Ethical implications of poor comparative effectiveness evidence: obligations in industry-research partnerships

Singh, Ilina; Naci, Huseyin; Miller, Jennifer E.; Caplan, Arthur L.; Cipriani, Andrea

doi:10.1016/s0140-6736(20)30413-x

Cited by 7 publications

(8 citation statements)

References 11 publications

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“…Vor allem helfen sie Patient:innen und Ärzt:innen, eine individuelle, informierte Entscheidungen zwischen alternativen Therapien zu treffen. Patient:innen, Ärzt:innen, HTA-Institute und damit auch die gesamte Gesellschaft stehen vor der Herausforderung, diese für sie notwendigen Informationen schnellstmöglich zu erlangen (Cipriani et al 2020;Naci et al 2020;Singh et al 2020).…”

Section: "Conditional Marketing Authorisation"unclassified

“…Obwohl auch das beginnende 21. Jahrhundert mit Cerivastatin (Lipobay ® ), Rofecoxib (Vioxx ® ) und anderen durch Arzneimittel-Skandale gezeichnet war (Suja et al 2015), ist durch beschleunigte Zulassungen weltweit ein Wandel in der Regulierung des Arzneimittelmarkts zu beobachten. Ein entscheidender Teil der Evidenzgenerierung wird dabei von der Phase vor der Zulassung auf die Phase nach der Zulassung verlagert -für mehr als die Hälfte aller Arzneimittel fehlen zum Zeitpunkt der Zulassung vergleichende Daten gegenüber Therapiealternativen (Naci et al 2020).…”

Section: Introductionunclassified

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Beschleunigte Zulassung von Arzneimitteln: Herausforderungen für Patient:innen, Datenqualität und faire Preise

Haas

Mayer

Tebinka-Olbrich

et al. 2021

Arzneimittel-Kompass 2021

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Zusammenfassung Zusammenfassung Beschleunigte Zulassungen stellen die Nutzenbewertung und Erstattung von Arzneimitteln vor besondere Herausforderungen. Der Artikel setzt sich in Form eines narrativen Reviews mit den Auswirkungen dieser Zulassungen auf Patient:innen, Angehörige der Gesundheitsberufe und die Solidargemeinschaft sowie mit den möglichen Lösungsstrategien auseinander. Die Autor:innen geben dazu anhand aktueller Literatur zunächst einen Überblick über die Zulassung von Arzneimitteln mit „conditional marketing authorisation“, „approval under exceptional circumstances“ sowie Orphan Drugs. Anschließend stellen sie den Einfluss beschleunigter Zulassungen auf die Nutzenbewertung dar und bewerten diesen. Zum Schluss beschreiben sie in diesem Zusammenhang Herausforderungen im Hinblick auf Arzneimittelpreise.

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Section: "Conditional Marketing Authorisation"unclassified

Section: Introductionunclassified

Beschleunigte Zulassung von Arzneimitteln: Herausforderungen für Patient:innen, Datenqualität und faire Preise

Haas

Mayer

Tebinka-Olbrich

et al. 2021

Arzneimittel-Kompass 2021

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“…Covid-19 has created an unprecedented need for living and trustworthy guidance based on comparative evidence. 47 Recent experience with Australia's National Covid-19 Clinical Evidence Taskforce shows how a comprehensive set of recommendations can be dynamically updated based on new evidence, facilitated by innovative processes and digitally structured data in interoperable platforms (eg, MAGICapp). 48 Such platforms allow for immediate global dissemination of recommendations, interactive evidence summaries, and decision aids that can be reused, adapted, or implemented.…”

Section: Translating Data Into Guidancementioning

confidence: 99%

Producing and using timely comparative evidence on drugs: lessons from clinical trials for covid-19

