Dataset Information

Applications for oncologic drugs: a descriptive analysis of the oncologic drugs advisory committee reviews.

ABSTRACT:

Background

Despite advances in cancer research, the majority of drug applications submitted to the U.S. Food and Drug Administration (FDA) are not approved. It is important to identify the concerns of the Oncologic Drugs Advisory Committee (ODAC) from rejected applications.

Methods

All applications referred to the ODAC from 2001 to 2012 were reviewed.

Results

Of 46 applications, 31 (67%) were for full and 15 (33%) were for supplemental approval, 34 (74%) were for solid and 12 (26%) were for hematologic tumors. In all, 22 (48%) were not approved. ODAC comments addressed missing or inadequate data (65%), excessive toxicity (55%), inappropriate study endpoints (45%), poor study design (40%), and insufficient sample size (30%). To define efficacy, 19 applications used response rates (RR) (median = 38%), and 19 applications used hazard ratios (HR) (median = 0.67). For all organ systems combined, the median cumulative grade 3 or 4 toxicity was 64%. Drugs with higher RR, lower HR, and lower toxicity were more likely to be approved versus other drugs (89% vs. 45%; p = .02). Over time (2001-2004, 2005-2008, 2009-2012), there was an increase in the following: number of applications submitted for review (from 11 to 12 to 23, respectively), number of approvals (from 6 to 6 to 12, respectively), and proportion of trials using progression-free survival as a primary endpoint (from 0% to 50% to 70%, respectively; p = .01).

Conclusion

Of all applications, common ODAC concerns included inadequate data, excessive toxicity, and inappropriate study endpoints. Over time, there was an approximate doubling of FDA application submissions and approved oncology drugs.

SUBMITTER: Chan JK

PROVIDER: S-EPMC3958455 | biostudies-literature | 2014 Mar

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Applications for oncologic drugs: a descriptive analysis of the oncologic drugs advisory committee reviews.

Chan John K JK Kiet Tuyen K TK Monk Bradley J BJ Young-Lin Nichole N Blansit Kevin K Kapp Daniel S DS Amanam Idoroenyi I

The oncologist 20140305 3

<h4>Background</h4>Despite advances in cancer research, the majority of drug applications submitted to the U.S. Food and Drug Administration (FDA) are not approved. It is important to identify the concerns of the Oncologic Drugs Advisory Committee (ODAC) from rejected applications.<h4>Methods</h4>All applications referred to the ODAC from 2001 to 2012 were reviewed.<h4>Results</h4>Of 46 applications, 31 (67%) were for full and 15 (33%) were for supplemental approval, 34 (74%) were for solid and ...[more]

PMID: 24599479

Similar Datasets

Project description:ObjectiveTo investigate the association between conflicts of interest and favourable recommendations in clinical guidelines, advisory committee reports, opinion pieces, and narrative reviews.DesignSystematic review.Eligibility criteriaStudies that compared the association between conflicts of interest and favourable recommendations of drugs or devices (eg, recommending a drug) in clinical guidelines, advisory committee reports, opinion pieces (eg, editorials), or narrative reviews.Data sourcesPubMed, Embase, Cochrane Methodology Register (from inception to February 2020), reference lists, Web of Science, and grey literature.Data extraction and analysisTwo authors independently extracted data and assessed the methodological quality of the studies. Pooled relative risks and 95% confidence intervals were estimated using random effects models (relative risk >1 indicates that documents with conflicts of interest more often had favourable recommendations than documents with no conflicts of interest). Financial and non-financial conflicts of interest were analysed separately, and the four types of documents were analysed separately (preplanned) and combined (post hoc).Results21 studies that analysed 106 clinical guidelines, 1809 advisory committee reports, 340 opinion pieces, and 497 narrative reviews were included. Unpublished data were received for 11 studies (eight full datasets and three summary datasets). 15 studies showed risk of confounding because the compared documents could differ in factors other than conflicts of interest (eg, different drugs used for different populations). The relative risk for associations between financial conflicts of interest and favourable recommendations for clinical guidelines was 1.26 (95% confidence interval 0.93 to 1.69; four studies of 86 clinical guidelines), for advisory committee reports was 1.20 (0.99 to 1.45; four studies of 629 advisory committee reports), for opinion pieces was 2.62 (0.91 to 7.55; four studies of 284 opinion pieces), and for narrative reviews was 1.20 (0.97 to 1.49; four studies of 457 narrative reviews). An analysis of all four types of documents combined supported these findings (1.26, 1.09 to 1.44). In one study that investigated specialty interests, the association between including radiologists as authors of guidelines and recommending routine breast cancer was: relative risk 2.10, 95% confidence interval 0.92 to 4.77; 12 clinical guidelines).ConclusionsWe interpret our findings to indicate that financial conflicts of interest are associated with favourable recommendations of drugs and devices in clinical guidelines, advisory committee reports, opinion pieces, and narrative reviews. Limitations of this review were risk of confounding in the included studies and the statistical imprecision of individual analyses of each document type. It is not certain whether non-financial conflicts of interest influence recommendations.Systematic review registrationCochrane Methodology Review Protocol MR000040.

