Abstract
BackgroundThe available evidence on the benefits and harms of novel drugs and therapeutic biologics at the time of approval is reported in publicly available documents provided by the US Food and Drug Administration (FDA). We aimed to create a comprehensive database providing the relevant information required to systematically analyze and assess this early evidence in meta-epidemiological research.MethodsWe designed a modular and flexible database of systematically collected data. We identified all novel cancer drugs and therapeutic biologics approved by the FDA between 2000 and 2016, recorded regulatory characteristics, acquired the corresponding FDA approval documents, identified all clinical trials reported therein, and extracted trial design characteristics and treatment effects. Herein, we describe the rationale and design of the data collection process, particularly the organization of the data capture, the identification and eligibility assessment of clinical trials, and the data extraction activities.DiscussionWe established a comprehensive database on the comparative effects of drugs and therapeutic biologics approved by the FDA over a time period of 17 years for the treatment of cancer (solid tumors and hematological malignancies). The database provides information on the clinical trial evidence available at the time of approval of novel cancer treatments. The modular nature and structure of the database and the data collection processes allow updates, expansions, and adaption for a continuous meta-epidemiological analysis of novel drugs.The database allows us to systematically evaluate benefits and harms of novel drugs and therapeutic biologics. It provides a useful basis for meta-epidemiological research on the comparative effects of innovative cancer treatments and continuous evaluations of regulatory developments.
Highlights
The available evidence on the benefits and harms of novel drugs and therapeutic biologics at the time of approval is reported in publicly available documents provided by the US Food and Drug Administration (FDA)
The current database will be used to describe the clinical trial evidence generated in the pre-marketing period, but the database can and will be updated and expanded for future meta-epidemiological analyses. What this adds to what is known Publicly available drug approval documents offer highly valuable information that is very useful for evidence syntheses and research-on-research projects
In step 2, we made an inventory of Randomized controlled trial (RCT) and Single-arm trial (SAT) reported in FDA approval documents, assessed their eligibility, and extracted trial design characteristics
Summary
The available evidence on the benefits and harms of novel drugs and therapeutic biologics at the time of approval is reported in publicly available documents provided by the US Food and Drug Administration (FDA). Cancer drug development is characterized by a perceived urgency to find novel treatments that improve patients’ survival and quality of life. Access to such beneficial treatments is considered paramount for patients with cancer. For drugs and therapeutic biologics that receive approval, the FDA reviews are made publicly available in the Drugs@FDA database as “approval packages” [3]. These packages provide a wealth of information on the evidence on benefits and harms of innovative treatments at the time of approval
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