Adaptive Licensing: Taking the Next Step in the Evolution of Drug Approval

Eichler, Hans‐Georg; Oye, Kenneth A.; Baird, Lynn G.; Abadie, Eric; Brown, Jeffrey S.; Drum, Chester L.; Ferguson, John G.; Garner, Sarah; Honig, Peter K.; Hukkelhoven, Mathias; Lim, Jeremy; Lim, Robyn; Lumpkin, Murray; Neil, Gary L.; O’Rourke, Brian; Pezalla, Edmund J.; Shoda, D.; Seyfert-Margolis, Vicki; Sigal, Ellen V.; Sobotka, J.; Tan, Derek S.; Unger, T. F.; Hirsch, Gigi

doi:10.1038/clpt.2011.345

Cited by 234 publications

(193 citation statements)

References 19 publications

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“…"Progressive health system decision making" of this kind could align well with thinking in the regulatory community on "progressive" or "adaptive" licensing (21), and build on existing approaches to managed entry or access with evidence development to create a system that better reflects the technology and evidence lifecycle. There are many practical problems to be addressed in putting such systems in place on the ground.…”

Section: Factoring Assessments Of Value Into Decisionsmentioning

confidence: 99%

Health Technology Assessment, Value-Based Decision Making, and Innovation

Henshall

Schuller

2013

Int J Technol Assess Health Care

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On behalf of the HTAi Policy ForumBackground: Identifying treatments that offer value and value for money is becoming increasingly important, with interest in how health technology assessment (HTA) and decision makers can take appropriate account of what is of value to patients and to society, and in the relationship between innovation and assessments of value. Methods: This study summarizes points from an Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations, discussions among attendees, and background papers. Results and Conclusions: Various perspectives on value were considered; most place patient health at the core of value. Wider elements of value comprise other benefits for: patients; caregivers; the health and social care systems; and society. Most decision-making systems seek to take account of similar elements of value, although they are assessed and combined in different ways. Judgment in decisions remains important and cannot be replaced by mathematical approaches. There was discussion of the value of innovation and of the effects of value assessments on innovation. Discussion also included moving toward "progressive health system decision making," an ongoing process whereby evidence-based decisions on use would be made at various stages in the technology lifecycle. Five actions are identified: (i) development of a general framework for the definition and assessment of value; development by HTA/coverage bodies and regulators of (ii) disease-specific guidance and (iii) further joint scientific advice for industry on demonstrating value; (iv) development of a framework for progressive licensing, usage, and reimbursement; and (v) promoting work to better adapt HTA, coverage, and procurement approaches to medical devices.

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Section: Factoring Assessments Of Value Into Decisionsmentioning

confidence: 99%

Health Technology Assessment, Value-Based Decision Making, and Innovation

Henshall

Schuller

2013

Int J Technol Assess Health Care

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“…We now report on the implementation of a new European Union (EU) initiative to improve the promotion and protection of public health through better planning for, and management of, drugs on the market. The new EU pharmacovigilance legislation became operational in July 2012, and after 18 months of operation we suggest that this initiative is starting to demonstrate results in terms of patient safety, and that there could be wider implications in terms of more safe and effective drugs being made available in the future 2,3 .…”

Section: I N K To O R I G I N a L A Rt I C L Ementioning

confidence: 88%

Proactively managing the risk of marketed drugs: experience with the EMA Pharmacovigilance Risk Assessment Committee

Arlett¹,

Portier²,

Lisa³

et al. 2014

Nat Rev Drug Discov

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“…Different evidentiary standards between regulators and payers may lead to divergent appraisals of benefit-risk versus cost-effectiveness (Littlejohns, 2009). This issue calls for good understanding and interaction between the two communities, possibly in the format of iterative discussions and agreement during drug development (Eichler et al, 2012).…”

Section: Similar Requirements Exist In Us Food Drug and Cosmeticmentioning

confidence: 99%

Assessment of benefits and risks in development of targeted therapies for cancer — The view of regulatory authorities

Pignatti¹,

Jönsson

Blumenthal

et al. 2014

Molecular Oncology

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A B S T R A C TDrug licensing and approval decisions involve the balancing of benefits against the risks (harms) in the presence of uncertainty. Typically, the benefits are estimated from primary efficacy endpoints from confirmatory (phase III) clinical trials although exceptions where promising early data from single-arm studies have led to accelerated approvals are not uncommon, particularly for cancer drugs.The challenge for regulators is to balance early evidence of efficacy that might support approval versus the need to establish clinical benefit based on conclusive evidence. Targeted agents offer the promise that knowledge about the mechanism of the disease will help identify patients with tumors likely to respond, resulting in higher efficacy and less toxicity, and earlier regulatory decisions based on convincing evidence of clinical benefit.In this paper, we describe methods and examples of benefit-risk assessment of targeted drugs, recent initiatives from EMA and FDA on improving communication about benefits and risks, and discuss future steps.Published by Elsevier B.V. on behalf of Federation of European Biochemical Societies.

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Adaptive Licensing: Taking the Next Step in the Evolution of Drug Approval

Cited by 234 publications

References 19 publications

Health Technology Assessment, Value-Based Decision Making, and Innovation

Health Technology Assessment, Value-Based Decision Making, and Innovation

Proactively managing the risk of marketed drugs: experience with the EMA Pharmacovigilance Risk Assessment Committee

Assessment of benefits and risks in development of targeted therapies for cancer — The view of regulatory authorities

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