Project description:Evidence-based decision making is a hallmark of effective veterinary clinical practice. Scoping reviews, systematic reviews, and meta-analyses all are methods intended to provide transparent and replicable ways of summarizing a body of research to address an important clinical or public health issue. As these methods increasingly are being used by researchers and read by practitioners, it is important to understand the distinction between these techniques and to understand what research questions they can, and cannot, address. This review provides an overview of scoping reviews, systematic reviews, and meta-analysis, including a discussion of the method and uses. A sample dataset and coding to conduct a simple meta-analysis in the statistical program R also are provided. Scoping reviews are a descriptive approach, designed to chart the literature around a particular topic. The approach involves an extensive literature search, following by a structured mapping, or charting, of the literature. The results of scoping reviews can help to inform future research by identifying gaps in the existing literature and also can be used to identify areas where there may be a sufficient depth of literature to warrant a systematic review. Systematic reviews are intended to address a specific question by identifying and summarizing all of the available research that has addressed the review question. Questions types that can be addressed by a systematic review include prevalence/incidence questions, and questions related to etiology, intervention efficacy, and diagnostic test accuracy. The systematic review process follows structured steps with multiple reviewers working in parallel to reduce the potential for bias. An extensive literature search is undertaken and, for each relevant study identified by the search, a formal extraction of data, including the effect size, and assessment of the risk of bias is performed. The results from multiple studies can be combined using meta-analysis. Meta-analysis provides a summary effect size, and allows heterogeneity of effect among studies to be quantified and explored. These evidence synthesis approaches can provide scientific input to evidence-based clinical decision-making for veterinarians and regulatory bodies, and also can be useful for identifying gaps in the literature to enhance the efficiency of future research in a topic area.

Project description:Summary This report compiles and summarizes all recommendations from CDC’s Advisory Committee on Immunization Practices (ACIP) for use of meningococcal vaccines in the United States. As a comprehensive summary and update of previously published recommendations, it replaces all previously published reports and policy notes. This report also contains new recommendations for administration of booster doses of serogroup B meningococcal (MenB) vaccine for persons at increased risk for serogroup B meningococcal disease. These guidelines will be updated as needed on the basis of availability of new data or licensure of new meningococcal vaccines. ACIP recommends routine vaccination with a quadrivalent meningococcal conjugate vaccine (MenACWY) for adolescents aged 11 or 12 years, with a booster dose at age 16 years. ACIP also recommends routine vaccination with MenACWY for persons aged ?2 months at increased risk for meningococcal disease caused by serogroups A, C, W, or Y, including persons who have persistent complement component deficiencies; persons receiving a complement inhibitor (e.g., eculizumab [Soliris] or ravulizumab [Ultomiris]); persons who have anatomic or functional asplenia; persons with human immunodeficiency virus infection; microbiologists routinely exposed to isolates of Neisseria meningitidis; persons identified to be at increased risk because of a meningococcal disease outbreak caused by serogroups A, C, W, or Y; persons who travel to or live in areas in which meningococcal disease is hyperendemic or epidemic; unvaccinated or incompletely vaccinated first-year college students living in residence halls; and military recruits. ACIP recommends MenACWY booster doses for previously vaccinated persons who become or remain at increased risk. In addition, ACIP recommends routine use of MenB vaccine series among persons aged ?10 years who are at increased risk for serogroup B meningococcal disease, including persons who have persistent complement component deficiencies; persons receiving a complement inhibitor; persons who have anatomic or functional asplenia; microbiologists who are routinely exposed to isolates of N. meningitidis; and persons identified to be at increased risk because of a meningococcal disease outbreak caused by serogroup B. ACIP recommends MenB booster doses for previously vaccinated persons who become or remain at increased risk. In addition, ACIP recommends a MenB series for adolescents and young adults aged 16–23 years on the basis of shared clinical decision-making to provide short-term protection against disease caused by most strains of serogroup B N. meningitidis.

Dataset Information

Applications for oncologic drugs: a descriptive analysis of the oncologic drugs advisory committee reviews.

Background

Methods

Results

Conclusion

Publications

Applications for oncologic drugs: a descriptive analysis of the oncologic drugs advisory committee reviews.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets