Real-world Evidence in Support of Oncology Product Registration: A Systematic Review of New Drug Application and Biologics License Application Approvals from 2015-2020

2021 ◽  
pp. clincanres.2639.2021
Author(s):  
Bhakti Arondekar ◽  
Mei Sheng Duh ◽  
Rachel H. Bhak ◽  
Maral DerSarkissian ◽  
Lynn Huynh ◽  
...  
Keyword(s):  
Systematic Review ◽  
Real World ◽  
Drug Application ◽  
New Drug ◽  
Biologics License Application ◽  
Real World Evidence ◽  
License Application ◽  
New Drug Application

scholarly journals REVIEW ON SPONSOR / APPLICANT MEETINGS WITH FDA: HIGHLIGHTS OF NEW GUIDANCE OVER EXISTING

2018 ◽  
Vol 4 (2) ◽  
pp. 19-26
Author(s):  
Charmy Kothari ◽  
Kavina Shah
Keyword(s):  
Clinical Trials ◽  
Management Practices ◽  
Drug Application ◽  
Development Program ◽  
The United States ◽  
United States Department ◽  
Biological Products ◽  
New Drug ◽  
License Application ◽  
New Drug Application

The United States Department of Health and Human Services has a federal agency called the Food and Drug Administration (FDA or USFDA). A pre-planned assembling of two or more people who have been together for the purpose of getting a common goal via verbal interaction is called a formal meeting. During development stage of any drug or biological products pharmaceutical companies face trouble for both scientific and regulatory point of view, here role of formal meetings comes. Formal meetings between sponsor or applicant and FDA are usually related to development and review of drug and biological products. Center for Drug evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) regulates the formal meetings. These meetings are applicable to Pre – Investigational New Drug Application, Pre – Biologics License Application, New Drug Application for drugs and biological products and not applicable to Abbreviated New Drug Applications (ANDA), application of medical devices and submission of biosimilar biological products. Meetings between FDA and sponsor or applicant are for resolution of dispute, clinical holds discussion, Assessment of protocols of clinical trial, during clinical trials, in between clinical trials – at the phase 1 ending or at the phase 2 ending, to discuss development program. The FDA has classified these formal meetings in different types based on the nature of the request, the information in the meeting request and each meeting type is handled through different procedures. The principles of Good Meeting Management Practices (GMMPs) must be maintained. There are specific requirements and procedures to request, prepare, schedule, conduct and document formal meetings. As the guidance documents for meetings are revised by FDA, Change in procedure and requirements takes place. Any pharmaceutical company need to be in line with new guidance requirements to avoid rejection. Formal meetings between sponsor or applicant and FDA save time, cost and will increase the probability of product approval.

Role of real-world evidence for oncology product registration in the United States: A review of approvals by the U.S. Food and Drug Administration from 2015 to 2019.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e14130-e14130
Author(s):  
Bhakti Arondekar ◽  
Rachel Bhak ◽  
Maral DerSarkissian ◽  
Lynn Huynh ◽  
Kelsey Wang ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Real World ◽  
Meta Analysis ◽  
A Priori ◽  
Drug Application ◽  
Clinical Trial Data ◽  
Retrospective Chart Review ◽  
The United States ◽  
Real World Evidence ◽  
License Application

e14130 Background: There are few concrete examples of real world evidence (RWE) used to support clinical development in regulatory filings despite growing interest in this field. This study systematically reviewed FDA oncology approvals to identify use cases of RWE. Methods: FDA’s new drug application (NDA) and biologics license application (BLA) approvals for oncology products from 2015-2019 were systematically reviewed. Among cases with RWE, data characterizing the submission and RWE details (data source, study design, FDA comments) were synthesized. Results: 93 approved NDAs and BLAs were identified; 6 included RWE in support of efficacy (see Table), approved on or after 2017, and were largely retrospective studies that contextualized results to pivotal trial, with primary endpoints overall survival (OS), overall response rate (ORR), and time to treatment discontinuation (TTD). Flatiron data were used in 3 of these as database analyses, 1 was an expanded access program (EAP), 1 was a meta-analysis, and 1 was a retrospective chart review. Conclusions: In the past 5 years, few FDA decisions incorporated RWE in oncology drug approvals. When used, RWE has been a complement rather than a supplement for clinical trial data. Early engagement, a priori protocol development, and robust research design (adjusting for bias, comparability to clinical trial population) remain key determinants for successful use of RWE in FDA decision making. [Table: see text]