Naci

Kesselheim

Røttingen

et al. 2020

BMJ

Self Cite

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Since the early days of the novel coronavirus outbreak, a record number of studies have been launched to test several repurposed and new medicines as potential treatments for covid-19. 1 An analysis by the news organisation STAT identified over 1000 clinical trials registered on ClinicalTrials.gov between January and June 2020. 2 This is a testament to the research and clinical community's commitment to identify effective treatments for covid-19. However, the large volume of studies may paradoxically limit the generation of robust evidence and complicate the formulation of trustworthy guidance and decisions related to drug use if the current research is duplicative and redundant or produces conflicting data. 3-5 Indeed, the multiplicity of research on candidate therapeutics for covid-19 has exposed important flaws and failures in the current evidence ecosystem. 6 7 Crucially, these limitations also affect the full spectrum of research on new health technologies. 8 9 Users of evidence across the healthcare system (patients, clinicians, health technology assessment bodies, guideline developers, payers) need timely data on how different treatments compare with each other in terms of their benefits and harms-their comparative effectiveness. Producing comparative evidence and ensuring its rapid translation into trustworthy guidance requires extensive coordination and collaboration between the researchers conducting clinical trials, those conducting comparative effectiveness assessments, and those producing guidance. 8 9 The experience of covid-19 highlights the difficulties in making comparative assessments and suggests areas for improvement.

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“…As the number of available medicines increases, high-quality evidence of comparative effectiveness becomes increasingly essential. This problem was the subject of two recent Lancet reviews and accompanying commentary that promoted comparative effectiveness research for public health [ 2 – 4 ].…”

Section: Introductionmentioning

confidence: 99%

Evaluating Diuretics in Normal Care (EVIDENCE): protocol of a cluster randomised controlled equivalence trial of prescribing policy to compare the effectiveness of thiazide-type diuretics in hypertension

Rogers

Flynn

Mackenzie

et al. 2021

Trials

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Introduction Healthcare systems must use treatments that are effective and safe. Regulators licensed many currently used older medications before introducing the stringent evidential requirements imposed on modern treatments. Also, there has been little encouragement to carry out within-class, head-to-head comparisons of licensed medicines. For commonly prescribed drugs, even small differences in effectiveness or safety could have significant public health implications. However, conventional clinical trials that randomise individual subjects are costly and unwieldy. Such trials are also often criticised as having low external validity. We describe an approach to rapidly generate externally valid evidence of comparative safety and effectiveness using the example of two widely used diuretics for the management of hypertension. Methods and analysis The EVIDENCE (Evaluating Diuretics in Normal Care) study has a prospective, cluster-randomised, open-label, blinded end-point design. By randomising prescribing policy in primary care practices, the study compares the safety and effectiveness of commonly used diuretics in treating hypertension. Participating practices are randomised 1:1 to a policy of prescribing either indapamide or bendroflumethiazide when clinically indicated. Suitable patients who are not already taking the policy diuretic are switched accordingly. All patients taking the study medications are written to explaining the rationale for changing the prescribing policy and notifying them they can opt-out of any switch. The prescribing policies’ effectiveness and safety will be compared using rates of major adverse cardiovascular events (hospitalisation with myocardial infarction, heart failure or stroke or cardiovascular death), routinely collected in national healthcare administrative datasets. The study will seek to recruit 250 practices to provide a study population of approximately 50,000 individuals with a mean follow-up time of two years. A primary intention-to-treat time-to-event analysis will be used to estimate the relative effect of the two policies. Ethics and dissemination EVIDENCE has been approved by the East of Scotland Research Ethics Service (17/ES/0016, current approved protocol version 5, 26 August 2021). The results will be disseminated widely in peer reviewed journals, guideline committees, National Health Service (NHS) organisations and patient groups. Trial registration ISRCTN46635087. Registered on 11 August 2017 (pre-recruitment).

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Ethical implications of poor comparative effectiveness evidence: obligations in industry-research partnerships

Cited by 7 publications

References 11 publications

Beschleunigte Zulassung von Arzneimitteln: Herausforderungen für Patient:innen, Datenqualität und faire Preise

Beschleunigte Zulassung von Arzneimitteln: Herausforderungen für Patient:innen, Datenqualität und faire Preise

Producing and using timely comparative evidence on drugs: lessons from clinical trials for covid-19

Evaluating Diuretics in Normal Care (EVIDENCE): protocol of a cluster randomised controlled equivalence trial of prescribing policy to compare the effectiveness of thiazide-type diuretics in hypertension

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