Many investigational drugs fail in late-stage clinical development. A better understanding of why can inform practice, regulatory decisions, and future research.To assess factors associated with approval or reasons for failure therapeutics phase 3 pivotal trials rates publication trial results.Using public sources commercial databases, we identified that entered between 1998 2008, follow-up through 2015. Agents were classified by therapeutic area, orphan designation status, fast track...

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Quality of life outcomes provide essential information for patients and physicians in oncology care. However, the validity progression-free survival (PFS) as a surrogate quality life, inclusion reporting endpoints clinical trials, is unclear. We performed retrospective study phase III trials drugs advanced or metastatic solid tumors published between 2010 2015. Correlation coefficient (r) area under ROC curve (AUC) association PFS positive were evaluated. Of 352 Phase 3 included, 190 (54%)...

Purpose The breakthrough therapy program was established in 2012 to expedite the development and review of new medicines. We evaluated times approval, efficacy, safety breakthrough-designated versus non-breakthrough-designated cancer drugs approved by US Food Drug Administration (FDA). Methods studied all FDA between January December 2017. Regulatory therapeutic characteristics (time pivotal trial efficacy end point, novelty mechanism action) were compared drugs. Random-effects...

<b>Objective</b>&nbsp;To evaluate safety alerts and recalls, publication of key trial outcomes, subsequent US approval high profile medical devices introduced in the European Union. <b>Design</b>&nbsp;Cohort study. <b>Setting</b>&nbsp;Novel cardiovascular, orthopedic, neurologic approved EU through Conformité Européenne marking between 2005 2010. <b>Data sources</b>&nbsp;Public commercial databases searched up to January 2016 for press releases announcements approvals; public Food Drug...

To characterize the therapeutic value of new drugs approved by US Food and Drug Administration (FDA) European Medicines Agency (EMA) association between these ratings regulatory approval through expedited programs.Retrospective cohort study.New FDA EMA 2007 2017, with follow-up 1 April 2020.Therapeutic was measured using five independent organizations (Prescrire health authorities Canada, France, Germany, Italy).Proportion rated as having high value; rating status.From 320 268 drugs,...

<h3>Importance</h3> The high cost of cancer medicines is a public health challenge. Policy makers in the US and Europe are debating reforms to drug pricing that would cover both prices new when entering market price increases after they launched. <h3>Objective</h3> To assess launch prices, postlaunch changes, clinical benefit drugs compared with 3 European countries (England, Germany, Switzerland). <h3>Design, Setting, Participants</h3> This economic evaluation identified all were approved...

Too often, the clinical trials that provide evidence on which drug approvals and medical practices are based fail to offer adequate support for decision making, because they include inappropriately narrow homogeneous study populations.The Journal has begun requiring investigators report representativeness of their participants, but what next steps?In this Perspective Roundtable,

Abstract Objective To analyze the therapeutic value of supplemental indications compared with first for drugs approved in US and Europe. Design Retrospective cohort study. Setting New by Food Drug Administration (FDA) European Medicines Agency (EMA) between 2011 2020. Main outcome measures Proportion rated as having high using ratings from French German national, independent health authorities. Results The study included 124 335 FDA 88 215 EMA 2020; largest subset was cancer disorders....

The number of new genome-targeted cancer drugs has increased, offering the possibility personalized therapy, often at a very high cost.

The accelerated approval pathway was developed to expedite US Food and Drug Administration (FDA) drug for life-threatening conditions based on changes unvalidated surrogate measures, with conversion regular then a required confirmatory trial. However, trials may not be completed in timely fashion. To analyze factors associated time approval. This cohort study of cancer drugs FDA-approved from 1992 2022 extracted pivotal trial characteristics, outcomes, safety data, status at labels published...

The US Food and Drug Administration (FDA) has 4 expedited programs to speed the development review of drugs treating serious diseases: (1) priority leads FDA in 6 months (vs 10 for standard review);(2) accelerated approval permits based on surrogate measures; (3) fast-track (4) breakthrough therapy are intended reduce duration clinical trials (Table ). 1 Clinical times these programs, particularly newly created program (enacted 2012), have not been comprehensively assessed.We analyzed within...

<h3>Importance</h3> The number of drugs approved through the accelerated approval or conditional marketing authorization pathways has increased with unclear evidence their therapeutic value. <h3>Objectives</h3> To assess value drug indications granted in US European Union (EU) overall and for cancer indications. <h3>Design, Setting, Participants</h3> This cohort study used public databases Food Drug Administration Medicines Agency to identify all (initial supplemental indications) only) EU...

Regulatory agencies have sought to speed up the review of new cancer medicines and reduce delays in approval between countries. We examined trends regulatory times association with clinical benefit for six jurisdictions: United States (Food Drug Administration [FDA]), European Union (European Medicines Agency [EMA]), Switzerland (Swissmedic), Japan (Pharmaceuticals Medical Devices [PMDA]), Canada (Health Canada), Australia (Therapeutic Goods Administration).We studied all drugs approved...

Limits on access to injectable generic drugs force providers use potentially less effective alternatives, current patients discontinue therapy, and some new receive more invasive interventions. Yet shortages of important remain frequent.

Background Few medicines have been approved for children, leading to rates of off-label prescribing reported be as high 90%. In 2007, the European Union adopted Paediatric Regulation, which mandates that pharmaceutical companies conduct paediatric studies all new medicines, unless granted a waiver. We aimed evaluate availability trial results from required under Regulation authorised in EU. Methods and findings The Medicines Agency (EMA) public database investigation plans was searched...

Many medicines prescribed to children have not been studied or formally approved for pediatric use. The Pediatric Research Equity Act of 2003 authorized the US Food and Drug Administration (FDA) require clinical studies.To evaluate characteristics, completion rate, transparency study design results mandatory postmarketing studies required under Act.A retrospective cohort was conducted new drugs indications by FDA between January 1, 2007, December 31, 2014, with follow-up through 2017....

This Viewpoint describes the provisions and limitations of prescription drug pricing reforms enacted by US Congress as part Inflation Reduction Act in August 2022.

BACKGROUND: Medical devices can be useful in a variety of diseases, but few have been specifically approved for use children. The 2007 Pediatric Device Safety and Improvement Act was passed to stimulate pediatric device development. current state trial evidence underpinning the approval remains poorly described. METHODS: We identified all high-risk (ie, class III) through premarket or humanitarian exemption pathways therapeutic children between 2008 2011. collected key information on...

This Viewpoint discusses the Pediatric Research Equity Act, a 2003 law authorizing US Food and Drug Administration to require study of new therapies for pediatric populations, proposes improvements as it comes up reauthorization in 2017.