Drug Development 101: A Primer

2020 ◽  
Vol 39 (5) ◽  
pp. 379-396
Author(s):  
Lorrene A. Buckley ◽  
Ilona Bebenek ◽  
Paul D. Cornwell ◽  
Aimee Hodowanec ◽  
Eric C. Jensen ◽  
...  
Keyword(s):  
Drug Development ◽  
Drug Application ◽  
Safety Testing ◽  
New Drug ◽  
Phase Development ◽  
Introductory Overview ◽  
Candidate Drug ◽  
License Application ◽  
Regulatory Submissions ◽  
New Drug Application

Drug development is a term used to define the entire process of bringing a new drug or device to market. It is an integrated, multidisciplinary endeavor that includes drug discovery, chemistry and pharmacology, nonclinical safety testing, manufacturing, clinical trials, and regulatory submissions. This report summarizes presentations of a workshop entitled “Drug Development 101,” held at the 39th Annual Meeting of the American College of Toxicology in West Palm Beach, Florida. The workshop was designed to provide an introductory overview of drug development. Experienced scientists from industry and government provided overviews of each area, with a focus on safety assessment, and described some of the challenges that can arise. The role of chemistry and manufacturing was discussed in the context of early- and late-stage product development and approaches to assess, control, and limit impurities. The toxicologic assessment was emphasized in early-phase development, from the selection of a candidate drug through the determination of a first-in-human starting dose. Clinical trial development was discussed in the context of regulatory requirements and expectations. The final topic of issues and considerations in the review processes of different types of submissions to Food and Drug Administration included advice for best practices in authoring good Investigational New Drug and New Drug Application/Biologic License Application submissions and interacting effectively with regulatory reviewers.

Utility of Bayesian Single-Arm Design in New Drug Application for Rare Cancers in Japan: A Case Study of Phase 2 Trial for Sarcoma

2018 ◽  
Vol 52 (3) ◽  
pp. 334-338 ◽  
Author(s):  
Akihiro Hirakawa ◽  
Tadaaki Nishikawa ◽  
Kan Yonemori ◽  
Taro Shibata ◽  
Kenichi Nakamura ◽  
...  
Keyword(s):  
Drug Application ◽  
Phase 2 ◽  
Rare Cancers ◽  
New Drug ◽  
Phase 2 Trial ◽  
New Drug Application

Systematic review of the real-world evidence of adalimumab safety in psoriasis registries

2018 ◽  
Vol 32 (12) ◽  
pp. 2126-2133 ◽  
Author(s):  
B. Strober ◽  
J. Crowley ◽  
R.G. Langley ◽  
K. Gordon ◽  
A. Menter ◽  
...  
Keyword(s):  
Systematic Review ◽  
Real World ◽  
The Real ◽  
Real World Evidence

scholarly journals FDA relations during drug development

2000 ◽  
Vol 2 (3) ◽  
pp. 213-217
Keyword(s):  
Drug Development ◽  
Drug Application ◽  
Development Program ◽  
The United States ◽  
Legal Aspects ◽  
Development Programs ◽  
New Drug ◽  
Regulatory Affairs ◽  
New Drug Application ◽  
Drug Development Program

Working closely and cooperatively with regulatory authorities during drug development is vital to successful drug development programs. In the United States, the drug development team includes not only members of the key disciplines of drug discovery, clinical research, regulatory affairs, marketing, chemistry, toxicology, and legal aspects, but also the Food and Drug Administration (FDA). New regulations encourage meetings at the pre-investigational new drug (pre-IND), end-of-phase-2, and pre-new drug application (pre-NDA) submission phases. Appropriate informal discussions via fax and telephone are also encouraged. By proactively interacting with the FDA, the pharmaceutical industry increases the probability of a successful drug development program.

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