OBJECTIVE In the last decade an increasing number of high-priced, new cancer treatments received marketing authorisation in Europe. What is actually known about the clinical benefit of those therapies at the time of approval needs to be elucidated in order to inform decisions about the use and reimbursement of these novel treatment options. Thus, the aim of the current analysis was to systemati...

A systematic analysis of clinical trials supporting rare cancer drug approvals may identify concepts and terms that can inform the effective design of prospective clinical trials for rare cancers. In this article, using annual incidence ≤6 of 100,000 individuals to define "rare cancer," we identified clinical trials for rare cancers, supporting U.S. Food and Drug Administration (FDA) drug appro...

Objective To determine the availability of data on overall survival and quality of life benefits of cancer drugs approved in Europe.Design Retrospective cohort study.Setting Publicly accessible regulatory and scientific reports on cancer approvals by the European Medicines Agency (EMA) from 2009 to 2013.Main outcome measures Pivotal and postmarketing trials of cancer drugs according to their de...

Objective To characterize the prospective controlled clinical studies for all novel drugs that were initially approved by the Food and Drug Administration on the basis of limited evidence.Design Systematic review.Data sources Drugs@FDA database and PubMed.Study inclusion All prospective controlled clinical studies published after approval for all novel drugs initially approved by the FDA betwee...

IMPORTANCE Many patients and physicians assume that the safety and effectiveness of newly approved therapeutic agents is well understood; however, the strength of the clinical trial evidence supporting approval decisions by the US Food and Drug Administration (FDA) has not been evaluated. OBJECTIVES To characterize pivotal efficacy trials (clinical trials that serve as the basis of FDA approv...

OBJECTIVE To characterize the types of comparators and endpoints used in efficacy trials for approvals of supplemental indications, compared with the data supporting these drugs' originally approved indications. DESIGN Systematic review. SETTING Publicly accessible data on supplemental indications approved by the US Food and Drug Administration from 2005 to 2014. MAIN OUTCOME MEASURES Typ...

progression [2]. In addition, the time gain is very large in cancers for which the response rate is used as a surrogate end point such as hematological malignancy [3]. Second, we are not discussing a surrogate end point for less serious disease like cardiovascular disease [4], but are discussing a surrogate end point supporting clinical trials of innovative new drugs of high unmet medical need ...

photorefractive keratectomy is a preprogrammed surface corneal refractive ablation which is aimed to reduce or eliminate myopia, hyperopia or astigmatism through the removal of the corneal surface epithelium and basement membrane and the application of excimer laser photoablation to bowman membrane and anterior stroma. the epithelium then begins to cover the wound, in direct contact with the an...

Increasingly, novel agents are approved for the treatment of lymphoma by US Food and Drug Administration (FDA) via accelerated approval using single arm trials surrogate efficacy measures, which leave uncertainty as to their clinical benefit. Methods: We reviewed FDA website [email protected] identify approvals, conversions withdrawals from 2013 2022. From summaries, we collected outcomes pivot...

– Additional analysis of trends for products launched between April 2013 and May 2014 • New molecular entities, formulations and combinations approved by the EMA (EC centralized approval) between January 2009 and May 2014 were included in the analysis. FDA approval dates were retrieved. • Time comparison for general medicines vs. orphan and oncology indications was made including shifts over ti...