) were managed according to guidelines in the drug-specific package insert. IRAEs included hypothyroidism, pneumonitis, hepatitis, dermatitis, adrenal insufficiency, colitis, and encephalitis. Conclusions: Education on toxicity grading and ease of accessibility to information regarding management of IRAEs are needed. Because clinicians were engaged, a survey to evaluate the current process succeeded with a high response rate. At baseline, there are significant gaps and variability in current practice. Interventions are underway to standardize documentation of grade and management of patients experiencing IRAEs.

Implementation of Question-based Review in support of Quality by Design streamlining the US FDA’s review process

2018 ◽  
Vol 14 (3) ◽  
pp. 129-134
Author(s):  
Alisha Desai ◽  
Jayanta Kumar Maji ◽  
Kanhoba Walavalkar ◽  
Priti J Mehta
Keyword(s):  
Quality By Design ◽  
Review Process ◽  
Critical Material ◽  
Technical Requirements ◽  
The Us ◽  
Product Profile ◽  
United States Food ◽  
Critical Material Attributes ◽  
States Food ◽  
Us Fda

Question-based Review (QbR) is a format proposed by United States Food and Drug Administration (US FDA) enhancing the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use's Common Technical Document (ICH CTD) format to streamline the submission process. It is a question–answer format applied to Quality Overall Summary section of the submission. The format includes putting up questions under every section, so the applicant can submit precise and accurate data for approval of the respective application. The QbR format can be applied to NDA, ANDA, and Type II DMF applications. The companion document available with Manual of Policy and Procedures 5015.10 (MaPP 5015.10) allows the reviewer to inspect the critical information in the data provided. It encourages applicants to encompass Quality by Design (QbD) in their development process. QbR gives a structure through which the data collected by applying QbD can be presented. For effective application of QbR format, the submission should be backed with thorough scientific knowledge, risk assessment data, and data integrity. The questions asked compel the applicant to provide justification for the various decisions made in the development phase. Also, questions regarding quality target product profile, critical quality attributes, critical material attributes, critical process parameters and design of experiment are covered under the QbR format. MaPP 5015.10 finalized by US FDA in 2014 clarifies the concept of QbR. There is MicroQbR available which includes questions confirming the sterility of the product. QbR is a step towards speeding up the review process with an intention to motivate the applicants to implement QbD to the project.

US FDA Warning Letters of CAPA Violations: A Review

2020 ◽  
Vol 7 (2) ◽  
pp. 85-92
Author(s):  
Pavan Deshpande ◽  
Rutuja Agawane ◽  
Sarath C. Tatikola ◽  
Surenahalli G. Vasantharaju
Keyword(s):  
United States ◽  
Drug Administration ◽  
The United States ◽  
Quality And Safety ◽  
Drug Substances ◽  
Warning Letter ◽  
End Products ◽  
United States Food ◽  
States Food ◽  
Us Fda

United States Food and Drug Administration (USFDA) is a federal agency functioning under United States Federal Executive Departments, which strives to regulate the food products and drug substances being manufactured or brought into US market, upholding Quality and Safety as prime goals. It takes care of its goals by inspecting firms which market products in the United States. It chalks out good manufacturing procedures for obtaining quality end-products. Based on inspections conducted and data collected thereby, those not abiding by rules shall be issued with Warning Letters and marketing license shall be cancelled for those who fail to justify the warning letter. This brings about discipline amongst manufacturers and sets a goal of quality that needs to be achieved to survive in market.

Acne Treatment With Light Absorbing Gold Microparticles and Optical Pulses: An Open‐Label European Multi‐Centered Study in Moderate to Moderately Severe Acne Vulgaris Patients

2019 ◽  
Vol 51 (8) ◽  
pp. 686-693 ◽  
Author(s):  
Christine Sofie Krohn Fuchs ◽  
Christiane Bay ◽  
Maurice Adatto ◽  
Hans Lomholt ◽  
Merete Haedersdal
Keyword(s):  
Acne Vulgaris ◽  
Optical Pulses ◽  
Open Label ◽  
Severe Acne ◽  
Acne Treatment
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