The Comparative Effectiveness of Innovative Treatments for Cancer (CEIT-Cancer) project: Rationale and design of the database and the collection of evidence available at approval of novel drugs

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.Electronic supplementary materialThe online version of this article (10.1186/s13063-018-2877-z) contains supplementary material, which is available to authorized users.

Characteristics and interpretation of subgroup analyses based on tumour characteristics in randomised trials testing target-specific anticancer drugs: design of a systematic survey

“…A database that our group has established for a related project will provide an initial list of target-specific drugs that we will extend during the screening. 30 Teams of two investigators, one of whom will be an oncologist, will assess final eligibility and, if needed, resolve disagreements by discussion or, if needed, third-party adjudication. The teams will also independently identify reported tumour characteristics used for subgroup analyses.…”

“…In previous empirical studies, investigators have successfully used similar methods, including systematic surveys of subgroup analyses in cancer trials, 4 assessing the strength of subgroup claims, 7 11 12 and qualitative analysis of clinical practice guidelines. 30 We anticipate some difficulties in rating the credibility of subgroup claims related to reporting quality. For instance, some trials will not provide a published protocol or insufficiently report the number of effect modifiers tested or numerical results.…”

Contrasting evidence to reimbursement reality for off-label use (OLU) of drug treatments in cancer care: rationale and design of the CEIT-OLU project

“…Here, we describe the rationale and design of the OLU-Cancer project as part of the Comparative Effectiveness of Innovative Treatments (CEIT) project 13. The CEIT project evaluates the generation of clinical trial evidence for novel drug treatments, in the case of OLU using a large sample of patients to provide empirical evidence on access to OLU in a highly diverse western healthcare system.…”

Contrasting Evidence to Reimbursement Reality for Off-label use (OLU) of Drug Treatments in Cancer Care – Rationale and Design of the CEIT-OLU-project

“…Here we describe the rationale and design of the OLU-Cancer project as part of the Comparative Effectiveness of Innovative Treatments (CEIT) project (13). The CEIT project evaluates the generation All rights reserved.…”

“…Here we describe the rationale and design of the OLU-Cancer project as part of the Comparative Effectiveness of Innovative Treatments (CEIT) project (13). The CEIT project evaluates the generation of clinical trial evidence for novel drug treatments, in the case of OLU using a large sample of patients to provide empirical evidence on access to OLU in a highly diverse Western health care system.…”